A Phase 2 interventional study of X4P-001 in WHIM Syndrome, sponsored by X4 Pharmaceuticals. Completed at 2 sites in 2 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2024-10-30.
Sponsored by X4 Pharmaceuticals · Phase 2, Interventional, and Treatment
This is a Phase 2 study with an initial 24-week Treatment Period and an Extension Phase. The primary objectives of this Phase 2 study are to determine the safety, tolerability, and dose selection of mavorixafor in participants with WHIM syndrome. Participants may continue treatment in an Extension Phase, if regionally applicable, until mavorixafor becomes available via an alternative mechanism (for example, drug is commercially available, an expanded access program, etc.) or until the study is terminated by the Sponsor for any reason.
X4 Pharmaceuticals is the lead sponsor of 13 studies on the registry; 2 are open to participants now.
Of its 10 completed or terminated interventional studies of FDA-regulated products, 3 (30%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Participants with a clinical diagnosis of WHIM syndrome must meet all of the following criteria to be eligible for study participation:
Exclusion Criteria:
Participants with any of the following will be excluded from participation in the study:
Has, at Screening, laboratory tests meeting one or more of the following criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Initial Treatment Phase: Participants will initiate treatment with mavorixafor at 50 milligrams (mg) once daily (QD) orally or a higher dose, with potential escalation based on area under the curve for absolute neutrophil count and absolute leukocyte count (AUCANC/ALC) values to a maximum total daily dose of 400 mg. Participants are expected to receive treatment for 24 weeks in the initial Treatment Period or until development of a treatment-limiting toxicity (TLT). Extension Phase: All participants will receive mavorixafor; the dose will not exceed 400 mg. In the Extension Phase, treatment may continue until mavorixafor becomes available via an alternative mechanism (for example, drug is commercially available, an expanded access program, etc.) or until the study is terminated by the sponsor.
Drug: X4P-001
Mavorixafor will be provided as either 25 mg or 100 mg capsules.
Also known as: AMD11070
Mean Value of the Area Under the Plasma Concentration-time Curve for Absolute Neutrophil Count (AUCANC)
AUCANC was collected over a 24-hour period above clinically meaningful thresholds for the mavorixafor-treated participants over 6 months. The absolute neutrophil count (ANC) clinically meaningful threshold was defined as ANC ≥ 600/microliter (μL).
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
All Visits: Average Per-Participant Value of the AUCANC
AUCANC was collected over a 24-hour period above clinically meaningful thresholds for the mavorixafor-treated participants over 6 months. The ANC clinically meaningful threshold was defined as ANC ≥ 600/μL. Data for this outcome measure are reported as an "All Visits" summary based on the mean of AUCs that is, the per-participant average of the AUCANC across the 3 visits where participant was treated with at least 300/400 mg dose. Time frame reported is based on data collection time points.
Time frame: Time 0 (-15 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
Mean Value of the Area Under the Plasma Concentration-time Curve for Absolute Lymphocyte Count (AUCALC)
AUCALC was collected over a 24-hour period above clinically meaningful thresholds for the mavorixafor-treated participants over 6 months. The absolute lymphocyte count (ALC) clinically meaningful threshold was defined as ALC ≥ 1000/μL.
Time frame: Time 0 (-15 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
All Visits: Average Per-Participant Value of the AUCALC
AUCALC was collected over a 24-hour period above clinically meaningful thresholds for the mavorixafor-treated participants over 6 months. The ALC clinically meaningful threshold was defined as ALC ≥ 1000/μL. Data for this outcome measure are reported as an "All Visits" summary based on the mean of AUCs that is, the per-participant average of the AUCALC across the 3 visits where participant was treated with at least 300/400 mg dose. Time frame reported is based on data collection time points.
Time frame: Time 0 (-15 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
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
An adverse event (AE) was defined as any untoward medical occurrence that developed or worsened in severity during the conduct of a clinical study and did not necessarily have a causal relationship to the study drug. SAEs included death, a life-threatening adverse event, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, a congenital anomaly or birth defect, or an important medical event that jeopardized the participant and required medical intervention to prevent 1 of the outcomes listed in this definition. A TEAE was defined as any AE that began or worsened in severity or frequency on or after the start of study drug through 10 days after the last dose of the study drug. A summary of serious and non-serious AEs regardless of causality is located in 'Reported Adverse Events module'.
