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Chemotherapy

Stem Cell Transplant + Cyclophosphamide for Chronic Granulomatous Disease

Phase 1 & 2

Waitlist Available

Led By Elizabeth M Kang, M.D.

Research Sponsored by National Institute of Allergy and Infectious Diseases (NIAID)

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Must have confirmed Chronic Granulomatous Disease.

Must have sufficient complications from underlying disease to warrant undergoing transplantation (either a history of or ongoing inflammation/CGD related autoimmunity OR a CGD related infection while on prophylaxis) OR or have a Quartile 1 and/or 2 residual oxidase production level.

Must not have

Eastern Cooperative Oncology Group (ECOG) or equivalent performance status greater than or equal to 3

Uncontrolled seizure disorder

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 5 years

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a new way to give a bone marrow transplant to people with a disease that affects white blood cell function.

Who is the study for?

This trial is for people aged 4-65 with Chronic Granulomatous Disease (CGD) who have serious complications from it or low enzyme levels. They need a matched stem cell donor, must be HIV negative, and able to stay near NIH post-transplant with a companion. Participants of childbearing potential must use contraception.

What is being tested?

The study tests high dose stem cell transplantation using drugs like Alemtuzumab, Busulfan, Sirolimus, Cyclophosphamide and Total Body Irradiation in CGD patients to replace faulty immune cells and reduce GvHD risk.

What are the potential side effects?

Possible side effects include reactions to the infusion of drugs or cells, organ damage due to drug toxicity or radiation exposure, increased infection risk from immunosuppressants, and fertility issues related to treatment.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have been diagnosed with Chronic Granulomatous Disease.

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I need a transplant due to severe complications or low oxidase production.

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I am between 4 and 65 years old.

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I have been diagnosed with Chronic Granulomatous Disease.

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I need a transplant due to severe complications or low enzyme levels in my blood.

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I have a donor match for a stem cell transplant.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I need help with my daily activities due to my health condition.

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I have seizures that are not controlled by medication.

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I am younger than 65 years old.

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I do not have any major illnesses or organ failures that would make me ineligible for a stem cell transplant.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and 5 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

To determine engraftment rates with the use of high cell doses, without increasing the risk of GvHD by using post-transplant cyclophosphamide and sirolimus in conjunction with a busulfan- based conditioning regimen. We will compare the incidence...

Secondary study objectives

To assess the level and kinetics of immune reconstitution (via chimerism) when using post- transplant cyclophosphamide

To further elucidate the factors involved in the development of GvHD and graft rejection/failure

To measure the engraftment rate and the engraftment kinetics using such a regimen