Retinitis PigmentosaPhase 2RETICELLNCT01560715
Autologous Bone Marrow-Derived Stem Cells Transplantation For Retinitis Pigmentosa
A Phase 2 study testing intravitreal injection of autologous bone marrow stem cells in people with Retinitis Pigmentosa, run by University of Sao Paulo at 1 site in Brazil.
Trial summary
The purpose of this study is to evaluate the short-term safety and efficacy of a single intravitreal injection of autologous bone marrow stem cells in patients with retinitis pigmentosa. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Retinitis Pigmentosa
- What is being tested?
- intravitreal injection of autologous bone marrow stem cells
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- 1 siteBrazil
- Study dates
- Jun 2011to Jun 2013
- Phase
- Phase 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 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- diagnosis of retinitis pigmentosa logarithm of minimum angle of resolution (logMAR) BCVA of 1.0 (Snellen equivalent, 20/200) or worse
- visual Field less than 20 degreees
Who cannot join
- previous ocular surgery other than cataract
- presence of cataract or other media opacity that would influence ocular fundus documentation and adequate ERG and visual field evaluation
- other ophthalmic disease like glaucoma and uveitis
- previous history of blood disorders like leukemia
- known allergy to fluorescein or indocyanine green
- known coagulation abnormalities or current use of anticoagulative medication other than aspirin.
Trial locations and contacts
1 site in 1 country.
Brazil1 site
Centro de Pesquisa Rubens Siqueira
São José do Rio Preto, São Paulo, 15010-100
About this study
Official title: Phase 2 Study Of Autologous Bone Marrow-Derived Stem Cells Transplantation For Retinitis Pigmentosa
The purpose of this study is to evaluate the short-term safety and efficacy of a single intravitreal injection of autologous bone marrow stem cells in patients with retinitis pigmentosa.
Read the detailed description
A prospective phase II, nonrandomized open-label study of retinitis pigmentosa patients with best-corrected ETDRS visual acuity (BCVA) worse than 20/200. Standardized ophthalmic evaluation will be perform at baseline and at weeks 1, 4,12 and 24 (±1) following intravitreal injection of 10 x 106 bone marrow stem cells/ 0,1ml . Three measures will be used to evaluate the short-term safety of intravitreal of ABMDSC: 1) severe visual loss, defined as a drop in 15 letters on ETDRS visual acuity scale; 2) decrease in ERG response; 3) decrease in 5 square degrees on visual field; secondary safety outcomes : 1) increase in intra-ocular inflammation defined herein as anterior chamber cells and flare higher than 3+ for more than 1 month after injection according to a classification described elsewhere ; 2) decrease in CMT more than 50um; 3)genesis of abnormal tissues (teratomas) or tumors; 4) qualitative changes in retinal or choroidal perfusion, like macular nonperfusion. Secondary outcome measures will be used to evaluate the short-term efficiency of intravitreal of ABMDSC: 1) improvement in ERG response; 2) increase in visual field: 3) increase in CSMT > 50um and not related to macular edema; 4) increase > 5 letters on BCVA
- Conditions
- Retinitis Pigmentosa
- Being tested
- intravitreal injection of autologous bone marrow stem cells
- Keywords
- Retinitis pigmentosa, stem cell, bone marrow, retinal dysprophy
- Medical subject headings
- Retinitis Pigmentosa
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
intravitreal injection of autologous bone marrow stem cellsBiological
Also called autologous bone marrow stem cells
One intravitreal injection of a 0.1-ml cell suspension containing around 10x106 bone marrow mononuclear stem cells(BMMSC). All treatments were performed by a single retinal specialist using topical proparacaine drops under sterile conditions (eyelid speculum and povidone-iodine). Autologous BMMSC were injected into the vitreous cavity using a 27 gauge needle inserted through the inferotemporal pars plana 3.0 - 3.5 mm posterior to the limbus. After the injection, central retinal artery perfusion was confirmed with indirect ophthalmoscopy. Patients were instructed to instill one drop of 0.3% ciprofloxacin into the injected eye four times daily for 1 week after the procedure.
| Study group | Role | What they receive |
|---|---|---|
| Experimental: Test group | Experimental | Retinitis pigmentosa patients with best-corrected visual acuity (BCVA) worse than 20/200 or visual field less than 20 degrees Receives: Biological: intravitreal injection of autologous bone marrow stem cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
ETDRS Visual acuity change
Measured: Day 1 to Day 365
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Mar 2012 (estimated)
- Study startJun 2011
- Primary completionJan 2013
- Study completionJun 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Retinitis PigmentosaStudies of the same condition, open and under way.
