Leber congenital amaurosis Advocacy Hub | UniteRare

At a Glance

Live from database

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7Recruiting trials

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45Specialists

Regulatory Watchboard

10 events

Aug 2025Psychotherapy Group for Parents of Children With LCA

Instituto de Genética Ocular — NA

TrialNOT YET RECRUITING

Jun 2025Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)

Laboratoires Thea — PHASE3

TrialRECRUITING

Oct 2023Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR)

HuidaGene Therapeutics Co., Ltd. — PHASE1, PHASE2

TrialRECRUITING

Sep 2023An Expanded Clinical Study Evaluating the AAV2-RPE65 Gene Therapy(LX101) in Patients With LCA

Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine — NA

TrialNOT YET RECRUITING

Jan 2023Leber Congenital Amaurosis Inherited Blindness of Gene Therapy Trial(LIGHT)

Xinhua Hospital, Shanghai Jiao Tong University School of Medicine — EARLY_PHASE1

TrialACTIVE NOT RECRUITING

Sep 2019Study of Subretinally Injected ATSN-101 Administered in Patients With Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D

Atsena Therapeutics Inc. — PHASE1, PHASE2

TrialACTIVE NOT RECRUITING

Apr 2019A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)

ProQR Therapeutics — PHASE2, PHASE3

TrialACTIVE NOT RECRUITING

Jun 2015Long-term Follow-up Study in Subjects Who Received Voretigene Neparvovec-rzyl (AAV2-hRPE65v2)

Spark Therapeutics, Inc.

TrialACTIVE NOT RECRUITING

Oct 2012Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis

Spark Therapeutics, Inc. — PHASE3

TrialACTIVE NOT RECRUITING

Nov 2010Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2

Spark Therapeutics, Inc. — PHASE1, PHASE2

TrialACTIVE NOT RECRUITING

Data from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Financial Assistance

FINANCIAL LANDSCAPE SUMMARY

Total programs

No financial assistance programs currently listed for Leber congenital amaurosis.
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Clinical Trial Landscape

7 active trials

2Phase 3

3PHASE1, PHASE2

1EARLY_PHASE1

1PHASE2, PHASE3

7Total recruiting

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Recent News & Research

No recent news articles indexed yet for Leber congenital amaurosis.
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Specialist Network

Top 6 by expertise

Albert M Maguire, MD, MD

Children's Hospital of Philadelphia

📍 PHILADELPHIA, PA

Score 291 trial1 pub

Robin R Ali, PhD

University College, London

Score 221 trial

Stephen R Russell, MD

University of Iowa

Score 211 trial

Josseline KAPLAN, MD

Necker-Enfants Malades Hospital, 75015 Paris. France

Score 181 trial

Michel WEBER, Professor

Nantes University Hospital

Score 101 trial1 pub

Michel Weber, MD

Nantes University Hospital

Score 91 trial

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