Clinical trials: Myelokathexis
WHIM Syndrome
A Retrospective and Prospective Natural History Study of Patients With WHIM Syndrome
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…
- Trial name
- NCT03087370
- Testing
- No intervention
- Sponsor
- X4 Pharmaceuticals
WHIM, Warts
Base-Edited Hematopoietic Stem/Progenitor Cell Gene Therapy for Treatment of CXCR4-WHIM
Background: Warts, hypogammaglobulinemia, infections and myelokathexis syndrome (WHIMs) is a rare disorder that affects the immune system. People with WHIMs can have severe infections all over their body.
- Conditions
- WHIM, Warts, Hypogammaglobulinemia, Immunodeficiency
- Trial name
- NCT07775313
- Testing
- Busulfan, Palifermin, Plerixafor
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
WHIM Syndrome
Efficacy and Safety Study of Mavorixafor in Participants With Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) Syndrome
This study has a double-blind, Randomized Placebo-Controlled Period and an Open-Label Period. The primary objective of the Randomized Placebo-Controlled Period is to demonstrate the efficacy of mavorixafor in participants with…
- Trial name
- NCT03995108
- Testing
- Mavorixafor
- Where
- 23 sites in United States, Australia and 11 more
- Volunteers
- 31 people
- Sponsor
- X4 Pharmaceuticals
Myelokathexis, Infections
Plerixafor Versus G-CSF in the Treatment of People With WHIM Syndrome
Background: WHIMS (Warts, Hypogammaglobulinemia, Infections, and Myelokathexis Syndrome) is a rare disease. It can cause cancers, infections, and warts. Researchers want to see if a drug called plerixafor can treat WHIMS.
- Conditions
- Myelokathexis, Infections, Neutropenia, Warts
- Trial name
- NCT02231879
- Testing
- Plerixafor, G-CSF
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)