Inborn Errors of Metabolism Clinical Trial
Official title:
Expanded Access Protocol for CaspaCIDe T Cells From An HLA-Partially Matched Related Donor After Negative Selection of TCR αβ+T Cells In Pediatric Patients Affected by Hematological and Other Disorders
Providing access of BPX-501 gene modified T cells and rimiducid to pediatric patients who do not meet the eligibility criteria of the BP-U-004 study.
This is an expanded access protocol of BPX-501 T cells infused after T cell-depleted HSCT in
pediatric patients with non-malignant hematologic disorders eligible for treatment on the
BP-U-004 study.
The purpose of this protocol is to provide access to the CaspaCIDe system combination product
(BPX-501 gene modified T cells and rimiducid) to patients on a case by case basis who do not
meet the BP-U-004 protocol eligibility criteria. BPX-501 infusion can enhance immune
reconstitution with the potential for reducing the severity and duration of severe acute
GVHD.
;
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Active, not recruiting |
NCT03548779 -
North Carolina Genomic Evaluation by Next-generation Exome Sequencing, 2
|
N/A | |
| Completed |
NCT01049854 -
CD34+Selection for Partially Matched Family or Matched Unrelated Adult Donor Transplant
|
Phase 2 | |
| Completed |
NCT00001596 -
Oral Pirfenidone for the Pulmonary Fibrosis of Hermansky-Pudlak Syndrome
|
Phase 2 | |
| Withdrawn |
NCT01003912 -
Fetal Umbilical Cord Blood (UCB) Transplant for Lysosomal Storage Diseases
|
Phase 1 | |
| Completed |
NCT00744692 -
Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders
|
Phase 1 | |
| Completed |
NCT00692926 -
Unrelated Umbilical Cord Blood Transplantation Augmented With ALDHbr Umbilical Cord Blood Cells
|
Phase 1 | |
| Recruiting |
NCT05818566 -
Orphan Drugs for Inherited Metabolic Diseases
|
||
| Completed |
NCT05330039 -
Characterization of Intestinal Microbiota in Children With Inborn Errors of Metabolism (IEM)
|
||
| Withdrawn |
NCT03866954 -
Trial of Erythrocyte Encapsulated Thymidine Phosphorylase In Mitochondrial Neurogastrointestinal Encephalomyopathy
|
Phase 2 | |
| Completed |
NCT03911089 -
A Collection of Case Studies in Infants With UCD to Evaluate Infant Growth and the Safety of a New Medical Food for UCD
|
N/A | |
| Completed |
NCT03058848 -
Evaluation of PKU Start
|
N/A | |
| Suspended |
NCT04399694 -
Identification and Characterization of Novel Non-Coding Variants That Contribute to Genetic Disorders
|
||
| Terminated |
NCT00654433 -
ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases
|
Phase 3 | |
| Completed |
NCT03168399 -
Evaluation of PKU Explore
|
N/A | |
| Recruiting |
NCT00078078 -
Clinical and Laboratory Study of Methylmalonic Acidemia
|
||
| Completed |
NCT00328159 -
Nutritional Therapy of the Deficits of Oxidation Mitochondrial of the Fatty Acids
|
N/A | |
| Completed |
NCT04309331 -
Market Research - Acceptability Trial for a New PKU Amino Acid Based Protein Substitute
|
N/A | |
| Completed |
NCT04709965 -
Evaluating Face-Recognition Technology in Syndrome Diagnosis
|
N/A | |
| Recruiting |
NCT06360913 -
Blood Spot and Urine Metabolomic Screening Applied to Rare Diseases
|
N/A | |
| Completed |
NCT00309400 -
The Early History of Universal Screening for Metabolic Disorders
|
N/A |