Ignite Creation Date:
2025-12-24 @ 8:02 PM
Ignite Modification Date:
2026-01-04 @ 3:43 AM
Study NCT ID:
NCT02282904
Status:
TERMINATED
Last Update Posted:
2020-05-12
First Post:
2014-11-04
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Haploidentical Transplant for People With Chronic Granulomatous Disease Using Post Transplant Cyclophosphamide
Sponsor:
National Institute of Allergy and Infectious Diseases (NIAID)
Organization:
Study Overview
Official Title:
Haploidentical Transplant for Patients With Chronic Granulomatous Disease (CGD) Using Post-Transplant Cyclophosphamide
Status:
TERMINATED
Status Verified Date:
2019-12-10
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
Background:
\- 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.
Objective:
\- To learn the effectiveness of using cyclophosphamide with a transplant from a partially matched donor in treating CGD.
Eligibility:
\- 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.
Design:
* Recipients will:
* 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.
\- 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.
Objective:
\- To learn the effectiveness of using cyclophosphamide with a transplant from a partially matched donor in treating CGD.
Eligibility:
\- 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.
Design:
* Recipients will:
* 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.
Detailed Description:
Allogeneic transplant using HLA matched donors, both related and unrelated, has proven curative for patients with various immunodeficiencies, including those with ongoing infections. However donor availability remains a limiting factor in the application of this treatment modality. The use of haploidentical donors has in the past been fraught with a greater rate of complications related to both higher rates of GvHD and delayed immunorecovery. Newer transplant regimens appear to have diminished these risks and improved outcomes. We propose using a subablative conditioning regimen followed by post-transplant cyclophosphamide for patients with CGD who do not have an HLA matched donor but whose circumstances necessitate the use of a potentially curative, albeit high-risk treatment modality.
Study Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
False
Is an FDA AA801 Violation?:
Secondary ID Infos
| Secondary ID | Type | Domain | Link | View |
|---|---|---|---|---|
| 15-I-0007 | OTHER | NIH | View |