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Trial record 2 of 689 for:    X4

A Trial of X4P-001 in Patients With WHIM Syndrome (X4P-001-MKKA)

This study is currently recruiting participants.
See Contacts and Locations
Verified February 2017 by X4 Pharmaceuticals
Sponsor:
Information provided by (Responsible Party):
X4 Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT03005327
First received: December 20, 2016
Last updated: February 8, 2017
Last verified: February 2017
  Purpose
The overall objective is to evaluate X4P-001 in the treatment of patients with WHIM Syndrome.

Condition Intervention Phase
WHIM Syndrome Drug: X4P-001 Drug: Placebo Phase 2 Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Randomized, Double-Blind, Placebo-Controlled, Multi-Center Trial of X4P-001 in Patients With WHIM (Warts, Hypogammaglobulinemia, Infections and Myelokathexis) Syndrome

Resource links provided by NLM:


Further study details as provided by X4 Pharmaceuticals:

Primary Outcome Measures:
  • Incidence of treatment-emergent adverse events (safety and tolerability) [ Time Frame: Up to 25 weeks, from time of enrollment through disease progression, study completion or early termination (Part A only) ]
  • Absolute Neutrophil Counts (ANC) over time and/or [ Time Frame: Up to 21 weeks, from time of enrollment (Part B Treatment) through Week 21 or early termination. ]
  • Absolute Lymphocyte Counts (ALC) over time. [ Time Frame: Up to 21 weeks, from time of enrollment (Part B Treatment) through Week 21 or early termination. ]

Secondary Outcome Measures:
  • Frequency of infections [ Time Frame: Up to 25 weeks, from time of enrollment (Part B Treatment) through study completion or early termination. ]
  • Severity of infections [ Time Frame: Up to 25 weeks, from time of enrollment (Part B Treatment) through study completion or early termination. ]
  • Area of skin involved with warts [ Time Frame: Up to 25 weeks, from time of enrollment (Part B Treatment) through study completion or early termination. ]
  • Severity of genital warts [ Time Frame: Up to 25 weeks, from time of enrollment (Part B Treatment) through study completion or early termination. ]
  • Antibody levels following revaccination [ Time Frame: Up to 25 weeks, from time of enrollment (Part B Treatment) through study completion or early termination. ]
  • Frequency of events requiring rescue therapy [ Time Frame: Up to 25 weeks, from time of enrollment (Part B Treatment) through study completion or early termination. ]

Estimated Enrollment: 33
Study Start Date: December 2016
Estimated Study Completion Date: August 2019
Estimated Primary Completion Date: May 2019 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: X4P-001
CXCR4 antagonist
Drug: X4P-001
CXCR4 antagonist
Other Name: AMD11070
Placebo Comparator: Placebo
Matched placebo capsules
Drug: Placebo
Matched placebo capsules

Detailed Description:

The primary objective of Phase 2 is to determine the safety, tolerability, and dose selection of X4P‑001 for Phase 3 in patients with WHIM syndrome.

The primary objective of Phase 3 is to demonstrate that X4P-001 is an effective treatment for patients with WHIM syndrome, as demonstrated by sustained increases in circulating neutrophils and lymphocytes that are associated with improvements in clinical manifestations of WHIM syndrome.

  Eligibility

Ages Eligible for Study:   13 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

To be eligible for this study, a patient must meet all of the following inclusion criteria:

  1. Be at least 18 years of age for Phase 2; at least 13 years of age for Phase 3.
  2. Has signed the current approved informed consent form; patients under 18 years of age will sign an approved informed assent form and must also have a signed parental/legal guardian consent.
  3. Has a genotype-confirmed mutation of CXCR4 consistent with WHIM syndrome.
  4. For women of childbearing potential and men, agree to use effective contraceptive methods from screening, through the study, and for at least 4 weeks after the last dose of study drug.
  5. Be willing and able to comply with this protocol.

Exclusion Criteria:

In the criteria below, "prior to Day 1" refers to Day 1 of treatment in Phase 2 and of the Observation Period in Phase 3. Patients with any of the following will be excluded from participation in the study:

  1. Has known systemic hypersensitivity to X4P-001.
  2. Is pregnant or nursing.
  3. Has, at screening, laboratory tests meeting one or more of the following criteria:

    • A positive or indeterminate screening test for antibody to human immunodeficiency virus (HIV) type 1 or 2.
    • A positive antibody test for hepatitis C virus (HCV), unless documented to have no detectable viral load on two independent samples.
    • A positive test for hepatitis B surface antigen (HBsAg).
  4. Has, at screening, safety laboratory tests meeting one or more of the following criteria:

    • Hemoglobin <8.0 g/dL
    • Platelets <75,000/μL
    • Creatinine >2.0 x the upper limit of normal (ULN)
    • Serum aspartate transaminase (AST) >2.5x ULN
    • Serum alanine transaminase (ALT) >2.5x ULN
    • Total bilirubin >1.5x ULN (unless due to Gilbert's Syndrome)
    • International normalized ratio (INR) >1.5x ULN
  5. Has, within 6 months prior to Day 1, received Plerixafor (open-label or blinded) as treatment of WHIM Syndrome.
  6. Has, within the 4 weeks prior to Day 1, had surgery requiring general anesthesia.
  7. Has, within 2 weeks prior to Day 1, received any of the following treatments:

    • G-CSF or granulocyte-macrophage colony-stimulating factor (GM-CSF)
    • Intravenous immunoglobulin (IVIG)
    • Corticosteroids (>10 mg prednisone equivalent per day)
    • Investigational therapies should be discussed with the Medical Monitor
  8. Has, within 2 weeks prior to Day 1, received a medication prohibited based on Cytochrome P450 3A4 (CYP3A4) and/or P-glycoprotein 1 (P‑gp) interaction.
  9. Has, at the planned initiation of study drug, an uncontrolled and active infection (excluding warts).
  10. Has any other medical or personal condition that, in the opinion of the Investigator, may potentially compromise the safety or compliance of the patient, or may preclude the patient's successful completion of the clinical study.
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT03005327

Contacts
Contact: Ramsey Johnson, MSM patientinfo@x4pharma.com

Locations
United States, Washington
University of Washingington Medical Center Recruiting
Seattle, Washington, United States, 98195
Sponsors and Collaborators
X4 Pharmaceuticals
Investigators
Study Director: Lu Gan, MD, PhD X4 Pharmaceuticals, Inc.
  More Information

Responsible Party: X4 Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03005327     History of Changes
Other Study ID Numbers: X4P-001-MKKA
Study First Received: December 20, 2016
Last Updated: February 8, 2017

Additional relevant MeSH terms:
Syndrome
Disease
Pathologic Processes

ClinicalTrials.gov processed this record on September 21, 2017