Autologous Bone Marrow-Derived Stem Cells Transplantation For Retinitis Pigmentosa

NCT ID: NCT01560715

Last Updated: 2017-07-21

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 50 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2011-06-30
• Study Completion Date: 2013-06-30

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

Conditions

Retinitis Pigmentosa

Study Design

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

Study Groups

Experimental: Test group

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

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 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
• 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.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 75 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

Sao Paulo University

Locations

Centro de Pesquisa Rubens Siqueira

São José do Rio Preto, São Paulo, Brazil

Countries

Brazil

References

Siqueira RC, Messias A, Voltarelli JC, Scott IU, Jorge R. Intravitreal injection of autologous bone marrow-derived mononuclear cells for hereditary retinal dystrophy: a phase I trial. Retina. 2011 Jun;31(6):1207-14. doi: 10.1097/IAE.0b013e3181f9c242.

Reference Type: BACKGROUND

PMID: 21293313 (View on PubMed)

Siqueira RC, Messias A, Messias K, Arcieri RS, Ruiz MA, Souza NF, Martins LC, Jorge R. Quality of life in patients with retinitis pigmentosa submitted to intravitreal use of bone marrow-derived stem cells (Reticell -clinical trial). Stem Cell Res Ther. 2015 Mar 14;6(1):29. doi: 10.1186/s13287-015-0020-6.

Reference Type: BACKGROUND

PMID: 25890251 (View on PubMed)

Siqueira RC. Stem cell therapy in retinal diseases? Rev Bras Hematol Hemoter. 2012;34(3):222-6. doi: 10.5581/1516-8484.20120054. No abstract available.

Reference Type: BACKGROUND

PMID: 23049424 (View on PubMed)

Siqueira RC, Messias A, Voltarelli JC, Messias K, Arcieri RS, Jorge R. Resolution of macular oedema associated with retinitis pigmentosa after intravitreal use of autologous BM-derived hematopoietic stem cell transplantation. Bone Marrow Transplant. 2013 Apr;48(4):612-3. doi: 10.1038/bmt.2012.185. Epub 2012 Sep 24. No abstract available.

Reference Type: BACKGROUND

PMID: 23000646 (View on PubMed)

Other Identifiers

Rubens Siqueira

Identifier Type: -

Identifier Source: org_study_id