Time frame: From first dose of study drug through 10 days after the last dose of the study drug (Maximum exposure: 1712 days)
| Milestone | X4P-001 50 to up to 400 mg/kg | X4P-001 100 to up to 400 mg/kg | X4P-001 200 to up to 400 mg/kg | X4P-001 300 to up to 400 mg/kg |
|---|---|---|---|---|
| Started | 2 | 2 | 2 | 2 |
| Received at least 1 dose of study drug | 2 | 2 | 2 | 2 |
| Completed | 2 | 1 | 1 | 2 |
| Not completed | 0 | 1 | 1 | 0 |
| Withdrew: Adverse event | 0 | 0 | 1 | 0 |
| Withdrew: Withdrawal by subject | 0 | 1 | 0 | 0 |
| Milestone | X4P-001 50 to up to 400 mg/kg | X4P-001 100 to up to 400 mg/kg | X4P-001 200 to up to 400 mg/kg | X4P-001 300 to up to 400 mg/kg |
|---|---|---|---|---|
| Started | 2 | 0 | 1 | 2 |
| Completed | 2 | 0 | 1 | 2 |
| Not completed | 0 | 0 | 0 | 0 |
AUCANC was collected over a 24-hour period above clinically meaningful thresholds for the mavorixafor-treated participants over 6 months. The absolute neutrophil count (ANC) clinically meaningful threshold was defined as ANC ≥ 600/microliter (μL).
| cells*hour/μL | X4P-001 50 mg | X4P-001 100 mg | X4P-001 150 mg | X4P-001 200 mg | X4P-001 300 mg | X4P-001 400 mg | X4P-001 300/400 mg |
|---|---|---|---|---|---|---|---|
| Week 5 | -12574.63 ± 156.094 | -6206.75 ± 4238.280 | — | -6949.58 | 24175.00 ± 777.817 | — | 24175.00 ± 777.817 |
| Week 13 | — | -11291.96 ± 1509.496 | — | -7575.21 ± 2701.443 | -2602.50 | — | -2602.50 |
| Week 21 | — | — | -8723.00 ± 2695.962 | — | 58118.02 ± 112851.505 | — | 58118.02 ± 112851.505 |
AUCANC was collected over a 24-hour period above clinically meaningful thresholds for the mavorixafor-treated participants over 6 months. The ANC clinically meaningful threshold was defined as ANC ≥ 600/μL. Data for this outcome measure are reported as an "All Visits" summary based on the mean of AUCs that is, the per-participant average of the AUCANC across the 3 visits where participant was treated with at least 300/400 mg dose. Time frame reported is based on data collection time points.
| cells*hour/μL | X4P-001 300/400 mg |
|---|---|
| All Visits: Average Per-Participant Value of the AUCANC | 27477.00 ± 55792.377 |
AUCALC was collected over a 24-hour period above clinically meaningful thresholds for the mavorixafor-treated participants over 6 months. The absolute lymphocyte count (ALC) clinically meaningful threshold was defined as ALC ≥ 1000/μL.
| cells*hour/μL | X4P-001 50 mg | X4P-001 100 mg | X4P-001 150 mg | X4P-001 200 mg | X4P-001 300 mg | X4P-001 400 mg | X4P-001 300/400 mg |
|---|---|---|---|---|---|---|---|
| Week 5 | -5839.54 ± 6485.407 | 6978.25 ± 7435.935 | — | 4783.33 | 21837.50 ± 24236.085 | — | 21837.50 ± 24236.085 |
| Week 13 | — | -1006.50 ± 10546.262 | — | 7023.83 ± 1603.482 | 2862.50 | — | 2862.50 |
| Week 21 | — | — | -2216.17 ± 8285.642 | — | 15331.35 ± 21250.202 | — | 15331.35 ± 21250.202 |
AUCALC was collected over a 24-hour period above clinically meaningful thresholds for the mavorixafor-treated participants over 6 months. The ALC clinically meaningful threshold was defined as ALC ≥ 1000/μL. Data for this outcome measure are reported as an "All Visits" summary based on the mean of AUCs that is, the per-participant average of the AUCALC across the 3 visits where participant was treated with at least 300/400 mg dose. Time frame reported is based on data collection time points.
| cells*hour/μL | X4P-001 300/400 mg |
|---|---|
| All Visits: Average Per-Participant Value of the AUCALC | 14232.17 ± 16789.700 |
An adverse event (AE) was defined as any untoward medical occurrence that developed or worsened in severity during the conduct of a clinical study and did not necessarily have a causal relationship to the study drug. SAEs included death, a life-threatening adverse event, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, a congenital anomaly or birth defect, or an important medical event that jeopardized the participant and required medical intervention to prevent 1 of the outcomes listed in this definition. A TEAE was defined as any AE that began or worsened in severity or frequency on or after the start of study drug through 10 days after the last dose of the study drug. A summary of serious and non-serious AEs regardless of causality is located in 'Reported Adverse Events module'.
