WHIM SyndromePhase 3NCT03995108
Efficacy and Safety Study of Mavorixafor in Participants With Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) Syndrome
A Phase 3 study testing Mavorixafor in people with WHIM Syndrome, run by X4 Pharmaceuticals at 23 sites in 13 countries.
Trial summary
This study has a double-blind, Randomized Placebo-Controlled Period and an Open-Label Period. The primary objective of the Randomized Placebo-Controlled Period is to demonstrate the efficacy of mavorixafor in participants with WHIM syndrome as assessed by increasing levels of circulating neutrophils compared with… Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- WHIM Syndrome
- What is being tested?
- Mavorixafor
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 31 participants
- Where
- 23 sitesin 13 countries
- Study dates
- 24 Oct 2019to 23 Dec 2025
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Completed the Randomized Period; or
- Granted Early Release from the Randomized Period.
Who cannot join
- 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.
Trial locations and contacts
23 sites in 13 countries.
23 sites in 13 countries
United States6 sites
Johns Hopkins University Medical Center
Baltimore, Maryland, 21224
University of Texas Southwestern Medical Center
Dallas, Texas, 75390-9020
University of Iowa
Iowa City, Iowa, 52242
University of California San Diego Health/Rady Children's Hospital
San Diego, California, 92123
University of Washington Medical Center
Seattle, Washington, 98195
California Dermatology Institute
Thousand Oaks, California, 91320
France3 sites
CHU de Lyon, Institut d'Hematologie et d'Oncologie Pediatrique
Lyon, Rhne, 69008
CHU Paris Est, Hôpital d'Enfants Armand-Trousseau
Paris, 75571
Hopital Necker-Enfants Malades
Paris, 75743
Australia2 sites
Wesley Hospital
Auchenflower, Queensland, 4006
Children's Health Queensland Hospital
South Brisbane, Queensland, 4101
Russia2 sites
Dmitry Rogachev National Research Center of Pediatric Hematology, Oncology and Immunology
Moscow, 117997
Academician I.P. Pavlov First Saint Petersburg State Medical University
Saint Pertersburg, 197022
Spain2 sites
Hospital Sant Joan de Deu Barcelona
Barcelona, Esplugues de Llobregat, 8950
Hospital Universitario Virgen del Rocío
Seville, Sevilla, 41013
Austria1 site
Medical University of Vienna - Medizinische Universität Wien
Vienna, 1090
Hungary1 site
University of Debrecen, Affiliated Department of Infectology
Debrecen, Hajdú-Bihar, H-4031
Italy1 site
Università degli Studi di Brescia, Scienze Cliniche e Sperimentali
Brescia, Piazza Del Mercato, 25123
Netherlands1 site
Emma Children's Hospital Academic Medical Center (AMC)
Amsterdam, 1105 AZ
South Korea1 site
Seoul National University Hospital, Children's Hospital
Seoul, 03080
Turkey (Türkiye)1 site
Cukurova University Faculty of Medicine
Sarıçam, Adana, 1330
About this study
Official title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Mavorixafor in Patients With WHIM Syndrome With Open-Label Extension
This study has a double-blind, Randomized Placebo-Controlled Period and an Open-Label Period. The primary objective of the Randomized Placebo-Controlled 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 Period, if regionally applicable, until mavorixafor becomes commercially available, or until the study is terminated by the Sponsor.
- Conditions
- WHIM Syndrome
- Being tested
- Mavorixafor, Placebo
- Medical subject headings
- WHIM syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MavorixaforDrug
Also called AMD11070, X4P-001
Mavorixafor provided as 100 mg capsules.
