Stem Cell Transplant + Cyclophosphamide for Chronic Granulomatous Disease

Recruiting in Palo Alto (17 mi)

+1 other location

Overseen by Elizabeth M Kang, M.D.

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Waitlist Available

Sponsor: National Institute of Allergy and Infectious Diseases (NIAID)

No Placebo Group

Breakthrough Therapy

Trial Summary

What is the purpose of this trial?

Chronic granulomatous disease (CGD) affects white blood cell function. Currently, the only curative treatment is bone marrow transplant to replace the abnormal stem cells with new ones (donor cells) capable of making a normal immune system. Transplant problems include graft versus host disease (GvHD) and graft rejection. With GvHD, donor cells attack the recipient s normal tissue. Researchers want to use preparation drugs and a high cell dose to increase graft success. They want to use 2 immunosuppressive drugs (cyclophosphamide and sirolimus) to lessen the risk of GvHD.

Eligibility Criteria

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.

Inclusion Criteria

Must provide a durable power of attorney for health care decisions to an appropriate adult relative or guardian in accordance to NIH -200 NIH Durable Power of Attorney for Health Care Decision Making.

If of child-bearing potential, must agree to consistently use contraception from one month prior to, and throughout, study participation, and for 3 months post-study. Acceptable forms of contraception are: Contraceptive pills or patch, Norplant , Depo-Provera , or other FDA-approved contraceptive method Male partner has previously undergone a vasectomy. Male participants will be advised to consistently use contraception throughout study participation and for 3 months post-transplant.

Must be HIV negative

Exclusion Criteria

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

My CRP levels were over 100 but are now under 100 after treatment.

I have seizures that are not controlled by medication.

Participant Groups

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.

Group I: 1 Active Control 6 Interventions

There is only one treatment arm for this study

Busulfan is already approved in United States, European Union, Canada, Japan for the following indications:

🇺🇸 Approved in United States as Busulfex for:

• Chronic myeloid leukemia
• Acute myeloid leukemia
• Malignant lymphoma
• Bone marrow transplantation conditioning

🇪🇺 Approved in European Union as Busulfan for:

• Chronic myeloid leukemia
• Acute myeloid leukemia
• Bone marrow transplantation conditioning

🇨🇦 Approved in Canada as Busulfex for:

• Chronic myeloid leukemia
• Acute myeloid leukemia
• Bone marrow transplantation conditioning

🇯🇵 Approved in Japan as Busulfan for:

• Chronic myeloid leukemia
• Acute myeloid leukemia
• Bone marrow transplantation conditioning

Find a Clinic Near You

Research Locations Nearby Select from list below to view details:

National Institutes of Health Clinical Center, 9000 Rockville Pike Bethesda, MD

National Institutes of Health Clinical Center Bethesda, MD

Who Is Running the Clinical Trial?

National Institute of Allergy and Infectious Diseases (NIAID) Lead Sponsor

References