NCT01068561

Brief 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.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
5

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started May 2009

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

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Study Timeline

Key milestones and dates

Study Start

First participant enrolled

May 1, 2009

Completed
8 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 1, 2010

Completed
1 month until next milestone

Study Completion

Last participant's last visit for all outcomes

February 1, 2010

Completed
9 days until next milestone

First Submitted

Initial submission to the registry

February 10, 2010

Completed
5 days until next milestone

First Posted

Study publicly available on registry

February 15, 2010

Completed
Last Updated

September 20, 2011

Status Verified

September 1, 2011

Enrollment Period

8 months

First QC Date

February 10, 2010

Last Update Submit

September 19, 2011

Conditions

Keywords

Retinitis pigmentosastem cellbone marrow

Outcome Measures

Primary Outcomes (1)

  • severe visual loss, defined as a drop in 15 letters on ETDRS visual acuity scale

    3-6 months

Study Arms (1)

Test group

EXPERIMENTAL

open-label study of retinitis pigmentosa patients with best-corrected visual acuity (BCVA) worse than 20/200.

Biological: intravitreal injection of autologous bone marrow stem cells

Interventions

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 mononuclear stem cells
Test group

Eligibility Criteria

Age18 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

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)

CPRS

São Jose Do Rio Preto, São Paulo, 15010-100, Brazil

Location

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

    Research Center Rubens Siqueira,Catanduva Medicine School and São Paulo University

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
MD,PhD

Study Record Dates

First Submitted

February 10, 2010

First Posted

February 15, 2010

Study Start

May 1, 2009

Primary Completion

January 1, 2010

Study Completion

February 1, 2010

Last Updated

September 20, 2011

Record last verified: 2011-09

Locations