PlaceboDrug
Placebo matching to mavorixafor capsules
| Study group | Role | What they receive |
|---|---|---|
| Mavorixafor | Experimental | 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 Placebo-Controlled Period. Adolescents weighing ≤50 kg will receive mavorixafor 200 mg QD. Participants who complete the Randomized Placebo-Controlled 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. Receives: Drug: Mavorixafor |
| Placebo | Placebo comparator | Participants will receive placebo matching to mavorixafor QD orally for 52 weeks in the Randomized Placebo-Controlled Period. Participants who complete the Randomized Placebo-Controlled 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. Receives: Drug: Mavorixafor; Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Randomized Placebo-Controlled Period: Time (in Hours) Above Threshold-Absolute Neutrophil Count (TAT-ANC in hours) of ≥ 500 Cells/Microliter (µL) over a 24-hour period
Measured: 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)
Measured: 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])
Secondary outcomes (52)
Randomized Placebo-Controlled Period: Time (in Hours) Above Threshold-Absolute Lymphocyte Count (TAT-ALC) of ≥ 1000 Cells/µL over a 24-hour period
Measured: 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 Placebo-Controlled Period: Composite Clinical Efficacy for Mavorixafor based on total infection score and total wart change score
Measured: Baseline up to Week 52
Randomized Placebo-Controlled Period: Change From Baseline in Total Warts Score at Week 52
Measured: Baseline, Week 52
Randomized Placebo-Controlled Period: Total Infection Score for Mavorixafor
Measured: Baseline up to Week 52
Randomized Placebo-Controlled Period: Time to Early Release
Measured: Baseline up to Week 52
Randomized Placebo-Controlled Period: TAT-ALC of ≥ 1000 Cells/µL in Participants With Lymphopenia
Measured: Baseline
Randomized Placebo-Controlled Period: Total Infection Score for Participants With Non-Immunoglobulin (non-Ig) Use (Percentage of Participants With Infections)
Measured: Baseline up to Week 52
Randomized Placebo-Controlled Period: Change in Total Wart Score at Baseline, Based on Clinical Global Impression of Change (CGI-C)
Measured: Baseline
Randomized Placebo-Controlled Period: Composite Clinical Efficacy (Total Infection Score and Total Wart Change Score) for Participants With Non-Ig Use
Composite clinical efficacy will be calculated using the total infection score and total wart change score for participants with warts at baseline or non-Ig use. It will be analyzed by a blinded, independent AC.
Measured: Baseline up to Week 52
Randomized Placebo-Controlled Period: Change in Total Wart Score at Baseline (CGI-C), Based on Local Dermatologist Review
Measured: Baseline
Randomized Placebo-Controlled Period: Participant Global Impression of Change (PGI-C)
Measured: Baseline up to Week 52
Randomized Placebo-Controlled Period: Participant Global Impression of Severity (PGI-S)
Measured: Baseline up to Week 52
Randomized Placebo-Controlled Period: Vaccine Titer Levels at Week 52 in Participants Vaccinated at Week 13, With Tetanus, Diphtheria, and Pertussis (Tdap) Including Pertussis Toxin, and Tetanus
Measured: Week 52
Randomized Placebo-Controlled 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)
Measured: Week 52
Randomized Placebo-Controlled Period: Change From Baseline in Clinical Global Impression of Severity (CGI-S), Based on Local Dermatologist Review
Measured: Baseline up to Week 52
Randomized Placebo-Controlled Period: Number of Participants with Infections
Measured: Baseline up to Week 52
Randomized Placebo-Controlled Period: Infection-Free Time
Measured: Baseline up to Week 52
Randomized Placebo-Controlled Period: Number of Days Lost From Work/School
Measured: Baseline up to Week 52
Randomized Placebo-Controlled Period: Quality of Life as Measured by 36-Item Short Form Survey Score
Measured: Baseline up to Week 52
Randomized Placebo-Controlled Period: Quality of Life as Measured by EuroQoL-5 Dimension-5 Level (EQ-5D-5L) Score
Measured: Baseline up to Week 52
Randomized Placebo-Controlled Period: Quality of Life as Measured by Life Quality Index (LQI) Score
Measured: Baseline up to Week 52
Randomized Placebo-Controlled Period: Quality of Life as Measured by Dermatology LQI Score
Measured: Baseline up to Week 52
Randomized Placebo-Controlled Period: Quality of Life as Measured by Pediatric Quality of Life Inventory (PedsQL) Score
Measured: Baseline up to Week 52
Randomized Placebo-Controlled Period: Change From Baseline in Anogenital (AG) Warts Based on Dermatologist CGI-C Assessment
Measured: Baseline to Week 52
Randomized Placebo-Controlled Period: Change From Baseline in Anogenital (AG) Warts Based on AG Wart Severity Assessment
