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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT03995108
Registration number
NCT03995108
Ethics application status
Date submitted
19/06/2019
Date registered
21/06/2019
Date last updated
2/05/2024
Titles & IDs
Public title
Efficacy and Safety Study of Mavorixafor in Participants With Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) Syndrome
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Scientific title
A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Mavorixafor in Patients With WHIM Syndrome With Open-Label Extension
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Secondary ID [1]
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2019-001153-10
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Secondary ID [2]
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X4P-001-103
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
WHIM Syndrome
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Condition category
Condition code
Other
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Research that is not of generic health relevance and not applicable to specific health categories listed above
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Mavorixafor
Treatment: Drugs - Placebo
Experimental: Mavorixafor - Participants (adults and adolescents [12 to 17 years of age weighing >50 kilograms [kg]) will receive mavorixafor 400 milligrams (mg) once daily (QD) orally for 52 weeks in the Randomized Placebo-Controlled Period. Adolescents weighing =50 kg will receive mavorixafor 200 mg QD. Participants who complete the Randomized Placebo-Controlled Period or are granted Early Release due to recurrent or significant infections, as adjudicated by a blinded, independent adjudication committee (AC), will be offered the opportunity to enroll in the Open-Label Period and receive treatment with mavorixafor 400 mg once daily orally until commercial availability or study termination by the Sponsor.
Placebo Comparator: Placebo - Participants will receive placebo matching to mavorixafor QD orally for 52 weeks in the Randomized Placebo-Controlled Period. Participants who complete the Randomized Placebo-Controlled Period or are granted Early Release due to recurrent or significant infections, as adjudicated by a blinded, independent AC, will be offered the opportunity to enroll in the Open-Label Period and receive treatment with mavorixafor 400 mg once daily orally until commercial availability or study termination by the Sponsor.
Treatment: Drugs: Mavorixafor
Mavorixafor provided as 100 mg capsules.
Treatment: Drugs: Placebo
Placebo matching to mavorixafor capsules
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Randomized Placebo-Controlled Period: Time (in Hours) Above Threshold-Absolute Neutrophil Count (TAT-ANC in hours) of = 500 Cells/Microliter (µL) over a 24-hour period
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Assessment method [1]
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Timepoint [1]
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Time 0 (pre-dose, up to 15 minutes prior), 30, 60, and 90 min (each ± 5 min) and 2, 3, 4, 8, 12, 16, and 24 hours (each ± 15 min) post-dose at Baseline, Weeks 13, 26, 39, and 52
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Primary outcome [2]
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Open-Label Period: Percentage of Participants With Adverse Events (AEs)
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Assessment method [2]
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Timepoint [2]
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From Day 1 (end of randomized period) up to end of study (30 days post-treatment in open-label period [Week 56 of open-label period])
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Secondary outcome [1]
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Randomized Placebo-Controlled Period: Time (in Hours) Above Threshold-Absolute Lymphocyte Count (TAT-ALC) of = 1000 Cells/µL over a 24-hour period
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Assessment method [1]
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Timepoint [1]
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Time 0 (pre-dose, up to 15 minutes prior), 30, 60, and 90 minutes (each ± 5 minutes) and 2, 3, 4, 8, 12, 16, and 24 hours (each ± 15 minutes) post-dose at Baseline, Weeks 13, 26, 39, and 52
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Secondary outcome [2]
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Randomized Placebo-Controlled Period: Composite Clinical Efficacy for Mavorixafor based on total infection score and total wart change score
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Assessment method [2]
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Timepoint [2]
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Baseline up to Week 52
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Secondary outcome [3]
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Randomized Placebo-Controlled Period: Change From Baseline in Total Warts Score at Week 52
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Assessment method [3]
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Timepoint [3]
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Baseline, Week 52
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Secondary outcome [4]
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Randomized Placebo-Controlled Period: Total Infection Score for Mavorixafor
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Assessment method [4]
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Timepoint [4]
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Baseline up to Week 52
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Secondary outcome [5]
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Randomized Placebo-Controlled Period: Time to Early Release
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Assessment method [5]
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Timepoint [5]
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Baseline up to Week 52
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Secondary outcome [6]
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Randomized Placebo-Controlled Period: TAT-ALC of = 1000 Cells/µL in Participants With Lymphopenia
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Assessment method [6]
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Timepoint [6]
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Baseline
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Secondary outcome [7]
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Randomized Placebo-Controlled Period: Total Infection Score for Participants With Non-Immunoglobulin (non-Ig) Use (Percentage of Participants With Infections)
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Assessment method [7]
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Timepoint [7]
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Baseline up to Week 52
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Secondary outcome [8]
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Randomized Placebo-Controlled Period: Change in Total Wart Score at Baseline, Based on Clinical Global Impression of Change (CGI-C)
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Assessment method [8]
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Timepoint [8]
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Baseline
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Secondary outcome [9]
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Randomized Placebo-Controlled Period: Composite Clinical Efficacy (Total Infection Score and Total Wart Change Score) for Participants With Non-Ig Use
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Assessment method [9]
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Composite clinical efficacy will be calculated using the total infection score and total wart change score for participants with warts at baseline or non-Ig use. It will be analyzed by a blinded, independent AC.
