NCT01560715

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
50

participants targeted

Target at P25-P50 for phase_2

Timeline
Completed

Started Jun 2011

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

June 1, 2011

Completed
10 months until next milestone

First Submitted

Initial submission to the registry

March 19, 2012

Completed
3 days until next milestone

First Posted

Study publicly available on registry

March 22, 2012

Completed
10 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 1, 2013

Completed
5 months until next milestone

Study Completion

Last participant's last visit for all outcomes

June 1, 2013

Completed
Last Updated

July 21, 2017

Status Verified

July 1, 2017

Enrollment Period

1.6 years

First QC Date

March 19, 2012

Last Update Submit

July 18, 2017

Conditions

Retinitis Pigmentosa

Keywords

Retinitis pigmentosastem cellbone marrowretinal dysprophy

Outcome Measures

Primary Outcomes (1)

  • ETDRS Visual acuity change

    Day 1 to Day 365

Study Arms (1)

Experimental: Test group

EXPERIMENTAL

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

Biological: intravitreal injection of autologous bone marrow stem cells

Interventions

intravitreal injection of autologous bone marrow stem cellsBIOLOGICAL

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.

Also known as: autologous bone marrow stem cells
Experimental: Test group

Eligibility Criteria

Age18 Years - 75 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Centro de Pesquisa Rubens Siqueira

São José do Rio Preto, São Paulo, 15010-100, Brazil

Location

Related Publications (4)

  • 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.

    PMID: 21293313BACKGROUND

  • 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.

    PMID: 25890251BACKGROUND

  • 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.

    PMID: 23049424BACKGROUND

  • 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.

    PMID: 23000646BACKGROUND

MeSH Terms

Conditions

Retinitis Pigmentosa

Condition Hierarchy (Ancestors)

Eye Diseases, HereditaryEye DiseasesRetinal DystrophiesRetinal DegenerationRetinal DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Rubens C Siqueira, MD,PhD

    Sao Paulo University

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
MD,PhD

Study Record Dates

First Submitted

March 19, 2012

First Posted

March 22, 2012

Study Start

June 1, 2011

Primary Completion

January 1, 2013

Study Completion

June 1, 2013

Last Updated

July 21, 2017

Record last verified: 2017-07

Locations

BR(1)