← Back to Search

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.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 5 years

Awards & highlights

Study 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.Check my eligibility

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.See study design

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

Select...

I have been diagnosed with Chronic Granulomatous Disease.

Select...

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

Select...

I am between 4 and 65 years old.

Select...

I have been diagnosed with Chronic Granulomatous Disease.

Select...

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

Select...

I have a donor match 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

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

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 outcome measures

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

Trial Design

1Treatment groups

Active Control

Group I: 1Active Control6 Interventions

There is only one treatment arm for this study

0 / 6Eligibility criteria met

Find a Location

Who is running the clinical trial?

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

3,268 Previous Clinical Trials

5,481,088 Total Patients Enrolled

Elizabeth M Kang, M.D.Principal InvestigatorNational Institute of Allergy and Infectious Diseases (NIAID)

11 Previous Clinical Trials

350 Total Patients Enrolled

Media Library

Busulfan (Chemotherapy) Clinical Trial Eligibility Overview. Trial Name: NCT02629120 — Phase 1 & 2

Chronic Granulomatous Disease Research Study Groups: 1

Chronic Granulomatous Disease Clinical Trial 2023: Busulfan Highlights & Side Effects. Trial Name: NCT02629120 — Phase 1 & 2

Busulfan (Chemotherapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT02629120 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is the age range for this experimental research accommodating seniors?

"This research endeavour is currently seeking individuals aged between 4 and 65 to take part."

Answered by AI

Are there still vacancies available in this medical experiment?

"As per clinicaltrials.gov, this research is actively recruiting participants and was initially publicized in December 17th 2015 with the most recent update occurring on November 2nd 2022."

Answered by AI

How many volunteers are participating in this experiment?

"Affirmative. Clinicaltrials.gov records that the clinical trial, whose opening date was 17 December 2015, is actively recruiting participants. Fifty individuals need to be sourced from 2 different medical centres."

Answered by AI

Are there any restrictions for participation in this clinical study?

"To partake in this clinical trial, study participants must have a prior transplant of chronic granulomatous disease and be aged between 4 and 65 years old. This medical research is enrolling up to 50 people."

Answered by AI

Could you detail the other experiments conducted utilizing 1?

"Currently, 962 clinical trials are underway, 172 of which have entered Phase 3. Philadelphia in Pennsylvania is the main hub for investigations into 1; however, there are 29046 sites on record that offer this medication to patients."

Answered by AI

What is the typical application of 1?

"1 has proven to be a highly effective treatment for lung cancer, as well as for organ transplantation, multiple sclerosis and acute myelocytic leukemia."

Answered by AI

Recent research and studies

formation/NLM/NIH, NCBI. “Gprobe”. 2017. Web. <https://github.com/ncbi/gprobe>..Journal

Gprobe

Information/NLM/NIH, NCBI. 2017. “Gprobe”. GitHub repository. https://github.com/ncbi/gprobe.

Experimental HematologyJournal

Busulfan Pharmacokinetics, Toxicity, and Low-dose Conditioning for Autologous Transplantation of Genetically Modified Hematopoietic Stem Cells in the Rhesus Macaque Model

Kang, Elizabeth M., Matthew M. Hsieh, Mark Metzger, Allen Krouse, Robert E. Donahue, Michel Sadelain, and John F. Tisdale. 2006. “Busulfan Pharmacokinetics, Toxicity, and Low-dose Conditioning for Autologous Transplantation of Genetically Modified Hematopoietic Stem Cells in the Rhesus Macaque Model”. Experimental Hematology. Elsevier BV. doi:10.1016/j.exphem.2005.10.010.

New England Journal of MedicineJournal

Treatment of Chronic Granulomatous Disease with Nonmyeloablative Conditioning and a T-cell–depleted Hematopoietic Allograft

Horwitz, Mitchell E., A. John Barrett, Margaret R. Brown, Charles S. Carter, Richard Childs, John I. Gallin, Ronald E. Gress, et al.. 2001. “Treatment of Chronic Granulomatous Disease with Nonmyeloablative Conditioning and a T-cell–depleted Hematopoietic Allograft”. New England Journal of Medicine. Massachusetts Medical Society. doi:10.1056/nejm200103223441203.

clinicaltrials.govStudy