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Results for “Pearson Marrow-pancreas Syndrome”

7 trials

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Showing 7 of 7 results

Testing effectiveness (Phase 2)Looking for participantsNCT06017869
What this trial is testing

Evaluate the Safety and Therapeutic Effects of a Single Intravenous Infusion (IV) of Autologous CD34+ Cells Enriched With Allogenic Placenta-derived Mitochondria in Patients With a Diagnosis of Pearson Syndrome (PS)

Who this might be right for
Mitochondrial DiseasesPearson Syndrome
Minovia Therapeutics Ltd. 6
Testing effectiveness (Phase 2)Ended earlyNCT02104336
What this trial is testing

Phase 2 Study of EPI-743 in Children With Pearson Syndrome

Who this might be right for
Pearson Syndrome
Edison Pharmaceuticals Inc 2
Not applicableLooking for participantsNCT05554835
What this trial is testing

Global Registry and Natural History Study for Mitochondrial Disorders

Who this might be right for
Mitochondrial DiseasesKearns-Sayre SyndromeMIDD+15 more
LMU Klinikum 6,000
Testing effectiveness (Phase 2)Study completedNCT03384420
What this trial is testing

Evaluate the Safety and Therapeutic Effects of Transplantation of MNV-BM-BLD in Pediatric Patients With Pearson Syndrome

Who this might be right for
Mitochondrial DiseasesPearson Syndrome
Minovia Therapeutics Ltd. 7
Not applicableStudy completedNCT02327364
What this trial is testing

Natural History of Pearson Syndrome

Who this might be right for
Pearson Syndrome
Sumit Parikh 11
Not applicableStudy completedNCT00499070
What this trial is testing

Assessing Immune Function in Young Patients With Cytopenia That Did Not Respond to Treatment

Who this might be right for
Dyskeratosis CongenitaFanconi AnemiaMyelodysplastic Syndromes+2 more
University Hospital Freiburg 119
Not applicableUnknownNCT05029843
What this trial is testing

SLSMDS Natural History Study

Who this might be right for
Pearson SyndromeSingle Large Scale Mitochondrial DNA Deletion Syndromes (SLSMDS)
The Champ Foundation 30