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Hematopoietic Stem Cell Transplantation
Haploidentical Transplant for Severe Aplastic Anemia
Phase 2
Waitlist Available
Led By Amr Qudeimat, MD
Research Sponsored by St. Jude Children's Research Hospital
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Adequate organ function defined as: Left ventricular ejection fraction > 40% or shortening fraction ≥ 25%. Creatinine clearance (CrCl) or glomerular filtration rate (GFR) ≥ 50 ml/ min/1.73m2. Forced vital capacity (FVC) ≥ 50% of predicted value; or pulse oximetry ≥ 92% on room air if patient is unable to perform pulmonary function testing. Karnofsky or Lansky (age-dependent) performance score ≥ 50. Bilirubin ≤ 3 times the upper limit of normal for age. Alanine aminotransferase (ALT) or Aspartate aminotransferase (AST) ≤ 5 times the upper limit of normal for age. Females and males of childbearing potential must agree to practice 2 effective methods of contraception at the same time or agree to abstinence until after the last dose of chemotherapy has been administered
Does not have a suitable HLA-matched sibling donor (MSD) or volunteer 10/10 HLA-matched unrelated donor (MUD) available in the necessary time for progenitor cell donation.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 1 year
Awards & highlights
Study Summary
This trial is for patients with Severe Aplastic Anemia who will receive a haploidentical transplantation (from a family member that is not fully matched). The primary objective is to assess the rate of engraftment at 30 days and overall survival (OS) and event free survival (EFS) at 1 year.
Who is the study for?
This trial is for patients under 21 with Severe Aplastic Anemia who have failed at least one immunosuppressive therapy, lack a fully matched donor, and have good organ function. Donors must be family members partially matching the patient (≥3 of 6 HLA types), willing to donate stem cells, and HIV negative. Pregnant or breastfeeding women can't participate.Check my eligibility
What is being tested?
The study tests haploidentical hematopoietic cell transplantation (HCT) in SAA patients using chemotherapy, radiation, transplanted cells from half-matched family donors, and additional white blood cell infusions. It aims to assess engraftment success at 30 days post-transplantation and survival rates after one year.See study design
What are the potential side effects?
Possible side effects include reactions to medication like Anti-Thymocyte Globulin or Cyclophosphamide such as fever or chills; organ inflammation due to G-CSF; low blood counts from Fludarabine; immune complications like graft-versus-host disease; infection risks due to weakened immunity.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I don't have a matching family or unrelated donor for a cell transplant.
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My potential donor is ready and can donate stem cells.
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I am 18 years old or older.
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I am 21 years old or younger.
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I have been diagnosed with severe aplastic anemia or a single blood cell deficiency, confirmed by specific blood and bone marrow tests.
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I have a family member who is at least a half match for a transplant.
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I have a family member who is a partial match for a donation.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 1 year
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~1 year
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Engraftment
Overall and event free survival
Secondary outcome measures
Graft rejection
Graft vs host disease
Immune reconstitution
+1 moreTrial Design
1Treatment groups
Experimental Treatment
Group I: Haploidentical HCTExperimental Treatment9 Interventions
To assess the safety and efficacy of haploidentical donor transplantation for patients with severe aplastic anemia who lack an available HLA-matched donor. The goal of this study is to develop a novel, reduced-toxicity, post-transplant pharmacologic immunosuppression (GVHD prophylaxis)- free, highly tolerogenic haploidentical transplant regimen that is associated with few post- transplant complications or late toxicities and is available promptly to all patients, irrespective of matched donor availability.
Cells for infusion are prepared using the CliniMACS System.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Coenzyme M
FDA approved
Filgrastim
FDA approved
Total Lymphoid Irradiation (TLI)
2008
Completed Phase 2
~450
Fludarabine
FDA approved
CliniMACS
2005
Completed Phase 3
~770
Cyclophosphamide
FDA approved
Find a Location
Who is running the clinical trial?
