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

NCT02282904 · Status: TERMINATED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 7

Last updated 2020-05-12

Study results available
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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.

Conditions

  • Chronic Granulomatous Disease

Interventions

DRUG

Sirolimus

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).

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

RADIATION

Total body 200cGy

Day -1

DRUG

Cyclophosphamide

14.5 mg/kg IV over one hour Day -6 and -5

DRUG

Fludarabine

30 mg/m2 over 30 minutes Day -6 through Day -2

DRUG

Busulfan

Busulfan 3.2 mg/kg IV once daily over 2-3 hours Day -4,-3,-2

Sponsors & Collaborators

  • National Institute of Allergy and Infectious Diseases (NIAID)

    lead NIH

Principal Investigators

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

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
2 Years
Max Age
65 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2014-10-23
Primary Completion
2019-04-10
Completion
2019-12-10
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02282904 on ClinicalTrials.gov