| Participants | X4P-001 50 mg | X4P-001 100 mg | X4P-001 150 mg | X4P-001 200 mg | X4P-001 300 mg | X4P-001 400 mg | Overall Participants |
|---|---|---|---|---|---|---|---|
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | 2 | 2 | 2 | 2 | 6 | 3 | 7 |
Collected over From first dose of study drug through 10 days after the last dose of the study drug (Maximum exposure: 1712 days). Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| X4P-001 50 mg | 0/2 (0%) | 0/2 (0%) | 2/2 (100%) |
| X4P-001 100 mg | 0/4 (0%) | 0/4 (0%) | 2/4 (50%) |
| X4P-001 150 mg | 0/3 (0%) | 0/3 (0%) | 2/3 (66.7%) |
| X4P-001 200 mg | 0/4 (0%) | 0/4 (0%) | 2/4 (50%) |
| X4P-001 300 mg | 0/7 (0%) | 2/7 (28.6%) | 6/7 (85.7%) |
| X4P-001 400 mg | 0/5 (0%) | 1/5 (20%) | 3/5 (60%) |
| Overall Participants | 0/8 (0%) | 3/8 (37.5%) | 7/8 (87.5%) |
| Event | X4P-001 50 mg | X4P-001 100 mg | X4P-001 150 mg | X4P-001 200 mg | X4P-001 300 mg | X4P-001 400 mg | Overall Participants |
|---|---|---|---|---|---|---|---|
| CellulitisInfections and infestations | 0/2 | 0/4 | 0/3 | 0/4 | 0/7 | 1/5 | 1/8 |
| Tibia fractureInjury, poisoning and procedural complications | 0/2 | 0/4 | 0/3 | 0/4 | 0/7 | 1/5 | 1/8 |
| PyrexiaGeneral disorders | 0/2 | 0/4 | 0/3 | 0/4 | 1/7 | 0/5 | 1/8 |
| BronchitisInfections and infestations | 0/2 | 0/4 | 0/3 | 0/4 | 1/7 | 0/5 | 1/8 |
| InfluenzaInfections and infestations | 0/2 | 0/4 | 0/3 | 0/4 | 1/7 | 0/5 | 1/8 |
| Event | X4P-001 50 mg | X4P-001 100 mg | X4P-001 150 mg | X4P-001 200 mg | X4P-001 300 mg | X4P-001 400 mg | Overall Participants |
|---|---|---|---|---|---|---|---|
| OtorrhoeaEar and labyrinth disorders | 1/2 | 0/4 | 0/3 | 0/4 | 0/7 | 0/5 | 1/8 |
| NauseaGastrointestinal disorders | 0/2 | 2/4 | 0/3 | 0/4 | 2/7 | 0/5 | 4/8 |
| NoduleGeneral disorders | 1/2 | 0/4 | 0/3 | 0/4 | 0/7 | 0/5 | 1/8 |
| Ear infectionInfections and infestations | 1/2 | 0/4 | 0/3 | 0/4 | 0/7 | 0/5 | 1/8 |
| NasopharyngitisInfections and infestations | 1/2 | 0/4 | 0/3 | 0/4 | 2/7 | 0/5 | 2/8 |
| SinusitisInfections and infestations | 1/2 | 0/4 | 0/3 | 0/4 | 2/7 | 2/5 | 3/8 |
| WheezingRespiratory, thoracic and mediastinal disorders | 1/2 | 0/4 | 0/3 | 0/4 | 0/7 | 0/5 | 1/8 |
| DyspepsiaGastrointestinal disorders | 0/2 | 1/4 | 0/3 | 0/4 | 0/7 | 2/5 | 3/8 |
| CellulitisInfections and infestations | 0/2 | 0/4 | 0/3 | 0/4 | 0/7 | 2/5 | 2/8 |
| Otitis mediaInfections and infestations | 0/2 | 0/4 | 0/3 | 0/4 | 1/7 | 2/5 | 3/8 |
The Safety Population included all participants who received at least 1 dose of the study medication (mavorixafor). Per prespecified analysis, data were collected for the 'Baseline Characteristics' as a single Arm/Group for any participants who received at least 1 dose of the study drug, regardless of their dose level. Due to the small number of participants per dose, data are reported for the 'Baseline Characteristics' as a single Arm/Group to maintain participant confidentiality.
| Age, Continuous(years) | X4P-001 |
|---|---|
| Mean | 35.5 ± 13.37 |
| Sex: Female, Male(Participants) | X4P-001 |
|---|---|
| Female | 6 |
| Male | 2 |
| Ethnicity (NIH/OMB)(Participants) | X4P-001 |
|---|---|
| Hispanic or Latino | 0 |
| Not Hispanic or Latino | 8 |
| Unknown or Not Reported | 0 |
| Race (NIH/OMB)(Participants) | X4P-001 |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 0 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 0 |
| White | 8 |
| More than one race | 0 |
| Unknown or Not Reported | 0 |
PDFs hosted by the registry, ClinicalTrials.gov.
Ran from Dec 2016 to primary completion in Jun 2022, 66 months.
Results were first posted in Oct 2024, 2 years 4 months after primary completion.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Oct 2024. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Studies of the drug tested here: Mavorixafor
WHIM syndrome
X4 Pharmaceuticals