Phase 2 N=15 Treatment

Matched Unrelated or Non-Genotype Identical Related Donor Transplantation For Chronic Granulomatous Disease

Chronic Granulomatous Disease

Bottom Line

View on ClinicalTrials.gov: NCT00578643 ↗

Enrolled (actual)

Serious AEs

60.0%

Results posted

Nov 2018

Primary outcome: Primary: Percentage of Participants With Engraftment — 100 percentage of participants

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: Busulfan (Drug); Alemtuzumab (Biological); Cyclophosphamide (Drug); Fludarabine (Drug); Cyclosporine (Drug); Stem Cell Infusion (Procedure)
Age: Pediatric, Adult, Older Adult
Sex: All
Sponsor: Baylor College of Medicine
Primary completion: Jul 2017

Outcome Measures

Outcome	Result	p-value
PRIMARY Percentage of Participants With Engraftment	100	—
SECONDARY Number of Patients That Have Complete Donor Chimerism After Transplant.	13	—
SECONDARY Number of Patients That Have Acute GVHD and Regimen Related Morbidity/Mortality Post Transplant.	8; 6; 0; 0	—
SECONDARY Number of Patients That Have Chronic GVHD and Regimen Related Morbidity/Mortality Post Transplant.	0; 13	—

Summary

This study is for patients with chronic granulomatous disease (CGD), which is a disorder of the immune system that puts them at risk for severe infections. CGD is caused by a genetic defect that stops or prevents the white blood cells from killing certain bacteria and fungi. This condition cannot presently be cured by standard treatment with drugs or surgery. Medicine including antibiotics, antifungals, and interferon gamma, may help some patients with CGD; however even with continuous treatment, most patients with CGD will have chronic and recurrent infections. Transfusion of white blood cells may help overcome infection, but white cell transfusions lead to allergic reactions and fever and the benefit of transfusion lasts only a matter of hours. Ultimately, chronic infections can damage or injure the body organs. Injury to the lung or liver can lead to lung or liver failure and death. Medicines used to treat infection can damage body organs too. Infections may become resistant to antibiotic or antifungal treatment, and infections not responding to treatment can be deadly. It is now known that under specific conditions and with special treatment, blood stem cells (the cells that make blood) can be transplanted from one person to another. Stem cell transplantation has been done for patients with CGD who have a healthy sibling and who share the same immune type (HLA type) as the patient. Stem cell transplantation allows healthy or normal white cells from the stem cell donor to grow or develop in the patient's bone marrow. These healthy white cells can fight infection and prevent future infections for a patient with CGD. Patients on this study will receive stem cells from a related or unrelated donor. The donor will be closely matched to the patient's immune type but the donor is not a sibling. The reason this treatment is investigational is that we do not know the likelihood of benefit that the patient will receive. It is possible that they will have great benefit, like some of the patients who have been transplanted from a brother or sister. It is possible that the side-effects of treatment may be too severe so that the transplant won't work. The purpose of this research study is to evaluate whether or not patients with CGD treated with a stem cell transplant from a non-matched and/or non-related donor can have a good outcome from the procedure with an acceptable number of side-effects.

Eligibility Criteria

INCLUSION CRITERIA

CGD patients as documented by an abnormal NBT assay in a male patient and/or abnormal NADPH enzyme mutation confirmed by genetic analysis with abnormal NBT.

Patients must not have an HLA genotype identical donor.

Patients must have a 5/6 or 6/6 HLA-matched unrelated donor or a 5/6 or 6/6 HLA phenotype-matched related donor.

Patients must have had at least one serious infection characteristic of those manifested in patients with CGD.

Patients must not have active infection. An active infection may include the following: 1) clinical findings consistent with an infection such as fever, cavitary organ lesions, osteomyelitis; 2) progression of presumed infection based upon findings of diagnostic imaging (two or more studies at least 1 month a part).

No cumulative organ dysfunction that, in the estimation of the treating physicians, will diminish the patient's likelihood to survive this procedure.

Negative pregnancy test for post-pubertal female patients.

Echocardiogram shortening fraction >/= 28%.

DLCO 50% or greater predicted or FEV1 >/= 50% predicted.

EXCLUSION CRITERIA

Active or uncontrolled infection (e.g. lung infection, cavitary organ lesions, osteomyelitis).

Markedly elevated C reactive protein or sedimentation rate relative to patient's baseline.

Invasive bone or bone marrow disease.

Lack of potential hematologic blood product donors in the past (related to McLeod phenotype).

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Data sourced from ClinicalTrials.gov (NCT00578643). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.