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

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ClinicalTrials.gov Identifier: NCT04925687
Recruitment Status : Recruiting
First Posted : June 14, 2021
Last Update Posted : August 23, 2021
Sponsor:
Collaborator:
Cures Within Reach
Information provided by (Responsible Party):
Susanna Park, MD, PhD, University of California, Davis

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.

Condition or disease Intervention/treatment Phase
Retinitis Pigmentosa Biological: Intravitreal autologous CD34+ cells Phase 1

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 4 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Pilot Study of the Feasibility and Safety of Intravitreal Autologous Bone Marrow CD34+ Stem Cell Therapy for Eyes With Vision Loss From Retinitis Pigmentosa
Actual Study Start Date : June 1, 2021
Estimated Primary Completion Date : June 30, 2022
Estimated Study Completion Date : June 30, 2022

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Intravitreal autologous CD34+ cells
Intravitreal injection of autologous CD34+ cells harvested from bone marrow under GMP conditions
Biological: Intravitreal autologous CD34+ cells
Autologous CD34+ cells harvested from bone marrow injected intravitreal




Primary Outcome Measures :
  1. Safety of intravitreal injection of autologous CD34+ cells [ Time Frame: 6 months ]
    Number and severity of Ocular and systemic adverse events

  2. Feasibility of intravitreal injection of autologous CD34+ cells [ Time Frame: Baseline ]
    Number of CD34+ cells harvested for intravitreal injection


Secondary Outcome Measures :
  1. Best corrected visual acuity [ Time Frame: 6 months ]
    Change in number of letters read in study eye from baseline

  2. Best corrected visual acuity [ Time Frame: 1 month ]
    Change in number of letters read in study eye from baseline

  3. Visual Field [ Time Frame: 6 months ]
    Change in visual field area in study eye from baseline

  4. Visual Field [ Time Frame: 1 month ]
    Change in visual field area in study eye from baseline

  5. Electroretinography [ Time Frame: 6 months ]
    Change in a and b wave amplitude in study eye from baseline

  6. Electroretinography [ Time Frame: 1 month ]
    Change in a and b wave amplitude in study eye from baseline

  7. National Eye Institute Vision Questionnaire [ Time Frame: 6 months ]
    Change in Vision Questionnaire compared to baseline

  8. National Eye Institute Vision Questionnaire [ Time Frame: 1 month ]
    Change in Vision Questionnaire compared to baseline

  9. Microperimetry [ Time Frame: 6 months ]
    Change in average threshold from baseline

  10. Microperimetry [ Time Frame: 6 months ]
    Change percent reduced sensitivity from baseline

  11. Microperimetry [ Time Frame: 1 month ]
    Change in average threshold from baseline

  12. Microperimetry [ Time Frame: 1 month ]
    Change in percent reduced sensitivity from baseline



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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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:

  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


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04925687


Contacts
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Contact: Cynthia Wallace 916-7346303 clwallace@ucdavis.edu
Contact: Denise Macias 916-7346303 dcmacias@ucdavis.edu

Locations
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United States, California
University of California Davis Recruiting
Sacramento, California, United States, 95817
Sub-Investigator: Jan Nolta, PhD         
Sub-Investigator: Gerhard Bauer         
Sub-Investigator: Mehrdad Abedi, MD         
Sub-Investigator: Ala Moshiri, MD PhD         
Sponsors and Collaborators
University of California, Davis
Cures Within Reach
Investigators
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Principal Investigator: Susanna Park, MD PhD University of California, Davis
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Responsible Party: Susanna Park, MD, PhD, Professor, University of California, Davis
ClinicalTrials.gov Identifier: NCT04925687    
Other Study ID Numbers: 1743714-2
First Posted: June 14, 2021    Key Record Dates
Last Update Posted: August 23, 2021
Last Verified: August 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Susanna Park, MD, PhD, University of California, Davis:
retinitis pigmentosa
stem cell
retinal degeneration
intravitreal
Additional relevant MeSH terms:
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Retinitis
Retinitis Pigmentosa
Retinal Diseases
Eye Diseases
Eye Diseases, Hereditary
Retinal Dystrophies
Retinal Degeneration
Genetic Diseases, Inborn