NCT06942572 · Splice Bio
A Phase 1/2, First-in-Human Dose Escalation/Expansion Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of a Subretinal Injection of SB-007 in Subjects With Stargardt Disease (STGD1)
(ASTRA)
What this study is about
This Phase 1/2 study will evaluate the safety, tolerability, and preliminary effectiveness of subretinal SB-007 administration to determine dose selection in subjects with Stargardt's Type 1 (STGD1). This is a conducted at multiple hospitals study which will enroll approximately 57 subjects, followed up over a 96 week period post treatment after a single administration of SB-007.
View original scientific description
This Phase 1/2 study will evaluate the safety, tolerability, and preliminary efficacy of subretinal SB-007 administration to determine dose selection in subjects with Stargardt's Type 1 (STGD1). This is a multicenter study which will enroll approximately 57 subjects, followed up over a 96 week period post treatment after a single administration of SB-007.
Interventions
GENETIC
SB-007
Subretinal Administration of SB-007
Primary outcome measures
Safety Measures
Time frame: 96 weeks
Safety and tolerability assessed by incidence and/or clinically significant changes in ocular and non-ocular AEs
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- To be eligible for study participation, subjects must meet the following criteria: 1. Provide written consent. Subjects under legal age will also provide informed assent according to guidelines set forth by the same. 2. Are male or female adolescents and adults, aged as follows: 1. In Part A, subjects will be ≥18 to ≤65\
- years (inclusive) 2. In Part B, subject age is planned as ≥12 to ≤65\
- years (inclusive) \*Subjects aged \>65 years may be eligible in Parts A and B, following discussion with, and approval by, the Medical Monitor. 3. Are able to understand and comply with the study procedures. 4. Have a diagnosis of STGD1 caused by bi-allelic pathogenic, or likely pathogenic, variants in the ABCA4 gene confirmed genotypically by an accredited genetic testing laboratory 5. Clinical evidence consistent with Stargardt Disease type 1. 6. For women of child-bearing potential (WOCBP), have a negative pregnancy test at Screening and, if due to receive active tre
Where
- Aurora, Colorado
- Miami, Florida
- Boston, Massachusetts
- Portland, Oregon
- Dallas, Texas
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Apr 29, 2025 · Source of record for eligibility and locations