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Stem Cell Transplantation
Stem Cell Transplant for Severe Aplastic Anemia
Phase 2
Recruiting
Led By Richard W Childs, M.D.
Research Sponsored by National Heart, Lung, and Blood Institute (NHLBI)
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Recipient: Patients diagnosed with one of the following hematologic diseases which are associated with reasonable longevity, shown to be curable by allogeneic BMT but where concern for a high procedural mortality with conventional BMT may delay or prevent such treatment: 1) Paroxysmal nocturnal hemoglobinuria (PNH) associated with life-threatening thrombosis, and/or cytopenia, and/or transfusion dependence and/or recurrent and debilitating hemolytic crisis 2) Severe aplastic anemia (SAA) or pure red cell aplasia (PRCA [acquired or congenital]) associated with transfusion dependence and/or neutropenia in patients who are not candidates for, or who have failed immunosuppressive therapy 3) Refractory anemia (RA) or RARS MDS patients who have associated transfusion dependence and/or neutropenia. Ages 4 to 80 (both inclusive), and weight >18kg Availability of HLA identical or single HLA locus mismatched family donor or 10/10 matched unrelated donor at the allelic level (HLA alleles A, B, C, DR, and DQ). 9/10 donors where all the HLA sequences have the same antigen/peptide binding domains in key exons to the patient. This can result in identical protein sequences between patient and donor. Allele mismatches in p and g groups can be considered acceptable due to the exact matching which exists in the binding domains. Telomere Length Testing Germline/Inherited gene panel in patients where a suspicion for a familial bone marrow failure syndrome (BMFS) exists, hTERC and hTERT, GATA2 mutation testing will be performed on protocol 04-H-0012 or performed elsewhere prior to enrolling on 04-H-0012.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 1 year
Awards & highlights
Study Summary
This trial is testing a new method of stem cell transplantation that may reduce the likelihood of severe side effects or transplant rejection.
Who is the study for?
This trial is for people aged 4-80 with severe blood diseases treatable by stem cell transplants, like severe anemia. They must weigh over 18kg and have a related donor or a matched unrelated donor available. It's not suitable for those who could be at high risk from traditional bone marrow transplants.Check my eligibility
What is being tested?
The study tests a new stem cell transplant method using G-CSF mobilized CD34+ cells combined with fewer non-mobilized white blood cells to potentially reduce complications. Donors give white blood cells and then stem cells after filgrastim injections; recipients get chemotherapy before the infusion.See study design
What are the potential side effects?
Possible side effects include reactions to the infusion, immune system suppression leading to increased infection risk, graft-versus-host disease where donated cells attack the body, and potential complications from chemotherapy drugs used.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowTimeline
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
Primary endpoint of this study is chronic GVHD by one year.
Trial Design
2Treatment groups
Experimental Treatment
Active Control
Group I: 1Experimental Treatment2 Interventions
Target doses: CD34+ cells 8 x 106/kg; CD3+ cells 2 x 107/kg
Group II: 2Active Control1 Intervention
Donor
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Who is running the clinical trial?
National Heart, Lung, and Blood Institute (NHLBI)Lead Sponsor
3,837 Previous Clinical Trials
47,851,474 Total Patients Enrolled
11 Trials studying Aplastic Anemia
922 Patients Enrolled for Aplastic Anemia
Richard W Childs, M.D.Principal InvestigatorNational Heart, Lung, and Blood Institute (NHLBI)
18 Previous Clinical Trials
8,636 Total Patients Enrolled
1 Trials studying Aplastic Anemia
500 Patients Enrolled for Aplastic Anemia
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:Research Study Groups:
This trial has the following groups:- Group 1: 1
- Group 2: 2
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.
Is the use of G-CSF from a donor FDA approved for treating PBC?
"While Phase 2 trials lack evidence surrounding a treatment's efficacy, there is some data supporting the safety of Donor derived G-CSF mobilized PBC. This gave it a score of 2."
Answered by AI
How many people are allowed to join this test group?
"That is correct. The page on clinicaltrials.gov says that the trial is looking for 120 patients from 8 hospitals or clinics. This particular study was posted on December 10th, 2010 and updated for the last time on November 10th, 2022."
Answered by AI
How can I sign up to participate in this experiment?
"This trial is currently looking for around 120 participants that have anemia, aplastic and are between 4 to 80 years old."
Answered by AI
Are people with the relevant condition still able to join this trial?
"That is correct, according to the listing on clinicaltrials.gov this study posted on December 10th 2010, is still recruiting patients. They are looking for a total of 120 participants who will be spread out between 8 different trial sites."
Answered by AI
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