Plerixafor Versus G-CSF in the Treatment of People With WHIM Syndrome

• Register: ClinicalTrials.gov
• Last refreshed on: 17 April 2023
• Main ID: NCT02231879
• Date of registration: 03/09/2014
• Prospective Registration: Yes
• Primary sponsor: National Institute of Allergy and Infectious Diseases (NIAID)
• Public title: Plerixafor Versus G-CSF in the Treatment of People With WHIM Syndrome
• Scientific title: A Phase III Double-Blind Randomized Crossover Study of Plerixafor Versus G-CSF in the Treatment of Patients With WHIM Syndrome.
• Date of first enrolment: October 14, 2014
• Target sample size: 20
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02231879
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Crossover Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2/Phase 3

Countries of recruitment

United States

Contacts

• Name: David H McDermott, M.D.
• Affiliation: National Institute of Allergy and Infectious Diseases (NIAID)

Key inclusion & exclusion criteria

- INCLUSION CRITERIA:

Subjects are eligible to enter the study if they meet all of the following criteria:

1. Age greater than or equal to 10 and less than or equal to 75 years.

2. Heterozygous mutation in the C-tail of CXCR4 in addition to a clinical diagnosis of
WHIMS.

3. Documented neutropenia with a baseline ANC below 1500 cells/microL of blood.

4. History of severe and/or recurrent infections.

5. Willingness to interrupt G-CSF medication, 2 days prior to study drug injection.

6. Must have a local medical provider for medical management.

7. Must be willing to provide blood, plasma, serum, and DNA samples for storage.

8. All study subjects must agree not to become pregnant or impregnate a female. Women of
childbearing potential must agree to take appropriate steps to avoid becoming pregnant
for the duration of the study. Participants in whom pregnancy is biologically possible
must use at least 2 study approved methods of contraception, one of which must be a
barrier method, and must continue contraception until 5 months after stopping the
study drug:

- Male or female condoms with a spermicide,

- Diaphragm or cervical cap with spermicide,

- Intrauterine device,

- Contraceptive pills or patch, Norplant, Depo-Provera or other FDA-approved
contraceptive,

- Male partner with vasectomy and documented aspermatogenic sterility.

9. Willingness to comply with the study medications, visits, and procedures, as deemed
necessary by the principal investigator (PI).

EXCLUSION CRITERIA:

If any of the following exclusion criteria are met, a subject will not be enrolled in this
study:

1. Neutropenia due to maturation defects in the myeloid lineage or a type of neutropenia,
which in the investigator s opinion, is unlikely to improve from the medication
administered in this study.

2. Pregnant or breast-feeding women.

3. Known hypersensitivity to plerixafor, G-CSF, or any components of the products.

4. Predisposition to or history of life-threatening cardiac arrhythmia.

5. Requiring dialysis or having markedly impaired renal function with a Creatinine
Clearance (CrCl) <15 mL/min.

6. Condition that in the investigator s opinion places a subject at undue risk by
participating in the study.



Age minimum: 10 Years
Age maximum: 75 Years
Gender: All

Health Condition(s) or Problem(s) studied

Infections

Warts

Hypogammaglobulinemia

Neutropenia

Myelokathexis

Intervention(s)

Drug: Plerixafor

Drug: G-CSF

Primary Outcome(s)

Difference in Total Infection Severity Score [Time Frame: Scores from 12 months treatment on each study drug]

Severity of Infection [Time Frame: Scores from 12 months treatment on each study drug]

Secondary ID(s)

14-I-0185

140185

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 07/04/2023
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02231879