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

July 28, 2025 updated by: X4 Pharmaceuticals

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Active, not recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
31

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
    • Queensland
      • Auchenflower, Queensland, Australia, 4006
        • Wesley Hospital
      • South Brisbane, Queensland, Australia, 4101
        • Children's Health Queensland Hospital
  • Austria
      • Wien, Austria, 1090
        • Medical University of Vienna - Medizinische Universität Wien
  • Denmark
      • Aarhus, Denmark, 8000
        • Aarhus University Hospital
  • France
      • Paris, France, 75743
        • Hôpital Necker-Enfants Malades
      • Paris Cedex 12, France, 75571
        • CHU Paris Est, Hôpital d'Enfants Armand-Trousseau
    • Rhne
      • Lyon, Rhne, France, 69008
        • CHU de Lyon, Institut d'Hematologie et d'Oncologie Pediatrique
  • Hungary
    • Hajdu-Bihar
      • Debrecen, Hajdu-Bihar, Hungary, H-4031
        • University of Debrecen, Affiliated Department of Infectology
  • Israel
      • Afula, Israel, 1834111
        • Haemek Medical Center
  • Italy
    • Piazza Del Mercato
      • Brescia, Piazza Del Mercato, Italy, 25123
        • Università degli Studi di Brescia, Scienze Cliniche e Sperimentali
  • Korea, Republic of
      • Seoul, Korea, Republic of, 03080
        • Seoul National University Hospital, Children's Hospital
  • Netherlands
      • Amsterdam, Netherlands, 1105 AZ
        • Emma Children's Hospital Academic Medical Center (AMC)
  • Russian Federation
      • Moscow, Russian Federation, 117997
        • Dmitry Rogachev National Research Center of Pediatric Hematology, Oncology and Immunology
      • Saint Pertersburg, Russian Federation, 197022
        • Academician I.P. Pavlov First Saint Petersburg State Medical University
  • Spain
    • Esplugues De Llobregat
      • Barcelona, Esplugues De Llobregat, Spain, 8950
        • Hospital Sant Joan de Deu Barcelona
    • Sevilla
      • Seville, Sevilla, Spain, 41013
        • Hospital Universitario Virgen del Rocio
  • Turkey
    • Adana
      • Sarıcam, Adana, Turkey, 1330
        • Cukurova University Faculty of Medicine
  • United States
    • California
      • San Diego, California, United States, 92123
        • University of California San Diego Health/Rady Children's Hospital
      • Thousand Oaks, California, United States, 91320
        • California Dermatology Institute
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • University of Iowa
    • Maryland
      • Baltimore, Maryland, United States, 21224
        • Johns Hopkins University Medical Center
    • Texas
      • Dallas, Texas, United States, 75390-9020
        • University of Texas Southwestern Medical Center
    • Washington
      • Seattle, Washington, United States, 98195
        • University of Washington Medical Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

12 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Double

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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 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.
Drug: Mavorixafor
Mavorixafor provided as 100 mg capsules.
Other Names:
  • AMD11070
  • X4P-001
Placebo Comparator: Placebo
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.
Drug: Mavorixafor
Mavorixafor provided as 100 mg capsules.
Other Names:
  • AMD11070
  • X4P-001
Drug: Placebo
Placebo matching to mavorixafor capsules

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
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
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])
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 Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
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
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
Baseline up to Week 52
Randomized Placebo-Controlled Period: Change From Baseline in Total Warts Score at Week 52
Time Frame: Baseline, Week 52
Baseline, Week 52
Randomized Placebo-Controlled Period: Total Infection Score for Mavorixafor
Time Frame: Baseline up to Week 52
Baseline up to Week 52
Randomized Placebo-Controlled Period: Time to Early Release
Time Frame: Baseline up to Week 52
Baseline up to Week 52
Randomized Placebo-Controlled Period: TAT-ALC of ≥ 1000 Cells/µL in Participants With Lymphopenia
Time Frame: Baseline
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
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
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
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.
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
Baseline
Randomized Placebo-Controlled Period: Participant Global Impression of Change (PGI-C)
Time Frame: Baseline up to Week 52
Baseline up to Week 52
Randomized Placebo-Controlled Period: Participant Global Impression of Severity (PGI-S)
Time Frame: Baseline up to Week 52
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
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
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
Baseline up to Week 52
Randomized Placebo-Controlled Period: Number of Participants with Infections
Time Frame: Baseline up to Week 52
Baseline up to Week 52
Randomized Placebo-Controlled Period: Infection-Free Time
Time Frame: Baseline up to Week 52
Baseline up to Week 52
Randomized Placebo-Controlled Period: Number of Days Lost From Work/School
Time Frame: Baseline up to Week 52
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
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
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
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
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
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
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
Baseline to Week 52
Randomized Placebo-Controlled Period: Number of Events Requiring Rescue Treatment Due to Infection
Time Frame: Baseline to Week 52
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
Baseline to Week 52
Randomized Placebo-Controlled Period: Number of Participants With Incidence of Newly Developed Warts
Time Frame: Baseline to Week 52
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
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
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
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
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
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
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
Baseline, Week 52
Randomized Placebo-Controlled Period: Number of Participants With AEs
Time Frame: Baseline up to Week 52
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
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
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
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
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
Time Frame: Baseline up to Week 52 of open-label period
Baseline up to Week 52 of open-label period
Open-Label Period: Percentage of Lymphocyte Responders
Time Frame: Baseline up to Week 52 of open-label period
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
Time Frame: Baseline up to Week 52 of open-label period
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
Time Frame: Year 1 of open-label period
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
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
Time Frame: Baseline, Week 52 of open-label period
Baseline, Week 52 of open-label period
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
Baseline, Week 52 of open-label period
Open-Label Period: Change From Baseline in Cutaneous Warts, Based on Local Dermatologist CGI-C
Time Frame: Baseline, Week 52 of open-label period
Baseline, Week 52 of open-label period
Open-Label Period: Change From Baseline in Cutaneous Warts, Based on Local Dermatologist CGI-S
Time Frame: Baseline, Week 52 of open-label period
Baseline, Week 52 of open-label period
Open-Label Period: Change Over Time in PGI-C
Time Frame: Baseline up to Week 52 of open-label period
Baseline up to Week 52 of open-label period
Open-Label Period: Change Over Time in PGI-S
Time Frame: Baseline up to Week 52 of open-label period
Baseline up to Week 52 of open-label period
Open-Label Period: Total Infection Score (Percentage of Participants With Infections)
Time Frame: Baseline up to Week 52 of open-label period
Baseline up to Week 52 of open-label period

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
X4 Pharmaceuticals

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Chief Medical Officer, X4 Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
October 24, 2019

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
December 1, 2025

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 1, 2025

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
June 19, 2019

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
June 19, 2019

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
June 21, 2019

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
July 29, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
July 28, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
July 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • X4P-001-103
  • 2019-001153-10 (EudraCT Number)
  • 4WHIM (Other Identifier: X4 Pharmaceuticals)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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