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OpenTrials
Completed

NCT Number: NCT03995108

Efficacy and Safety Study of Mavorixafor in Participants With Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) Syndrome

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.

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Key information

Conditions

WHIM Syndrome

Age range

12 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Wesley Hospital, Auchenflower, Queensland, Australia

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

for the Randomized Placebo-Controlled Period :

  • 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.

Treatment and study plan

Mavorixafor

Drug

Mavorixafor provided as 100 mg capsules.

Other names: AMD11070, X4P-001

Placebo

Drug

Placebo matching to mavorixafor capsules

Primary outcomes

  1. 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

  2. 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])

Secondary outcomes

  1. 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

  2. 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

  3. Randomized Placebo-Controlled Period: Change From Baseline in Total Warts Score at Week 52

    Time frame: Baseline, Week 52

  4. Randomized Placebo-Controlled Period: Total Infection Score for Mavorixafor

    Time frame: Baseline up to Week 52

  5. Randomized Placebo-Controlled Period: Time to Early Release

    Time frame: Baseline up to Week 52

  6. Randomized Placebo-Controlled Period: TAT-ALC of ≥ 1000 Cells/µL in Participants With Lymphopenia

    Time frame: Baseline

  7. 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

  8. Randomized Placebo-Controlled Period: Change in Total Wart Score at Baseline, Based on Clinical Global Impression of Change (CGI-C)

    Time frame: Baseline

  9. 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

    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.

  10. Randomized Placebo-Controlled Period: Change in Total Wart Score at Baseline (CGI-C), Based on Local Dermatologist Review

    Time frame: Baseline

  11. Randomized Placebo-Controlled Period: Participant Global Impression of Change (PGI-C)

    Time frame: Baseline up to Week 52

  12. Randomized Placebo-Controlled Period: Participant Global Impression of Severity (PGI-S)

    Time frame: Baseline up to Week 52

  13. 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

  14. 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

  15. 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

  16. Randomized Placebo-Controlled Period: Number of Participants with Infections

    Time frame: Baseline up to Week 52

  17. Randomized Placebo-Controlled Period: Infection-Free Time

    Time frame: Baseline up to Week 52

  18. Randomized Placebo-Controlled Period: Number of Days Lost From Work/School

    Time frame: Baseline up to Week 52

  19. Randomized Placebo-Controlled Period: Quality of Life as Measured by 36-Item Short Form Survey Score

    Time frame: Baseline up to Week 52

  20. 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

  21. Randomized Placebo-Controlled Period: Quality of Life as Measured by Life Quality Index (LQI) Score

    Time frame: Baseline up to Week 52

  22. Randomized Placebo-Controlled Period: Quality of Life as Measured by Dermatology LQI Score

    Time frame: Baseline up to Week 52

  23. Randomized Placebo-Controlled Period: Quality of Life as Measured by Pediatric Quality of Life Inventory (PedsQL) Score

    Time frame: Baseline up to Week 52

  24. Randomized Placebo-Controlled Period: Change From Baseline in Anogenital (AG) Warts Based on Dermatologist CGI-C Assessment

    Time frame: Baseline to Week 52

  25. Randomized Placebo-Controlled Period: Change From Baseline in Anogenital (AG) Warts Based on AG Wart Severity Assessment

    Time frame: Baseline to Week 52

  26. Randomized Placebo-Controlled Period: Number of Events Requiring Rescue Treatment Due to Infection

    Time frame: Baseline to Week 52

  27. Randomized Placebo-Controlled Period: Number of Participants With Incidence and Duration of Hospitalizations Due to Infection

    Time frame: Baseline to Week 52

  28. Randomized Placebo-Controlled Period: Number of Participants With Incidence of Newly Developed Warts

    Time frame: Baseline to Week 52

  29. 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

  30. Randomized Placebo-Controlled Period: Percentage of Neutrophil Responders

    Time frame: Baseline up to Week 52

  31. 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

  32. 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

  33. Randomized Placebo-Controlled Period: Percentage of Lymphocyte Responders

    Time frame: Baseline up to Week 52

  34. 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

  35. Absolute and Fold Change From Baseline in Absolute T, B and Natural Killer Lymphocyte at Week 52

    Time frame: Baseline, Week 52

  36. Randomized Placebo-Controlled Period: Number of Participants With AEs

    Time frame: Baseline up to Week 52

  37. 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

  38. 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

  39. 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

  40. 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

  41. Open-Label Period: Percentage of Neutrophil Responders

    Time frame: Baseline up to Week 52 of open-label period

  42. Open-Label Period: Percentage of Lymphocyte Responders

    Time frame: Baseline up to Week 52 of open-label period

  43. Open-Label Period: Absolute and Fold Change From Baseline in Total ALC, AMC, ANC, and WBC at Week 52

    Time frame: Baseline up to Week 52 of open-label period

  44. 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

  45. 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

  46. Open-Label Period: Change From Baseline in Cutaneous Warts at Week 52, Based on Central Review of CGI-C

    Time frame: Baseline, Week 52 of open-label period

  47. Open-Label Period: Change From Baseline in Cutaneous Warts, Based on Central Review of CGI-S

    Time frame: Baseline, Week 52 of open-label period

  48. Open-Label Period: Change From Baseline in Cutaneous Warts, Based on Local Dermatologist CGI-C

    Time frame: Baseline, Week 52 of open-label period

  49. Open-Label Period: Change From Baseline in Cutaneous Warts, Based on Local Dermatologist CGI-S

    Time frame: Baseline, Week 52 of open-label period

  50. Open-Label Period: Change Over Time in PGI-C

    Time frame: Baseline up to Week 52 of open-label period

  51. Open-Label Period: Change Over Time in PGI-S

    Time frame: Baseline up to Week 52 of open-label period

  52. Open-Label Period: Total Infection Score (Percentage of Participants With Infections)

    Time frame: Baseline up to Week 52 of open-label period

Sponsors and collaborators

Lead sponsor

X4 Pharmaceuticals

Industry

Registry information

Official study title

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Mavorixafor in Patients With WHIM Syndrome With Open-Label Extension

Important dates

Study start
2019
Primary completion
2025
Study completion
2025
First posted
Jun 21, 2019
Registry last updated
Jul 6, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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