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NCT ID: NCT03639844 No longer available - Clinical trials for Inborn Errors of Metabolism

BPX-501 T Cells Infused Post Stem Cell Transplant in Pediatrics With Non-Malignant Disorders Ineligible for BPU004 Study

Start date: n/a
Phase:
Study type: Expanded Access

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.

NCT ID: NCT01461304 No longer available - Barth Syndrome Clinical Trials

Compassionate Use of Triheptanoin (C7) for Inherited Disorders of Energy Metabolism

Start date: n/a
Phase:
Study type: Expanded Access

This is a compassionate use study to allow patients already taking triheptanoin (C7) through previous studies to continue to receive the supplement. It will also allow triheptanoin supplementation in patients with qualifying disorders if they are failing conventional therapy.