Efficacy and Safety Study of Mavorixafor in Participants With Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) Syndrome
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ClinicalTrials.gov Identifier: NCT03995108 |
Recruitment Status :
Recruiting
First Posted : June 21, 2019
Last Update Posted : March 16, 2021
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Condition or disease | Intervention/treatment | Phase |
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WHIM Syndrome | Drug: Mavorixafor Drug: Placebo | Phase 3 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 28 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | Double (Participant, Investigator) |
Primary Purpose: | Treatment |
Official Title: | A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Mavorixafor in Patients With WHIM Syndrome With Open-Label Extension |
Actual Study Start Date : | October 17, 2019 |
Estimated Primary Completion Date : | September 2021 |
Estimated Study Completion Date : | October 2022 |

Arm | Intervention/treatment |
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Experimental: Mavorixafor
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.
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Drug: Mavorixafor
Mavorixafor provided as 100 mg capsules.
Other Name: AMD11070, X4P-001 |
Placebo Comparator: Placebo
Participants will receive placebo matching to mavorixafor QD orally for 52 weeks in the Randomized Period. Participants who complete the Randomized Period or are granted Early Release due to recurrent or significant infections, as adjudicated by a blinded, independent 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.
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Drug: Mavorixafor
Mavorixafor provided as 100 mg capsules.
Other Name: AMD11070, X4P-001 Drug: Placebo Placebo matching to mavorixafor capsules |
- Randomized Period: Time (in Hours) Above Absolute Neutrophil Count (ANC) Threshold of 500 Cells/Microliter (µL) [ Time Frame: Time 0 (pre-dose, up to 15 minutes prior), 30, 60, and 90 min (each ± 5 min) and 2, 3, 4, 8, 12, 16, and 24 hours (each ± 15 min) post-dose at Baseline, Weeks 13, 26, 39, and 52 ]
- Open-Label Period: Percentage of Participants With Adverse Events (AEs) [ Time Frame: From Day 1 (end of randomized period) up to end of study (30 days post-treatment in open-label period [Week 56 of open-label period]) ]
- Randomized Period: Area Under the Curve for ANC (AUCANC) Using Trapezoidal Method [ Time Frame: Time 0 (pre-dose, up to 15 minutes prior), 30, 60, and 90 minutes (each ± 5 minutes) and 2, 3, 4, 8, 12, 16, and 24 hours (each ± 15 minutes) post-dose at Baseline, Weeks 13, 26, 39, and 52 ]
- Randomized Period: Infection Rate (Percentage of Participants With Infections) Based on Infections Adjudicated by a Blinded, Independent AC [ Time Frame: Baseline up to Week 52 ]
- Randomized Period: Vaccine Titer Levels at Week 52 in Participants Vaccinated at Week 13, With Tetanus, Diphtheria, and Pertussis (Tdap) Including pertussis Toxin, and Tetanus [ Time Frame: Week 52 ]
- Randomized Period: Vaccine Titer Levels at Week 52 for Human Papillomavirus (HPV) 16 and HPV 18 in Participants Receiving Vaccinations With HPV 9-Valent Vaccine, Recombinant (Gardasil®9) [ Time Frame: Week 52 ]
- Randomized Period: Change From Baseline in Cutaneous Warts at Week 52, Based on Dermatologist Clinical Global Impression of Change (CGI-C) [ Time Frame: Baseline, Week 52 ]
- Randomized Period: Time to Early Release as Confirmed by Blinded Independent AC [ Time Frame: Baseline up to Week 52 ]
- Randomized Period: Percentage of Neutrophil Responders [ Time Frame: Baseline up to Week 52 ]
- Randomized Period: Mavorixafor Treatment Group: AUCANC [ Time Frame: Time 0 (pre-dose, up to 15 minutes prior), 30, 60, and 90 minutes (each ± 5 minutes) and 2, 3, 4, 8, 12, 16, and 24 hours (each ± 15 minutes) post-dose at Baseline, Weeks 13, 26, 39, and 52 ]
- Randomized Period: Time (in Hours) Above Absolute Lymphocyte Count (ALC) Threshold of 1000 Cells/µL [ Time Frame: Time 0 (pre-dose, up to 15 minutes prior), 30, 60, and 90 minutes (each ± 5 minutes) and 2, 3, 4, 8, 12, 16, and 24 hours (each ± 15 minutes) post-dose at Baseline, Weeks 13, 26, 39, and 52 ]
- Randomized Period: Area Under the Curve for ALC (AUCALC) [ Time Frame: Time 0 (pre-dose, up to 15 minutes prior), 30, 60, and 90 minutes (each ± 5 minutes) and 2, 3, 4, 8, 12, 16, and 24 hours (each ± 15 minutes) post-dose at Baseline, Weeks 13, 26, 39, and 52 ]
