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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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18 September 2023 |
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Main ID: |
NCT03005327 |
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Date of registration:
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20/12/2016 |
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Prospective Registration:
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No |
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Primary sponsor: |
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Public title:
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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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Scientific title:
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A Phase 2, Open-Label, Multi-Center Trial of Mavorixafor in Patients With WHIM Syndrome |
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Date of first enrolment:
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December 2016 |
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Target sample size:
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8 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/ct2/show/NCT03005327 |
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Study type:
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Interventional |
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Study design:
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Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 2
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Countries of recruitment
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Australia
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United States
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Key inclusion & exclusion criteria
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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.
Age minimum:
18 Years
Age maximum:
N/A
Gender:
All
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Health Condition(s) or Problem(s) studied
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WHIM Syndrome
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Intervention(s)
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Drug: X4P-001
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Primary Outcome(s)
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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]
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Secondary ID(s)
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2016-005028-26
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X4P-001-MKKA
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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