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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Rubens Camargo Siqueira, University of Sao Paulo
ClinicalTrials.gov Identifier:
NCT01560715
First received: March 19, 2012
Last updated: January 12, 2014
Last verified: January 2014

March 19, 2012
January 12, 2014
June 2011
January 2013   (final data collection date for primary outcome measure)
ETDRS Visual acuity change [ Time Frame: Day 1 to Day 365 ] [ Designated as safety issue: Yes ]
Same as current
Complete list of historical versions of study NCT01560715 on ClinicalTrials.gov Archive Site
Not Provided
Not Provided
Not Provided
Not Provided
 
Autologous Bone Marrow-Derived Stem Cells Transplantation For Retinitis Pigmentosa
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.

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

Interventional
Phase 2
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Retinitis Pigmentosa
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
Experimental: Experimental: Test group
Retinitis pigmentosa patients with best-corrected visual acuity (BCVA) worse than 20/200 or visual field less than 20 degrees
Intervention: Biological: intravitreal injection of autologous bone marrow stem cells
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.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
50
June 2013
January 2013   (final data collection date for primary outcome measure)

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.
Both
18 Years to 75 Years
No
Contact information is only displayed when the study is recruiting subjects
Brazil
 
NCT01560715
Rubens Siqueira
Yes
Rubens Camargo Siqueira, University of Sao Paulo
University of Sao Paulo
Not Provided
Principal Investigator: Rubens C Siqueira, MD,PhD Sao Paulo University
University of Sao Paulo
January 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP