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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 : June 21, 2019
Sponsor:
Information provided by (Responsible Party):
X4 Pharmaceuticals

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.

Condition or disease Intervention/treatment Phase
WHIM Syndrome Drug: X4P-001 Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 15 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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 : September 2022
Estimated Study Completion Date : September 2022

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
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.

Drug: X4P-001
Mavorixafor will be provided as either 25 mg or 100 mg capsules.
Other Name: AMD11070, Mavorixafor




Primary Outcome Measures :
  1. 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 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

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


Locations
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United States, Washington
University of Washingington Medical Center
Seattle, Washington, United States, 98195
Australia, Victoria
St. Vincent's Hospital
Fitzroy, Victoria, Australia, 3065
Sponsors and Collaborators
X4 Pharmaceuticals
Investigators
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Study Director: Chief Medical Officer X4 Pharmaceuticals

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Responsible Party: X4 Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03005327     History of Changes
Other Study ID Numbers: X4P-001-MKKA
2016-005028-26 ( EudraCT Number )
First Posted: December 29, 2016    Key Record Dates
Last Update Posted: June 21, 2019
Last Verified: June 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Syndrome
Disease
Pathologic Processes