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Timepoint [9]
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Baseline up to Week 52
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Secondary outcome [10]
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Randomized Placebo-Controlled Period: Change in Total Wart Score at Baseline (CGI-C), Based on Local Dermatologist Review
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Assessment method [10]
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Timepoint [10]
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Baseline
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Secondary outcome [11]
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Randomized Placebo-Controlled Period: Participant Global Impression of Change (PGI-C)
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Assessment method [11]
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Timepoint [11]
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Baseline up to Week 52
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Secondary outcome [12]
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Randomized Placebo-Controlled Period: Participant Global Impression of Severity (PGI-S)
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Assessment method [12]
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Timepoint [12]
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Baseline up to Week 52
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Secondary outcome [13]
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Randomized Placebo-Controlled Period: Vaccine Titer Levels at Week 52 in Participants Vaccinated at Week 13, With Tetanus, Diphtheria, and Pertussis (Tdap) Including Pertussis Toxin, and Tetanus
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Assessment method [13]
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Timepoint [13]
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Week 52
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Secondary outcome [14]
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Randomized Placebo-Controlled Period: Vaccine Titer Levels at Week 52 for Human Papillomavirus (HPV) 16 and HPV 18 in Participants Receiving Vaccinations With HPV 9-Valent Vaccine, Recombinant (Gardasil®9)
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Assessment method [14]
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Timepoint [14]
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Week 52
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Secondary outcome [15]
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Randomized Placebo-Controlled Period: Change From Baseline in Clinical Global Impression of Severity (CGI-S), Based on Local Dermatologist Review
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Assessment method [15]
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Timepoint [15]
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Baseline up to Week 52
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Secondary outcome [16]
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Randomized Placebo-Controlled Period: Number of Participants with Infections
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Assessment method [16]
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Timepoint [16]
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Baseline up to Week 52
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Secondary outcome [17]
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Randomized Placebo-Controlled Period: Infection-Free Time
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Assessment method [17]
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Timepoint [17]
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Baseline up to Week 52
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Secondary outcome [18]
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Randomized Placebo-Controlled Period: Number of Days Lost From Work/School
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Assessment method [18]
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Timepoint [18]
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Baseline up to Week 52
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Secondary outcome [19]
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Randomized Placebo-Controlled Period: Quality of Life as Measured by 36-Item Short Form Survey Score
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Assessment method [19]
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Timepoint [19]
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Baseline up to Week 52
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Secondary outcome [20]
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Randomized Placebo-Controlled Period: Quality of Life as Measured by EuroQoL-5 Dimension-5 Level (EQ-5D-5L) Score
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Assessment method [20]
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Timepoint [20]
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Baseline up to Week 52
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Secondary outcome [21]
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Randomized Placebo-Controlled Period: Quality of Life as Measured by Life Quality Index (LQI) Score
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Assessment method [21]
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Timepoint [21]
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Baseline up to Week 52
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Secondary outcome [22]
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Randomized Placebo-Controlled Period: Quality of Life as Measured by Dermatology LQI Score
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Assessment method [22]
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Timepoint [22]
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Baseline up to Week 52
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Secondary outcome [23]
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Randomized Placebo-Controlled Period: Quality of Life as Measured by Pediatric Quality of Life Inventory (PedsQL) Score
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Assessment method [23]
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Timepoint [23]
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Baseline up to Week 52
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Secondary outcome [24]
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Randomized Placebo-Controlled Period: Change From Baseline in Anogenital (AG) Warts Based on Dermatologist CGI-C Assessment
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Assessment method [24]
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Timepoint [24]
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Baseline to Week 52
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Secondary outcome [25]
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Randomized Placebo-Controlled Period: Change From Baseline in Anogenital (AG) Warts Based on AG Wart Severity Assessment
