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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
No Placebo-Only Group
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.
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.
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
Study objectives can provide a clearer picture of what you can expect from a treatment.Primary study objectives
Overall Survival
Secondary study objectives
Decreased engraftment syndrome.
Engraftment without GvHD
Transplantation, Homologous
+2 moreAwards & Highlights
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Trial 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 4
~1710
Sirolimus
2013
Completed Phase 4
~2750
Cyclophosphamide
2010
Completed Phase 4
~2310
Alemtuzumab
2004
Completed Phase 4
~1880
Find a Location
Who is running the clinical trial?
National Institute of Allergy and Infectious Diseases (NIAID)Lead Sponsor
3,323 Previous Clinical Trials
5,364,718 Total Patients Enrolled
Elizabeth M Kang, M.D.Principal InvestigatorNational Institute of Allergy and Infectious Diseases (NIAID)
11 Previous Clinical Trials
353 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.
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