Eligibility Module
The Eligibility Module contains detailed information about who can participate in the clinical trial. This includes eligibility criteria, age restrictions, gender requirements, healthy volunteer status, and study population descriptions, helping researchers understand who is eligible to participate in the study.
Eligibility Module path is as follows:
Study -> Protocol Section -> Eligibility Module
Eligibility Module
Ignite Creation Date:
2025-12-25 @ 4:06 AM
Ignite Modification Date:
2025-12-25 @ 4:06 AM
NCT ID:
NCT03384420
Eligibility Criteria:
Inclusion Criteria
1. Patient diagnosed with Pearson Syndrome, as verified by molecular identification of a defect in the mitochondrial DNA.
2. Normal maternal mitochondria as verified by mtDNA sequencing.
3. Males and females between 3 years or older and up to 18th birthday.
4. Patient is transfusion independent.
5. Patient has at least one of the following systematic involvements:
1. High baseline lactate levels
2. Episodes of metabolic crisis in the last year before pre-screening
3. Renal failure (not dependent on dialysis) or evidence of proximal tubulopathy
4. Growth retardation or failure to thrive
Exclusion Criteria
1. Absence of detectable mitochondria mutation or deletion.
2. Patient or patient's mother have a positive test for microbiologic
3. Patient is unable to undergo leukapheresis.
4. Patient suffers from chronic severe infection, malignant disease or any other disease or condition that may risk the patient or interfere with the ability to interpret the study results.
5. Patient has been treated previously with any cell or gene therapy.
6. Patient has participated in another clinical treatment trial or received other experimental medications outside of a clinical trial within 1 month prior to start of this study.
Healthy Volunteers:
False
Sex:
ALL
Minimum Age:
3 Years
Maximum Age:
18 Years
Study:
NCT03384420
Study Brief:
Protocol Section:
NCT03384420