Study of ALK-001 on the Progression of Stargardt Disease
Recruiting
Phase 3Interventional Study
Stargardt Disease
Pivotal Trial
This treatment is in the last trial phase before FDA approval.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
Ready to participate?
Review the details below, then apply to join this clinical trial.
At a Glance
Age
8 – 45
Sex
Any
Trial phase
Phase 3
Study type
Interventional
Purpose
Treatment
Participants needed
230 (estimated)
Sponsor
Alkeus Pharmaceuticals, Inc. · Industry
Who this trial is looking for
Think this trial could be right for you?
Answer a few quick questions to see if you may meet the eligibility requirements.
This study evaluates the efficacy and safety of investigational study drug ALK-001 in participants 8 to 45 years of age, inclusive, with symptoms and signs of autosomal recessive Stargardt disease (STGD)
Trial Locations
Loading…
Loading trial locations…
Facility
City
State
Country
Status
Eligibility Criteria
Inclusion Criteria
1. Male or female, 8 to 45 years of age (inclusive) on the day of screening.
2. Female participants of childbearing potential, as well as fertile male participants with female partners of childbearing potential, must be willing to comply with the protocol-defined methods of contr…
Inclusion Criteria
1. Male or female, 8 to 45 years of age (inclusive) on the day of screening.
2. Female participants of childbearing potential, as well as fertile male participants with female partners of childbearing potential, must be willing to comply with the protocol-defined methods of contraception from the time of consent until 90 days and 30 days, respectively, after the last dose of IMP.
3. Have a clinical diagnosis of typical autosomal recessive STGD macular dystrophy
4. Have provided a genetic report by any regional accredited organization that provides certified testing indicating the presence of disease-causing mutation.
5. Have signed and dated the informed consent forms (ICFs; or assent as appropriate) to participate.
Exclusion Criteria
1. Has taken disallowed items (supplements containing vitamin A or beta-carotene, liver-based products, or prescription oral retinoid medications) within 30 days of randomization.
2. Is lactating, pregnant, or has a positive serum or urine pregnancy test at screening or at randomization, or is planning to become pregnant during the course of study. A male participant who wishes to father a child during the course of the study.
3. Has ever participated in any gene therapy, cell therapy, or device study to treat STGD, unless documented confirmation of participation in the placebo arm with no surgery is provided. Has participated in a drug study to treat STGD within the past 6 months.
4. Has participated in any drug study to treat any other condition within 5 half-lives of the investigational drug prior to screening, unless documented confirmation of participation in the placebo arm is provided. Has participated in an investigational device study within 30 days prior to screening or longer if, in the Investigator's judgment, the device could affect study outcomes.
5. Anticipates participating in any other drug or device study within the duration of the study.
Contacts
For trial questions: trials@alkeuspharma.com or 877-255-7476
Picking one helps us show the most relevant trials first.
Your results are loading in the background — you can
change this any time from the results page.
Set your location to continue
Find My Trials uses your location to surface clinical trials near you.
Add your city or zip code to your profile and try again.