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Antibody
JSP191 Conditioning for Immunodeficiency
Phase 2
Recruiting
Led By Danielle E Pregent-Arnold, M.D.
Research Sponsored by National Cancer Institute (NCI)
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, +2, and +3 years post-transplant
Awards & highlights
Study Summary
This trial tests a new drug to see if it can make stem cell transplants for GATA2 deficiency safer. People aged 6-70 with the disease are eligible. Tests, a catheter, meds, & radiation will be used. Hospitalization & follow-up visits required.
Who is the study for?
This trial is for people aged 6-70 with GATA2 deficiency, a condition affecting immune function and increasing cancer risk. Eligible participants must have certain blood cell counts, organ function within specific limits, and an available matched stem cell donor. They should be able to stay near the hospital for at least 100 days post-transplant with a caregiver.Check my eligibility
What is being tested?
The study tests JSP191's ability to make hematopoietic stem cell transplants safer in GATA2 deficiency patients. It involves screening, conditioning (including JSP191 administration), transplanting donor stem cells, and taking other drugs to prevent side effects. Participants will be closely monitored for three years.See study design
What are the potential side effects?
Potential side effects include reactions related to the central venous catheter placement, medication-induced issues like liver or kidney problems from Tacrolimus or Mycophenolate Mofetil, low blood counts due to Cyclophosphamide or Fludarabine, and risks associated with total body irradiation.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ +1, +2, and +3 years post-transplant
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~+1, +2, and +3 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
To determine whether allogeneic hematopoietic cell transplantation with JSP191-based conditioning results in sustained donor engraftment by 100 days post-transplant in participants with GATA2 deficiency
Secondary outcome measures
3-year event-free survival
3-year incidence of grade III-IV acute and moderate to severe chronic graft versus host disease
3-year incidence of secondary graft failure
+3 moreTrial Design
2Treatment groups
Experimental Treatment
Group I: Arm BExperimental Treatment8 Interventions
JSP191, Fludarabine, Cyclophosphamide, Total Body Irradiation
Group II: Arm AExperimental Treatment7 Interventions
JSP191, Fludarabine, Total Body Irradiation
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Fludarabine
2012
Completed Phase 3
~1100
Mycophenolate Mofetil
1997
Completed Phase 4
~2380
Tacrolimus
2011
Completed Phase 4
~4740
Total Body Irradiation
2006
Completed Phase 3
~820
Hematopoietic Cell Transplant
2016
Completed Phase 2
~140
Cyclophosphamide
1995
Completed Phase 3
~3770
Find a Location
Who is running the clinical trial?
National Cancer Institute (NCI)Lead Sponsor
13,672 Previous Clinical Trials
40,926,409 Total Patients Enrolled
Danielle E Pregent-Arnold, M.D.Principal InvestigatorNational Cancer Institute (NCI)
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I have a harmful GATA2 gene mutation.I am between 6 and 70 years old.I do not have a history of blood cancer (except MDS), severe allergies, active cancer (except virus-driven or MDS), HIV, pregnancy, serious illness, or social issues affecting treatment compliance.
Research Study Groups:
This trial has the following groups:- Group 1: Arm A
- Group 2: Arm B
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.
Has Arm A been granted regulatory clearance from the FDA?
"Arm A's safety score is 2, since it is currently undergoing Phase 2 trials. This implies that there has been some evidence of the drug being safe but none regarding its efficacy yet."
Answered by AI
Are participants being recruited for this research endeavor?
"Clinicaltrials.gov indicates that this trial is not taking new enrolments; the initial post was on June 22nd 2023 and it had been last updated two weeks prior, on June 16th 2023. However, at present there are 82 other trials actively seeking participants."
Answered by AI
Do I conform to the requirements of this trial's enrollment criteria?
"To qualify for this research study, applicants must be between 6 and 70 years old and demonstrate a deficiency in gata2. A total of 32 participants are needed to complete the trial."
Answered by AI
Does this investigation permit applicants aged seventy or over?
"This trial's criteria for admittance requires that participants must be between 6 and 70 years old."
Answered by AI
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