St. Jude Children's Research HospitalLead Sponsor
428 Previous Clinical Trials
5,306,592 Total Patients Enrolled
2 Trials studying Aplastic Anemia
108 Patients Enrolled for Aplastic Anemia
Amr Qudeimat, MDPrincipal InvestigatorSt. Jude Children's Research Hospital
1 Previous Clinical Trials
14 Total Patients Enrolled
Akshay Sharma, MBBSPrincipal InvestigatorSt. Jude Children's Research Hospital
2 Previous Clinical Trials
40 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I don't have a matching family or unrelated donor for a cell transplant.You had a life-threatening reaction (like anaphylaxis) to ATG in the past, which means you cannot use it again.My potential donor is ready and can donate stem cells.I am 18 years old or older.I do not have an ongoing infection that isn't getting better with treatment.I have been diagnosed with dyskeratosis congenita.My bone marrow test shows changes linked to early or actual MDS.You are not currently breastfeeding a baby.I am 21 years old or younger.I have been diagnosed with Fanconi anemia, confirmed by specific testing.I have had a stem cell transplant from a donor.I have been diagnosed with severe aplastic anemia or a single blood cell deficiency, confirmed by specific blood and bone marrow tests.I have not received Alemtuzumab or ATG in the last 2 weeks.I have severe aplastic anemia and did not respond well to or relapsed after immunosuppressive therapy.I have had a solid organ transplant.My related donors have been checked and do not have telomere disease.I have antibodies against a donor organ.I have a family member who is at least a half match for a transplant.I am not pregnant or breastfeeding.I have had cancer before, but it was either treated over 5 years ago, was a basal cell carcinoma that was removed, or was a treated cervical carcinoma in situ.I have been diagnosed with myelodysplastic syndrome.I have a family member who is a partial match for a donation.
Research Study Groups:
This trial has the following groups:- Group 1: Haploidentical HCT
Awards:
This trial has 3 awards, including:- All Individual Drugs Already Approved - Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.
- Approved for 20 Other Conditions - This treatment demonstrated efficacy for 20 other conditions.
- 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.
Are there any harmful side effects associated with Total Lymphoid Irradiation (TLI)?
"Total Lymphoid Irradiation (TLI) has some evidence backing its safety, but as this is only a Phase 2 trial, there is no data yet to support efficacy. Therefore, it received a score of 2."
Answered by AI
Are new participants being accepted into this research project at present?
"Yes, this study is still looking for volunteers. According to the clinicaltrials.gov website, the trial was initially posted on 1/21/2021 and last updated on 8/26/2022."
Answered by AI
How many total participants are needed for this research project?
"The information available on clinicaltrials.gov verifies that this study is currently looking for 21 individuals to fill the 1 remaining locations. This particular trial was first posted on January 21st, 2021 and was most recently updated on August 26th, 2022."
Answered by AI
Why was this clinical trial designed in the first place?
"The primary goal of this trial, as measured over the course of 1 year, is engraftment. Additionally, we will track secondary outcomes like immune reconstitution (defined as the order and magnitude of leukocyte subtype recovery), graft rejection (the rate of secondary rejections post-transplant), and viral reactivation (occurrence of CMV, EBV or adenovirus post-transplant)."
Answered by AI
What is the research precedent for Total Lymphoid Irradiation (TLI)?
"As of now, there are over one-thousand studies investigating Total Lymphoid Irradiation (TLI). Out of those 183 live clinical trials are in the final stage. Most of the research surrounding TLI is based in Philadelphia, but this treatment is being studied at 30287 locations worldwide."
Answered by AI
What are some benefits of Total Lymphoid Irradiation (TLI)?
"Total Lymphoid Irradiation (TLI) is a medical procedure that has shown efficacy in the treatment of lung cancers. Additionally, this therapy can be used to treat conditions such as mobilization of hematopoietic stem cells therapy, immunosuppressive treatment, and multiple sclerosis."
Answered by AI
Who else is applying?
What site did they apply to?
St. Jude Children's Research Hospital
What portion of applicants met pre-screening criteria?
Met criteria
How many prior treatments have patients received?
2
Why did patients apply to this trial?
I am currently on cyclosporine and revolad since 2 and a half months ago with poor results, hoping for clinical trial.
PatientReceived 2+ prior treatments
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