Autologous Bone Marrow-Derived Stem Cells Transplantation For Retinitis Pigmentosa

Phase 1

Completed

• Conditions: Retinitis Pigmentosa
• Interventions: Biological: intravitreal injection of autologous bone marrow stem cells

• Registration Number: NCT01068561
• Lead Sponsor: University of Sao Paulo

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

Study Timeline

• Study Start: 2009-05
• Primary Completion: 2010-01
• Study Completion: 2010-02
• Study First Submitted: 2010-02-10
• Study First Submitted QC: 2010-02-12
• Study First Posted: 2010-02-15
• Status Verified: 2011-09
• Last Update Submitted: 2011-09-19
• Last Update Posted: 2011-09-20

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 5

Inclusion Criteria

• diagnosis of retinitis pigmentosa
• logarithm of minimum angle of resolution (logMAR) BCVA of 1.0 (Snellen equivalent, 20/200) or worse

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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.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Test group	intravitreal injection of autologous bone marrow stem cells	open-label study of retinitis pigmentosa patients with best-corrected visual acuity (BCVA) worse than 20/200.

Primary Outcome Measures

Name	Time	Method
severe visual loss, defined as a drop in 15 letters on ETDRS visual acuity scale	3-6 months

Trial Locations

Locations (1)

CPRS; 🇧🇷 São Jose do Rio Preto, SP, Brazil