- Randomized Period: Percentage of Lymphocyte Responders [ Time Frame: Baseline up to Week 52 ]
- Change From Baseline in Total ALC, Absolute Monocyte Count (AMC), ANC, and White Blood Cell (WBC) at Week 52 [ Time Frame: Baseline, Week 52 ]
- Randomized Period: Change From Baseline in Clinical Global Impression of Severity (CGI-S) Score at Week 52 [ Time Frame: Baseline, Week 52 ]
- Randomized Period: Infection Characteristics, The Number of Participants With Severe Infections [ Time Frame: Baseline up to Week 52 ]
- Randomized Period: Infection Duration [ Time Frame: Baseline up to Week 52 ]
- Randomized Period: Infection-Free Time [ Time Frame: Baseline up to Week 52 ]
- Randomized Period: Number of Days Lost From Work/School [ Time Frame: Baseline up to Week 52 ]
- Randomized Period: Quality of Life as Measured by 36-Item Short Form Survey Score [ Time Frame: Baseline up to Week 52 ]
- Randomized Period: Quality of Life as Measured by Pediatric Quality of Life Inventory (PedsQL) Score [ Time Frame: Baseline up to Week 52 ]
- Randomized Period: Quality of Life as Measured by EuroQoL-5 Dimension-5 Level (EQ-5D-5L) Score [ Time Frame: Baseline up to Week 52 ]
- Randomized Period: Quality of Life as Measured by Life Quality Index (LQI) Score [ Time Frame: Baseline up to Week 52 ]
- Randomized Period: Quality of Life as Measured by Dermatology LQI Score [ Time Frame: Baseline up to Week 52 ]
- Randomized Period: Pharmacokinetics (PK), Maximum Observed Plasma Concentration (Cmax) of Mavorixafor [ Time Frame: Time 0 (pre-dose, up to 15 minutes prior), 30, 60, and 90 minutes (each ± 5 minutes) and 2, 3, 4, 8, 12, 16, and 24 hours (each ± 15 minutes) post-dose at Weeks 13, 26, 39, and 52; and 4 hours post-dose at Baseline ]
- Randomized Period: PK, Time to Reach Cmax (Tmax) of Mavorixafor [ Time Frame: Time 0 (pre-dose, up to 15 minutes prior), 30, 60, and 90 min (each ± 5 minutes) and 2, 3, 4, 8, 12, 16, and 24 hours (each ± 15 minutes) post-dose at Weeks 13, 26, 39, and 52; and 4 hours post-dose at Baseline ]
- Randomized Period: PK, Half-Life of (T1/2) of Mavorixafor [ Time Frame: Time 0 (pre-dose, up to 15 minutes prior), 30, 60, and 90 minutes (each ± 5 minutes) and 2, 3, 4, 8, 12, 16, and 24 hours (each ± 15 minutes) post-dose at Weeks 13, 26, 39, and 52; and 4 hours post-dose at Baseline ]
- Randomized Period: PK, Area Under the Curve (AUC) of Mavorixafor [ Time Frame: Time 0 (pre-dose, up to 15 minutes prior), 30, 60, and 90 minutes (each ± 5 minutes) and 2, 3, 4, 8, 12, 16, and 24 hours (each ± 15 minutes) post-dose at Weeks 13, 26, 39, and 52; and 4 hours post-dose at Baseline ]
- Open-Label Period: Vaccine Titer Levels During the First Year of Open-Label Period, in Participants Vaccinated With Tdap During the Study Including Pertusis Toxin and Tetanus [ Time Frame: Year 1 of open-label period ]
- Open-Label Period: Vaccine Titer Levels During the First Year of the Open-Label Period for HPV 16 and HPV 18 in Participants Receiving Vaccinations With HPV 9-Valent Vaccine, Recombinant (Gardasil®9) During the Study [ Time Frame: Year 1 of open-label period ]
- Open-Label Period: Change From Baseline in Cutaneous Warts at Week 52, Based on Dermatologist CGI-C [ Time Frame: Baseline, Week 52 of open-label period ]
- Open-Label Period: Infection Rate (Percentage of Participants With Infections) Based on Infections Adjudicated by an AC [ Time Frame: Baseline up to Week 52 of open-label period ]

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Ages Eligible for Study: | 12 Years and older (Child, Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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.

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): NCT03995108
Contact: X4 Pharmaceuticals | 857-529-5779 | patientinfo@x4pharma.com |

Study Director: | Chief Medical Officer | X4 Pharmaceuticals |
Responsible Party: | X4 Pharmaceuticals |
ClinicalTrials.gov Identifier: | NCT03995108 |
Other Study ID Numbers: |
X4P-001-103 2019-001153-10 ( EudraCT Number ) 4WHIM ( Other Identifier: X4 Pharmaceuticals ) |
First Posted: | June 21, 2019 Key Record Dates |
Last Update Posted: | March 16, 2021 |
Last Verified: | March 2021 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Syndrome Disease Pathologic Processes |