Clinical TrialID 6611

Deoxynucleosides Pyrimidines as Treatment for Mitochondrial Depletion Syndrome

NCT04802707

Mitochondrial Diseases|Mitochondrial Encephalomyopathy|Mitochondrial Encephalopathy|Mitochondrial DNA Depletion|Mitochondrial Metabolism Disorders

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Record Fields

Scalar fields from the final clinical_trial record.

Clinical Trial Id
6611
Evidence Id
6611
Core Evidence Id
6611
Source Clinical Trial Id
6598
Herb2 Clinical Trial Id
HBCT006598
Nct Id
NCT04802707
Title
Deoxynucleosides Pyrimidines as Treatment for Mitochondrial Depletion Syndrome
Status
Recruiting
Phase
Phase 2
Study Result
No
Study Condition
Mitochondrial Diseases|Mitochondrial Encephalomyopathy|Mitochondrial Encephalopathy|Mitochondrial DNA Depletion|Mitochondrial Metabolism Disorders
Study Type
Interventional
Study Design
Allocation: NA|Intervention Model: SINGLE_GROUP|Masking: NONE|Primary Purpose: TREATMENT
Intervention
COMBINATION_PRODUCT: deoxycytidine and deoxythymidine
Intervention Allocation
Intervention Model
Single_Group
Intervention Mask
Intervention Purpose
Treatment
Gender
All
Age
Child, Adult
Enrollment
50
Outcome Measure
Rate of Responder versus Non-Responder Status with investigational product, "Responder" defined as having ≥ 2 of (1) electroencephalography EEG improvement, (2) decreased seizure frequency, (3) cognitive improvement, (4) caregiver impression of improvement, (5) clinical improvement, (6) Normal Organics and Metabolism functions Description of the primary variable(s) The primary efficacy endpoint is the composite cluster of the first occurrence, over the duration of study, of Mitochondria Depletion Syndrome., 104 weeks
Sponsor Collaborator
McGill University Health Centre/Research Institute of the McGill University Health Centre
Funded By
OTHER
Location
Research InstituMcGill University Health Centre - Children Hospital of Montreal, Montréal, Quebec, H4A 3J1, Canada
Other Id
2021-7654 dC-dT-MDS
Start Date
2021-10-18
Primary Completion Date
2025-12-31
Completion Date
2026-06-30
First Posted
2021-03-17
Results First Posted
Last Update Posted
2024-03-13
Study Document
Study Url
https://clinicaltrials.gov/study/NCT04802707
Title Cn
Title En
Deoxynucleosides Pyrimidines as Treatment for Mitochondrial Depletion Syndrome
Bilingual Status
semi_complete