Trial · NCT03005327
A Dose Determination and Safety Study of X4P-001 (Mavorixafor) in Participants With Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) Syndrome
NCT03005327, Phase 2, Completed, X4 Pharmaceuticals; results first posted 2024-10-30
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Dose Determination and Safety Study of X4P-001 (Mavorixafor) in Participants With Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) Syndrome. Trials, record NCT03005327. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03005327
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Dose Determination and Safety Study of X4P-001 (Mavorixafor) in Participants With Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) Syndrome
- NCT number
- NCT03005327
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 8 participants, actual
- Lead sponsor
- X4 Pharmaceuticals
- Conditions
- WHIM Syndrome
- Interventions
- X4P-001
- First posted
- 2016-12-29
- Last updated
- 2024-10-30
- Other registries' IDs
- eudract:2016-005028-26
- Results first posted
- 2024-10-30
Source ClinicalTrials.gov
Registered outcomes5
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Mean Value of the Area Under the Plasma Concentration-time Curve for Absolute Neutrophil Count (AUCANC) | 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 | 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). |
| Primary | All Visits: Average Per-Participant Value of the AUCANC | 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 | 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. |
| Primary | Mean Value of the Area Under the Plasma Concentration-time Curve for Absolute Lymphocyte Count (AUCALC) | 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 | 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. |
| Primary | All Visits: Average Per-Participant Value of the AUCALC | 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 | 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. |
| Primary | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | From first dose of study drug through 10 days after the last dose of the study drug (Maximum exposure: 1712 days) | 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'. |
Source ClinicalTrials.gov
Outcomes5
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Mean Value of the Area Under the Plasma Concentration-time Curve for Absolute Neutrophil Count (AUCANC) | 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 | Overall Number of Participants Analyzed=number of participants in safety population (all participants who received at least 1 dose of study medication). 'Number analyzed'=number of participants with data above clinically meaningful thresholds at specified timepoints. Participants included in 1 category may be different than participants in other categories. Data for this Outcome Measure were collected by dose level; therefore, participants may have been included in more than 1 arm (dose level). | cells*hour/μL | MEAN |
| PRIMARY | All Visits: Average Per-Participant Value of the AUCANC | 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 | The Safety Population included all participants who received at least 1 dose of the study medication (mavorixafor). Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure. | cells*hour/μL | MEAN |
| PRIMARY | Mean Value of the Area Under the Plasma Concentration-time Curve for Absolute Lymphocyte Count (AUCALC) | 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 | Overall Number of Participants Analyzed=number of participants in safety population (all participants who received at least 1 dose of study medication). 'Number analyzed'=number of participants with data above clinically meaningful thresholds at specified timepoints. Participants included in 1 category may be different than participants in other categories. Data for this Outcome Measure were collected by dose level; therefore, participants may have been included in more than 1 arm (dose level). | cells*hour/μL | MEAN |
| PRIMARY | All Visits: Average Per-Participant Value of the AUCALC | 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 | The Safety Population included all participants who received at least 1 dose of the study medication (mavorixafor). Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure. | cells*hour/μL | MEAN |
| PRIMARY | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | From first dose of study drug through 10 days after the last dose of the study drug (Maximum exposure: 1712 days) | The Safety Population included all participants who received at least 1 dose of the study medication (mavorixafor). Data for this Outcome Measure were collected by dose level; therefore, participants may have been included in more than one arm (dose level). | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group33
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Mean Value of the Area Under the Plasma Concentration-time Curve for Absolute Neutrophil Count (AUCANC) | X4P-001 50 mg | Week 5 | -12574.63 | Standard Deviation 156.094 | cells*hour/μL |
