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Phase 1 Study of Intravitreal Autologous CD34+ Stem Cell Therapy for Retinitis Pigmentosa (BMSCRP1)

Primary Purpose

Retinitis Pigmentosa

Status

Active

Phase

Phase 1

Locations

United States

Study Type

Interventional

Intervention

Intravitreal autologous CD34+ cells

About this trial

This is an interventional treatment trial for Retinitis Pigmentosa focused on measuring retinitis pigmentosa, stem cell, retinal degeneration, intravitreal

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

1. Age >18 years of age 2. Visual Acuity at Presentation of 20/100 to Counting Fingers or constriction of peripheral visual field 360 degrees to <10 degrees by perimetry if BCVA is better than 20/100, duration of vision loss > 3 months 3. Vision loss due to retinitis pigmentosa 4. The study eye has the worse visual acuity or peripheral constriction. 14. Able and willing to sign consent 15. Able to keep follow-up appointments for at least 6 months

Exclusion Criteria:

Other concurrent optic nerve or retinal disease in study eye affecting vision
History of active retinal or choroidal neovascularization or macular edema in study eye requiring on-going intravitreal or periocular therapy in study eye
Active eye or systemic infection
Significant media opacity in the study eye precluding view of the fundus for examination, photography or optical coherence tomography (OCT)

9. Other cause for vision loss in the study eye 10. History of coagulopathy or other hematologic abnormality 11. Use of coumadin within 3 days of enrollment 12. Concurrent immunosuppressive therapy 13. History of allergy to fluorescein dye

Sites / Locations

University of California Davis

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Intravitreal autologous CD34+ cells

Arm Description

Intravitreal injection of autologous CD34+ cells harvested from bone marrow under GMP conditions

Outcomes

Primary Outcome Measures

Safety of intravitreal injection of autologous CD34+ cells

Number and severity of Ocular and systemic adverse events

Feasibility of intravitreal injection of autologous CD34+ cells

Number of CD34+ cells harvested for intravitreal injection

Secondary Outcome Measures

Best corrected visual acuity

Change in number of letters read in study eye from baseline

Best corrected visual acuity

Change in number of letters read in study eye from baseline

Visual Field

Change in visual field area in study eye from baseline

Visual Field

Change in visual field area in study eye from baseline

Electroretinography

Change in a and b wave amplitude in study eye from baseline

Electroretinography

Change in a and b wave amplitude in study eye from baseline

National Eye Institute Vision Questionnaire

Change in Vision Questionnaire compared to baseline

National Eye Institute Vision Questionnaire

Change in Vision Questionnaire compared to baseline

Microperimetry

Change in average threshold from baseline

Microperimetry

Change percent reduced sensitivity from baseline

Microperimetry

Change in average threshold from baseline

Microperimetry

Change in percent reduced sensitivity from baseline

Full Information

NCT ID

NCT04925687

First Posted

June 2, 2021

Last Updated

April 19, 2023

Sponsor

University of California, Davis

Collaborators

Cures Within Reach, Retina Society

1. Study Identification

Unique Protocol Identification Number

NCT04925687

Brief Title

Phase 1 Study of Intravitreal Autologous CD34+ Stem Cell Therapy for Retinitis Pigmentosa

Acronym

BMSCRP1

Official Title

Phase 1 Study of the Feasibility and Safety of Intravitreal Autologous Bone Marrow CD34+ Stem Cell Therapy for Eyes With Vision Loss From Retinitis Pigmentosa

Study Type

Interventional

2. Study Status

Record Verification Date

September 2022

Overall Recruitment Status

Active, not recruiting

Study Start Date

June 1, 2021 (Actual)

Primary Completion Date

November 30, 2023 (Anticipated)