Measured: Baseline to Week 52
Randomized Placebo-Controlled Period: Number of Events Requiring Rescue Treatment Due to Infection
Measured: Baseline to Week 52
Randomized Placebo-Controlled Period: Number of Participants With Incidence and Duration of Hospitalizations Due to Infection
Measured: Baseline to Week 52
Randomized Placebo-Controlled Period: Number of Participants With Incidence of Newly Developed Warts
Measured: Baseline to Week 52
Randomized Placebo-Controlled Period: Area Under the Curve for ANC (AUCANC) Using Trapezoidal Method
Measured: 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 Placebo-Controlled Period: Percentage of Neutrophil Responders
Measured: Baseline up to Week 52
Randomized Placebo-Controlled Period: Mavorixafor Treatment Group: AUCANC
Measured: 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 Placebo-Controlled Period: Area Under the Curve for ALC (AUCALC)
Measured: 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 Placebo-Controlled Period: Percentage of Lymphocyte Responders
Measured: Baseline up to Week 52
Randomized Placebo-Controlled Period: Change From Baseline in Total ALC, Absolute Monocyte Count (AMC), ANC, and White Blood Cell (WBC) at Week 52
Measured: Baseline, Week 52
Absolute and Fold Change From Baseline in Absolute T, B and Natural Killer Lymphocyte at Week 52
Measured: Baseline, Week 52
Randomized Placebo-Controlled Period: Number of Participants With AEs
Measured: Baseline up to Week 52
Randomized Placebo-Controlled Period: Pharmacokinetics (PK), Maximum Observed Plasma Concentration (Cmax) of Mavorixafor
Measured: 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 Placebo-Controlled Period: PK, Time to Reach Cmax (Tmax) of Mavorixafor
Measured: 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 Placebo-Controlled Period: PK, Half-Life of (T1/2) of Mavorixafor
Measured: 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 Placebo-Controlled Period: PK, Area Under the Curve (AUC) of Mavorixafor
Measured: 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: Percentage of Neutrophil Responders
Measured: Baseline up to Week 52 of open-label period
Open-Label Period: Percentage of Lymphocyte Responders
Measured: Baseline up to Week 52 of open-label period
Open-Label Period: Absolute and Fold Change From Baseline in Total ALC, AMC, ANC, and WBC at Week 52
Measured: Baseline up to Week 52 of open-label period
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
Measured: 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
Measured: Year 1 of open-label period
Open-Label Period: Change From Baseline in Cutaneous Warts at Week 52, Based on Central Review of CGI-C
Measured: Baseline, Week 52 of open-label period
Open-Label Period: Change From Baseline in Cutaneous Warts, Based on Central Review of CGI-S
Measured: Baseline, Week 52 of open-label period
Open-Label Period: Change From Baseline in Cutaneous Warts, Based on Local Dermatologist CGI-C
Measured: Baseline, Week 52 of open-label period
Open-Label Period: Change From Baseline in Cutaneous Warts, Based on Local Dermatologist CGI-S
Measured: Baseline, Week 52 of open-label period
Open-Label Period: Change Over Time in PGI-C
Measured: Baseline up to Week 52 of open-label period
Open-Label Period: Change Over Time in PGI-S
Measured: Baseline up to Week 52 of open-label period
Open-Label Period: Total Infection Score (Percentage of Participants With Infections)
Measured: Baseline up to Week 52 of open-label period
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Jun 2019
- Study start24 Oct 2019
- Primary completion23 Dec 2025
- Study completion23 Dec 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for WHIM SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 31 participants (actual)
Sponsor and investigators
- Lead sponsor
- X4 Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Chief Medical Officer, Study director, X4 Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03995108
- Sponsor's protocol ID
- X4P-001-103
- Other IDs
- 2019-001153-10, 4WHIM, 2019
- Status last verified
- Jul 2026
- First submitted
- 19 Jun 2019
- Last updated
- 6 Jul 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Badolato R, Alsina L, Azar A, Bertrand Y, Bolyard AA, Dale D, Deya-Martinez A, Dickerson KE, Ezra N, Hasle H, Kang HJ, Kiani-Alikhan S, Kuijpers TW, Kulagin A, Langguth D, Levin C, Neth O, Olbrich P, Peake J, Rodina Y, Rutten CE, Shcherbina A, Tarrant TK, Vossen MG, Wysocki CA, Belschner A, Bridger GJ, Chen K, Dubuc S, Hu Y, Jiang H, Li S, MacLeod R, Stewart M, Taveras AG, Yan T, Donadieu J. A phase 3 randomized trial of mavorixafor, a CXCR4 antagonist, for WHIM syndrome. Blood. 2024 Jul 4;144(1):35-45. doi: 10.1182/blood.2023022658.