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Assessment method [25]
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Timepoint [25]
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Baseline to Week 52
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Secondary outcome [26]
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Randomized Placebo-Controlled Period: Number of Events Requiring Rescue Treatment Due to Infection
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Assessment method [26]
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Timepoint [26]
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Baseline to Week 52
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Secondary outcome [27]
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Randomized Placebo-Controlled Period: Number of Participants With Incidence and Duration of Hospitalizations Due to Infection
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Assessment method [27]
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Timepoint [27]
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Baseline to Week 52
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Secondary outcome [28]
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Randomized Placebo-Controlled Period: Number of Participants With Incidence of Newly Developed Warts
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Assessment method [28]
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Timepoint [28]
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Baseline to Week 52
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Secondary outcome [29]
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Randomized Placebo-Controlled Period: Area Under the Curve for ANC (AUCANC) Using Trapezoidal Method
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Assessment method [29]
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Timepoint [29]
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Time 0 (pre-dose, up to 15 minutes prior), 30, 60, and 90 minutes (each ± 5 minutes) and 2, 3, 4, 8, 12, 16, and 24 hours (each ± 15 minutes) post-dose at Baseline, Weeks 13, 26, 39, and 52
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Secondary outcome [30]
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Randomized Placebo-Controlled Period: Percentage of Neutrophil Responders
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Assessment method [30]
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Timepoint [30]
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Baseline up to Week 52
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Secondary outcome [31]
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Randomized Placebo-Controlled Period: Mavorixafor Treatment Group: AUCANC
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Assessment method [31]
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Timepoint [31]
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Time 0 (pre-dose, up to 15 minutes prior), 30, 60, and 90 minutes (each ± 5 minutes) and 2, 3, 4, 8, 12, 16, and 24 hours (each ± 15 minutes) post-dose at Baseline, Weeks 13, 26, 39, and 52
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Secondary outcome [32]
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Randomized Placebo-Controlled Period: Area Under the Curve for ALC (AUCALC)
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Assessment method [32]
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Timepoint [32]
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Time 0 (pre-dose, up to 15 minutes prior), 30, 60, and 90 minutes (each ± 5 minutes) and 2, 3, 4, 8, 12, 16, and 24 hours (each ± 15 minutes) post-dose at Baseline, Weeks 13, 26, 39, and 52
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Secondary outcome [33]
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Randomized Placebo-Controlled Period: Percentage of Lymphocyte Responders
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Assessment method [33]
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Timepoint [33]
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Baseline up to Week 52
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Secondary outcome [34]
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Randomized Placebo-Controlled Period: Change From Baseline in Total ALC, Absolute Monocyte Count (AMC), ANC, and White Blood Cell (WBC) at Week 52
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Assessment method [34]
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Timepoint [34]
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Baseline, Week 52
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Secondary outcome [35]
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Absolute and Fold Change From Baseline in Absolute T, B and Natural Killer Lymphocyte at Week 52
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Assessment method [35]
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Timepoint [35]
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Baseline, Week 52
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Secondary outcome [36]
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Randomized Placebo-Controlled Period: Number of Participants With AEs
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Assessment method [36]
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Timepoint [36]
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Baseline up to Week 52
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Secondary outcome [37]
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Randomized Placebo-Controlled Period: Pharmacokinetics (PK), Maximum Observed Plasma Concentration (Cmax) of Mavorixafor
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Assessment method [37]
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Timepoint [37]
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Time 0 (pre-dose, up to 15 minutes prior), 30, 60, and 90 minutes (each ± 5 minutes) and 2, 3, 4, 8, 12, 16, and 24 hours (each ± 15 minutes) post-dose at Weeks 13, 26, 39, and 52; and 4 hours post-dose at Baseline
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Secondary outcome [38]
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Randomized Placebo-Controlled Period: PK, Time to Reach Cmax (Tmax) of Mavorixafor
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Assessment method [38]
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Timepoint [38]
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Time 0 (pre-dose, up to 15 minutes prior), 30, 60, and 90 min (each ± 5 minutes) and 2, 3, 4, 8, 12, 16, and 24 hours (each ± 15 minutes) post-dose at Weeks 13, 26, 39, and 52; and 4 hours post-dose at Baseline
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Secondary outcome [39]
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Randomized Placebo-Controlled Period: PK, Half-Life of (T1/2) of Mavorixafor
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Assessment method [39]
