Haploidentical Transplant for People With Chronic Granulomatous Disease Using Post Transplant Cyc… (NCT02282904) | Clinical Trial Compass
TerminatedPhase 1/2
Haploidentical Transplant for People With Chronic Granulomatous Disease Using Post Transplant Cyclophosphamide
United States7 participantsStarted 2014-10-23
Plain-language 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.
Who can participate
Age range
2 Years – 65 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
* 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 f…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
To Determine the Efficacy of This Allogeneic Transplant Approach in Reconstituting Normal Hematopoiesis and Reversing the Clinical Phenotype of CGD
Timeframe: 5 years
Trial details
NCT IDNCT02282904
SponsorNational Institute of Allergy and Infectious Diseases (NIAID)