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A Study of Mavorixafor in Participants With Congenital Neutropenia and Chronic Idiopathic Neutropenia Disorders

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ClinicalTrials.gov Identifier: NCT04154488
Recruitment Status : Recruiting
First Posted : November 6, 2019
Last Update Posted : December 5, 2022
Sponsor:
Information provided by (Responsible Party):
X4 Pharmaceuticals

Brief Summary:
This is a 2-part study of mavorixafor in participants diagnosed with chronic neutropenia. The main goal of Part 1 (Phase 1b) is to help researchers learn more about how the investigational medicine, mavorixafor, impacts people living with chronic neutropenia (including congenital, idiopathic, and cyclic). In Part 2 (Phase 2), the safety and tolerability of chronic dosing of mavorixafor will be evaluated in a larger participant population and the impact of 6-month chronic dosing of mavorixafor on participant neutropenia.

Condition or disease Intervention/treatment Phase
Neutropenia Drug: Mavorixafor Phase 1 Phase 2

Detailed Description:

Part 1: Participants will receive one oral dose of mavorixafor and be monitored for 8 hours to see if neutrophil cell counts increase.

Part 2: Eligible participants from Part 1 can directly roll-over. Participants will receive once daily oral dosing of mavorixafor for 6 months and be monitored throughout to see if neutrophil cell counts increase.

Study visits can be conducted at-home or at one of many study clinic locations, depending on the participant's preference.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 50 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 1b/2, Open-Label, Multicenter Study of Mavorixafor in Patients With Congenital Neutropenia and Chronic Neutropenia Disorders
Actual Study Start Date : October 16, 2020
Estimated Primary Completion Date : June 2025
Estimated Study Completion Date : July 2025


Arm Intervention/treatment
Experimental: Mavorixafor

Part 1: Adult participants and adolescent participants who weigh more than 50 kilograms (kg) will receive mavorixafor 400 milligrams (mg) (4 capsules of 100 mg each), orally once on Day 1. Adolescents weighing less than or equal to 50 kg will receive mavorixafor 200 mg (2 capsules of 100 mg each), orally once on Day 1.

Part 2: Eligible participants from Part 1 will receive once daily dosing of mavorixafor for 6 months.

Drug: Mavorixafor
Mavorixafor capsules will be administered per dose and schedule specified in the arm.
Other Name: X4P-001




Primary Outcome Measures :
  1. Number of Participants With Treatment-Emergent Adverse Events (TEAEs) After a Single Dose of Mavorixafor [ Time Frame: Baseline through Day 1 and 7 days follow-up ]
  2. Number of Participants With Treatment-Emergent Adverse Events (TEAEs) After Multiple Doses of Mavorixafor [ Time Frame: Baseline through Month 6 and 30 days follow-up ]
  3. Change From Baseline in Absolute Neutrophil Count (ANC) to 8 hours Post-dose On Day 1 [ Time Frame: Baseline, 8 hours Post-dose On Day 1 ]
  4. Change From Baseline in ANC to Month 6 [ Time Frame: Baseline, Month 6 ]

Secondary Outcome Measures :
  1. Serum Concentration of Mavorixafor in Relation to ANC and Area Under the Curve (AUC) for ANC (AUCANC) [ Time Frame: 0 (pre-dose), 60 minutes and 2, 3, 4, 6, and 8 hours post-dose on Day 1 ]
  2. Serum Concentrations of Mavorixafor [ Time Frame: 0 (pre-dose) up to Month 6 ]
  3. Change from Baseline in Absolute Lymphocyte Count (ALC) [ Time Frame: Baseline, Month 6 ]
  4. Change from Baseline in Total White Blood Cells (WBC) [ Time Frame: Baseline, Month 6 ]
  5. Change from Baseline in Absolute Monocyte (AMC) [ Time Frame: Baseline, Month 6 ]
  6. AUC of ANC (AUCANC) [ Time Frame: Baseline up to Month 6 ]
  7. AUC of ALC (AUCALC) [ Time Frame: Baseline up to Month 6 ]
  8. AUC of AMC (AUCAMC) [ Time Frame: Baseline up to Month 6 ]
  9. AUC of WBC (AUCWBC) [ Time Frame: Baseline up to Month 6 ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

For all participants (Parts 1 and 2):

  • Sign the informed consent form (ICF) and be willing and able to comply with the protocol.
  • Weigh ≥15 kg
  • Agree to use a highly effective form of contraception if sexually active.
  • Participants may be eligible for the study whether they are on or off granulocyte-colony stimulating factor (G-CSF) treatment.

    • Note: Participants who are on G-CSF must be on a stable dose for ≥14 days prior to the Baseline visit and should not have an ANC ≥10,000 cells/μL.
    • Note: Participants who are not on G-CSF must be off for ≥14 days prior to the Baseline visit and have an ANC ≤1000 cells/µL at the Screening visit.
    • Note: Participants with Shwachman-Diamond syndrome, Cohensyndrome, and warts, hypogammaglobulinemia, infections and myelokathexis syndrome are eligible. Other types of chronic neutropenic disorders may also be eligible for enrollment upon discussion and approval with Sponsor and Study Medical Monitor.
  • Have been diagnosed with chronic neutropenia for ≥6 months prior to the Screening visit that is not attributable to medications, active or recent (≤3 months) infections, or malignant cause.

