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

This study is currently recruiting participants.

Verified March 2012 by University of Sao Paulo

Sponsor:

Information provided by (Responsible Party):

Rubens Camargo Siqueira, University of Sao Paulo

ClinicalTrials.gov Identifier:

NCT01560715

First received: March 19, 2012

Last updated: March 20, 2012

Last verified: March 2012

History of Changes

Tracking Information

First Received Date ^ICMJE

March 19, 2012

Last Updated Date

March 20, 2012

Start Date ^ICMJE

June 2011

Estimated Primary Completion Date

June 2012 (final data collection date for primary outcome measure)

Current Primary Outcome Measures ^ICMJE

ETDRS Visual acuity change [ Time Frame: Day 1 to Day 365 ] [ Designated as safety issue: Yes ]

Original Primary Outcome Measures ^ICMJE

Same as current

Change History

Complete list of historical versions of study NCT01560715 on ClinicalTrials.gov Archive Site

Current Secondary Outcome Measures ^ICMJE

Not Provided

Original Secondary Outcome Measures ^ICMJE

Not Provided

Current Other Outcome Measures ^ICMJE

Not Provided

Original Other Outcome Measures ^ICMJE

Not Provided

Descriptive Information

Brief Title ^ICMJE

Autologous Bone Marrow-Derived Stem Cells Transplantation For Retinitis Pigmentosa

Official Title ^ICMJE

Phase 2 Study Of Autologous Bone Marrow-Derived Stem Cells Transplantation For Retinitis Pigmentosa

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.

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 Type ^ICMJE

Interventional

Study Phase

Phase 2

Study Design ^ICMJE

Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment

Condition ^ICMJE

Retinitis Pigmentosa

Intervention ^ICMJE

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

Study Arm (s)

Experimental: Experimental: Test group

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

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

Publications *

Not Provided

* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.

Recruitment Information

Recruitment Status ^ICMJE

Recruiting

Estimated Enrollment ^ICMJE

Estimated Completion Date

June 2013

Estimated Primary Completion Date

June 2012 (final data collection date for primary outcome measure)

Eligibility Criteria ^ICMJE

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.

Gender

Both

Ages

18 Years to 75 Years

Accepts Healthy Volunteers

Contacts ^ICMJE

Contact: Rubens C Siqueira, MD,PhD

55(17)32140896

rubenssiqueira@terra.com.br

Location Countries ^ICMJE

Brazil

Administrative Information

NCT Number ^ICMJE

NCT01560715

Other Study ID Numbers ^ICMJE

Rubens Siqueira

Has Data Monitoring Committee

Yes

Responsible Party

Rubens Camargo Siqueira, University of Sao Paulo

Study Sponsor ^ICMJE

University of Sao Paulo

Collaborators ^ICMJE

Not Provided

Investigators ^ICMJE

Principal Investigator:

Rubens C Siqueira, MD,PhD

Sao Paulo University

Information Provided By

University of Sao Paulo

Verification Date

March 2012

^ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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