Haploidentical Transplant for People With Chronic Granulomatous Disease Using Post Transplant Cyclophosphamide

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02282904
• Date of registration: 04/11/2014
• Prospective Registration: No
• Primary sponsor: National Institute of Allergy and Infectious Diseases (NIAID)
• Public title: Haploidentical Transplant for People With Chronic Granulomatous Disease Using Post Transplant Cyclophosphamide
• Scientific title: Haploidentical Transplant for Patients With Chronic Granulomatous Disease (CGD) Using Post-Transplant Cyclophosphamide
• Date of first enrolment: October 23, 2014
• Target sample size: 7
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT02282904
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1/Phase 2

Countries of recruitment

United States

Contacts

• Name: Elizabeth M Kang, M.D.
• Affiliation: National Institute of Allergy and Infectious Diseases (NIAID)

Key inclusion & exclusion criteria

- INCLUSION CRITERIA:

- Must have sufficient complications from underlying disease to warrant undergoing
transplantation

- Ages 2 years - 65 years

- No appropriate HLA matched donor (available donor has greater than 1 mismatch or the
single mismatch is not at DQ for unrelated donors (including cord blood products), or
no available 6 out of 6 HLA matched related donor), or patients who may have an
unrelated donor, but whose clinical status is such that the time required to obtain an
unrelated donor would be life threatening.

- HLA haploidentical family donor graft available.

- Ability to comprehend and willingness to sign the informed consent or have a
parent/guardian consent if the donor is a minor; assent being obtained from minors as
appropriate

- Must be HIV negative

- Must not be pregnant (confirmed by a negative serum beta-human chorionic gonadotropin
(Beta-hCG) for women of child-bearing potential) or breastfeeding

- Must be able to stay within one hour s travel of the NIH for the first 3 months after
transplantation and have a family member or other designated companion to stay with
during the post-transplant period.

- Must provide a durable power of attorney for health care decisions to an appropriate
adult relative or guardian in accordance to NIH Form-200 NIH Durable Power of Attorney
for Health Care Decision Making.

- Where appropriate, subjects must agree to use contraception for 3 months
post-transplant

EXCLUSION CRITERIA:

- Major anticipated illness or organ failure incompatible with survival from
Allo-transplant

- Inadequate collection from prospective donors.



Age minimum: 2 Years
Age maximum: 65 Years
Gender: All

Health Condition(s) or Problem(s) studied

Chronic Granulomatous Disease

Intervention(s)

Drug: Cyclophosphamide

Radiation: Total body 200cGy

Drug: Fludarabine

Drug: Cyclophosphamide post transplant

Biological: Donor peripheral blood stem cells.

Drug: Busulfan

Drug: Sirolimus

Primary Outcome(s)

To Determine the Efficacy of This Allogeneic Transplant Approach in Reconstituting Normal Hematopoiesis and Reversing the Clinical Phenotype of CGD [Time Frame: 5 years]

Secondary Outcome(s)

To Determine the Safety of This Allogeneic HSCT Approach in Patients With CGD Including Transplant Related Toxicity, the Incidence of Acute and Chronic Graft-versus-host Disease, Immune Reconstitution, Overalland Disease-free Survival. [Time Frame: 1 year post transplant]

Secondary ID(s)

150007

15-I-0007

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 01/05/2020
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02282904