Autologous Bone Marrow-Derived Stem Cells Transplantation For Retinitis PigmentosaThe purpose of this study is to evaluate the short-term safety of a single intravitreal injection of autologous bone marrow stem cells in patients with retinitis pigmentosa...
Date First Received: February 10, 2010
Last Updated: February 17, 2010
Verified by: University of Sao Paulo, January 2010
Clinical Trial Phase: Phase 1 | Start Date: May 2009
Overall Status: Completed
Estimated Enrollment: 5
Brief Summary
Official Title: “Phase 1 Study Of Autologous Bone Marrow-Derived Stem Cells Transplantation For Retinitis Pigmentosa”
Condition Keyword(s):
Retinitis Pigmentosa
Intervention(s):
Biological: intravitreal injection of autologous bone marrow stem cells
The purpose of this study is to evaluate the short-term safety of a single intravitreal injection of autologous bone marrow stem cells in patients with retinitis pigmentosa.
Study Type: Interventional
Study Design: Treatment, Non-Randomized, Open Label, Placebo Control, Single Group Assignment, Safety Study
Study Primary Completion Date: January 2010
Detailed Clinical Trial Description
A prospective phase I, 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
Intervention(s) in this Clinical Trial
Biological: intravitreal injection of 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.
Arms, Groups and Cohorts in this Clinical Trial
Experimental: Test group
open-label study of retinitis pigmentosa patients with best-corrected visual acuity (BCVA) worse than 20/200.
Outcome Measures for this Clinical Trial
Primary Measures
severe visual loss, defined as a drop in 15 letters on ETDRS visual acuity scale
Time Frame: 3-6 months
Safety Issue?: Yes
Criteria for Participation in this Clinical Trial
Inclusion Criteria:
diagnosis of retinitis pigmentosa
logarithm of minimum angle of resolution (logMAR) BCVA of 1.0 (Snellen equivalent, 20/200) or worse
Exclusion Criteria:
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.
Gender Eligibility for this Clinical Trial: Both
Minimum Age for this Clinical Trial: 18 Years
Maximum Age for this Clinical Trial: N/A
Are Healthy Volunteers Accepted for this Clinical Trial?: No
Clinical Trial Sponsor Information
Lead Sponsor: University of Sao Paulo
Overall Clinical Trial Officials and Contacts
Rubens C Siqueira, MD,PhD Principal Investigator Research Center Rubens Siqueira,Catanduva Medicine School and São Paulo University
Additional Information
Information obtained from ClinicalTrials.gov on February 18, 2010
Link to the current ClinicalTrials.gov record. http://clinicaltrials.gov/show/NCT01068561
Study ID Number: CPRS
ClinicalTrials.gov Identifier: NCT01068561
Health Authority: Brazil: National Committee of Ethics in Research
Clinical Trials Authorship and Review
Clinical Trials content is provided directly by the U.S. National Institutes of Health via ClinicalTrials.gov and is not reviewed separately by ClinicalTrialsFeeds.org. Every page of specific clinical trials information contains a unique identifier which can be used to find further details directly |
