← Back to Search

Gene Therapy

Gene Therapy with Light-Stimulating Glasses for Retinitis Pigmentosa (PIONEER Trial)

Phase 1 & 2

Recruiting

Research Sponsored by GenSight Biologics

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Diagnosis of non-syndromic RP confirmed on full-field ERG

Diagnosis of non-syndromic RP based on history, mid-peripheral visual dysfunction, and fundoscopic appearance

Timeline

Screening 3 weeks

Treatment Varies

Follow Up week 52/year 1

Awards & highlights

PIONEER Trial Summary

This trial is testing a gene therapy called GS030-DP for safety and tolerability in people with a disease called Retinitis Pigmentosa. The therapy is given as a single injection and then the person wears special glasses that provide light stimulation.

Who is the study for?

Adults aged 18-75 with non-syndromic Retinitis Pigmentosa (RP), confirmed by specific tests, who have not had gene therapy before or significant eye surgery within the last 3 months. Participants should have a certain range of visual acuity and refractive error, as well as an appropriate interpupillary distance.Check my eligibility

What is being tested?

The trial is testing GS030-DP, a new gene therapy given through an injection in the eye, alongside GS030-MD, special glasses that provide light stimulation. The study aims to find out how safe these treatments are and what doses are tolerable for people with RP.See study design

What are the potential side effects?

Potential side effects aren't specified here but generally could include discomfort at the injection site, inflammation in the eye, headache from light stimulation or vision changes due to treatment.

PIONEER Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

My retinitis pigmentosa diagnosis was confirmed with an ERG test.

Select...

I have been diagnosed with non-syndromic retinitis pigmentosa.

Select...

I am between 18 and 75 years old.

PIONEER Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ week 52/year 1

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~week 52/year 1

This trial's timeline: 3 weeks for screening, Varies for treatment, and week 52/year 1 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

The safety and tolerability of escalating doses of GS030-DP administered via a single IVT and repeated light stimulation using GS030-MD in subjects with non-syndromic Retinitis Pigmentosa

Secondary outcome measures

Evaluate immune response to recombinant adeno associated viral vector, derived from serotype 2 (rAAV2.7m8) and ChR tdT protein.

Evaluate the treatment effect of GS030 as assessed by QoL

Therapeutic procedure

+2 more

PIONEER Trial Design

1Treatment groups

Experimental Treatment

Group I: CohortExperimental Treatment1 Intervention

3 dose escalation cohorts (low, medium and high dose) with 3 subjects per cohort followed by an extension cohort at the highest-well tolerated dose with 3 to 9 subjects.

0 / 3Eligibility criteria met

Find a Location

Who is running the clinical trial?

GenSight BiologicsLead Sponsor

8 Previous Clinical Trials

303 Total Patients Enrolled

Media Library

GS030-DP (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT03326336 — Phase 1 & 2

Retinitis Pigmentosa Research Study Groups: Cohort

Retinitis Pigmentosa Clinical Trial 2023: GS030-DP Highlights & Side Effects. Trial Name: NCT03326336 — Phase 1 & 2

GS030-DP (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03326336 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is recruitment for this research currently open?

"According to clinicaltrials.gov, enrollment for this experiment is open - it was initially posted on the 26th of September 2018, and the latest update was made in July 25th 2022."

Answered by AI

Are octogenarians being considered for the trial?

"This clinical trial is open to individuals aged 18-75. If a research participant falls outside of this range, there are 12 available studies for those under 18 and 44 for people over 65 years old."

Answered by AI

For what demographic is this research particularly appropriate?

"This clinical trial is seeking 15 individuals with retinitis pigmentosa between 18 and 75 years of age. Vital requirements include: an age range from 18 to no more than 75, a diagnosis of non-syndromic RP based on medical records, visual acuity worse than CF (depending on dose status data), refractive error ranging -6 diopters to +6 diopters in the study eye, and other exclusion criteria."

Answered by AI

What is the enrollment number of individuals taking part in this trial?

"Affirmative. According to the information provided by clinicaltrials.gov, this experiment is currently recruiting participants and was initially posted on September 26th 2018 with its most recent update occuring in July 25th 2022. Fifteen candidates are being sought out from a single medical facility."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Dose-escalation Study to Evaluate the Safety and Tolerability of GS030 in Subjects With Retinitis Pigmentosa

2018. "Dose-escalation Study to Evaluate the Safety and Tolerability of GS030 in Subjects With Retinitis Pigmentosa". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03326336.

All > Retinitis Pigmentosa