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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
Sponsored by
University of California, Davis
About
Eligibility
Locations
Arms
Outcomes
Full info

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:

  1. Other concurrent optic nerve or retinal disease in study eye affecting vision
  2. History of active retinal or choroidal neovascularization or macular edema in study eye requiring on-going intravitreal or periocular therapy in study eye
  3. Active eye or systemic infection
  4. 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

First Posted
June 2, 2021
Last Updated
April 19, 2023
Sponsor
University of California, Davis
Collaborators
Cures Within Reach, Retina Society
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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
No
Data Monitoring Committee
No

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
No
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
No

Learn more about this trial

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

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