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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/NCT03005327




Registration number
NCT03005327
Ethics application status
Date submitted
20/12/2016
Date registered
29/12/2016
Date last updated
7/09/2023

Titles & IDs
Public title
A Dose Determination and Safety Study of X4P-001 (Mavorixafor) in Participants With Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) Syndrome
Scientific title
A Phase 2, Open-Label, Multi-Center Trial of Mavorixafor in Patients With WHIM Syndrome
Secondary ID [1] 0 0
2016-005028-26
Secondary ID [2] 0 0
X4P-001-MKKA
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
WHIM Syndrome 0 0
Condition category
Condition code
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - X4P-001

Experimental: X4P-001 - Initial Treatment Phase: Participants will initiate treatment with mavorixafor at 50 milligrams (mg) once daily (QD) orally or a higher dose, with potential escalation based on area under the curve for absolute neutrophil count and absolute leukocyte count (AUCANC/ALC) values to a maximum total daily dose of 400 mg. Participants are expected to receive treatment for 24 weeks in the initial Treatment Period or until development of a treatment-limiting toxicity (TLT).
Extension Phase: All participants will receive mavorixafor; the dose will not exceed 400 mg. In the Extension Phase, treatment may continue until mavorixafor becomes available via an alternative mechanism (for example, drug is commercially available, an expanded access program, etc.) or until the study is terminated by the sponsor.


Treatment: Drugs: X4P-001
Mavorixafor will be provided as either 25 mg or 100 mg capsules.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Mean AUCANC and/or AUCALC
Timepoint [1] 0 0
Time 0 (-15 minutes [min] pre-dose), 30, 60, and 90 min (each ± 5 min) and 2, 3, 4, 8, 12, 16, and 24 hours (each ±15 min) at Weeks 5, 13, and 21

Eligibility
Key inclusion criteria
Participants with a clinical diagnosis of WHIM syndrome must meet all of the following
criteria to be eligible for study participation:

1. Be at least 18 years of age.

2. Has signed the current approved informed consent form.

3. Has a genotype-confirmed mutation of chemokine receptor type 4 (CXCR4) consistent with
WHIM syndrome.

4. Agree to use effective contraception.

5. Be willing and able to comply with this protocol.

6. Has confirmed ANC less than or equal to (=) 400/µL or ALC =650/µL or both.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Participants with any of the following will be excluded from participation in the study:

1. Has known systemic hypersensitivity to the mavorixafor drug substance or its inactive
ingredients.

2. Is pregnant or nursing.

3. Has a known history of a positive serology or viral load for human immunodeficiency
virus (HIV) or a known history of acquired immunodeficiency syndrome (AIDS).

4. Has, at Screening, laboratory tests meeting one or more of the following criteria:

- A positive antibody test for hepatitis C virus (HCV), unless documented to have
no detectable viral load on 2 independent samples.

- A positive test for hepatitis B surface antigen (HBsAg).

5. Has any medical or personal condition that, 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
N/A
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
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/12/2016
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
16/06/2022
Sample size
Target
Accrual to date
Final
8
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
St. Vincent's Hospital - Fitzroy
Recruitment postcode(s) [1] 0 0
3065 - Fitzroy
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Washington

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

Ethics approval
Ethics application status

Summary
Brief summary
This is a Phase 2 study with an initial 24-week Treatment Period and an Extension Phase. The
primary objectives of this Phase 2 study are to determine the safety, tolerability, and dose
selection of mavorixafor in participants with WHIM syndrome. Participants are allowed to
continue treatment in an Extension Phase, if regionally applicable, until it becomes
commercially available or until the study is terminated by the Sponsor.
Trial website
https://clinicaltrials.gov/ct2/show/NCT03005327
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT03005327