- More trials run by University of Sao PauloThe 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 50 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Sao Paulo
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Rubens Camargo Siqueira, University of Sao Paulo
- Investigators
- Rubens C Siqueira, MD,PhD, Principal investigator, Sao Paulo University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01560715
- Sponsor's protocol ID
- Rubens Siqueira
- Acronym
- RETICELL
- Status last verified
- Jul 2017
- First submitted
- 19 Mar 2012
- Last updated
- 21 Jul 2017
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Siqueira RC, Messias A, Voltarelli JC, Scott IU, Jorge R. Intravitreal injection of autologous bone marrow-derived mononuclear cells for hereditary retinal dystrophy: a phase I trial. Retina. 2011 Jun;31(6):1207-14. doi: 10.1097/IAE.0b013e3181f9c242.
PubMed 21293313Siqueira RC, Messias A, Messias K, Arcieri RS, Ruiz MA, Souza NF, Martins LC, Jorge R. Quality of life in patients with retinitis pigmentosa submitted to intravitreal use of bone marrow-derived stem cells (Reticell -clinical trial). Stem Cell Res Ther. 2015 Mar 14;6(1):29. doi: 10.1186/s13287-015-0020-6.
PubMed 25890251Siqueira RC. Stem cell therapy in retinal diseases? Rev Bras Hematol Hemoter. 2012;34(3):222-6. doi: 10.5581/1516-8484.20120054. No abstract available.
PubMed 23049424Siqueira RC, Messias A, Voltarelli JC, Messias K, Arcieri RS, Jorge R. Resolution of macular oedema associated with retinitis pigmentosa after intravitreal use of autologous BM-derived hematopoietic stem cell transplantation. Bone Marrow Transplant. 2013 Apr;48(4):612-3. doi: 10.1038/bmt.2012.185. Epub 2012 Sep 24. No abstract available.
PubMed 23000646
Study at a glance
From the registry record, last updated by the sponsor on 21 Jul 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 2 Study Of Autologous Bone Marrow-Derived Stem Cells Transplantation For Retinitis Pigmentosa
- ClinicalTrials.gov ID
- NCT01560715
- Acronym / sponsor ID
- RETICELL / Rubens Siqueira
- Conditions
- Retinitis Pigmentosa
- Keywords
- Retinitis pigmentosa, stem cell, bone marrow, retinal dysprophy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 50 participants (actual)
- Lead sponsor
- University of Sao PauloUniversity, hospital or other
- Study start
- Jun 2011
- Primary completion
- Jan 2013 (actual)
- Study completion
- Jun 2013 (actual)
- First posted
- 22 Mar 2012 (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.
The purpose of this study is to evaluate the short-term safety and efficacy of a single intravitreal injection of autologous bone marrow stem cells in patients with retinitis pigmentosa.
