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WHIM Syndrome

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

Sponsored by X4 Pharmaceuticals

About this trial

Last updated 7 years ago

Study ID

X4-WHIM-NTHX

Status

Withdrawn

Type

Observational

Placebo

No

Accepting

18-75 Years
All
All
All

Not accepting

Not accepting
Healthy Volunteers

Trial Timing

Ended 3 years ago

What is this trial about?

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.

What are the participation requirements?

Yes

Inclusion Criteria

1. Has a confirmed clinical diagnosis of WHIM syndrome.

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 consent.

3. Be willing and able to comply with the study protocol.

No

Exclusion Criteria

1. Has, within 6 months prior to Day 1, received a CXCR4 antagonist.

2. Currently participating in an investigational study for treatment of WHIM.

3. 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.