WithdrawnNot Applicable

A Retrospective and Prospective Natural History Study of Patients With WHIM Syndrome

Stopped: Company Decision

0Started 2018-06

Plain-language summary

This natural history study is a prospective and retrospective, observational study of WHIM patients. WHIM syndrome is a rare, genetic, primary immunodeficiency disorder (a disorder in which the body's immune system does not function properly). WHIM is an acronym for some of the symptoms of the disorder - Warts, Hypogammaglobulinemia (low levels of certain antibodies), Infections and Myelokathexis (too many white blood cells in the bone marrow).This study includes 10-year retrospective (Retrospective Phase) and up to 5-year prospective (Prospective Phase) components.

Who can participate

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Has a confirmed clinical diagnosis of WHIM syndrome.
✓. 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 consent.
✓. Be willing and able to comply with the study protocol.

Exclusion criteria

✕. Has, within 6 months prior to Day 1, received a CXCR4 antagonist.
✕. Currently participating in an investigational study for treatment of WHIM.
✕. 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.

What they're measuring

Incidence of infections

Timeframe: Up to five years, from time of enrollment through study completion or early termination

Severity of infections

Timeframe: Up to five years, from time of enrollment through study completion or early termination

Incidence of warts

Timeframe: Up to five years, from time of enrollment through study completion or early termination

Severity of warts

Timeframe: Up to five years, from time of enrollment through study completion or early termination

Change in quality of life over time

Timeframe: Up to five years, from time of enrollment through study completion or early termination

Change in quality of life over time

Timeframe: Up to five years, from time of enrollment through study completion or early termination

Change in quality of life over time

Timeframe: Up to five years, from time of enrollment through study completion or early termination

Trial details

NCT IDNCT03087370

SponsorX4 Pharmaceuticals

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2022-03

View on ClinicalTrials.gov More WHIM Syndrome trials

Plain-language summary

Who can participate

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Has a confirmed clinical diagnosis of WHIM syndrome.
✓. 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 consent.
✓. Be willing and able to comply with the study protocol.

Exclusion criteria

✕. Has, within 6 months prior to Day 1, received a CXCR4 antagonist.
✕. Currently participating in an investigational study for treatment of WHIM.
✕. 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.

What they're measuring

Incidence of infections

Timeframe: Up to five years, from time of enrollment through study completion or early termination

Severity of infections

Timeframe: Up to five years, from time of enrollment through study completion or early termination

Incidence of warts

Timeframe: Up to five years, from time of enrollment through study completion or early termination

Severity of warts

Timeframe: Up to five years, from time of enrollment through study completion or early termination

Change in quality of life over time

Timeframe: Up to five years, from time of enrollment through study completion or early termination

Change in quality of life over time

Timeframe: Up to five years, from time of enrollment through study completion or early termination

Change in quality of life over time

Timeframe: Up to five years, from time of enrollment through study completion or early termination