50 Participants Needed

Natural History of PRPF31 Mutation-Associated Retinal Dystrophy

Recruiting at 6 trial locations
GN
LV
LP
CG
Overseen ByClare Guerrero
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

What You Need to Know Before You Apply

What is the purpose of this trial?

The purpose of this study is to characterize the natural history through temporal systemic evaluation of subjects identified with PRPF31 mutation-associated retinal dystrophy, also called retinitis pigmentosa type 11, or RP11.Assessments will be completed to measure and evaluate structural and functional visual changes including those impacting patient quality of life associated with this inherited retinal condition and observing how these changes evolve over time.

Who Is on the Research Team?

SM

Sreenivasu Mudumba, PhD

Principal Investigator

PYC Therapeutics

Are You a Good Fit for This Trial?

Inclusion Criteria

Male or female, ≥ 10 years of age at baseline (Visit 2).
Have a clinical and molecular diagnosis of PRPF31 mutation-associated retinal dystrophy.
Are willing to comply with the instructions and attend all scheduled study visits.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

4 weeks
1 visit (in-person)

Baseline Assessment

Initial assessments including ophthalmic exams, imaging studies, and electrophysiological testing

1 visit
1 visit (in-person)

Follow-up

Participants are monitored for changes in visual function and retinal structure every 16 weeks for the first year and every 24 weeks for up to 4 years

4 years
Regular visits every 16-24 weeks

What Are the Treatments Tested in This Trial?

Interventions

  • None

Find a Clinic Near You

Who Is Running the Clinical Trial?

PYC Therapeutics

Lead Sponsor

Trials
7
Recruited
170+
Unbiased ResultsWe believe in providing patients with all the options.
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