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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06056297




Registration number
NCT06056297
Ethics application status
Date submitted
20/09/2023
Date registered
28/09/2023

Titles & IDs
Public title
A Study of Mavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders Who Are Experiencing Recurrent and/or Serious Infections
Scientific title
A Phase 3, Randomized, Double-blind, Placebo-controlled, Multicenter Study of Mavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders Who Are Experiencing Recurrent and/or Serious Infections
Secondary ID [1] 0 0
2023-508482-32-00
Secondary ID [2] 0 0
X4P-001-110
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Neutropenia 0 0
Condition category
Condition code
Blood 0 0 0 0
Other blood disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Mavorixafor
Treatment: Drugs - Placebo

Experimental: Mavorixafor - Participants will receive mavorixafor orally once daily starting from Day 1 through Week 52.

Placebo comparator: Placebo - Participants will receive placebo to match mavorixafor orally once daily starting from Day 1 through Week 52.


Treatment: Drugs: Mavorixafor
Mavorixafor will be administered per schedule specified in the arm description.

Treatment: Drugs: Placebo
Placebo will be administered per schedule specified in the arm description.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Annualized Infection Rate Based on Infections Adjudicated by Blinded, Independent Adjudication Committee (BIAC) During the Treatment Period
Timepoint [1] 0 0
Up to 52 Weeks
Primary outcome [2] 0 0
Number of Participants Meeting the Definition of a Positive Absolute Neutrophil Count (ANC) Response
Timepoint [2] 0 0
Baseline, Week 52
Secondary outcome [1] 0 0
Infection Severity Based on Common Terminology Criteria for Adverse Events (CTCAE) as Assessed by a BIAC During the Treatment Period
Timepoint [1] 0 0
Up to 52 Weeks
Secondary outcome [2] 0 0
Infection Duration Based on Duration of Infections Adjudicated by a BIAC During the Treatment Period in Those Participants who Developed Infections
Timepoint [2] 0 0
Up to 52 Weeks
Secondary outcome [3] 0 0
Antibiotic Use Due to Infection, Characterized by the Frequency of Antibiotic Use During the Treatment Period
Timepoint [3] 0 0
Up to 52 Weeks
Secondary outcome [4] 0 0
Oral Ulcers, as Assessed by Presence or Absence of Ulcers During the Treatment Period
Timepoint [4] 0 0
Up to 52 Weeks
Secondary outcome [5] 0 0
Change From Baseline in Patient Reported Outcomes Measurement Information System Short Form (PROMIS SF) Fatigue Questionnaire Total Score
Timepoint [5] 0 0
Baseline, Week 52

Eligibility
Key inclusion criteria
Key

* Diagnosis of congenital or acquired primary autoimmune and idiopathic chronic neutropenic disorder =6 months prior to the screening visit that is not attributable to medications, active or recent infections or malignancy.
* Congenital Neutropenia, including but not limited to these classifications:

1. Isolated with a permanent (non-cyclic) presentation, for example, ELANE, CSF3R, CXCR2, WAS
2. Associated with extra-hematological manifestations, for example, Barth syndrome, Cohen syndrome, G6PC3, Kostmann disease
3. Associated with metabolic disorders, for example, glycogen storage disease 1b (GSD1b)
4. Shwachman-Diamond syndrome
* Acquired Primary Neutropenia

1. Chronic idiopathic neutropenia
2. Primary autoimmune neutropenia. Other chronic neutropenia (CN) disorders that may be eligible for enrollment can be clarified and approved upon discussion with Study Medical Monitor and Sponsor.
* Have a confirmed trough ANC <1500 cells/µL during the screening visit (single ANC measurement) and at baseline visit (mean ANC over 6 hours) held at least 2 weeks prior to Day 1 dosing, with no clinical evidence of infection.
* Prior history of recurrent and/or serious infections during the 12 months preceding the screening visit (that is, suffering sequelae of chronic neutropenia), as defined by having at least 2 infections in the last 12 months that meet at least 1 of the following criteria:
* Infection requiring the use of antibiotics (intravenous [IV]/oral/topical)
* Infection requiring a visit to healthcare facility (including but not limited to emergency room visit, urgent care facility, primary care physician's office, or in-patient hospitalization).
* Participants who are on G-CSF or other active background therapy must have been receiving these therapies during the previous 12 months while continuing to suffer from infections, be on a stable dose and dosing schedule for =4 weeks prior to screening visit and remain on this dose and dosing schedule throughout the study (unless ANC >10,000 cells/µL for =4 weeks).
* Participants must be willing to keep their G-CSF or other background therapy doses/regimens stable (other than for safety reasons) for the duration of the study.

