Haploidentical Transplant for People With Chronic Granulomatous Disease Using Post Transplant Cyclophosphamide
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|ClinicalTrials.gov Identifier: NCT02282904|
Recruitment Status : Terminated
First Posted : November 5, 2014
Results First Posted : May 1, 2020
Last Update Posted : May 12, 2020
- Chronic Granulomatous Disease (CGD) causes immune system problems. Treatment is usually a bone marrow transplant from a fully matched donor. Researchers want to try using partially matched donors for patients who do not have a fully matched donor available. The researchers will also use the drug cyclophosphamide to try to improve the outcomes when using a partially matched donor.
- To learn the effectiveness of using cyclophosphamide with a transplant from a partially matched donor in treating CGD.
- Recipients: age 2-65 with CGD with an ongoing infection that has not been cured by standard treatment and no fully matched donor available in an appropriate timeframe.
- be admitted to the hospital 2 weeks before transplant.
- be screened with blood and urine tests, breathing and heart health tests, X-rays, and/or magnetic resonance imaging. They may have a bone marrow aspiration and biopsy.
- meet with a social worker and dentist.
- get chemotherapy, radiation, and other medicines.
- get an intravenous (IV) catheter in their chest.
- have the transplant.
- get more medicines and standard supportive care.
- have blood drawn frequently.
- have to stay in the Washington, D.C. area for 3 months post-transplant.
- be followed closely for the first 6 months, and then less frequently for at least 5 years.
|Condition or disease||Intervention/treatment||Phase|
|Chronic Granulomatous Disease||Drug: Sirolimus Biological: Donor peripheral blood stem cells. Drug: Cyclophosphamide post transplant Radiation: Total body 200cGy Drug: Cyclophosphamide Drug: Fludarabine Drug: Busulfan||Phase 1 Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||7 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Haploidentical Transplant for Patients With Chronic Granulomatous Disease (CGD) Using Post-Transplant Cyclophosphamide|
|Study Start Date :||October 23, 2014|
|Actual Primary Completion Date :||April 10, 2019|
|Actual Study Completion Date :||December 10, 2019|
Experimental: CGD Recipient
CGD patients that will undergo haplo transplantation with post-transplant cyclophosphamide as described
For pediatric patients: Begin sirolimus 1 mg/m2 PO q4h for 3 doses, then 1 mg/m2 once a day (QD). For adult patients, begin sirolimus 5 mg PO q4h for 3 doses, then 5 mg once a day (QD).
Doses may be adjusted to maintain trough levels between 8-14 ng/ml. Recipients will take sirolimus from Day +5 to at least Day 100 (minimum).
Other Name: Rapamycin
Biological: Donor peripheral blood stem cells.
Infuse donor graft.
Drug: Cyclophosphamide post transplant
50 mg/kg/d IV infused over 90 minutes. Day +3 and +4
Other Name: Cytoxan post transplant
Radiation: Total body 200cGy
14.5 mg/kg IV over one hour Day -6 and -5
Other Name: Cytoxan
30 mg/m2 over 30 minutes Day -6 through Day -2
Other Name: Fludara
Busulfan 3.2 mg/kg IV once daily over 2-3 hours Day -4,-3,-2
Other Name: Busulfex
- To Determine the Efficacy of This Allogeneic Transplant Approach in Reconstituting Normal Hematopoiesis and Reversing the Clinical Phenotype of CGD [ Time Frame: 5 years ]Patient will have donor chimerism of greater than 20% and resolution of infection or autoimmunity at end of follow up
- 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 ]1. Stable chimerism as indicated by 30-50% myeloid engraftment and 50% lymphoid engraftment as assessed by 1 year post transplant. 2. Immune reconstitution levels with DHR as a marker of normal neutrophil function by 1 year post transplant. 3. GvHD grades of less than 3.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02282904
|United States, Maryland|
|National Institutes of Health Clinical Center, 9000 Rockville Pike|
|Bethesda, Maryland, United States, 20892|
|Principal Investigator:||Elizabeth M Kang, M.D.||National Institute of Allergy and Infectious Diseases (NIAID)|