Clinical trial NCT01560715: Autologous Bone Marrow-Derived Stem Cells Transplantation For Retinitis Pigmentosa
Official title: Phase 2 Study Of Autologous Bone Marrow-Derived Stem Cells Transplantation For Retinitis Pigmentosa
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Retinitis Pigmentosa
Keywords: Retinitis pigmentosa
stem cell
bone marrow
retinal dysprophy
Lead sponsor: University of Sao Paulo
Sponsor class: OTHER
First posted: 2012-03-22
Enrollment: 50
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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

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.

Primary outcomes:
measure: ETDRS Visual acuity change
timeFrame: Day 1 to Day 365
Secondary outcomes:
Other outcomes:
Locations:
city: Sao Jose do Rio Preto
country: Brazil
facility: Centro de Pesquisa Rubens Siqueira
state: SP
Study officials:
affiliation: Sao Paulo University
name: Rubens C Siqueira, MD,PhD
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.
citation: 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.
citation: 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.
citation: 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.