Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders
Recruiting
Phase 1
Phase 2
Interventional Study
Severe Sickle Cell Disease
Bone Marrow Failure Syndromes
Metabolic Disorders
Immunologic Disorders
Hemoglobinopathies
Non-malignant Disorders
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 1 Day – 21
- Sex
- Any
- Trial phase
- Phase 1/2
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 29 (estimated)
- Sponsor
- Washington University School of Medicine · Other
Think this trial could be right for you?
Answer a few quick questions to see if you may meet the eligibility requirements.
Check Your Eligibility
About This Trial
This study is designed to estimate the efficacy and toxicity of familial HLA mismatched bone marrow transplants in patients with non-malignant disease who are less than 21 years of age and could benefit from the procedure.
Trial Locations
Loading…
Loading trial locations…
Eligibility Criteria
Inclusion Criteria:
* Nonmalignant disorder requiring bone marrow transplant including bone marrow failure syndromes, metabolic disorders, immunologic disorders, or hemoglobinopathy
* For patients with sickle cell disease, must have one of the following severe manifestations:
1. Overt or silent …
Contacts