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| ID | Type | Description | Link |
|---|---|---|---|
| 2016-005028-26 | EudraCT Number |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| X4P-001 | Experimental | 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| X4P-001 | Drug | Mavorixafor will be provided as either 25 mg or 100 mg capsules. |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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 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 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 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. |
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Inclusion Criteria:
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 known systemic hypersensitivity to the mavorixafor drug substance or its inactive ingredients.
Is pregnant or nursing.
Has a known history of a positive serology or viral load for human immunodeficiency virus (HIV) or a known history of acquired immunodeficiency syndrome (AIDS).
Has, at Screening, laboratory tests meeting one or more of the following criteria:
Has any medical or personal condition that, 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.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Washington Medical Center | Seattle | Washington | 98195 | United States | ||
| St. Vincent's Hospital |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 32870250 | Derived | Dale DC, Firkin F, Bolyard AA, Kelley M, Makaryan V, Gorelick KJ, Ebrahim T, Garg V, Tang W, Jiang H, Skerlj R, Beaussant Cohen S. Results of a phase 2 trial of an oral CXCR4 antagonist, mavorixafor, for treatment of WHIM syndrome. Blood. 2020 Dec 24;136(26):2994-3003. doi: 10.1182/blood.2020007197. |
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Each participant was treated at doses that were increased up to a maximum total daily dose of 400 milligrams (mg) based on their individual area under the curve for absolute neutrophil count and absolute leukocyte count (AUCANC/ALC) values.
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| ID | Title | Description |
|---|---|---|
| FG000 | X4P-001 50 to up to 400 mg/kg | Participants received X4P-001 (mavorixafor) orally once daily (QD) at doses between 50 mg and 400 mg for the Initial Treatment Period (24 weeks) and could continue participation in the open-label Extension Phase until experiencing a treatment-limiting toxicity (TLT) event, X4P-001 was available commercially, or until the study was terminated by the Sponsor. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Initial Treatment Period (24 Weeks) |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Dec 21, 2020 | May 21, 2024 |
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| 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'. | From first dose of study drug through 10 days after the last dose of the study drug (Maximum exposure: 1712 days) |
| Fitzroy |
| Victoria |
| 3065 |
| Australia |
| FG001 | X4P-001 100 to up to 400 mg/kg | Participants received X4P-001 (mavorixafor) orally QD at doses between 100 mg and 400 mg for the Initial Treatment Period (24 weeks) and could continue participation in the open-label Extension Phase until experiencing a TLT event, X4P-001 was available commercially, or until the study was terminated by the Sponsor. |
| FG002 | X4P-001 200 to up to 400 mg/kg | Participants received X4P-001 (mavorixafor) orally QD at doses between 200 mg and 400 mg for the Initial Treatment Period (24 weeks) and could continue participation in the open-label Extension Phase until experiencing a TLT event, X4P-001 was available commercially, or until the study was terminated by the Sponsor. |
| FG003 | X4P-001 300 to up to 400 mg/kg | Participants received X4P-001 (mavorixafor) orally QD at doses between 300 mg and 400 mg for the Initial Treatment Period (24 weeks) and could continue participation in the open-label Extension Phase until experiencing a TLT event, X4P-001 was available commercially, or until the study was terminated by the Sponsor. |
| Received at Least 1 Dose of Study Drug |
|
| COMPLETED |
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| NOT COMPLETED |
|
|
| Extension Phase (56 Months) |
|
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.
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| ID | Title | Description |
|---|---|---|
| BG000 | X4P-001 | Participants received X4P-001 (mavorixafor) orally QD at doses between 50 mg and 400 mg for the Initial Treatment Period (24 weeks) and could continue participation in the open-label Extension Phase until experiencing a TLT event, X4P-001 was available commercially, or until the study was terminated by the Sponsor. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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). | 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). | Posted | Mean | Standard Deviation | cells*hour/μL | 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 |
|
|
| ||||||||||||||||||||||||||||||||||||||
| Primary | 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. | 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. | Posted | Mean | Standard Deviation | cells*hour/μL | 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 |
|
| |||||||||||||||||||||||||||||||||||||||
| Primary | 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. | 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). | Posted | Mean | Standard Deviation | cells*hour/μL | 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 |
| ||||||||||||||||||||||||||||||||||||||||
| Primary | 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. | 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. | Posted | Mean | Standard Deviation | cells*hour/μL | 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 |
|
| |||||||||||||||||||||||||||||||||||||||
| Primary | 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'. | 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). | Posted | Count of Participants | Participants | From first dose of study drug through 10 days after the last dose of the study drug (Maximum exposure: 1712 days) |
|
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). All-Cause Mortality and Adverse Event data were collected by dose level; therefore, participants may have been included in more than one arm (dose level).
