Autologous Bone Marrow-Derived Stem Cells Transplantation For Retinitis Pigmentosa

NCT ID: NCT01068561

Last Updated: 2011-09-20

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 5 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2009-05-31
• Study Completion Date: 2010-02-28

Brief Summary

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.

Detailed 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

Conditions

Retinitis Pigmentosa

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Test group

open-label study of retinitis pigmentosa patients with best-corrected visual acuity (BCVA) worse than 20/200.

Group Type: EXPERIMENTAL

intravitreal injection of autologous bone marrow stem cells

Intervention Type: BIOLOGICAL

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.

Interventions

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.

Intervention Type: BIOLOGICAL

Other Intervention Names

Autologous bone marrow mononuclear stem cells

Eligibility Criteria

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.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

University of Sao Paulo

OTHER

Sponsor Role: lead

Responsible Party

Rubens Camargo Siqueira

MD,PhD

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Rubens C Siqueira, MD,PhD

Role: PRINCIPAL_INVESTIGATOR

Research Center Rubens Siqueira,Catanduva Medicine School and São Paulo University

Locations

CPRS

São Jose Do Rio Preto, São Paulo, Brazil

Countries

Brazil

Other Identifiers

CPRS

Identifier Type: -

Identifier Source: org_study_id