A Phase 1/2 interventional study of HG004 in Leber Congenital Amaurosis and Inherited Retinal Diseases Caused by RPE65 Mutations, sponsored by HuidaGene Therapeutics Co., Ltd.. Status unknown at 3 sites in 2 countries. Open to participants aged 6 Years to 50 Years. Per ClinicalTrials.gov, last updated 2024-09-19.
Sponsored by HuidaGene Therapeutics Co., Ltd. · Phase 1/2, Interventional, and Treatment
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Among 23 studies in Leber Congenital Amaurosis with a site in United States — 4 open to participants now, 9 under way, 3 of them phase 3. Leber Congenital Amaurosis · United States.
Among the 23 studies in Leber Congenital Amaurosis with a site in United States, this one is the 8th largest by planned enrollment of the 16 interventional ones that give a count, is the only one that started in 2023, is one of 9 in phase 1/2, and was the 21st registered.
32 studies on the registry are indexed under Leber Congenital Amaurosis; 4 are open to participants now.
Browse Leber Congenital Amaurosis studies →4 of the 23 studies in Leber Congenital Amaurosis with a site in United States are open now. Recruiting first, the newest first:
Opus Genetics, Inc · Sites in United States
Ages 18+
Laboratoires Thea · Sites in United States, Australia, Belgium and 7 more countries
Ages 6+
Foundation Fighting Blindness · Sites in United States
Healthy volunteers
Sanford Health · Sites in United States
A study can be open while its sites in United States have not opened yet or have stopped. Only those recruiting at a site in United States →
Of the 4 Leber Congenital Amaurosis studies recruiting now, 4 set no upper age limit and none stop at 75 or younger; 3 take children under 18; 1 takes healthy volunteers.
This study: 6 Years to 50 Years · all sexes · no healthy volunteers.
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23 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 13 · Phase 2 10 · Phase 3 3
First in 2007; this study’s year is marked. 1 started in 2023.
This study's planned enrollment of 20 is above the median of 15 across 23 interventional studies indexed under Leber Congenital Amaurosis.
Median enrollment 18 across the 16 interventional studies, 59 across the 7 observational. Of the 12 that completed or were terminated, 3 (25%) have results posted.
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20 of 23 have an industry lead sponsor. The 10 most frequent:
HuidaGene Therapeutics Co., Ltd. is the lead sponsor of 7 studies on the registry; 4 are open to participants now.
HuidaGene Therapeutics Co., Ltd. has 1 study filed under Leber Congenital Amaurosis — fewer than ten, so there is no comparison to draw.
3 of the studies in Leber Congenital Amaurosis with a site in United States that are under way reach primary completion within a year, 1 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT06891443Phase 3Laboratoires TheaStudy to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION) | 2027-11 |
| NCT05203939OcugenStudy to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis | 2027-03 |
| NCT01208389Spark Therapeutics, Inc.Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2 | 2027-10 |
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Among the 23 studies in Leber Congenital Amaurosis with a site in United States, from their registry updates:
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The purpose of the study is to determine whether HG004 as gene therapy is safe and effective for the treatment of Leber Congenital Amaurosis caused by mutations in RPE65 gene.
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Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Drug: HG004
Low dose Medium dose High dose
Incidence of ocular and non-ocular adverse events
Time frame: 52 weeks
Change from baseline in Best Corrected Visual Acuity (BCVA) of letters based on the Early Treatment Diabetic Retionpathy Study (ETDRS) chart
Time frame: 52 weeks
Change from baseline in visual fields of full-field stimulus threshold test in log cd.s/m2.
Time frame: 52 weeks
Plan to share: No
No publications or documents are linked to this record.
Started Oct 2023 (actual). Primary completion is estimated for Dec 2025, 26 months after the start.
How these figures are worked out, and which studies are left out of them.
This study is status unknown, as verified in Sep 2024. You cannot join it, but the record below documents what was studied.
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HuidaGene Therapeutics Co., Ltd.