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Monoclonal Antibodies
Stem Cell Transplant + Drug Therapy for Chronic Granulomatous Disease
Phase 1 & 2
Recruiting
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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 1 years post-transplant.
Awards & highlights
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.
Who is the study for?
This trial is for people aged 4-65 with Chronic Granulomatous Disease (CGD) who have complications from the disease or a specific level of oxidase production. They must not be HIV positive, pregnant, or breastfeeding and should agree to use contraception. Participants need an HLA-matched stem cell donor and must stay close to the NIH post-transplant.Check my eligibility
What is being tested?
The study tests if a combination of drugs including Busulfan, Sirolimus, Cyclophosphamide, Alemtuzumab plus cytokine antagonists Emapalumab-Izsg and Tocilizumab can improve stem cell transplant success in CGD patients. It includes pre-transplant medication, total body irradiation, and follow-up for five years.See study design
What are the potential side effects?
Potential side effects may include immune system reactions due to Alemtuzumab, organ damage from radiation exposure during Total Body Irradiation (TBI), kidney issues from Busulfan or Cyclophosphamide use; infection risk could increase due to weakened immunity.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 1 years post-transplant.
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~1 years post-transplant.
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Overall Survival
Secondary outcome measures
Decreased engraftment syndrome.
Engraftment without GvHD
Transplantation, Homologous
+2 moreTrial Design
2Treatment groups
Experimental Treatment
Active Control
Group I: Arm 2 / Group 2 High Risk.Experimental Treatment7 Interventions
Group 2 (High risk group will receive tociluzumab at day -24, a repeat dose at day -19, Alemtuzumab at day -9,-8,-7,-6,-5; Busulfan at day -4 and -3, Emapalumab at Day -1, matched donor PBSC infusion, and post transplant cyclophosphamide at Day +3 and +4
Group II: Arm 1 / Group 1 Standard RiskActive Control7 Interventions
Group 1 (Standard group) will receive tociluzumab at Day -10, Alemtuzumab on Day -9,-8,-7,-6,-5; Busulfan on Day -4, and -3, TBI, matched donor PBSC infusion, and Post transplant Cyclophosphamide.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Busulfan
2008
Completed Phase 3
~1120
Sirolimus
2013
Completed Phase 4
~2750
Cyclophosphamide
1995
Completed Phase 3
~3770
Alemtuzumab
2004
Completed Phase 4
~1890
Find a Location
Who is running the clinical trial?
National Institute of Allergy and Infectious Diseases (NIAID)Lead Sponsor
3,269 Previous Clinical Trials
5,481,437 Total Patients Enrolled
Elizabeth M Kang, M.D.Principal InvestigatorNational Institute of Allergy and Infectious Diseases (NIAID)
11 Previous Clinical Trials
344 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I do not have an active TB infection.I have been diagnosed with Chronic Granulomatous Disease (CGD).I have a donor match for my transplant.My lung function tests show severe limitations.I am younger than 65 years old.I do not have any major illnesses that would make a stem cell transplant too risky.I have someone to help me after my transplant when I'm not in the hospital.My CRP levels will be checked 6 to 7 weeks before my transplant.I need a transplant due to severe complications or low enzyme levels in my immune system.I am between 4 and 65 years old.I understand the study and agree to participate, or if I'm a minor, my guardian agrees.I agree to use birth control before, during, and after the study for the required time.I can stay within 1 hour's travel of the NIH for 3 months after my transplant.
Research Study Groups:
This trial has the following groups:- Group 1: Arm 1 / Group 1 Standard Risk
- Group 2: Arm 2 / Group 2 High Risk.
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
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."
Answered by AI
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."
Answered by AI
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."
Answered by AI
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."
Answered by AI
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