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rAAV2tYF-CB-hRS1

Phase 1

X-linked Retinoschisis | Monoclonal antibody | Other |Elevance Health, Inc.|Last Updated: Jun 12, 2023

Success Probability

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Market & Valuation

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Trial Design

CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment27

FDA Designations

No designations recorded

Clinical trial landscape

rAAV2tYF-CB-hRS1 · 1 trial · 1 indication

Phase 1 1
NCT02416622Safety and Efficacy of rAAV-hRS1 in Patients With X-linked Retinoschisis (XLRS)X-linked Retinoschisis
COMPLETED27 Analytics
PHASE1COMPLETED
Safety and Efficacy of rAAV-hRS1 in Patients With X-linked Retinoschisis (XLRS)
X-linked RetinoschisisUnlock trial analytics

Study Endpoints

Primary Endpoints

Number of Participants Experiencing Adverse Events
From Day 0 to Month 12 (12 months)

Number of participants with ocular adverse events, assessed by standard ophthalmic examination, including slit lamp biomicroscopy, tonometry, and indirect ophthalmoscopy.

Secondary Endpoints

Change From Baseline in Best Corrected Visual Acuity (BCVA)
From Day 0 to Month 12 (12 Months)
Change From Baseline in Schisis Cavity Size on Optical Coherence Tomography (OCT)
From Day 0 to Month 12 (12 months)
Change From Baseline in B-wave Amplitude in Electroretinogram (ERG) Responses
From Day 0 to Month 12 (12 months)
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Study Design & Arms

AllocationNON_RANDOMIZED
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Groups 1A and 1BEXPERIMENTALSubjects at least 18 y/o treated with a lower dose of rAAV2tYF-CB-hRS1 study drug.
Groups 2 and 2AEXPERIMENTALSubjects at least 6 y/o treated with a middle dose of rAAV2tYF-CB-hRS1 study drug.
Group 3EXPERIMENTALSubjects at least 18 y/o treated with a higher dose of rAAV2tYF-CB-hRS1 study drug.
Group 4EXPERIMENTALSubjects at least 6 y/o treated with a maximum tolerated dose of rAAV2tYF-CB-hRS1 study drug determined for Groups 1A, 1B, 2 and 3.

Interventions

NameTypeDescription
rAAV2tYF-CB-hRS1BIOLOGICALadeno-associated virus vector expressing retinoschisin
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Eligibility Criteria

Age Range6 Years to N/A
SexMALE
Healthy VolunteersNo
Study Sites9

Inclusion Criteria include: 1. Retinal disease consistent with a diagnosis of XLRS and documented mutations in the RS1 gene 2. Male individual at least 18 years of age (dose escalation phase) or at least 6 years of age (maximum tolerated dose phase), 3. Able to perform tests of visual and retinal f...

Countries:United States
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Frequently asked questions about rAAV2tYF-CB-hRS1

What is rAAV2tYF-CB-hRS1 used for?

rAAV2tYF-CB-hRS1 is an investigational gene therapy being developed for the treatment of X-linked Retinoschisis, a genetic eye disorder that causes vision loss. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities.

Who is developing rAAV2tYF-CB-hRS1?

rAAV2tYF-CB-hRS1 is being developed by Elevance Health, Inc., a company traded on the stock exchange under the ticker symbol ELV. The drug is currently in Phase 1 clinical trials for X-linked Retinoschisis.

What phase is rAAV2tYF-CB-hRS1 in?

rAAV2tYF-CB-hRS1 is in Phase 1 clinical development. It is an investigational drug and has not yet been approved by the FDA or any other regulatory agency. The drug is being studied for the treatment of X-linked Retinoschisis.

What clinical trials is rAAV2tYF-CB-hRS1 in?

rAAV2tYF-CB-hRS1 has been studied in one clinical trial with the identifier NCT02416622. This Phase 1 trial, titled 'Safety and Efficacy of rAAV-hRS1 in Patients With X-linked Retinoschisis (XLRS)', has been completed and enrolled 27 male participants in the United States.

Is rAAV2tYF-CB-hRS1 the same as rAAV-hRS1?

Yes, rAAV2tYF-CB-hRS1 is also known as rAAV-hRS1. The clinical trial NCT02416622 uses the name rAAV-hRS1 to refer to the same investigational gene therapy being developed for X-linked Retinoschisis.