PubMed 38643510
Study at a glance
From the registry record, last updated by the sponsor on 6 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Mavorixafor in Patients With WHIM Syndrome With Open-Label Extension
- ClinicalTrials.gov ID
- NCT03995108
- Acronym / sponsor ID
- X4P-001-103
- Conditions
- WHIM Syndrome
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 31 participants (actual)
- Lead sponsor
- X4 PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 24 Oct 2019 (actual)
- Primary completion
- 23 Dec 2025 (actual)
- Study completion
- 23 Dec 2025 (actual)
- First posted
- 21 Jun 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study has a double-blind, Randomized Placebo-Controlled Period and an Open-Label Period. The primary objective of the Randomized Placebo-Controlled 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 Period, if regionally applicable, until mavorixafor becomes commercially available, or until the study is terminated by the Sponsor.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Mavorixafor | Experimental | Drug: MavorixaforParticipants (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 Placebo-Controlled Period. Adolescents weighing ≤50 kg will receive mavorixafor 200 mg QD. Participants who complete the Randomized Placebo-Controlled 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. |
| Placebo | Placebo comparator | Drug: Mavorixafor, Drug: PlaceboParticipants will receive placebo matching to mavorixafor QD orally for 52 weeks in the Randomized Placebo-Controlled Period. Participants who complete the Randomized Placebo-Controlled 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. |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- Completed the Randomized Period; or
- Granted Early Release from the Randomized Period.
Key exclusion criteria (3 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MavorixaforAlso: AMD11070, X4P-001Mavorixafor provided as 100 mg capsules. | Drug | Mavorixafor, Placebo |
| PlaceboPlacebo matching to mavorixafor capsules | Drug | Placebo |
Primary outcome
Randomized Placebo-Controlled Period: Time (in Hours) Above Threshold-Absolute Neutrophil Count (TAT-ANC in hours) of ≥ 500 Cells/Microliter (µL) over a 24-hour period
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])
52 secondary outcomes
Randomized Placebo-Controlled Period: Time (in Hours) Above Threshold-Absolute Lymphocyte Count (TAT-ALC) of ≥ 1000 Cells/µL over a 24-hour period
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 Placebo-Controlled Period: Composite Clinical Efficacy for Mavorixafor based on total infection score and total wart change score
Time frame: Baseline up to Week 52
Randomized Placebo-Controlled Period: Change From Baseline in Total Warts Score at Week 52
Time frame: Baseline, Week 52
Randomized Placebo-Controlled Period: Total Infection Score for Mavorixafor
Time frame: Baseline up to Week 52
Randomized Placebo-Controlled Period: Time to Early Release
Time frame: Baseline up to Week 52
Randomized Placebo-Controlled Period: TAT-ALC of ≥ 1000 Cells/µL in Participants With Lymphopenia
Time frame: Baseline
Randomized Placebo-Controlled Period: Total Infection Score for Participants With Non-Immunoglobulin (non-Ig) Use (Percentage of Participants With Infections)
Time frame: Baseline up to Week 52
Randomized Placebo-Controlled Period: Change in Total Wart Score at Baseline, Based on Clinical Global Impression of Change (CGI-C)
Time frame: Baseline
Randomized Placebo-Controlled Period: Composite Clinical Efficacy (Total Infection Score and Total Wart Change Score) for Participants With Non-Ig Use
Time frame: Baseline up to Week 52
Randomized Placebo-Controlled Period: Change in Total Wart Score at Baseline (CGI-C), Based on Local Dermatologist Review
Time frame: Baseline
Randomized Placebo-Controlled Period: Participant Global Impression of Change (PGI-C)
Time frame: Baseline up to Week 52
Randomized Placebo-Controlled Period: Participant Global Impression of Severity (PGI-S)
Time frame: Baseline up to Week 52
Randomized Placebo-Controlled 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 Placebo-Controlled 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 Placebo-Controlled Period: Change From Baseline in Clinical Global Impression of Severity (CGI-S), Based on Local Dermatologist Review
Time frame: Baseline up to Week 52
