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:
2025-12-24 @ 10:58 PM
Ignite Modification Date:
2025-12-24 @ 10:58 PM
NCT ID:
NCT06017869
Eligibility Criteria:
Inclusion Criteria:
1. Male or female participants aged from 1 to 18 years old.
2. Diagnosis of Pearson Syndrome (current or history) as verified by molecular identification of deletion in mtDNA of peripheral blood. Participants are diagnosed with PS Participant can be in either the PS manifestations of the disease or may have transitioned to Kearns Sayre Syndrome (KSS) manifestations but has a history of PS.
3. Participants have failure to thrive (height SDS smaller than -1)
4. Participants should have at least 12 months' history of body weight and height and calculated GFR (from creatinine) before treatment.
5. Body weight ≥ 10 kg.
6. Participants' living parent(s) and/or legal guardian(s) able to understand and provide voluntary written informed consent.
7. Participants' parents or legal guardian have a good understanding of the study and nature of the procedure and are expected to be able to comply with study visit schedules and caregiver assessments without difficulty.
8. Participants' parents or legal guardian provides written informed consent prior to study participation.
9. Participants are medically able to undergo the study interventions as determined by the Investigator.
Exclusion criteria:
1. History of infection with HIV-1, HIV-2, or HTLV I/II.
2. Participants have any active infection.
3. Participants have been diagnosed with Myelodysplastic Syndrome, by FISH and/or karyotype.
4. Participants are unable to undergo apheresis.
5. Participants have known hypersensitivity to murine proteins or iron-dextran.
6. Participants have severe chronic infection.
7. Participants have disease or conditions that may risk the participant or interfere with the ability to interpret the study results.
8. History of malignancy.
9. History of treatment with gene therapy, allogeneic bone marrow or cord blood transplantation.
10. Participants have had a change in growth hormone regimen in less than 2 years prior to treatment.
11. Participants have participated in another clinical trial or received other experimental medications outside a clinical trial within 1 month prior to start of this study.
12. Participants who are pregnant or intend to become pregnant in the next 12 months.
13. In the opinion of the Investigator, the participant is unsuitable for participating in the study for any reason.
Healthy Volunteers:
False
Sex:
ALL
Minimum Age:
1 Year
Maximum Age:
18 Years
Study:
NCT06017869
Study Brief:
Protocol Section:
NCT06017869