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | X4P-001 50 mg | Participants received X4P-001 50 mg orally QD. | 0 | 2 | 0 | 2 | 2 | 2 |
| EG001 | X4P-001 100 mg | Participants received X4P-001 100 mg orally QD. | 0 | 4 | 0 | 4 | 2 | 4 |
| EG002 | X4P-001 150 mg | Participants received X4P-001 150 mg orally QD. | 0 | 3 | 0 | 3 | 2 | 3 |
| EG003 | X4P-001 200 mg | Participants received X4P-001 200 mg orally QD. | 0 | 4 | 0 | 4 | 2 | 4 |
| EG004 | X4P-001 300 mg | Participants received X4P-001 300 mg orally QD. | 0 | 7 | 2 | 7 | 6 | 7 |
| EG005 | X4P-001 400 mg | Participants received X4P-001 400 mg orally QD. | 0 | 5 | 1 | 5 | 3 | 5 |
| EG006 | Overall Participants | Participants received X4P-001 (mavorixafor) orally QD at doses between 50 mg and 400 mg for the Initial Treatment Period (24 weeks) and could continue participation in the open-label Extension Phase until experiencing a TLT event, X4P-001 was available commercially, or until the study was terminated by the Sponsor. | 0 | 8 | 3 | 8 | 7 | 8 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Pyrexia | General disorders | MedDRA 23.1 | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
| |
| Tibia fracture | Injury, poisoning and procedural complications | MedDRA 23.1 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Eustachian tube obstruction | Ear and labyrinth disorders | MedDRA 23.1 | Systematic Assessment |
| |
| Otorrhoea | Ear and labyrinth disorders | MedDRA 23.1 | Systematic Assessment |
| |
| Conjunctivitis allergic | Eye disorders | MedDRA 23.1 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 23.1 | Systematic Assessment |
| |
| Aphthous ulcer | Gastrointestinal disorders | MedDRA 23.1 | Systematic Assessment |
| |
| Dry mouth | Gastrointestinal disorders | MedDRA 23.1 | Systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA 23.1 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 23.1 | Systematic Assessment |
| |
| Chills | General disorders | MedDRA 23.1 | Systematic Assessment |
| |
| Nodule | General disorders | MedDRA 23.1 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 23.1 | Systematic Assessment |
| |
| Cholecystitis | Hepatobiliary disorders | MedDRA 23.1 | Systematic Assessment |
| |
| Arthritis infective | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
| |
| Conjunctivitis | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
| |
| Ear infection | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
| |
| Folliculitis | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
| |
| Genital herpes simplex | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
| |
| Hordeolum | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
| |
| Infected bite | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
| |
| Localised infection | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
| |
| Lower respiratory tract infection | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
| |
| Otitis media | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
| |
| Otitis media acute | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
| |
| Papilloma viral infection | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
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| Respiratory tract infection | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
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| Sinusitis | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
| |
| Skin infection | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
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| Upper respiratory tract infection | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
| |
| Viral infection | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
| |
| Animal bite | Injury, poisoning and procedural complications | MedDRA 23.1 | Systematic Assessment |
| |
| Animal scratch | Injury, poisoning and procedural complications | MedDRA 23.1 | Systematic Assessment |
| |
| Arthropod bite | Injury, poisoning and procedural complications | MedDRA 23.1 | Systematic Assessment |
| |
| Incision site ulcer | Injury, poisoning and procedural complications | MedDRA 23.1 | Systematic Assessment |
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| Limb injury | Injury, poisoning and procedural complications | MedDRA 23.1 | Systematic Assessment |
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| Muscle strain | Injury, poisoning and procedural complications | MedDRA 23.1 | Systematic Assessment |