| Mean Value of the Area Under the Plasma Concentration-time Curve for Absolute Neutrophil Count (AUCANC) | X4P-001 100 mg | Week 5 | -6206.75 | Standard Deviation 4238.280 | cells*hour/μL |
| Mean Value of the Area Under the Plasma Concentration-time Curve for Absolute Neutrophil Count (AUCANC) | X4P-001 200 mg | Week 5 | -6949.58 | cells*hour/μL | |
| Mean Value of the Area Under the Plasma Concentration-time Curve for Absolute Neutrophil Count (AUCANC) | X4P-001 300 mg | Week 5 | 24175.00 | Standard Deviation 777.817 | cells*hour/μL |
| Mean Value of the Area Under the Plasma Concentration-time Curve for Absolute Neutrophil Count (AUCANC) | X4P-001 300/400 mg | Week 5 | 24175.00 | Standard Deviation 777.817 | cells*hour/μL |
| Mean Value of the Area Under the Plasma Concentration-time Curve for Absolute Neutrophil Count (AUCANC) | X4P-001 100 mg | Week 13 | -11291.96 | Standard Deviation 1509.496 | cells*hour/μL |
| Mean Value of the Area Under the Plasma Concentration-time Curve for Absolute Neutrophil Count (AUCANC) | X4P-001 200 mg | Week 13 | -7575.21 | Standard Deviation 2701.443 | cells*hour/μL |
| Mean Value of the Area Under the Plasma Concentration-time Curve for Absolute Neutrophil Count (AUCANC) | X4P-001 300 mg | Week 13 | -2602.50 | cells*hour/μL | |
| Mean Value of the Area Under the Plasma Concentration-time Curve for Absolute Neutrophil Count (AUCANC) | X4P-001 300/400 mg | Week 13 | -2602.50 | cells*hour/μL | |
| Mean Value of the Area Under the Plasma Concentration-time Curve for Absolute Neutrophil Count (AUCANC) | X4P-001 150 mg | Week 21 | -8723.00 | Standard Deviation 2695.962 | cells*hour/μL |
1 to 10 of 33Source ClinicalTrials.gov
See all 33 results by groupParticipant flow28
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Initial Treatment Period (24 Weeks) | STARTED | X4P-001 50 to up to 400 mg/kg | 2 | |
| Initial Treatment Period (24 Weeks) | STARTED | X4P-001 100 to up to 400 mg/kg | 2 | |
| Initial Treatment Period (24 Weeks) | STARTED | X4P-001 200 to up to 400 mg/kg | 2 | |
| Initial Treatment Period (24 Weeks) | STARTED | X4P-001 300 to up to 400 mg/kg | 2 | |
| Initial Treatment Period (24 Weeks) | Received at Least 1 Dose of Study Drug | X4P-001 50 to up to 400 mg/kg | 2 | |
| Initial Treatment Period (24 Weeks) | Received at Least 1 Dose of Study Drug | X4P-001 100 to up to 400 mg/kg | 2 | |
| Initial Treatment Period (24 Weeks) | Received at Least 1 Dose of Study Drug | X4P-001 200 to up to 400 mg/kg | 2 | |
| Initial Treatment Period (24 Weeks) | Received at Least 1 Dose of Study Drug | X4P-001 300 to up to 400 mg/kg | 2 | |
| Initial Treatment Period (24 Weeks) | COMPLETED | X4P-001 50 to up to 400 mg/kg | 2 | |
| Initial Treatment Period (24 Weeks) | COMPLETED | X4P-001 100 to up to 400 mg/kg | 1 |
1 to 10 of 28Source ClinicalTrials.gov
See all 28 participant flowAdverse events462
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Eustachian tube obstruction | Ear and labyrinth disorders | X4P-001 50 mg | 0 | 2 | |
| other | Eustachian tube obstruction | Ear and labyrinth disorders | X4P-001 100 mg | 0 | 4 | |
| other | Eustachian tube obstruction | Ear and labyrinth disorders | X4P-001 150 mg | 0 | 3 | |
| other | Eustachian tube obstruction | Ear and labyrinth disorders | X4P-001 200 mg | 0 | 4 | |
| other | Eustachian tube obstruction | Ear and labyrinth disorders | X4P-001 300 mg | 1 | 7 | |
| other | Eustachian tube obstruction | Ear and labyrinth disorders | X4P-001 400 mg | 0 | 5 | |
| other | Eustachian tube obstruction | Ear and labyrinth disorders | Overall Participants | 1 | 8 | |
| other | Otorrhoea | Ear and labyrinth disorders | X4P-001 50 mg | 1 | 2 | |
| other | Otorrhoea | Ear and labyrinth disorders | X4P-001 100 mg | 0 | 4 | |
| other | Otorrhoea | Ear and labyrinth disorders | X4P-001 150 mg | 0 | 3 |
1 to 10 of 462Source ClinicalTrials.gov
See all 462 adverse eventsAdverse event totals21
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | X4P-001 50 mg | 0 | 2 |
| Total, all-cause mortality | X4P-001 100 mg | 0 | 4 |
| Total, all-cause mortality | X4P-001 150 mg | 0 | 3 |
| Total, all-cause mortality | X4P-001 200 mg | 0 | 4 |
| Total, all-cause mortality | X4P-001 300 mg | 0 | 7 |
| Total, all-cause mortality | X4P-001 400 mg | 0 | 5 |
| Total, all-cause mortality | Overall Participants | 0 | 8 |
| Total, other adverse events | X4P-001 50 mg | 2 | 2 |
| Total, other adverse events | X4P-001 100 mg | 2 | 4 |
| Total, other adverse events | X4P-001 150 mg | 2 | 3 |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 adverse event totalsPapers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 32870250 | DERIVED |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
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The record and each of its lists are JSON, twenty rows a page. No key.
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