Leber Congenital Amaurosis

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9 Leber Congenital Amaurosis Trials Near You

Power is an online platform that helps thousands of Leber Congenital Amaurosis patients discover FDA-reviewed trials every day. Every trial we feature meets safety and ethical standards, giving patients an easy way to discover promising new treatments in the research stage.

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No Placebo
Highly Paid
Stay on Current Meds
Pivotal Trials (Near Approval)
Breakthrough Medication
The purpose of this study is to evaluate the safety, tolerability and efficacy of a single escalating doses of EDIT-101 administered via subretinal injection in participants with LCA10 caused by a homozygous or compound heterozygous mutation involving c.2991+1655A\>G in intron 26 of the CEP290 gene ("LCA10-IVS26").
No Placebo Group

Trial Details

Trial Status:Active Not Recruiting
Trial Phase:Phase 1, 2
Age:3+

34 Participants Needed

This trial tests OCU400, an eye injection, in patients with specific genetic mutations causing RP and LCA. The treatment aims to correct faulty genes to prevent vision loss.
No Placebo Group

Trial Details

Trial Status:Active Not Recruiting
Trial Phase:Phase 1, 2
Age:6+

22 Participants Needed

The study is a Phase 3, open-label, randomized controlled trial of gene therapy intervention by subretinal administration of AAV2-hRPE65v2 (voretigene neparvovec-rzyl). At least twenty-four subjects, three years of age or older, will be recruited. The intervention group will receive AAV2-hRPE65v2 at either The Children's Hospital of Philadelphia or University of Iowa to determine if it improves visual and retinal function in individuals with RPE65 gene mutations.
No Placebo Group
Pivotal Trial (Near Approval)

Trial Details

Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:3+

31 Participants Needed

This trial is testing a new gene therapy injected under the retina to help people with a specific genetic eye condition that causes vision loss. The therapy aims to replace faulty genes with healthy ones to improve vision. Mutations in the RPE65 gene are one of the causes of RP and Leber congenital amaurosis (LCA), a form of RP present at birth.
No Placebo Group

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 1, 2
Age:13+

15 Participants Needed

Primary Objective: To evaluate the safety and tolerability of ascending doses of ATSN-101 administered as a unilateral subretinal injection in patients with Leber Congenital Amaurosis (LCA) caused by autosomal recessive guanylate cyclase 2D (GUCY2D) mutations (GUCY2D-LCA). Secondary Objective: To evaluate the efficacy of ascending doses of ATSN-101 administered as a unilateral subretinal injection in patients with GUCY2D-LCA.
No Placebo Group

Trial Details

Trial Status:Active Not Recruiting
Trial Phase:Phase 1, 2
Age:6+

15 Participants Needed

The study is a follow-on to a Phase 1 dose-escalation and safety study.
No Placebo Group

Trial Details

Trial Status:Active Not Recruiting
Trial Phase:Phase 1, 2
Age:8+

12 Participants Needed

The purpose of this double-masked, randomized, placebo-controlled, paired-eye study is to evaluate the efficacy, safety and tolerability of Sepofarsen in subjects with Leber Congenital Amaurosis (LCA) due to the c.2991+1655A\>G (p.Cys998X) mutation in the CEP290.
Pivotal Trial (Near Approval)

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 3
Age:6+

32 Participants Needed

A recombinant adeno-associated virus serotype 2 (rAAV2) vector has been altered to carry the human RPE65 (hRPE65) gene. This vector has been shown to restore vision in animal models that resemble human RPE65-associated Leber congenital amaurosis (LCA), an incurable retinal degeneration that causes severe vision loss. The proposed study is an open label, Phase I clinical trial of subretinal rAAV2-CBSB-hRPE65 administration to individuals with RPE65-associated retinal disease. Five cohorts will be included in this trial. Cohorts 1, 2 and 4 will consist of individuals 18 years of age and older. Cohorts 3 and 5 will consist of individuals between the ages of 8 and 17, inclusive. Enrollment in Cohorts 3 and 5 will begin only after confirming the safety of rAAV2-CBSB-hRPE65 administration in the older groups of participants. This trial will lead to a greater understanding of the safety and thereby potential value of gene transfer in RPE65-associated retinal disease and will have implications for other forms of retinal degenerative disease amenable to this type of intervention. The goal of this clinical trial is to determine the safety of uniocular subretinal administration of rAAV2-CBSB-hRPE65 in individuals with RPE65-associated retinal disease. Ocular and systemic toxicity will be assessed prior to and following vector administration to determine if there are adverse changes that may be associated with vector administration.
No Placebo Group

