Optic Atrophy, Hereditary, Leber clinical trials

The registry lists 44 studies for Optic Atrophy, Hereditary, Leber across more than 10 countries. 9 are recruiting or about to start. 32 of them are mainly about Optic Atrophy, Hereditary, Leber (how this is decided). Per ClinicalTrials.gov, last updated 2026-10-21.

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Status

Completed: 21
Recruiting: 7
Terminated: 5
No longer available: 3
Active, not recruiting: 3
Not yet recruiting: 2

Phase

Phase 2: 15
Phase 3: 11
Phase 1: 9
Phase 4: 1

Where

United States: 23
France: 17
United Kingdom: 11
Germany: 10
Italy: 10
Belgium: 8

Who runs them

Santhera Pharmaceuticals (8) · GenSight Biologics (8) · Laboratoires Thea (3) · University Hospital, Angers (2) · Wuhan Neurophth Biotechnology Limited Company (2) · Neurophth Therapeutics Inc (2) · Amgen (2) · Randy Kardon (1)

What is being tested here

Drugs under test in Optic Atrophy, Hereditary, Leber studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted

Drug	Open	Phase 3	All
Sepofarsen	1	2	4
Idebenone	0	1	3
Cysteamine	0	0	2
Elamipretide	0	0	1
QPI-1007	0	0	1
Cyclosporine	0	0	1

Coming up

4phase 3 studies under way

5studies finish main data collection within a year1 of them phase 3

Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.

All 5, phase 3 first

Nov 2027Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION) · Phase 3 · Laboratoires Thea
Dec 2026North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC) · Columbia University
Jan 2027Metabolomics Analysis According to the Retinal Nerve Fiber Layer in Patients With NOHL Mutations (MétabOCT) · Hôpital Necker-Enfants Malades
Jul 2027Stem Cell Ophthalmology Treatment Study II · MD Stem Cells
Oct 2027Optic Nerve Head Evaluation Through Multimodal Blood Flow Analysis: a Prospective Observational, Multi-center Study · Barboni

Who these studies take

Of the 7 Optic Atrophy, Hereditary, Leber studies recruiting now, 4 (57%) set no upper age limit and 2 (29%) stop at 75 or younger. 4 (57%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 3 (43%) take healthy volunteers.

Recruiting studies that take each age, of 7
Age	Women	Men
10	2 (29%)	2 (29%)
18	7 (100%)	7 (100%)
30	7 (100%)	7 (100%)
40	7 (100%)	7 (100%)
50	7 (100%)	7 (100%)
65	6 (86%)	6 (86%)
75	6 (86%)	6 (86%)
85	5 (71%)	5 (71%)

Across all 44 studies, recruiting or not: 36% stop at 75 or younger and 25% at 65; 70% take children; 0% are for women only and 0% for men only; 16% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.

Studies

Recruiting first, then most recently updated. Showing 44 of 44. search them.