A Dose Determination and Safety Study of X4P-001 (Mavorixafor) in Participants With Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) Syndrome
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|ClinicalTrials.gov Identifier: NCT03005327|
Recruitment Status : Active, not recruiting
First Posted : December 29, 2016
Last Update Posted : August 14, 2020
|Condition or disease||Intervention/treatment||Phase|
|WHIM Syndrome||Drug: X4P-001||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||15 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase 2, Open-Label, Multi-Center Trial of Mavorixafor in Patients With WHIM Syndrome|
|Actual Study Start Date :||December 2016|
|Estimated Primary Completion Date :||December 2022|
|Estimated Study Completion Date :||December 2022|
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.
Mavorixafor will be provided as either 25 mg or 100 mg capsules.
Other Name: AMD11070, Mavorixafor
- Mean AUCANC and/or AUCALC [ Time Frame: 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 ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03005327
|United States, Washington|
|University of Washingington Medical Center|
|Seattle, Washington, United States, 98195|
|St. Vincent's Hospital|
|Fitzroy, Victoria, Australia, 3065|
|Study Director:||Chief Medical Officer||X4 Pharmaceuticals|