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Active clinical trials for "Stargardt Disease"

Results 1-10 of 43

Oral Metformin for Treatment of ABCA4 Retinopathy

ABCA4 RetinopathyStargardt Disease2 more

Background: ABCA4 retinopathy is a genetic disease in which the ABCA4 protein is absent or faulty. It can cause waste material to collect in the eye and may cause cells to die. The cell death can lead to vision loss. Researchers want to see if an oral drug called metformin can help. Objective: To see if metformin is safe and possibly helps to slow the rate of ABCA4 retinopathy. Eligibility: People age 12 and older who have ABCA4 retinopathy and have problems with their vision. Design: Participants will be screened under a separate protocol. Participants will have a medical and family history. They will complete a questionnaire about their vision and daily activities. They will have a physical exam. They may have blood drawn through a needle in the arm. Participants will have an eye exam. Their pupils may be dilated with eye drops. Their retina may be photographed. Participants will have a visual field test. They will sit in front of a large dome and press a button when they see a light within the dome. Participants will have an electroretinogram. It examines the function of the retina. They will sit in the dark for 30 minutes. Then their eyes will be numbed with eye drops. They will wear contact lenses that can sense signals from the retinas. They will watch flashing lights. Participants will have optical coherence tomography. This non-invasive procedure makes pictures of the retina. Participants will have fundus autofluorescence. A bright blue light will be shone into their eye. Participants will take metformin by mouth for 24 months. Participants will have study visits every 6 months. Participation will last for at least 36 months....

Recruiting33 enrollment criteria

Study to Assess the Safety and Efficacy of OCU410ST for Stargardt Disease

Stargardt Disease

This is a Phase 1/2 Study to Assess the Safety and Efficacy of OCU410ST for Stargardt Disease. This is a multicenter study, which will be conducted in two phases and will enroll up to a total of 42 subjects.

Recruiting20 enrollment criteria

Phase 2 Tolerability and Effects of ALK-001 on Stargardt Disease

Stargardt DiseaseStargardt Macular Degeneration2 more

The purpose of this study is to determine the long term safety and tolerability of ALK-001 (C20-D3-retinyl acetate), and to explore the effects of ALK-001 on the progression of Stargardt disease in patients between the ages of 8 and 70 years old. Funding Source - FDA OOPD

Recruiting19 enrollment criteria

Stem Cell Ophthalmology Treatment Study II

Retinal DiseaseAge-Related Macular Degeneration17 more

This study will evaluate the use of autologous bone marrow derived stem cells (BMSC) for the treatment of retinal and optic nerve damage or disease.

Recruiting12 enrollment criteria

Open-Label Extension: Tolerability and Effects of ALK-001 on Stargardt Disease (TEASE)

Stargardt DiseaseStargardt Macular Degeneration2 more

The purpose of this open-label, multicenter study is to determine the long-term safety, pharmacokinetics and effects of ALK-001 (C20-D3-retinyl acetate) on the progression of Stargardt disease. This study is an extension of NCT02402660 and enrolls participants who are at least 8 years old. Enrollment is by invitation only. Funding Source - FDA OOPD

Enrolling by invitation14 enrollment criteria

This is a Dose-finding Study Followed by 2-year Extension Study to Evaluate Safety and Tolerability...

Stargardt Disease

Stargardt disease 1 (STGD1) is the most prevalent form of juvenile macular degeneration. It is caused by a rare, inherited autosomal recessive trait, leading to severe and irreversible blindness by the first or second decade of life. Earlier onset of the disease is related to a rapid vision loss, while patients with a later onset tend to have a better prognosis. This study will enrol subjects aged 12-18 years old with a confirmed clinical diagnosis of Stargardt disease type 1 (STGD1). This study will include 2 phases, the phase 1b portion is to determine the optimal dose for phase 2 based on the extent of retinol binding protein 4 (RBP4) reduction after 2 cycles of tinlarebant treatment. The phase 2 portion will evaluate the safety and efficacy of a single daily dose of tinlarebant over a 24-month treatment period.

Active4 enrollment criteria

Safety and Effects of a Single Intravitreal Injection of vMCO-010 Optogenetic Therapy in Subjects...

Stargardt Disease

The purpose of the study is to evaluate the safety and effects of a single intravitreal injection of virally-carried Multi-Characteristic Opsin (vMCO-010) in Subjects with Stargardt Disease

Active27 enrollment criteria

Zimura Compared to Sham in Patients With Autosomal Recessive Stargardt Disease (STGD1)

Stargardt Disease 1

To evaluate the safety and efficacy of Zimura™ (complement factor C5 inhibitor) compared to Sham in subjects with autosomal recessive Stargardt disease 1 (STGD1).

Active14 enrollment criteria

Study to Evaluate the Safety and Efficacy of Tinlarebant in the Treatment of Stargardt Disease in...

Stargardt Disease 1

The primary objective of this trial is to assesses the efficacy of tinlarebant in slowing the rate of growth of atrophic lesion(s) in adolescent subjects with STGD1

Active10 enrollment criteria

Phase I/II Follow-up Study of SAR422459 in Patients With Stargardt's Macular Degeneration

Stargardt's Disease

Primary Objective: To evaluate the long-term safety and tolerability of SAR422459 in patients with Stargardt's Macular Degeneration. Secondary Objective: To assess: Safety Biological activity

Active7 enrollment criteria
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