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Timepoint [39]
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Time 0 (pre-dose, up to 15 minutes prior), 30, 60, and 90 minutes (each ± 5 minutes) and 2, 3, 4, 8, 12, 16, and 24 hours (each ± 15 minutes) post-dose at Weeks 13, 26, 39, and 52; and 4 hours post-dose at Baseline
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Secondary outcome [40]
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Randomized Placebo-Controlled Period: PK, Area Under the Curve (AUC) of Mavorixafor
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Assessment method [40]
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0
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Timepoint [40]
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Time 0 (pre-dose, up to 15 minutes prior), 30, 60, and 90 minutes (each ± 5 minutes) and 2, 3, 4, 8, 12, 16, and 24 hours (each ± 15 minutes) post-dose at Weeks 13, 26, 39, and 52; and 4 hours post-dose at Baseline
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Secondary outcome [41]
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Open-Label Period: Percentage of Neutrophil Responders
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Assessment method [41]
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Timepoint [41]
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Baseline up to Week 52 of open-label period
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Secondary outcome [42]
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Open-Label Period: Percentage of Lymphocyte Responders
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Assessment method [42]
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Timepoint [42]
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Baseline up to Week 52 of open-label period
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Secondary outcome [43]
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Open-Label Period: Absolute and Fold Change From Baseline in Total ALC, AMC, ANC, and WBC at Week 52
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Assessment method [43]
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Timepoint [43]
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Baseline up to Week 52 of open-label period
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Secondary outcome [44]
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Open-Label Period: Vaccine Titer Levels During the First Year of Open-Label Period, in Participants Vaccinated With Tdap During the Study Including Pertusis Toxin and Tetanus
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Assessment method [44]
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0
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Timepoint [44]
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Year 1 of open-label period
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Secondary outcome [45]
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Open-Label Period: Vaccine Titer Levels During the First Year of the Open-Label Period for HPV 16 and HPV 18 in Participants Receiving Vaccinations With HPV 9-Valent Vaccine, Recombinant (Gardasil®9) During the Study
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Assessment method [45]
0
0
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Timepoint [45]
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Year 1 of open-label period
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Secondary outcome [46]
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Open-Label Period: Change From Baseline in Cutaneous Warts at Week 52, Based on Central Review of CGI-C
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Assessment method [46]
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0
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Timepoint [46]
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Baseline, Week 52 of open-label period
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Secondary outcome [47]
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Open-Label Period: Change From Baseline in Cutaneous Warts, Based on Central Review of CGI-S
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Assessment method [47]
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0
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Timepoint [47]
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Baseline, Week 52 of open-label period
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Secondary outcome [48]
0
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Open-Label Period: Change From Baseline in Cutaneous Warts, Based on Local Dermatologist CGI-C
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Assessment method [48]
0
0
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Timepoint [48]
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Baseline, Week 52 of open-label period
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Secondary outcome [49]
0
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Open-Label Period: Change From Baseline in Cutaneous Warts, Based on Local Dermatologist CGI-S
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Assessment method [49]
0
0
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Timepoint [49]
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Baseline, Week 52 of open-label period
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Secondary outcome [50]
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Open-Label Period: Change Over Time in PGI-C
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Assessment method [50]
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Timepoint [50]
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Baseline up to Week 52 of open-label period
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Secondary outcome [51]
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Open-Label Period: Change Over Time in PGI-S
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Assessment method [51]
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Timepoint [51]
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Baseline up to Week 52 of open-label period
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Secondary outcome [52]
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Open-Label Period: Total Infection Score (Percentage of Participants With Infections)
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Assessment method [52]
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Timepoint [52]
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Baseline up to Week 52 of open-label period
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Eligibility
Key inclusion criteria
Inclusion Criteria for the Randomized Placebo-Controlled Period :
- Have signed the current approved informed consent form. Participants under 18 years of
age (in the Netherlands and other applicable regions, participants under 16 years of
age) will sign an approved informed assent form and must also have a signed
parental/legal guardian consent.