Key
Minimum age
12 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* A diagnosis of secondary neutropenia including those due to:

1. Hypersplenism
2. Infection
3. Malignancy
4. Autoimmune disease, for example, systemic lupus erythematosus, rheumatoid arthritis, irritable bowel disease, graft-versus-host disease, thyroid disease
5. Nutritional deficiency, for example, vitamin B12, folic acid, copper, caloric malnutrition
6. Drug-induced cause, for example, chemotherapy, clozapine, antiretrovirals, antibiotics, monoclonal antibodies.
* A diagnosis of any of the following:

1. Aplastic anemia
2. Warts, hypogammaglobulinemia, infections, and myelokathexis (WHIM) syndrome
3. Certain CNs, including but not limited to these classifications are excluded:

1. Isolated with a cyclic presentation, for example, elastase, neutrophil expressed (ELANE)
2. Associated with immune dysregulation, for example, common variable immunodeficiency (CVID), autoimmune lymphoproliferative syndrome (ALPS), familial hemophagocytic lymphohistiocytosis, Chédiak-Higashi syndrome, GATA-binding protein 2 (GATA2) deficiency syndrome
3. Associated with bone marrow failure, for example, Fanconi anemia, Diamond-Blackfan anemia
4. Neutropenia associated with a Duffy-null phenotype (formerly known as benign ethnic neutropenia). However, a participant with an autosomal dominant pathogenic variant in a gene associated with CN on a Duffy-null background may be eligible for inclusion
* A medical or personal condition that may potentially compromise the safety of the participant, may preclude the participant's successful completion of the clinical study, or could, in the opinion of the Investigator or the Sponsor, interfere with the objectives of the study.
* Received more than 1 dose of mavorixafor in the past.
* Received CXCR4 antagonist (other than mavorixafor) in the past 6 months.
* Participants taking pegylated-G-CSF unless they have a diagnosis of congenital neutropenia confirmed at screening.
* Participant is currently taking or have taken other investigational drug at least 30 days prior to the screening visit.

Note: Other protocol-defined inclusion and exclusion criteria may apply.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
6/06/2024
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/08/2026
Actual
Sample size
Target
150
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Icon Cancer Centre Southport - Southport
Recruitment postcode(s) [1] 0 0
4215 - Southport
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Michigan
Country [3] 0 0
United States of America
State/province [3] 0 0
Missouri
Country [4] 0 0
Argentina
State/province [4] 0 0
San Miguel de Tucumán
Country [5] 0 0
Czechia
State/province [5] 0 0
Brno
Country [6] 0 0
Czechia
State/province [6] 0 0
Prague
Country [7] 0 0
France
State/province [7] 0 0
Paris
Country [8] 0 0
France
State/province [8] 0 0
Rennes
Country [9] 0 0
Georgia
State/province [9] 0 0
Tbilisi
Country [10] 0 0
Germany
State/province [10] 0 0
Dresden
Country [11] 0 0
Greece
State/province [11] 0 0
Iraklio
Country [12] 0 0
Greece
State/province [12] 0 0
Patra
Country [13] 0 0
Hungary
State/province [13] 0 0
Budapest
Country [14] 0 0
Hungary
State/province [14] 0 0
Szombathely
Country [15] 0 0
Italy
State/province [15] 0 0
Ancona
Country [16] 0 0
Italy
State/province [16] 0 0
Genova
Country [17] 0 0
Italy
State/province [17] 0 0
Meldola
Country [18] 0 0
Italy
State/province [18] 0 0
Monza
Country [19] 0 0
Portugal
State/province [19] 0 0
Aveiro
Country [20] 0 0
Portugal
State/province [20] 0 0
Coimbra
Country [21] 0 0
Romania
State/province [21] 0 0
Bucharest
Country [22] 0 0
Romania
State/province [22] 0 0
Târgu-Mures
Country [23] 0 0
Serbia
State/province [23] 0 0
Belgrade
Country [24] 0 0
Spain
State/province [24] 0 0
Barcelona
Country [25] 0 0
Spain
State/province [25] 0 0
Cáceres
Country [26] 0 0
Spain
State/province [26] 0 0
Córdoba
Country [27] 0 0
Spain
State/province [27] 0 0
Madrid
Country [28] 0 0
Spain
State/province [28] 0 0
Sevilla
Country [29] 0 0
Turkey
State/province [29] 0 0
Samsun
Country [30] 0 0
Ukraine
State/province [30] 0 0
Oblast
Country [31] 0 0
Ukraine
State/province [31] 0 0
Kyiv
Country [32] 0 0
Ukraine
State/province [32] 0 0
Ternopil
Country [33] 0 0
United Kingdom
State/province [33] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
X4 Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Chief Medical Officer
Address 0 0
X4 Pharmaceuticals
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Patient Affairs and Advocacy
Address 0 0
Country 0 0
Phone 0 0
857-529-5779
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT06056297