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RG2133 (2',3',5'-Tri-O-Acetyluridine) in Mitochondrial Disease

An Open-Label Dose-Escalation Phase I Study to Asses the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of RG2133 (2',3',5'-Tri-O-Acetyluridine) in the Treatment of Inherited Mitochondrial Diseases

Status
Terminated
Phases
Phase 1
Study type
Interventional
Source
ClinicalTrials.gov
Registry ID
NCT00060515
Enrollment
12
Registered
2003-05-08
Start date
Unknown
Completion date
Unknown
Last updated
2005-08-05

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Mitochondrial Diseases

Brief summary

The objective of the study is to determine the safety and tolerability of RG2133 in patients with Mitochondrial Disease.

Interventions

DRUGRG2133 (2',3',5'-tri-O-acetyluridine)

Sponsors

Repligen Corporation
Lead SponsorINDUSTRY

Study design

Allocation
NON_RANDOMIZED
Primary purpose
TREATMENT

Eligibility

Sex/Gender
ALL
Age
3 Years to No maximum
Healthy volunteers
No

Inclusion criteria

Mitochondrial Disease

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026