| |
| Post procedural contusion | Injury, poisoning and procedural complications | MedDRA 23.1 | Systematic Assessment |
| |
| Procedural pain | Injury, poisoning and procedural complications | MedDRA 23.1 | Systematic Assessment |
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| Scratch | Injury, poisoning and procedural complications | MedDRA 23.1 | Systematic Assessment |
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| Skin abrasion | Injury, poisoning and procedural complications | MedDRA 23.1 | Systematic Assessment |
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| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 23.1 | Systematic Assessment |
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| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 23.1 | Systematic Assessment |
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| Rotator cuff syndrome | Musculoskeletal and connective tissue disorders | MedDRA 23.1 | Systematic Assessment |
| |
| Temporomandibular joint syndrome | Musculoskeletal and connective tissue disorders | MedDRA 23.1 | Systematic Assessment |
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| Uterine spasm | Reproductive system and breast disorders | MedDRA 23.1 | Systematic Assessment |
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| Vulva cyst | Reproductive system and breast disorders | MedDRA 23.1 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 23.1 | Systematic Assessment |
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| Nasal dryness | Respiratory, thoracic and mediastinal disorders | MedDRA 23.1 | Systematic Assessment |
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| Rales | Respiratory, thoracic and mediastinal disorders | MedDRA 23.1 | Systematic Assessment |
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| Wheezing | Respiratory, thoracic and mediastinal disorders | MedDRA 23.1 | Systematic Assessment |
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| Dermatitis psoriasiform | Skin and subcutaneous tissue disorders | MedDRA 23.1 | Systematic Assessment |
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| Dry skin | Skin and subcutaneous tissue disorders | MedDRA 23.1 | Systematic Assessment |
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| Erythema | Skin and subcutaneous tissue disorders | MedDRA 23.1 | Systematic Assessment |
| |
| Palmar erythema | Skin and subcutaneous tissue disorders | MedDRA 23.1 | Systematic Assessment |
| |
| Rash pruritic | Skin and subcutaneous tissue disorders | MedDRA 23.1 | Systematic Assessment |
| |
| Skin irritation | Skin and subcutaneous tissue disorders | MedDRA 23.1 | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Chief Medical Officer | X4 Pharmaceuticals | (857) 529-8300 | patientinfo@x4pharma.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Feb 16, 2022 | May 21, 2024 | SAP_001.pdf |
| ID | Term |
|---|---|
| C536697 | WHIM syndrome |
Not provided
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| ID | Term |
|---|---|
| C494414 | mavorixafor |
Not provided
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| Unknown or Not Reported |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
|
| Week 13 |
|
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| Week 21 |
|
|
|
Participants received X4P-001 150 mg orally QD.
| OG003 | X4P-001 200 mg | Participants received X4P-001 200 mg orally QD. |
| OG004 | X4P-001 300 mg | Participants received X4P-001 300 mg orally QD. |
| OG005 | X4P-001 400 mg | Participants received X4P-001 400 mg orally QD. |
| OG006 | X4P-001 300/400 mg | Participants received X4P-001 300 mg or 400 mg orally QD. |
|
|
|
Participants received X4P-001 100 mg orally QD.
| OG002 | X4P-001 150 mg | Participants received X4P-001 150 mg orally QD. |
| OG003 | X4P-001 200 mg | Participants received X4P-001 200 mg orally QD. |
| OG004 | X4P-001 300 mg | Participants received X4P-001 300 mg orally QD. |
| OG005 | X4P-001 400 mg | Participants received X4P-001 400 mg orally QD. |
| OG006 | Overall Participants | Participants received X4P-001 (mavorixafor) orally QD at doses between 50 mg and 400 mg for the Initial Treatment Period (24 weeks) and could continue participation in the open-label Extension Phase until experiencing a TLT event, X4P-001 was available commercially, or until the study was terminated by the Sponsor. |
|
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