Randomized Placebo-Controlled Period: Number of Participants with Infections
Time frame: Baseline up to Week 52
Randomized Placebo-Controlled Period: Infection-Free Time
Time frame: Baseline up to Week 52
Randomized Placebo-Controlled Period: Number of Days Lost From Work/School
Time frame: Baseline up to Week 52
Randomized Placebo-Controlled Period: Quality of Life as Measured by 36-Item Short Form Survey Score
Time frame: Baseline up to Week 52
Randomized Placebo-Controlled Period: Quality of Life as Measured by EuroQoL-5 Dimension-5 Level (EQ-5D-5L) Score
Time frame: Baseline up to Week 52
Randomized Placebo-Controlled Period: Quality of Life as Measured by Life Quality Index (LQI) Score
Time frame: Baseline up to Week 52
Randomized Placebo-Controlled Period: Quality of Life as Measured by Dermatology LQI Score
Time frame: Baseline up to Week 52
Randomized Placebo-Controlled Period: Quality of Life as Measured by Pediatric Quality of Life Inventory (PedsQL) Score
Time frame: Baseline up to Week 52
Randomized Placebo-Controlled Period: Change From Baseline in Anogenital (AG) Warts Based on Dermatologist CGI-C Assessment
Time frame: Baseline to Week 52
Randomized Placebo-Controlled Period: Change From Baseline in Anogenital (AG) Warts Based on AG Wart Severity Assessment
Time frame: Baseline to Week 52
Randomized Placebo-Controlled Period: Number of Events Requiring Rescue Treatment Due to Infection
Time frame: Baseline to Week 52
Randomized Placebo-Controlled Period: Number of Participants With Incidence and Duration of Hospitalizations Due to Infection
Time frame: Baseline to Week 52
Randomized Placebo-Controlled Period: Number of Participants With Incidence of Newly Developed Warts
Time frame: Baseline to Week 52
Randomized Placebo-Controlled 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 Placebo-Controlled Period: Percentage of Neutrophil Responders
Time frame: Baseline up to Week 52
Randomized Placebo-Controlled 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 Placebo-Controlled 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 Placebo-Controlled Period: Percentage of Lymphocyte Responders
Time frame: Baseline up to Week 52
Randomized Placebo-Controlled Period: Change From Baseline in Total ALC, Absolute Monocyte Count (AMC), ANC, and White Blood Cell (WBC) at Week 52
Time frame: Baseline, Week 52
Absolute and Fold Change From Baseline in Absolute T, B and Natural Killer Lymphocyte at Week 52
Time frame: Baseline, Week 52
Randomized Placebo-Controlled Period: Number of Participants With AEs
Time frame: Baseline up to Week 52
Randomized Placebo-Controlled 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 Placebo-Controlled 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 Placebo-Controlled 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 Placebo-Controlled 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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Jul 2026.
- Derived from this study: PubMed 38643510Badolato R, Alsina L, Azar A, Bertrand Y, Bolyard AA, Dale D, Deya-Martinez A, Dickerson KE, Ezra N, Hasle H, Kang HJ, Kiani-Alikhan S, Kuijpers TW, Kulagin A, Langguth D, Levin C, Neth O, Olbrich P, Peake J, Rodina Y, Rutten CE, Shcherbina A, Tarrant TK, Vossen MG, Wysocki CA, Belschner A, Bridger GJ, Chen K, Dubuc S, Hu Y, Jiang H, Li S, MacLeod R, Stewart M, Taveras AG, Yan T, Donadieu J. A phase 3 randomized trial of mavorixafor, a CXCR4 antagonist, for WHIM syndrome. Blood. 2024 Jul 4;144(1):35-45. doi: 10.1182/blood.2023022658.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Chief Medical Officer study director
X4 Pharmaceuticals
23 sites in 13 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Mavorixafor in people with WHIM Syndrome, run by X4 Pharmaceuticals at 23 sites in 13 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 6 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 23 sites in United States, France, Australia, Russia, Spain and 8 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Randomized Placebo-Controlled Period: Time (in Hours) Above Threshold-Absolute Neutrophil Count (TAT-ANC in hours) of ≥ 500 Cells/Microliter (µL) over a 24-hour period (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). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03995108, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Jul 2026.