Retinitis PigmentosaPhase 1BMSCRP1NCT04925687
Phase 1 Study of Intravitreal Autologous CD34+ Stem Cell Therapy for Retinitis Pigmentosa
A Phase 1 study testing Intravitreal autologous CD34+ cells in people with Retinitis Pigmentosa, run by University of California, Davis at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Retinitis Pigmentosa
- What is being tested?
- Intravitreal autologous CD34+ cells
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Jun 2021to 1 Nov 2023
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
University of California Davis
Sacramento, California, 95817
About this study
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
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.
- Conditions
- Retinitis Pigmentosa
- Being tested
- Intravitreal autologous CD34+ cells
- Keywords
- retinitis pigmentosa, stem cell, retinal degeneration, intravitreal
- Medical subject headings
- Retinitis Pigmentosa, Retinal Degeneration
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Intravitreal autologous CD34+ cellsBiological
Autologous CD34+ cells harvested from bone marrow injected intravitreal
| Study group | Role | What they receive |
|---|---|---|
| Intravitreal autologous CD34+ cells | Experimental | Intravitreal injection of autologous CD34+ cells harvested from bone marrow under GMP conditions Receives: Biological: Intravitreal autologous CD34+ cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety of intravitreal injection of autologous CD34+ cells
Number and severity of Ocular and systemic adverse events
Measured: 6 months
Feasibility of intravitreal injection of autologous CD34+ cells
Number of CD34+ cells harvested for intravitreal injection
Measured: Baseline
Secondary outcomes (12)
Best corrected visual acuity
Change in number of letters read in study eye from baseline
Measured: 6 months
Best corrected visual acuity
Change in number of letters read in study eye from baseline
Measured: 1 month
Visual Field
Change in visual field area in study eye from baseline
Measured: 6 months
Visual Field
Change in visual field area in study eye from baseline
Measured: 1 month
Electroretinography
Change in a and b wave amplitude in study eye from baseline
Measured: 6 months
Electroretinography
Change in a and b wave amplitude in study eye from baseline
Measured: 1 month
National Eye Institute Vision Questionnaire
Change in Vision Questionnaire compared to baseline
Measured: 6 months
National Eye Institute Vision Questionnaire
Change in Vision Questionnaire compared to baseline
Measured: 1 month
Microperimetry
Change in average threshold from baseline
Measured: 6 months
Microperimetry
Change percent reduced sensitivity from baseline
Measured: 6 months
Microperimetry
Change in average threshold from baseline
Measured: 1 month
Microperimetry
Change in percent reduced sensitivity from baseline
Measured: 1 month
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Jun 2021
- Study start1 Jun 2021
- Primary completion1 Nov 2023
- Study completion1 Nov 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Retinitis PigmentosaStudies of the same condition, open and under way.
- More trials run by University of California, DavisThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of California, Davis
- Sponsor type
- University, hospital or other
- Collaborators
- Cures Within Reach, The Retina Society
- Responsible party
- Sponsor
- Investigators
- Susanna Park, MD PhD, Principal investigator, University of California, Davis
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04925687
- Sponsor's protocol ID
- 1743714
- Acronym
- BMSCRP1
- Status last verified
- May 2024
- First submitted
- 2 Jun 2021
- Last updated
- 23 Sep 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Sep 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT04925687
- Acronym / sponsor ID
- BMSCRP1 / 1743714
- Conditions
- Retinitis Pigmentosa
- Keywords
- retinitis pigmentosa, stem cell, retinal degeneration, intravitreal
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 4 participants (actual)
- Lead sponsor
- University of California, DavisUniversity, hospital or other
- Collaborators
- Cures Within Reach, The Retina Society
- Study start
- 1 Jun 2021 (actual)
- Primary completion
- 1 Nov 2023 (actual)
- Study completion
- 1 Nov 2023 (actual)
- First posted
- 14 Jun 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Intravitreal autologous CD34+ cells | Experimental | Biological: Intravitreal autologous CD34+ cellsIntravitreal injection of autologous CD34+ cells harvested from bone marrow under GMP conditions |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (1 listed)
- 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
Key exclusion criteria (5 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Intravitreal autologous CD34+ cellsAutologous CD34+ cells harvested from bone marrow injected intravitreal | Biological | Intravitreal autologous CD34+ cells |
Primary outcome
Safety of intravitreal injection of autologous CD34+ cells
Time frame: 6 months
Number and severity of Ocular and systemic adverse events
Feasibility of intravitreal injection of autologous CD34+ cells
Time frame: Baseline
Number of CD34+ cells harvested for intravitreal injection
12 secondary outcomes
Best corrected visual acuity
Time frame: 6 months
Best corrected visual acuity
Time frame: 1 month
Visual Field
Time frame: 6 months
Visual Field
Time frame: 1 month
Electroretinography
Time frame: 6 months
Electroretinography
Time frame: 1 month
National Eye Institute Vision Questionnaire
Time frame: 6 months
National Eye Institute Vision Questionnaire
Time frame: 1 month
Microperimetry
Time frame: 6 months
Microperimetry
Time frame: 6 months
Microperimetry
Time frame: 1 month
Microperimetry
Time frame: 1 month
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Sep 2024.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Susanna Park, MD PhD principal investigator
University of California, Davis
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Intravitreal autologous CD34+ cells in people with Retinitis Pigmentosa, run by University of California, Davis at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 23 Sep 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety of intravitreal injection of autologous CD34+ cells (time frame: 6 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Sep 2024.