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

University of Sao Paulo

Status and phase: Completed; Phase 2

Conditions: Retinitis Pigmentosa

Treatments: Biological: intravitreal injection of autologous bone marrow stem cells

Study type: Interventional

Funder types: Other

Identifiers

NCT01560715

Rubens Siqueira

Details and patient eligibility

About

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.

Full 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

Enrollment: 50 patients

Sex: All

Ages: 18 to 75 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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50 participants in 1 patient group

Experimental: Test group

Experimental group

Description:

Retinitis pigmentosa patients with best-corrected visual acuity (BCVA) worse than 20/200 or visual field less than 20 degrees

Treatment:

Biological: intravitreal injection of autologous bone marrow stem cells