Trial Details

Trial Status:Active Not Recruiting
Trial Phase:Phase 1
Age:8+

15 Participants Needed

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.
No Placebo Group

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 1, 2
Age:6 - 50

20 Participants Needed

Why Other Patients Applied

"I was diagnosed with stage 4 pancreatic cancer three months ago, metastatic to my liver, and I have been receiving and responding well to chemotherapy. My blood work revealed that my tumor markers have gone from 2600 in the beginning to 173 as of now, even with the delay in treatment, they are not going up. CT Scans reveal they have been shrinking as well. However, chemo is seriously deteriorating my body. I have 4 more treatments to go in this 12 treatment cycle. I am just interested in learning about my other options, if any are available to me."

ID
Pancreatic Cancer PatientAge: 40

"My orthopedist recommended a half replacement of my right knee. I have had both hips replaced. Currently have arthritis in knee, shoulder, and thumb. I want to avoid surgery, and I'm open-minded about trying a trial before using surgery as a last resort."

HZ
Arthritis PatientAge: 78

"I have dealt with voice and vocal fold issues related to paralysis for over 12 years. This problem has negatively impacted virtually every facet of my life. I am an otherwise healthy 48 year old married father of 3 living. My youngest daughter is 12 and has never heard my real voice. I am now having breathing issues related to the paralysis as well as trouble swallowing some liquids. In my research I have seen some recent trials focused on helping people like me."

AG
Paralysis PatientAge: 50

"I changed my diet in 2020 and I’ve lost 95 pounds from my highest weight (283). I am 5’3”, female, and now 188. I still have a 33 BMI. I've been doing research on alternative approaches to continue my progress, which brought me here to consider clinical trials."

WR
Obesity PatientAge: 58

"I've tried several different SSRIs over the past 23 years with no luck. Some of these new treatments seem interesting... haven't tried anything like them before. I really hope that one could work."

ZS
Depression PatientAge: 51

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We started Power when my dad was diagnosed with multiple myeloma, and I struggled to help him access the latest immunotherapy. Hopefully Power makes it simpler for you to explore promising new treatments, during what is probably a difficult time.

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Bask GillCEO at Power
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Frequently Asked Questions

How much do Leber Congenital Amaurosis clinical trials pay?

Each trial will compensate patients a different amount, but $50-100 for each visit is a fairly common range for Phase 2–4 trials (Phase 1 trials often pay substantially more). Further, most trials will cover the costs of a travel to-and-from the clinic.

How do Leber Congenital Amaurosis clinical trials work?

After a researcher reviews your profile, they may choose to invite you in to a screening appointment, where they'll determine if you meet 100% of the eligibility requirements. If you do, you'll be sorted into one of the treatment groups, and receive your study drug. For some trials, there is a chance you'll receive a placebo. Across Leber Congenital Amaurosis trials 30% of clinical trials have a placebo. Typically, you'll be required to check-in with the clinic every month or so. The average trial length for Leber Congenital Amaurosis is 12 months.

How do I participate in a study as a "healthy volunteer"?

Not all studies recruit healthy volunteers: usually, Phase 1 studies do. Participating as a healthy volunteer means you will go to a research facility several times over a few days or weeks to receive a dose of either the test treatment or a "placebo," which is a harmless substance that helps researchers compare results. You will have routine tests during these visits, and you'll be compensated for your time and travel, with the number of appointments and details varying by study.

What does the "phase" of a clinical trial mean?

The phase of a trial reveals what stage the drug is in to get approval for a specific condition. Phase 1 trials are the trials to collect safety data in humans. Phase 2 trials are those where the drug has some data showing safety in humans, but where further human data is needed on drug effectiveness. Phase 3 trials are in the final step before approval. The drug already has data showing both safety and effectiveness. As a general rule, Phase 3 trials are more promising than Phase 2, and Phase 2 trials are more promising than phase 1.

Do I need to be insured to participate in a Leber Congenital Amaurosis medical study?

Clinical trials are almost always free to participants, and so do not require insurance. The only exception here are trials focused on cancer, because only a small part of the typical treatment plan is actually experimental. For these cancer trials, participants typically need insurance to cover all the non-experimental components.

What are the newest Leber Congenital Amaurosis clinical trials?

Most recently, we added Sepofarsen for Leber Congenital Amaurosis, Gene Therapy for Leber Congenital Amaurosis and Gene Therapy for Leber Congenital Amaurosis to the Power online platform.

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