Phase I Trial of Ocular Subretinal Injection of a Recombinant Adeno-Associated Virus (rAAV2-VMD2-hMERTK) Gene Vector to Patients With Retinal Disease Due to MERTK Mutations

Trial of Ocular Subretinal Injection of a Recombinant Adeno-Associated Virus (rAAV2-VMD2-hMERTK) Gene Vector to Patients With Retinal Disease Due to MERTK Mutations

Sponsors

Lead sponsor: Fowzan Alkuraya

Collaborator: King Khaled Eye Specialist Hospital
King Faisal Specialist Hospital & Research Center

Source King Khaled Eye Specialist Hospital
Brief Summary

A recombinant adeno-associated virus serotype 2 (rAAV2) vector has been altered to carry the human MERTK (hMERTK) gene. This vector has been shown to restore vision in animal models that resemble human MERTK-associated Retinitis Pigmentosa (RP), an incurable retinal degeneration that causes severe vision loss. The proposed study is an open label, Phase I clinical trial of subretinal rAAV2-VMD2-hMERTK administration to individuals with MERTK-associated retinal disease. This trial will lead to a greater understanding of the safety and thereby potential value of gene transfer in MERTK-associated retinal disease and will have implications for other forms of retinal degenerative disease amenable to this type of intervention.

Overall Status Recruiting
Start Date August 2011
Completion Date August 2023
Primary Completion Date August 2014
Phase Phase 1
Study Type Interventional
Primary Outcome
Measure Time Frame
Ocular and Systemic safety of uniocular subretinal administration of rAAV2-VMD2-hMERTK in individuals with MERTK-associated retinal disease 12 yrs
Secondary Outcome
Measure Time Frame
Visual Outcome 12 yrs
Enrollment 6
Condition
Intervention

Intervention type: Genetic

Intervention name: Recombinant Adeno-Associated Virus

Description: Ocular Subretinal Injection of a Recombinant Adeno-Associated Virus

Arm group label: Recombinant Adeno-Associated Virus

Eligibility

Criteria:

Inclusion Criteria:

- MERTK-associated retinal disease;

- VA: 20/100 or less in worse eye

- Ability to perform tests of visual and retinal function;

- Good general health based on a complete physical examination and hematology and chemistry studies performed at a pre-treatment evaluation;

- Ability to comply with research procedures;

Exclusion Criteria:

- Pre-existing eye conditions that would preclude the planned surgery or interfere with the interpretation of study endpoints or surgical complications (for example, glaucoma, corneal or lenticular opacities);

- Complicating systemic diseases (such as medical conditions causing immunosuppression) that would preclude the gene transfer, ocular surgery or known sensitivity or allergy to medications planned for use in the peri-operative period;

- Use of anti-platelet agents that may alter coagulation within 7 days prior to study agent administration;

- Use of immunosuppressive medications;

- Pregnancy or breastfeeding;

- Individuals (males and females) of childbearing potential who are unwilling to use effective contraception for 1 year following agent administration and barrier contraception for 3 months following agent administration;

- Any other condition that would prevent a subject from completing follow-up examinations during the course of the study and that, in the opinion of the investigator, makes the subject unsuitable for the study.

- Current, or recent (within the past 30 days, or 10 half lives of the drug) participation, in any other research protocol involving investigational agents or therapies.

- Recent (within past 6 months) receipt of an investigational biologic therapeutic agent.Subjects will not be excluded based on their gender, race or ethnicity.

Gender: All

Minimum age: 14 Years

Maximum age: 70 Years

Healthy volunteers: No

Overall Official
Last Name Role Affiliation
Fowzan S Alkuraya, MD Principal Investigator King Faisal Specialist Hospital & Research Center
Overall Contact

Last name: Fowzan S Alkuraya, MD

Phone: +966 1 442 7875

Email: [email protected]

Location
facility status contact investigator King Khaled Eye Specialist Hospital Deepak Edward, MD 96614821234 1362 [email protected] Emad Abboud, MD Principal Investigator
Location Countries

Saudi Arabia

Verification Date

November 2011

Responsible Party

Responsible party type: Sponsor-Investigator

Investigator affiliation: King Khaled Eye Specialist Hospital

Investigator full name: Fowzan Alkuraya

Investigator title: Head, Developmental Genetics Unit

Keywords
Has Expanded Access No
Condition Browse
Number Of Arms 1
Arm Group

Arm group label: Recombinant Adeno-Associated Virus

Arm group type: Experimental

Study Design Info

Allocation: N/A

Intervention model: Single Group Assignment

Primary purpose: Treatment

Masking: None (Open Label)

Source: ClinicalTrials.gov