Part 2 only:

  • Participants who have completed Part 1 and exhibit a positive response to treatment.
  • Participant has a history of symptomatic chronic neutropenia confirmed by the Investigator.

Key Exclusion Criteria (Parts 1 and 2):

  • Known systemic hypersensitivity to the mavorixafor drug substance or its inactive ingredients.
  • Is pregnant, breastfeeding, or plans to become pregnant over the next 8 months.
  • Known history of a positive serology or viral load for human immunodeficiency virus (HIV) or a known history of acquired immune deficiency syndrome.
  • Known active SARS-CoV-2 virus (COVID-19) infection or a positive test within the local accepted clinical and governmental guidelines for a communicable window.
  • At the Screening Visit, has a laboratory test result meeting one or more of the following criteria:

    • Positive hepatitis C virus (HCV) antibodies with confirmation by HCV-ribonucleic acid polymerase chain reaction reflex testing.
    • Positive hepatitis B surface antigen (HBsAg) or hepatitis B core antibody (HBcAb).
    • Note: If a participant tests negative for HBsAg but positive for HBcAb, the participant would be considered eligible if the participant tests positive for antibody to HBsAg reflex testing.
  • At the Screening visit, has a laboratory test result meeting one or more of the following criteria:

    • Hemoglobin <9.0 grams/deciliter (g/dL)
    • Platelets <30,000/μL
    • Estimated glomerular filtration rate (eGFR) ≤60 mL/minute/1.73 meter (m)^2, as estimated by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation
    • Serum aspartate transaminase >2.5 x upper limit of normal (ULN)
    • Serum alanine transaminase >2.5 x ULN
    • Total bilirubin >1.5 x ULN (unless due to Gilbert's syndrome, in which case total bilirubin greater than or equal to (≥) 3.0 x ULN and direct bilirubin >1.5 x ULN)
  • ≤14 days before Day 1, received any of the following treatments:

    • Systemic glucocorticoids (>5 mg prednisone equivalent per day).
    • Medication prohibited based on cytochrome P450 (CYP), P-glycoprotein, OAT1, OCT2, or MATE1 potential for interaction.
  • Has an infection requiring use of systemic antibiotics ≤4 weeks before the Baseline visit.
  • Has a medical or personal condition that may potentially compromise the safety or compliance of the participant, or may preclude the participant's successful completion of the clinical study or that in the opinion of the Investigator or the Sponsor could interfere with the objectives of the study.
  • Has had major surgery ≤4 weeks before the Baseline visit.
  • Inability to ingest mavorixafor capsules.
  • Has an active malignancy or history of (≤5 years prior to enrollment) in the study of solid, metastatic, or hematologic malignancy. Exception: basal cell carcinoma in situ of the skin that has been adequately treated.
  • Diagnosed or has suspected congenital long QT syndrome. Any history of clinically significant ventricular arrhythmias (such as ventricular tachycardia, ventricular fibrillation, or torsades de pointes); any history of arrhythmia will be discussed with the sponsor's medical monitor before participant's entry into the study.
  • Prolonged corrected QT interval using Fridericia's formula at the Screening visit electrocardiogram (ECG) (>450 milliseconds [ms])

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04154488


Contacts
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Contact: Patient Affairs and Advocacy 857-529-5779 clinicaltrialinfo@x4pharma.com

Locations
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United States, California
Parexel International Withdrawn
Glendale, California, United States, 91206
United States, Florida
USF Health Department of Pediatrics Recruiting
Saint Petersburg, Florida, United States, 33701
Contact: Jolan Walter         
United States, Iowa
University of Iowa Hospital and Clinics Recruiting
Iowa City, Iowa, United States, 52242
Contact: Anjali Sharathkumar         
United States, Maryland
Harbor Hospital Withdrawn
Baltimore, Maryland, United States, 21225
United States, Michigan
University of Michigan Recruiting
Ann Arbor, Michigan, United States, 48109
Contact: Kelly Jo Walkovich         
United States, Missouri
Washington University School of Medicine Recruiting
Saint Louis, Missouri, United States, 63110
Contact: David Wilson         
United States, New York
Northwell Feinstein Institutes for Medical Research Withdrawn
Manhasset, New York, United States, 11030
United States, Ohio
Cleveland Clinic Withdrawn
Cleveland, Ohio, United States, 44195
United States, Texas
University of Texas, Southwestern Not yet recruiting
Dallas, Texas, United States, 75235
Contact: Kathryn Dickerson         
United States, Washington
University of Washington Recruiting
Seattle, Washington, United States, 98195
Contact: David C. Dale         
Sponsors and Collaborators
X4 Pharmaceuticals
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Responsible Party: X4 Pharmaceuticals
ClinicalTrials.gov Identifier: NCT04154488    
Other Study ID Numbers: X4P-001-104
First Posted: November 6, 2019    Key Record Dates
Last Update Posted: December 5, 2022
Last Verified: November 2022

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by X4 Pharmaceuticals:
Mavorixafor
Chronic congenital neutropenia
Chronic idiopathic neutropenia
SCN
CIN
CXCR4
Neutropenia glycogen storage disease type 1b
GSD1b
G6PC3
SLC37A4
GATA2
Additional relevant MeSH terms:
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Neutropenia
Agranulocytosis
Leukopenia
Leukocyte Disorders
Hematologic Diseases