A Dose Determination and Safety Study of X4P-001 (Mavorixafor) in Participants With Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) Syndrome

WHIM Syndrome Clinical Trial

Official title:

A Phase 2, Open-Label, Multi-Center Trial of Mavorixafor in Patients With WHIM Syndrome

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03005327
• Other study ID #: X4P-001-MKKA
• Secondary ID: 2016-005028-26
• Status: Completed
• Phase: Phase 2
• Start date: December 2016
• Est. completion date: June 16, 2022

Study information

• Verified date: September 2023
• Source: X4 Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 8
• Est. completion date: June 16, 2022
• Est. primary completion date: June 16, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

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.

Related Conditions & MeSH terms

• Syndrome
• WHIM Syndrome

Intervention

Drug:
• X4P-001
Mavorixafor will be provided as either 25 mg or 100 mg capsules.

Locations

Country	Name	City	State
Australia	St. Vincent's Hospital	Fitzroy	Victoria
United States	University of Washingington Medical Center	Seattle	Washington

Sponsors (1)

Lead Sponsor	Collaborator
X4 Pharmaceuticals

Countries where clinical trial is conducted

United States,  Australia, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Mean AUCANC and/or AUCALC		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