Intravitreal Bone Marrow-Derived Stem Cells in Patients With Macular Degeneration (AMDCELL)

The purpose of this study is to evaluate the behavior of intravitreal injection of of autologous bone marrow stem cells in patients with age related macular degeneration.

Study Overview

• Status: Completed
• Conditions: Age Related Macular Degeneration and Stargartd
• Intervention / Treatment: Other: intravitreal injection of autologous bone marrow stem cells

Detailed Description

A prospective phase I/II, nonrandomized open-label study of agen related macular degeneration and Stargartd 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 bone marrow stem cells: 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 Bone Marrow Stem Cells: 1) improvement in ERG response; 2) increase in visual field: 3) increase in CSMT > 50um and not related to macular edemar or choroidal neovascularization; 4) increase > 5 letters on BCVA

• Study Type: Interventional
• Enrollment (Actual): 20
• Phase: Not Applicable

Contacts and Locations

Study Locations

• Brazil
  • SP
    • Sao Jose do Rio Preto, SP, Brazil, 15010-100
      • Rubens Siqueira Research Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 80 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Male or female AMD and Stargartd patients 18 to 80 years old, inclusive.
• In either eye, diagnosis of geographic atrophy or choroidal neovascularization due to AMD
• ETDRS best corrected visual acuity of 60 letters or worse in the study eye.

Exclusion Criteria:

• Retinal disease other than AMD in study eye which, in the investigator's opinion, may pose a safety risk or interfere with the study.
• Choroidal neovascularization due to a cause other than AMD.
• In the study eye, media opacity that interferes with fundus imaging or is likely to require surgery during the study period.
• Any of the following treatments to the study eye within 14 days prior to dosing: ranibizumab (Lucentis), bevacizumab (Avastin), pegaptanib (Macugen), or other VEGF inhibitor; OR likely requirement for one of the above treatments within 14 days of LFG316 administration.
• Any of the following within 30 days prior to dosing: photodynamic therapy treatment in the study eye; extrafoveal or juxtafoveal thermal laser photocoagulation in the study eye; systemic or topical (ophthalmic) steroid use in the study eye; bacterial keratitis in the study eye; OR intraocular surgery (including cataract surgery) in the fellow eye.
• Any of the following within 90 days prior to dosing: intraocular surgery (including cataract surgery) in the study eye; OR intravitreal or periocular corticosteroid injection in the study eye.
• Participation in another interventional clinical study, or use of any experimental treatment for AMD or any other investigational new drug within 12 weeks prior to the start of study treatment. Clinical trials solely involving over-the-counter vitamins, supplements, or diets will not exclude the patients from study participation.
• Any medical condition likely to interfere with the patient's participation in the study, or likely to cause serious adverse events during the study.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: stem cell group; intravitreal injection of autologous bone marrow stem cells	Other: intravitreal injection of autologous bone marrow stem cells; intravitreal injection of autologous bone marrow stem cells; Other Names: Cell Therapy

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
ETDRS Visual acuity change	Primary safety outcome included visual acuity loss of 15 or more ETDRS letters after treatment	Day 1 to Day 365

Collaborators and Investigators

• Sponsor: University of Sao Paulo
• Investigators: Principal Investigator: Rubens C Siqueira, MD,PhD, University of Sao Paulo

Study record dates

Study Major Dates

• Study Start: August 1, 2011
• Primary Completion (Actual): September 1, 2013
• Study Completion (Actual): December 1, 2015

Study Registration Dates

• First Submitted: September 20, 2011
• First Submitted That Met QC Criteria: January 22, 2012
• First Posted (Estimate): January 25, 2012

Study Record Updates

• Last Update Posted (Actual): July 21, 2017
• Last Update Submitted That Met QC Criteria: July 18, 2017
• Last Verified: July 1, 2017

More Information

Terms related to this study

• Keywords: Macular Degeneration
• Additional Relevant MeSH Terms: Eye Diseases; Retinal Degeneration; Retinal Diseases; Macular Degeneration
• Other Study ID Numbers: Sao Paulo University