- Have a genotype-confirmed mutation of chemokine (C-X-C motif) receptor 4 (CXCR4)
consistent with WHIM phenotype.
- Agree to use a highly effective form of contraception.
- Be willing and able to comply with the protocol.
- Have confirmed ANC =400 cells/µL during screening, obtained while participant has no
clinical evidence of infection.
Inclusion Criteria for the Open-Label Period:
- Completed the Randomized Period; or
- Granted Early Release from the Randomized Period.
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Minimum age
12
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
- Has known systemic hypersensitivity to the mavorixafor drug substance, its inactive
ingredients, or the placebo.
- Is pregnant or breastfeeding.
- Has any medical or personal condition, which in the opinion of the Investigator may
potentially compromise the safety or compliance of the participant or may preclude the
participant's successful completion of the clinical study.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people analysing the results/data
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Active, not recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
24/10/2019
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
1/12/2024
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Actual
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Sample size
Target
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Accrual to date
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Final
31
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Recruitment in Australia
Recruitment state(s)
QLD
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Recruitment hospital [1]
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Wesley Hospital - Auchenflower
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Recruitment hospital [2]
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Children's Health Queensland Hospital - South Brisbane
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Recruitment postcode(s) [1]
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0
4006 - Auchenflower
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Recruitment postcode(s) [2]
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0
4101 - South Brisbane
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Recruitment outside Australia
Country [1]
0
0
United States of America
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State/province [1]
0
0
California
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Country [2]
0
0
United States of America
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State/province [2]
0
0
Iowa
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Country [3]
0
0
United States of America
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State/province [3]
0
0
Maryland
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Country [4]
0
0
United States of America
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State/province [4]
0
0
Texas
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Country [5]
0
0
United States of America
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State/province [5]
0
0
Washington
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Country [6]
0
0
Austria
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State/province [6]
0
0
Wien
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Country [7]
0
0
Denmark
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State/province [7]
0
0
Aarhus
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Country [8]
0
0
France
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State/province [8]
0
0
Rhne
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Country [9]
0
0
France
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State/province [9]
0
0
Paris Cedex 12
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Country [10]
0
0
France
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State/province [10]
0
0
Paris
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Country [11]
0
0
Hungary
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State/province [11]
0
0
Hajdu-Bihar
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Country [12]
0
0
Israel
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State/province [12]
0
0
Afula
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Country [13]
0
0
Italy
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State/province [13]
0
0
Piazza Del Mercato
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Country [14]
0
0
Korea, Republic of
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State/province [14]
0
0
Seoul
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Country [15]
0
0
Netherlands
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State/province [15]
0
0
Amsterdam
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Country [16]
0
0
Russian Federation
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State/province [16]
0
0
Moscow
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Country [17]
0
0
Russian Federation
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State/province [17]
0
0
Saint Pertersburg
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Country [18]
0
0
Spain
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State/province [18]
0
0
Esplugues De Llobregat
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Country [19]
0
0
Spain
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State/province [19]
0
0
Sevilla
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Country [20]
0
0
Turkey
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State/province [20]
0
0
Adana
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
X4 Pharmaceuticals
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
This study has a double-blind, Randomized Placebo-Controlled Period and an Open-Label Period.
The primary objective of the Randomized Placebo-Controlled Period is to demonstrate the
efficacy of mavorixafor in participants with WHIM syndrome as assessed by increasing levels
of circulating neutrophils compared with placebo, and relative to a clinically meaningful
threshold. The primary objective of the Open-Label Period is to evaluate the safety and
tolerability of mavorixafor in participants with WHIM syndrome. Participants are allowed to
continue treatment in the Open-Label Period, if regionally applicable, until mavorixafor
becomes commercially available, or until the study is terminated by the Sponsor.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT03995108
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Principal investigator
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X4 Pharmaceuticals
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Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT03995108
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