Study Completion Date

November 30, 2023 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

University of California, Davis

Collaborators

Cures Within Reach, Retina Society

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

Brief Summary

In this Phase 1 open-labeled prospective study, one eye of each participant with vision loss from retinitis pigmentosa will be administered intravitreal injection of autologous CD34+ stem cells harvested from bone marrow. Each participant will be examined serially for 6 months after study injection to determine safety and feasibility of this intervention.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Retinitis Pigmentosa

Keywords

retinitis pigmentosa, stem cell, retinal degeneration, intravitreal

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

4 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Intravitreal autologous CD34+ cells

Arm Type

Experimental

Arm Description

Intravitreal injection of autologous CD34+ cells harvested from bone marrow under GMP conditions

Intervention Type

Biological

Intervention Name(s)

Intravitreal autologous CD34+ cells

Intervention Description

Autologous CD34+ cells harvested from bone marrow injected intravitreal

Primary Outcome Measure Information:

Title

Safety of intravitreal injection of autologous CD34+ cells

Description

Number and severity of Ocular and systemic adverse events

Time Frame

6 months

Title

Feasibility of intravitreal injection of autologous CD34+ cells

Description

Number of CD34+ cells harvested for intravitreal injection

Time Frame

Baseline

Secondary Outcome Measure Information:

Title

Best corrected visual acuity

Description

Change in number of letters read in study eye from baseline

Time Frame

6 months

Title

Best corrected visual acuity

Description

Change in number of letters read in study eye from baseline

Time Frame

1 month

Title

Visual Field

Description

Change in visual field area in study eye from baseline

Time Frame

6 months

Title

Visual Field

Description

Change in visual field area in study eye from baseline

Time Frame

1 month

Title

Electroretinography

Description

Change in a and b wave amplitude in study eye from baseline

Time Frame

6 months

Title

Electroretinography

Description

Change in a and b wave amplitude in study eye from baseline

Time Frame

1 month

Title

National Eye Institute Vision Questionnaire

Description

Change in Vision Questionnaire compared to baseline

Time Frame

6 months

Title

National Eye Institute Vision Questionnaire

Description

Change in Vision Questionnaire compared to baseline

Time Frame

1 month

Title

Microperimetry

Description

Change in average threshold from baseline

Time Frame

6 months

Title

Microperimetry

Description

Change percent reduced sensitivity from baseline

Time Frame

6 months

Title

Microperimetry

Description

Change in average threshold from baseline

Time Frame

1 month

Title

Microperimetry

Description

Change in percent reduced sensitivity from baseline

Time Frame

1 month

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: 1. Age >18 years of age 2. Visual Acuity at Presentation of 20/100 to Counting Fingers or constriction of peripheral visual field 360 degrees to <10 degrees by perimetry if BCVA is better than 20/100, duration of vision loss > 3 months 3. Vision loss due to retinitis pigmentosa 4. The study eye has the worse visual acuity or peripheral constriction. 14. Able and willing to sign consent 15. Able to keep follow-up appointments for at least 6 months Exclusion Criteria: Other concurrent optic nerve or retinal disease in study eye affecting vision History of active retinal or choroidal neovascularization or macular edema in study eye requiring on-going intravitreal or periocular therapy in study eye Active eye or systemic infection Significant media opacity in the study eye precluding view of the fundus for examination, photography or optical coherence tomography (OCT) 9. Other cause for vision loss in the study eye 10. History of coagulopathy or other hematologic abnormality 11. Use of coumadin within 3 days of enrollment 12. Concurrent immunosuppressive therapy 13. History of allergy to fluorescein dye

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Susanna Park, MD PhD

Organizational Affiliation

University of California, Davis

Official's Role

Principal Investigator

Facility Information:

Facility Name

University of California Davis

City

Sacramento

State/Province

California

ZIP/Postal Code

95817

Country

United States

12. IPD Sharing Statement

Plan to Share IPD

Learn more about this trial

Phase 1 Study of Intravitreal Autologous CD34+ Stem Cell Therapy for Retinitis Pigmentosa

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