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Leber Congenital Amaurosis: early-phase trial registry
2 of 7 indexed Leber Congenital Amaurosis trials are currently recruiting, with more weight in Phase 1-2.
Corpus placement: #2,497 of 28,707 conditions by registered trial count.
7 US clinical trials · 2 currently recruiting · #2,497 of 28,707 conditions by registered trial count
Leber Congenital Amaurosis: 7 US clinical trials tracked, 2 recruiting.
Leber Congenital Amaurosis is the subject of 7 registered US clinical trials on ClinicalTrials.gov, 2 of them currently open to new participants. 2 are in Phase 3-4 (later-stage) and 5 in Phase 1-2 (earlier-stage). The most active sponsor is Spark Therapeutics, running 2 of these trials.
Enrollment posture brief
Registry condition leber-congenital-amaurosis
OPEN 2 | RECR 29% | LATE 2 | EARLY 5 | RANK #2497 | SPON 2
Volume-matched condition peers
Nearest open-share peer: Acromegaly (29% recruiting · 14 trials)
Leber Congenital Amaurosis lists 2 open slots (29% of 7 indexed trials). Peers are matched on registered volume, not therapeutic-area browse lists. How peer matching works →
Key findings
What ClinicalTrials.gov does not surface for Leber Congenital Amaurosis on its own pages , computed from the registry mirror as of 2026-08-08. Each line carries its own denominator so it can be quoted as it stands.
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Leber Congenital Amaurosis is recruiting below its size-band peers.
2 of 7 indexed trials (28.6%) are open to enrollment, against a 46.5% average across 27,806 conditions with fewer than 20 trials. The peer set uses the same ClinicalTrials.gov-derived counts as this page.
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One sponsor concentrates Leber Congenital Amaurosis research.
Spark Therapeutics accounts for 2 of 7 trials (28.6%). 6 distinct sponsors appear on at least one study for this condition label.
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Most Leber Congenital Amaurosis trials plan to enrol fewer than 100 people.
Median target enrolment is 22 among the 7 trials that report a genuine target (sentinel values excluded). 7 of those (100%) plan for fewer than 100 participants.
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Leber Congenital Amaurosis research is mostly interventional.
7 interventional and 0 observational studies make up this index (100% interventional). Study type is sponsor-reported on ClinicalTrials.gov.
Open and recent trials
7 total, page 1 of 1
Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)
Laboratoires Thea
NCT06891443
Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR)
HuidaGene Therapeutics Co.
NCT05906953
A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)
ProQR Therapeutics
NCT03913143
Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis
Spark Therapeutics
NCT00999609
Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis
Ocugen
NCT05203939
Study of Subretinally Injected ATSN-101 Administered in Patients With Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D
Atsena Therapeutics
NCT03920007
Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2
Spark Therapeutics
NCT01208389
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 1 | 4 |
| Phase 2 | 1 |
| Phase 3 | 2 |
Top Sponsors
Named interventions
Most-linked intervention names on Leber Congenital Amaurosis records (sponsor-reported on ClinicalTrials.gov). Counts are trial links, not unique products.
US sites by state
Facility locations filed on Leber Congenital Amaurosis trials. A trial with sites in several states counts in each.
Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.
Related
What to do with this Leber Congenital Amaurosis page
7 registered trials, 2 currently recruiting, is a starting point for a conversation, not a diagnosis or a recommendation.
- 2 Leber Congenital Amaurosis trials are currently recruiting, check eligibility criteria before contacting a site. Browse recruiting trials
- Spark Therapeutics sponsors the most Leber Congenital Amaurosis trials on record, review their full trial history before evaluating a specific study. See Spark Therapeutics's trials
- Bring the specific trial ID (NCT number) to your own doctor before enrolling, this page is a directory, not medical guidance. Read the trial-finding guide
Trial and recruiting counts reflect ClinicalTrials.gov registry status as of the data vintage above; individual trial eligibility and enrollment status can change.
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This Leber Congenital Amaurosis condition card totals 7 registered trials; most-linked intervention sepofarsen; top US site state Pennsylvania. See our editorial standards & corrections policy, the methodology behind these numbers, the data changelog, or report a data error.