- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT06056297
A Study of Mavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders Who Are Experiencing Recurrent and/or Serious Infections
A Phase 3, Randomized, Double-blind, Placebo-controlled, Multicenter Study of Mavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders Who Are Experiencing Recurrent and/or Serious Infections
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
Study Type
Enrollment (Estimated)
Phase
- Phase 3
Contacts and Locations
Study Contact
- Name: Patient Affairs and Advocacy
- Phone Number: 857-529-5779
- Email: clinicaltrialinfo@x4pharma.com
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
- Child
- Adult
- Older Adult
Accepts Healthy Volunteers
Description
Key Inclusion Criteria:
- Diagnosis of congenital or acquired primary autoimmune and idiopathic chronic neutropenic disorder ≥6 months prior to the screening visit that is not attributable to medications, active or recent infections or malignancy.
- Have a confirmed trough ANC <1500 cells/µL during the screening visit (single ANC measurement) and at baseline visit (mean ANC over 6 hours) held at least 2 weeks prior to Day 1 dosing, with no clinical evidence of infection.
Prior history of recurrent and/or serious infections during the 12 months preceding the screening visit (that is, suffering sequelae of chronic neutropenia), as defined by having at least 2 infections in the last 12 months that meet at least 1 of the following criteria:
- Infection requiring the use of antibiotics (intravenous [IV]/oral/topical)
- Infection requiring a visit to healthcare facility (including but not limited to emergency room visit, urgent care facility, primary care physician's office, or in-patient hospitalization).
- Participants who are on G-CSF or other active background therapy must have been receiving these therapies for ≥12 months, be on a stable dose and dosing schedule for ≥4 weeks prior to screening visit and remain on this dose and dosing schedule throughout the study (unless ANC >10,000 cells/µL for ≥4 weeks).
- Participants must be willing to keep their G-CSF or other background therapy doses/regimens stable (other than for safety reasons) for the duration of the study.
Key Exclusion Criteria:
A diagnosis of secondary neutropenia including those due to:
- Hypersplenism
- Infection
- Malignancy
- Autoimmune disease, for example, systemic lupus erythematosus, rheumatoid arthritis, irritable bowel disease, graft-versus-host disease, thyroid disease
- Nutritional deficiency, for example, vitamin B12, folic acid, copper, caloric malnutrition
- Drug-induced cause, for example, chemotherapy, clozapine, antiretrovirals, antibiotics, monoclonal antibodies.
A diagnosis of any of the following:
- Aplastic anemia
- Warts, hypogammaglobulinemia, infections, and myelokathexis (WHIM) syndrome
Certain Congenital Neutropenias, including but not limited to these classifications are excluded:
- Isolated with a cyclic presentation, for example, elastase, neutrophil expressed (ELANE)
- Associated with immune dysregulation, for example, autoimmune lymphoproliferative syndrome, Familial hemophagocytic lymphohistiocytosis, Chédiak-Higashi syndrome
- Associated with bone marrow failure, for example, Fanconi Anemia, Diamond-Blackfan anemia, Telomere diseases
- Neutropenia associated with a Duffy-null phenotype (formerly known as benign ethnic neutropenia).
- A medical or personal condition that may potentially compromise the safety of the participant, may preclude the participant's successful completion of the clinical study, or could, in the opinion of the Investigator or the Sponsor, interfere with the objectives of the study.
- Received more than 1 dose of mavorixafor in the past.
- Received C-X-C chemokine receptor 4 (CXCR4) antagonist (other than mavorixafor) in the past 6 months.
- Participants taking pegylated-G-CSF unless they have a diagnosis of congenital neutropenia confirmed at screening.
- Participant is currently taking or have taken other investigational drug at least 30 days prior to the screening visit.
Note: Other protocol-defined inclusion and exclusion criteria may apply.
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: Quadruple
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Mavorixafor
Participants will receive mavorixafor orally once daily starting from Day 1 through Week 52.
|
Mavorixafor will be administered per schedule specified in the arm description.
Other Names:
|
Placebo Comparator: Placebo
Participants will receive placebo matching to mavorixafor orally once daily starting from Day 1 through Week 52.
|
Placebo will be administered per schedule specified in the arm description.
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Infection Rate Based on Infections Adjudicated by Blinded, Independent Adjudication Committee (AC) During the 12-Month Treatment Period
Time Frame: Baseline up to Month 12
|
Baseline up to Month 12
|
|
Proportion of Participants Meeting the Definition of a Positive Absolute Neutrophil Count (ANC) Response During the First 3 Months
Time Frame: Baseline up to Month 3
|
Positive ANC response: ANC ≥1500 cells/microliter (µL), with the exception of participants with Baseline ANC <500 cells/µL; and ≥2-fold increase in ANC from baseline, for participants with baseline ANC < 500 cells/μL.
|
Baseline up to Month 3
|
Secondary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Infection Duration Based on Duration of Infections Adjudicated by Blinded, Independent AC During the 12-Month Treatment Period in Those Participants who Developed Infections
Time Frame: Baseline up to Month 12
|
Baseline up to Month 12
|
Infection Severity Based on Severity of Infections Adjudicated by a Blinded Independent AC During the 12-Month Treatment Period
Time Frame: Baseline up to Month 12
|
Baseline up to Month 12
|
Antibiotic Use Due to Infection, Characterized by the Frequency of Antibiotic Use During the 12-Month Treatment Period
Time Frame: Baseline up to Month 12
|
Baseline up to Month 12
|
Change From Baseline to Week 52 in Patient Reported Outcomes Measurement Information System Short Form (PROMIS SF) Fatigue Questionnaire Total Score
Time Frame: Baseline, Week 52
|
Baseline, Week 52
|
Oral Ulcers, as Assessed by Presence or Absence of Ulcers During the 12-Month Treatment Period
Time Frame: Baseline up to Month 12
|
Baseline up to Month 12
|
Collaborators and Investigators
Sponsor
Investigators
- Study Director: Chief Medical Officer, X4 Pharmaceuticals
Study record dates
Study Major Dates
Study Start (Estimated)
Primary Completion (Estimated)
Study Completion (Estimated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Estimated)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- X4P-001-110
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
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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