Detailed description
A prospective phase II, nonrandomized open-label study of retinitis pigmentosa patients with best-corrected ETDRS visual acuity (BCVA) worse than 20/200. Standardized ophthalmic evaluation will be perform at baseline and at weeks 1, 4,12 and 24 (±1) following intravitreal injection of 10 x 106 bone marrow stem cells/ 0,1ml . Three measures will be used to evaluate the short-term safety of intravitreal of ABMDSC: 1) severe visual loss, defined as a drop in 15 letters on ETDRS visual acuity scale; 2) decrease in ERG response; 3) decrease in 5 square degrees on visual field; secondary safety outcomes : 1) increase in intra-ocular inflammation defined herein as anterior chamber cells and flare higher than 3+ for more than 1 month after injection according to a classification described elsewhere ; 2) decrease in CMT more than 50um; 3)genesis of abnormal tissues (teratomas) or tumors; 4) qualitative changes in retinal or choroidal perfusion, like macular nonperfusion. Secondary outcome measures will be used to evaluate the short-term efficiency of intravitreal of ABMDSC: 1) improvement in ERG response; 2) increase in visual field: 3) increase in CSMT > 50um and not related to macular edema; 4) increase > 5 letters on BCVA
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Experimental: Test group | Experimental | Biological: intravitreal injection of autologous bone marrow stem cellsRetinitis pigmentosa patients with best-corrected visual acuity (BCVA) worse than 20/200 or visual field less than 20 degrees |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- diagnosis of retinitis pigmentosa logarithm of minimum angle of resolution (logMAR) BCVA of 1.0 (Snellen equivalent, 20/200) or worse
- visual Field less than 20 degreees
Key exclusion criteria (6 listed)
- previous ocular surgery other than cataract
- presence of cataract or other media opacity that would influence ocular fundus documentation and adequate ERG and visual field evaluation
- other ophthalmic disease like glaucoma and uveitis
- previous history of blood disorders like leukemia
- known allergy to fluorescein or indocyanine green
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 injection of autologous bone marrow stem cellsAlso: autologous bone marrow stem cellsOne intravitreal injection of a 0.1-ml cell suspension containing around 10x106 bone marrow mononuclear stem cells(BMMSC). All treatments were performed by a single retinal specialist using topical proparacaine drops under sterile conditions (eyelid speculum and povidone-iodine). Autologous BMMSC were injected into the vitreous cavity using a 27 gauge needle inserted through the inferotemporal pars plana 3.0 - 3.5 mm posterior to the limbus. After the injection, central retinal artery perfusion was confirmed with indirect ophthalmoscopy. Patients were instructed to instill one drop of 0.3% ciprofloxacin into the injected eye four times daily for 1 week after the procedure. | Biological | Experimental: Test group |
Primary outcome
ETDRS Visual acuity change
Time frame: Day 1 to Day 365
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 21 Jul 2017.
- Background: PubMed 21293313Siqueira RC, Messias A, Voltarelli JC, Scott IU, Jorge R. Intravitreal injection of autologous bone marrow-derived mononuclear cells for hereditary retinal dystrophy: a phase I trial. Retina. 2011 Jun;31(6):1207-14. doi: 10.1097/IAE.0b013e3181f9c242.
- Background: PubMed 25890251Siqueira RC, Messias A, Messias K, Arcieri RS, Ruiz MA, Souza NF, Martins LC, Jorge R. Quality of life in patients with retinitis pigmentosa submitted to intravitreal use of bone marrow-derived stem cells (Reticell -clinical trial). Stem Cell Res Ther. 2015 Mar 14;6(1):29. doi: 10.1186/s13287-015-0020-6.
- Background: PubMed 23049424Siqueira RC. Stem cell therapy in retinal diseases? Rev Bras Hematol Hemoter. 2012;34(3):222-6. doi: 10.5581/1516-8484.20120054. No abstract available.
- Background: PubMed 23000646Siqueira RC, Messias A, Voltarelli JC, Messias K, Arcieri RS, Jorge R. Resolution of macular oedema associated with retinitis pigmentosa after intravitreal use of autologous BM-derived hematopoietic stem cell transplantation. Bone Marrow Transplant. 2013 Apr;48(4):612-3. doi: 10.1038/bmt.2012.185. Epub 2012 Sep 24. No abstract available.
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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.
Rubens C Siqueira, MD,PhD principal investigator
Sao Paulo University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing intravitreal injection of autologous bone marrow stem cells in people with Retinitis Pigmentosa, run by University of Sao Paulo at 1 site in Brazil.
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 2 inclusion and 6 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 21 Jul 2017. 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 Brazil. 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 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?
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?
ETDRS Visual acuity change (time frame: Day 1 to Day 365). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01560715, which also lists 4 linked publications.
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 21 Jul 2017.