A Phase 1/2 interventional study of voretigene neparvovec-rzyl in Leber Congenital Amaurosis, sponsored by Spark Therapeutics, Inc.. Active, not recruiting at 1 site in United States. Open to participants aged 8 Years and older. Per ClinicalTrials.gov, last updated 2025-04-29.
Sponsored by Spark Therapeutics, Inc. (part of Hoffmann-La Roche) · 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 12th largest by planned enrollment of the 16 interventional ones that give a count, is one of 2 that started in 2010, is one of 9 in phase 1/2, and was the 6th 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: 8 Years and older · all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
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. 2 started in 2010.
This study's planned enrollment of 12 is below 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:
This study is registered under Spark Therapeutics, Inc., one of the names counted as Hoffmann-La Roche. Under that name alone:
Spark Therapeutics, Inc. is the lead sponsor of 14 studies on the registry; none are open to participants now.
Hoffmann-La Roche has 5 studies 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 study is a follow-on to a Phase 1 dose-escalation and safety study.
The study is a follow-on to a Phase 1 dose-escalation and safety study (closed to enrollment as of June 2009). Up to twelve adults and children with a molecular diagnosis of biallelic RPE65 mutations, who have participated in the earlier Phase 1 study, and who meet all study eligibility criteria, will receive AAV2-hRPE65v2 vector in the previously uninjected, contralateral eye to evaluate the safety of bilateral, sequential subretinal administration of AAV2-hRPE65v2.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
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.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
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.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Administration of study agent (AAV2-hRPE65v2) to the previously, uninjected contralateral eye:
Biological: voretigene neparvovec-rzyl
One time, subretinal administration of 1.5E11 vg AAV2-hRPE65v2 vector in 300 microliters to the contralateral, previously uninjected eye.
Also known as: AAV2-hRPE65v2
Adverse events as a measure of safety and tolerability
The primary outcome measures are safety and tolerability. Secondary outcome measure(s) include changes in visual function as measured by subjective, psychophysical tests and by objective, physiologic tests.
Time frame: 15 years
Visual acuity
The primary outcome measures are safety and tolerability. Secondary outcome measure(s) include changes in visual function as measured by subjective, psychophysical tests and by objective, physiologic tests.
Time frame: 15 years
Visual field
The primary outcome measures are safety and tolerability. Secondary outcome measure(s) include changes in visual function as measured by subjective, psychophysical tests and by objective, physiologic tests.
Time frame: 15 years
Pupillary light response
The primary outcome measures are safety and tolerability. Secondary outcome measure(s) include changes in visual function as measured by subjective, psychophysical tests and by objective, physiologic tests.
Time frame: 15 years
Mobility testing
The primary outcome measures are safety and tolerability. Secondary outcome measure(s) include changes in visual function as measured by subjective, psychophysical tests and by objective, physiologic tests.
Time frame: 15 years
Full-field light threshold sensitivity testing
The primary outcome measures are safety and tolerability. Secondary outcome measure(s) include changes in visual function as measured by subjective, psychophysical tests and by objective, physiologic tests.
Time frame: 15 years
Contrast sensitivity
The primary outcome measures are safety and tolerability. Secondary outcome measure(s) include changes in visual function as measured by subjective, psychophysical tests and by objective, physiologic tests.
Time frame: 15 years
Plan to share: No
Started Nov 2010 (actual). Primary completion is estimated for Oct 2027, 203 months after the start.
How these figures are worked out, and which studies are left out of them.
This study is active, not recruiting, as verified in Dec 2024. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Voretigene neparvovec