Autologous Bone Marrow-Derived Stem Cells Transplantation For Retinitis Pigmentosa

Retinitis Pigmentosa Clinical Trial

Official title:

Phase 1 Study Of Autologous Bone Marrow-Derived Stem Cells Transplantation For Retinitis Pigmentosa

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01068561
• Other study ID #: CPRS
• Status: Completed
• Phase: Phase 1
• First received: February 10, 2010
• Last updated: September 19, 2011
• Start date: May 2009
• Est. completion date: February 2010

Study information

• Verified date: September 2011
• Source: University of Sao Paulo
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 5
• Est. completion date: February 2010
• Est. primary completion date: January 2010
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Retinitis
• Retinitis Pigmentosa

Intervention

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.

Locations

Country	Name	City	State
Brazil	CPRS	São Jose do Rio Preto	SP

Sponsors (1)

Lead Sponsor	Collaborator
University of Sao Paulo

Country where clinical trial is conducted

Brazil, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	severe visual loss, defined as a drop in 15 letters on ETDRS visual acuity scale		3-6 months