Brief Title
Efficacy and Safety Study of Mavorixafor in Participants With Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) Syndrome
Official Title
A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Mavorixafor in Patients With WHIM Syndrome With Open-Label Extension
Brief Summary
This study has a double-blind, placebo-controlled Randomized Period and an Open-Label extension Period. The primary objective of the Randomized 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 extension Period, if regionally applicable, until mavorixafor becomes commercially available, or until the study is terminated by the Sponsor.
Study Phase
Phase 3
Study Type
Interventional
Primary Outcome
Randomized Period: Time (in Hours) Above Absolute Neutrophil Count (ANC) Threshold of 500 Cells/Microliter (µL)
Secondary Outcome
Randomized Period: Area Under the Curve for ANC (AUCANC) Using Trapezoidal Method
Condition
WHIM Syndrome
Intervention
Mavorixafor
Study Arms / Comparison Groups
Mavorixafor
Description: 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 Period. Adolescents weighing ≤50 kg will receive mavorixafor 200 mg QD. Participants who complete the Randomized 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.
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
28
Start Date
October 17, 2019
Completion Date
October 2022
Primary Completion Date
September 2022
Eligibility Criteria
Inclusion Criteria: - 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. 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. Exclusion Criteria for the Open-Label Period: - Participants who experience any treatment-limiting toxicity (TLT) will be excluded from participating in the Open-Label Period.
Gender
All
Ages
12 Years - N/A
Accepts Healthy Volunteers
No
Contacts
Chief Medical Officer, ,
Location Countries
Australia
Location Countries
Australia
Administrative Informations
NCT ID
NCT03995108
Organization ID
X4P-001-103
Secondary IDs
2019-001153-10
Responsible Party
Sponsor
Study Sponsor
X4 Pharmaceuticals
Study Sponsor
Chief Medical Officer, Study Director, X4 Pharmaceuticals
Verification Date
October 2021