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Autologous Bone Marrow-Derived Stem Cells Transplantation For Retinitis Pigmentosa (RETICELL)

This study is currently recruiting participants.
Verified March 2012 by University of Sao Paulo
Sponsor:
Information provided by (Responsible Party):
Rubens Camargo Siqueira, University of Sao Paulo
ClinicalTrials.gov Identifier:
NCT01560715
First received: March 19, 2012
Last updated: March 20, 2012
Last verified: March 2012
History of Changes
  Purpose

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.


Condition Intervention Phase
Retinitis Pigmentosa
 Biological: intravitreal injection of autologous bone marrow stem cells
 Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Phase 2 Study Of Autologous Bone Marrow-Derived Stem Cells Transplantation For Retinitis Pigmentosa

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by University of Sao Paulo:

Primary Outcome Measures:
  • ETDRS Visual acuity change [ Time Frame: Day 1 to Day 365 ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 50
Study Start Date: June 2011
Estimated Study Completion Date: June 2013
Estimated Primary Completion Date: June 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Experimental: Test group
Retinitis pigmentosa patients with best-corrected visual acuity (BCVA) worse than 20/200 or visual field less than 20 degrees
 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.
Other Name: autologous bone marrow stem cells

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

  Eligibility

Ages Eligible for Study:   18 Years to 75 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 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

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.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01560715

Contacts
Contact: Rubens C Siqueira, MD,PhD 55(17)32140896 rubenssiqueira@terra.com.br

Locations
Brazil
Centro de Pesquisa Rubens Siqueira Recruiting
Sao Jose do Rio Preto, SP, Brazil, 15010-100
Contact: Rubens Siqueira, MD,PhD     55(17)32140896        
Principal Investigator: Rubens C Siqueira, MD,PhD            
Sub-Investigator: Andre Messias, MD,PhD            
Sub-Investigator: Rodrigo Jorge, MD,PhD            
Sub-Investigator: Julio C Voltarelli, MD,PhD            
Sponsors and Collaborators
University of Sao Paulo
Investigators
Principal Investigator: Rubens C Siqueira, MD,PhD Sao Paulo University
  More Information

No publications provided

Responsible Party: Rubens Camargo Siqueira, MD,PhD, University of Sao Paulo
ClinicalTrials.gov Identifier: NCT01560715     History of Changes
Other Study ID Numbers: Rubens Siqueira
Study First Received: March 19, 2012
Last Updated: March 20, 2012
Health Authority: Brazil: National Committee of Ethics in Research

Keywords provided by University of Sao Paulo:
Retinitis pigmentosa
stem cell
bone marrow
retinal dysprophy

Additional relevant MeSH terms:
Retinitis
Retinitis Pigmentosa
Retinal Diseases
Eye Diseases
 Eye Diseases, Hereditary
Retinal Dystrophies
Retinal Degeneration
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on May 16, 2013