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Anti-metabolites
Fludarabine RIC for Bone Marrow Failure Syndrome
Phase < 1
Recruiting
Led By Timothy Olson, MD, PhD
Research Sponsored by Children's Hospital of Philadelphia
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Patients 0-22 years with acquired aplastic anemia or a diagnosed inherited bone marrow failure syndrome
Patients must have a fully Human leukocyte antigen (HLA)-matched (10/10) related donor
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 1 year post transplant
Awards & highlights
Study Summary
This trial is testing whether a less intense form of chemotherapy can help patients with aplastic anemia or inherited bone marrow failure syndromes to receive a bone marrow transplant from a related donor.
Who is the study for?
This trial is for young people (ages 0-22) with bone marrow failure syndromes like aplastic anemia or inherited conditions causing low blood counts. They need a fully matched related bone marrow donor, good organ function, and no uncontrolled infections. Pregnant females or those without a suitable donor are excluded.Check my eligibility
What is being tested?
The study tests if fludarabine-based reduced intensity conditioning helps patients with acquired aplastic anemia or inherited bone marrow failures successfully accept transplants from matched donors.See study design
What are the potential side effects?
Fludarabine may cause side effects such as nausea, fatigue, weakened immune system leading to increased infection risk, liver issues, and rarely severe lung problems.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I am 22 or younger with a rare disorder affecting my bone marrow.
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I have a relative who is a perfect match for my transplant needs.
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I have severe aplastic anemia confirmed by specific tests and blood counts.
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My kidney, liver, and heart functions are within normal ranges.
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I can do most activities but may need help.
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I have a family member who is a match for a bone marrow donation.
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I do not have any untreated infections.
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I am 22 years old or younger.
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I need regular blood or platelet transfusions, or I have a low white blood cell count.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ up to 1 year post transplant
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 1 year post transplant
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Rate of graft failure
Time to neutrophil engraftment
Transplant-related mortality
Secondary outcome measures
Rate of disease free survival
Rate of overall survival
Trial Design
3Treatment groups
Experimental Treatment
Group I: Inherited Bone Marrow Failure Syndrome no Trilineage AplasiaExperimental Treatment1 Intervention
Patients with inherited bone marrow failure (iBMF) syndromes without trilineage aplasia includes those with diagnoses of Severe Congenital Neutropenia, Diamond-Blackfan Anemia, and related conditions. Patients will receive a matched related donor bone marrow transplant following conditioning with thymoglobulin, busulfan and fludarabine.
Group II: Inherited Bone Marrow Failure Syndrome + Trilineage AplasiaExperimental Treatment1 Intervention
Patients with inherited bone marrow failure (iBMF) syndromes with trilineage aplasia includes those with diagnoses of Fanconi Anemia, Dyskeratosis Congenita, and related conditions. Patients will receive a matched related donor bone marrow transplant following conditioning with fludarabine, cyclophosphamide, thymoglobulin.
Group III: Acquired Aplastic Anemia (AA)Experimental Treatment1 Intervention
Patients with severe or very severe acquired aplastic anemia (AA). Patients will receive a matched related donor bone marrow transplant following reduced intensity conditioning (RIC) including thymoglobulin (ATG), fludarabine and dose-reduced cyclophosphamide.
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Who is running the clinical trial?
Children's Hospital of PhiladelphiaLead Sponsor
708 Previous Clinical Trials
8,581,278 Total Patients Enrolled
Timothy Olson, MD, PhDPrincipal Investigator - Children's Hospital of Philadelphia
Children's Hospital of Philadelphia
2 Previous Clinical Trials
70 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I do not have any untreated serious infections.I have been diagnosed with Myelodysplastic syndrome.I am 22 or younger with a rare disorder affecting my bone marrow.I have a relative who is a perfect match for my transplant needs.I have severe aplastic anemia confirmed by specific tests and blood counts.I am capable of becoming pregnant and have a negative pregnancy test.My kidney, liver, and heart functions are within normal ranges.I can do most activities but may need help.I have a family member who is a match for a bone marrow donation.I do not have a fully matched family donor for a transplant.I do not have any untreated infections.I am 22 years old or younger.I need regular blood or platelet transfusions, or I have a low white blood cell count.I don't have a family donor for a bone marrow transplant.I have PNH without bone marrow failure.
Research Study Groups:
This trial has the following groups:- Group 1: Inherited Bone Marrow Failure Syndrome no Trilineage Aplasia
- Group 2: Acquired Aplastic Anemia (AA)
- Group 3: Inherited Bone Marrow Failure Syndrome + Trilineage Aplasia
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.
How extensive is the recruitment for this examination?
"Affirmative, according to clinicaltrials.gov the research endeavor is currently recruiting members for participation; it was first announced on April 1st 2015 and last modified on May 6th 2022. This medical trial requires 25 individuals from a single site."
Answered by AI
Are there still vacancies available in this clinical trial?
"Per the clinicaltrials.gov portal, recruitment for this trial is in progress. This study was initially advertised on April 1st 2015 and its criteria were most recently revised on May 6th 2022."
Answered by AI
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