Retinitis PigmentosaPhase 1/2NCT02709876
Autologous Bone Marrow-Derived CD34+, CD133+, and CD271+ Stem Cell Transplantation for Retinitis Pigmentosa
A Phase 1/2 study testing Stem Cell Transplantation in people with Retinitis Pigmentosa, run by Stem Cells Arabia at 1 site in Jordan.
Trial summary
A single arm, single center trial to evaluate the safety and efficacy of autologous purified populations of bone-marrow derived stem cells in patients with Retinitis Pigmentosa (BM-SCs) through a 48 month follow up period. Read more about this study
- Who is it for?
- Ages 18 to 55 years
- Conditions the study is for
- Retinitis Pigmentosa
- What is being tested?
- Stem Cell Transplantation
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- 1 siteJordan
- Study dates
- Apr 2014to Mar 2021
- Phase
- Phase 1/2
- 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 to 55 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Retinitis pigmentosa patients diagnosed by ophthalmologists
- Visual acuity (measured with ETDRS) less than or equal to 20/70 and visual field below 30° central in both eyes.
- Best corrected visual acuity less than 6/120 by Snellen visual acuity chart
Who cannot join
- Other eye conditions that could mask the interpretation of the results
- Unable to return for follow up
- Underlying diseases including asthma, heart failure, myocardial infarction, liver failure, renal failure
- Pregnant and lactating women
Trial locations and contacts
1 site in 1 country.
About this study
A single arm, single center trial to evaluate the safety and efficacy of autologous purified populations of bone-marrow derived stem cells in patients with Retinitis Pigmentosa (BM-SCs) through a 48 month follow up period.
Read the detailed description
Retinitis pigmentosa (RP) is an inherited disorder of the photoreceptor cells in the retina. Patients may lose vision since they were young or later in life. Currently, there are more than 60 genes identified as the cause of this condition, one of which, RPE65, has been studied in several gene therapy trials for Leber congenital amaurosis with promising results. Another treatment approach for RP is stem cell therapy. Studies in animal models of RP have shown that subretinal injection of bone marrow-derived mesenchymal stem cells may delay degenerative changes of photoreceptor cells. This is a single arm, single center trial to assess the safety and efficacy of purified adult autologous bone marrow derived CD34+, CD133+, and CD271+ stem cells through a 48 month follow-up period. The combination of these three cell types was based on their diverse potentialities to differentiate into specific functional cell types to regenerate damaged retinal tissue, and the availability of clinical-grade purification system (CliniMACS) and microbeads to purify the target cell populations in clinically-approved methods.
- Conditions
- Retinitis Pigmentosa
- Being tested
- Stem Cell Transplantation
- Medical subject headings
- Retinitis Pigmentosa
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Stem Cell TransplantationBiological
Bone marrow-derived CD34+, CD133+, CD271+ stem cells in 1.0 ml normal saline will be injected into the vitreous cavity.
| Study group | Role | What they receive |
|---|---|---|
| Stem Cells | Experimental | Intravitreal Injection of bone marrow derived CD34+, CD133+, CD271+ stem cells. Receives: Biological: Stem Cell Transplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
ETDRS Visual acuity change
Measured: 12 months from baseline
Secondary outcomes (3)
Quality of Life: Questionnaire VFQ-25 (Visual Function Questionnaire-25)
Measured: 12 months from baseline
Color Vision: Ishihara Color Test
Measured: 12 months from baseline
Contrast sensitivity: CSV-1000E
Measured: 12 months from baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Mar 2016 (estimated)
- Study startApr 2014
- Primary completionSep 2020 (estimated)
- Study completionMar 2021 (estimated)
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.
- 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Stem Cells Arabia
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02709876
- Sponsor's protocol ID
- SCA-RP1
- Status last verified
- Mar 2020
- First submitted
- 26 Feb 2016
- Last updated
- 17 Mar 2020
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 17 Mar 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Autologous Bone Marrow-Derived CD34+, CD133+, and CD271+ Stem Cell Transplantation for Retinitis Pigmentosa
- ClinicalTrials.gov ID
- NCT02709876
- Acronym / sponsor ID
- SCA-RP1
- Conditions
- Retinitis Pigmentosa
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Stem Cells ArabiaUniversity, hospital or other
- Study start
- Apr 2014
- Primary completion
- Sep 2020 (estimated)
- Study completion
- Mar 2021 (estimated)
- First posted
- 16 Mar 2016 (estimated)
- 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.
A single arm, single center trial to evaluate the safety and efficacy of autologous purified populations of bone-marrow derived stem cells in patients with Retinitis Pigmentosa (BM-SCs) through a 48 month follow up period.
Detailed description
Retinitis pigmentosa (RP) is an inherited disorder of the photoreceptor cells in the retina. Patients may lose vision since they were young or later in life. Currently, there are more than 60 genes identified as the cause of this condition, one of which, RPE65, has been studied in several gene therapy trials for Leber congenital amaurosis with promising results. Another treatment approach for RP is stem cell therapy. Studies in animal models of RP have shown that subretinal injection of bone marrow-derived mesenchymal stem cells may delay degenerative changes of photoreceptor cells. This is a single arm, single center trial to assess the safety and efficacy of purified adult autologous bone marrow derived CD34+, CD133+, and CD271+ stem cells through a 48 month follow-up period. The combination of these three cell types was based on their diverse potentialities to differentiate into specific functional cell types to regenerate damaged retinal tissue, and the availability of clinical-grade purification system (CliniMACS) and microbeads to purify the target cell populations in clinically-approved methods.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Stem Cells | Experimental | Biological: Stem Cell TransplantationIntravitreal Injection of bone marrow derived CD34+, CD133+, CD271+ stem cells. |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (3 listed)
- Retinitis pigmentosa patients diagnosed by ophthalmologists
- Visual acuity (measured with ETDRS) less than or equal to 20/70 and visual field below 30° central in both eyes.
- Best corrected visual acuity less than 6/120 by Snellen visual acuity chart
Key exclusion criteria (4 listed)
- Other eye conditions that could mask the interpretation of the results
- Unable to return for follow up
- Underlying diseases including asthma, heart failure, myocardial infarction, liver failure, renal failure
- Pregnant and lactating women
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 |
|---|---|---|
| Stem Cell TransplantationBone marrow-derived CD34+, CD133+, CD271+ stem cells in 1.0 ml normal saline will be injected into the vitreous cavity. | Biological | Stem Cells |
Primary outcome
ETDRS Visual acuity change
Time frame: 12 months from baseline
3 secondary outcomes
Quality of Life: Questionnaire VFQ-25 (Visual Function Questionnaire-25)
Time frame: 12 months from baseline
Color Vision: Ishihara Color Test
Time frame: 12 months from baseline
Contrast sensitivity: CSV-1000E
Time frame: 12 months from baseline
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Mar 2020.
- Individual participant dataSharing planned: Yes
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Stem Cell Transplantation in people with Retinitis Pigmentosa, run by Stem Cells Arabia at 1 site in Jordan.
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 3 inclusion and 4 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: status not confirmed, as of the sponsor's last update on 17 Mar 2020. 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 Jordan. 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 combined 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.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
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?
ETDRS Visual acuity change (time frame: 12 months from baseline). 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 17 Mar 2020.