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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
First received
Last updated
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
Health authority
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.

Study Design


Related Conditions & MeSH terms


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
See also
  Status Clinical Trial Phase
Active, not recruiting NCT03995108 - Efficacy and Safety Study of Mavorixafor in Participants With Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) Syndrome Phase 3
Withdrawn NCT03087370 - A Retrospective and Prospective Natural History Study of Patients With WHIM Syndrome