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

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ClinicalTrials.gov Identifier: NCT01560715
Recruitment Status : Completed
First Posted : March 22, 2012
Last Update Posted : July 21, 2017
Information provided by (Responsible Party):

Study Description
Brief 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.

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

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

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 50 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase 2 Study Of Autologous Bone Marrow-Derived Stem Cells Transplantation For Retinitis Pigmentosa
Study Start Date : June 2011
Primary Completion Date : January 2013
Study Completion Date : June 2013

Arms and Interventions

Arm Intervention/treatment
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

Outcome Measures

Primary Outcome Measures :
  1. ETDRS Visual acuity change [ Time Frame: Day 1 to Day 365 ]

Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 75 Years   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01560715

Centro de Pesquisa Rubens Siqueira
Sao Jose do Rio Preto, SP, Brazil, 15010-100
Sponsors and Collaborators
University of Sao Paulo
Principal Investigator: Rubens C Siqueira, MD,PhD Sao Paulo University
More Information

Responsible Party: Rubens Camargo Siqueira, MD,PhD, University of Sao Paulo
ClinicalTrials.gov Identifier: NCT01560715     History of Changes
Other Study ID Numbers: Rubens Siqueira
First Posted: March 22, 2012    Key Record Dates
Last Update Posted: July 21, 2017
Last Verified: July 2017

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

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