NCT03862846

A Study of the Safety of REN001 in Patients With Primary Mitochondrial Myopathy

Terminated Phase 1 Last updated 16 July 2020

What this trial tests

Phase 1 trial testing REN001 in Primary Mitochondrial Myopathy in 23 participants. Terminated before completion.

Timeline

10 May 2019

Primary endpoint
23 April 2020

23 April 2020

Quick facts

Lead sponsor	Reneo Pharma Ltd
Phase	Phase 1
Status	Terminated
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	23
Start date	10 May 2019
Primary completion	23 April 2020
Estimated completion	23 April 2020
Sites	2 locations across United Kingdom

Drugs / interventions tested

• REN001 — full drug profile →

Conditions studied

• Primary Mitochondrial Myopathy — all drugs for Primary Mitochondrial Myopathy →

Sponsor

Reneo Pharma Ltd — full company profile →

Who can join

16 and older, any sex, with Primary Mitochondrial Myopathy. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

The purpose of this study is to assess REN001 safety in subjects with primary mitochondrial myopathy

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Pharmacological advances in mitochondrial therapy.
   Singh A, Faccenda D, Campanella M. · · 2021 · cited 88× · PMID 33647769 · DOI 10.1016/j.ebiom.2021.103244
2. Current and Emerging Clinical Treatment in Mitochondrial Disease.
   Tinker RJ, Lim AZ, Stefanetti RJ, McFarland R. · · 2021 · cited 64× · PMID 33646563 · DOI 10.1007/s40291-020-00510-6
3. Clinical trials in mitochondrial disorders, an update.
   Almannai M, El-Hattab AW, Ali M, Soler-Alfonso C, et al · · 2020 · cited 56× · PMID 33129691 · DOI 10.1016/j.ymgme.2020.10.002
4. Moving towards clinical trials for mitochondrial diseases.
   Pitceathly RDS, Keshavan N, Rahman J, Rahman S. · · 2021 · cited 52× · PMID 32618366 · DOI 10.1002/jimd.12281
5. PGC1s and Beyond: Disentangling the Complex Regulation of Mitochondrial and Cellular Metabolism.
   Coppi L, Ligorio S, Mitro N, Caruso D, et al · · 2021 · cited 31× · PMID 34199142 · DOI 10.3390/ijms22136913
6. Mitochondrial Dysfunction in Atrial Fibrillation-Mechanisms and Pharmacological Interventions.
   Muszyński P, Bonda TA. · · 2021 · cited 29× · PMID 34071563 · DOI 10.3390/jcm10112385
7. Challenges and opportunities to bridge translational to clinical research for personalized mitochondrial medicine.
   Gropman AL, Uittenbogaard MN, Chiaramello AE. · · 2024 · cited 11× · PMID 38266483 · DOI 10.1016/j.neurot.2023.e00311
8. Therapies for Mitochondrial Disease: Past, Present, and Future.
   Ball M, van Bergen NJ, Compton AG, Thorburn DR, et al · · 2025 · cited 3× · PMID 40714961 · DOI 10.1002/jimd.70065