Pheripheral blood stem cells for Chronic Granulomatous Disease

Phase-Based Progress Estimates
1
Effectiveness
1
Safety
National Institutes of Health Clinical Center, Bethesda, MDChronic Granulomatous DiseasePheripheral blood stem cells - Biological
Eligibility
4 - 65
All Sexes
What conditions do you have?
Select

Study Summary

This trial is testing a new drug treatment to see if it can improve the success rates of stem cell transplants in people with CGD.

Eligible Conditions
  • Chronic Granulomatous Disease

Treatment Effectiveness

Effectiveness Progress

1 of 3

Study Objectives

1 Primary · 5 Secondary · Reporting Duration: 1 years post-transplant.

1 years post-transplant.
Overall Survival
30 days post transplant.
Decreased engraftment syndrome.
Year 1
To assess kinetics of engraftment
Year 2
To assess the infection rates in the setting of Alemtuzumab (Campath-1H) and post-transplant cyclophosphamide with the addition of an Il-6 antagonist and TNF inhibitor.
Year 1
Engraftment without GvHD
Eligibility determination for protocol enrollment
Transplantation, Homologous

Trial Safety

Safety Progress

1 of 3

Trial Design

2 Treatment Groups

Arm 1 / Group 1 Standard Risk
1 of 2
Arm 2 / Group 2 High Risk.
1 of 2

Active Control

Experimental Treatment

50 Total Participants · 2 Treatment Groups

Primary Treatment: Pheripheral blood stem cells · No Placebo Group · Phase 1 & 2

Arm 2 / Group 2 High Risk.Experimental Group · 7 Interventions: Emapalumab-Izsg, Cyclophosphamide, Busulfan, Sirolimus, Alemtuzumab, Pheripheral blood stem cells, Tociluzumab · Intervention Types: Drug, Drug, Drug, Drug, Drug, Biological, Drug
Arm 1 / Group 1 Standard RiskActiveComparator Group · 7 Interventions: Cyclophosphamide, Busulfan, Sirolimus, Alemtuzumab, Pheripheral blood stem cells, Tociluzumab, Total Body Irradiation · Intervention Types: Drug, Drug, Drug, Drug, Biological, Drug, Drug
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Cyclophosphamide
1995
Completed Phase 3
~3920
Busulfan
2008
Completed Phase 3
~1110
Sirolimus
2013
Completed Phase 4
~2750
Alemtuzumab
2004
Completed Phase 4
~1980

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: 1 years post-transplant.

Who is running the clinical trial?

National Institute of Allergy and Infectious Diseases (NIAID)Lead Sponsor
3,165 Previous Clinical Trials
4,936,971 Total Patients Enrolled
Elizabeth M Kang, M.D.Principal InvestigatorNational Institute of Allergy and Infectious Diseases (NIAID)
10 Previous Clinical Trials
334 Total Patients Enrolled

Eligibility Criteria

Age 4 - 65 · All Participants · 13 Total Inclusion Criteria

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References

Frequently Asked Questions

What is the total sample size of this experiment?

"Affirmative, clinicaltrials.gov data indicates that this medical trial is actively recruiting its targeted 50 patients from a single site. It has been available since 8th July 2022 and was last modified on 23rd July 2022." - Anonymous Online Contributor

Unverified Answer

Who is able to partake in this clinical analysis?

"To be considered for this study, applicants must possess granuloma and have a minimum age of 4 years old up to 65. Approximately 50 people will join the trial." - Anonymous Online Contributor

Unverified Answer

Are there any additional opportunities for enrolment in this clinical research?

"Affirmative, the data published on clinicaltrials.gov affirms that this research project is actively searching for volunteers. It was initially posted on July 8th 2022 and recently amended on July 23rd 2022. The investigation seeks 50 participants from just one site." - Anonymous Online Contributor

Unverified Answer

Does this medical research accept participants who have reached the age of majority?

"This medical trial is seeking participants who are between 4 and 65 years old." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.