ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 2 of 767 for:    X4

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03005327
Recruitment Status : Recruiting
First Posted : December 29, 2016
Last Update Posted : January 10, 2018
Sponsor:
Information provided by (Responsible Party):
X4 Pharmaceuticals

Brief Summary:
The overall objective is to evaluate X4P-001 in the treatment of patients with WHIM Syndrome.

Condition or disease Intervention/treatment Phase
WHIM Syndrome Drug: X4P-001 Drug: Placebo Phase 2 Phase 3

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.


Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 42 participants
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
Study Start Date : December 2016
Estimated Primary Completion Date : May 2019
Estimated Study Completion Date : August 2019

Resource links provided by the National Library of Medicine

U.S. FDA Resources

Arm Intervention/treatment
Experimental: X4P-001
CXCR4 antagonist
Drug: X4P-001
CXCR4 antagonist
Other Name: AMD11070
Placebo Comparator: Placebo
Matched placebo capsules
Drug: Placebo
Matched placebo capsules



Primary Outcome Measures :
  1. 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) ]
  2. 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. ]
  3. 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 :
  1. Frequency of infections [ Time Frame: Up to 25 weeks, from time of enrollment (Part B Treatment) through study completion or early termination. ]
  2. Severity of infections [ Time Frame: Up to 25 weeks, from time of enrollment (Part B Treatment) through study completion or early termination. ]
  3. Number of warts [ Time Frame: Up to 25 weeks, from time of enrollment (Part B Treatment) through study completion or early termination. ]
  4. Severity of genital warts [ Time Frame: Up to 25 weeks, from time of enrollment (Part B Treatment) through study completion or early termination. ]
  5. Antibody levels following revaccination [ Time Frame: Up to 25 weeks, from time of enrollment (Part B Treatment) through study completion or early termination. ]
  6. 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. ]


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.


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

Inclusion Criteria:

Patients with a clinical diagnosis of WHIM syndrome must meet all of the following criteria to be eligible for study participation:

  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. Agree to use effective contraception as follows:

    • women of childbearing potential (WOCBP) agree to use highly effective contraceptive methods from Screening, through the study, and for at least 4 weeks after the last dose of study drug (see Section 7.4.1.1 for the definition of non-childbearing potential.)
    • males, agree to use a condom with any WOCBP sexual partner from Day 1 of study treatment, through the study, and 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 a known history of a positive serology or viral load for HIV or a known history of AIDS.
  4. Has, at Screening, laboratory tests meeting one or more of the following criteria:

    • 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).
  5. 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
  6. Has, within 2 months prior to Day 1 for Phase 2 and within 6 months prior to Day 1 for Phase 3, received Plerixafor (open-label or blinded) as treatment of WHIM Syndrome.
  7. Has, within the 4 weeks prior to Day 1, had surgery requiring general anesthesia.
  8. Has, within 2 weeks prior to Day 1, received any of the following treatments:

    • G-CSF or granulocyte-macrophage colony-stimulating factor (GM-CSF)
    • Immunoglobulin - Intravenous or subcutaneous (unless deemed necessary by the treating physician and after agreement from the Sponsor)
    • Corticosteroids (>10 mg prednisone equivalent per day)
    • Investigational therapies should be discussed with the Medical Monitor
  9. 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.
  10. Has, at the planned initiation of study drug, an uncontrolled and active infection (excluding warts).
  11. 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.

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): NCT03005327


Contacts
Contact: Ramsey Johnson, MSM 857-529-8308 patientinfo@x4pharma.com
Contact: Tarek Ebrahim, MD 857-529-8304 patientinfo@x4pharma.com

Locations
United States, Florida
University of South Florida Recruiting
Saint Petersburg, Florida, United States, 33701
United States, Michigan
University of Michigan Recruiting
Ann Arbor, Michigan, United States, 48109
United States, Washington
University of Washingington Medical Center Recruiting
Seattle, Washington, United States, 98195
Australia, Victoria
St. Vincent's Hospital Recruiting
Fitzroy, Victoria, Australia, 3065
Sponsors and Collaborators
X4 Pharmaceuticals
Investigators
Study Director: Sudha Parasuraman, MD X4 Pharmaceuticals

Responsible Party: X4 Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03005327     History of Changes
Other Study ID Numbers: X4P-001-MKKA
First Posted: December 29, 2016    Key Record Dates
Last Update Posted: January 10, 2018
Last Verified: January 2018

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Syndrome
Disease
Pathologic Processes