NCT02903576

Brief Summary

This study is a Phase I/II , open label,non randomized, prospective study to determine the safety of human embryonic stem cell derived Retinal pigmented epithelium (hESC RPE) sub retinal injections versus hESC RPE seeded on a polymeric substrate implanted in the sub retinal space,

Trial Health

87
On Track

Trial Health Score

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

Enrollment
15

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Aug 2015

Longer than P75 for phase_1

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

August 1, 2015

Completed
7 months until next milestone

First Submitted

Initial submission to the registry

March 7, 2016

Completed
6 months until next milestone

First Posted

Study publicly available on registry

September 16, 2016

Completed
2.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 1, 2019

Completed
5 months until next milestone

Study Completion

Last participant's last visit for all outcomes

June 1, 2019

Completed
Last Updated

July 22, 2020

Status Verified

August 1, 2017

Enrollment Period

3.4 years

First QC Date

March 7, 2016

Last Update Submit

July 20, 2020

Conditions

Outcome Measures

Primary Outcomes (1)

  • Incidence of Treatment-Emergent Adverse Events of sub retinal implantation of stem cell derived retinal pigmented epithelium in the sub retinal space.

    Incidence of surgical related side effects: Retinal detachment, Ocular inflammation, Increase in intraocular pressure, Infection( endophthalmitis), Loss of vision due to surgical, related complications

    1 year

Secondary Outcomes (1)

  • Incidence of side effects related to the treatment itself( injection and implantation of sub retinal stem cell related RPE)

    1 year

Study Arms (2)

injection of hESC-RPE in suspension

ACTIVE COMPARATOR

6 patients will receive cell suspension injections on the sub retinal space prior to the surgeries, to access safety

Procedure: injection of hESC-RPE in suspension

injection hESC-RPE seeded in a substrate

ACTIVE COMPARATOR

15 patients will receive a sub-retinal implantation of a polymeric scaffold seeded hesc- RPE in monolayer

Procedure: injection hESC-RPE seeded in a substrate

Interventions

The first six patients will receive a sub retinal injection of hESC-RPE in solution after a complete pars plana vitrectomy to access safety of the cell implant alone.

Also known as: Human Embryonic Stem Cell - Retinal Pigmented Epithelium
injection of hESC-RPE in suspension

Fifteen patients will receive a sub-retinal implantation of embryonic stem cell derived retinal pigmented epithelium seeded in a polymeric substrate to access safety of substrate seeded with RPE

Also known as: Human Embryonic Stem Cell seeded in a polymeric substrate
injection hESC-RPE seeded in a substrate

Eligibility Criteria

Age18 Years - 90 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Patients with AMD ( Dry , Wet after failure of treatment, disciform scars)
  • Patients with Stargardt's Disease BCVA on the selected eye: worse than 20/200

You may not qualify if:

  • Other ophthalmological diseases( Glaucoma, Diabetic Retinopathy, Previous retinal surgery, uveitis)
  • Systemic diseases with contraindication for surgical procedures with local anaesthesia

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Federal University of Sao Paulo

São Paulo, 04023-062, Brazil

Location

MeSH Terms

Conditions

Macular DegenerationStargardt Disease

Interventions

Suspensions

Condition Hierarchy (Ancestors)

Retinal DegenerationRetinal DiseasesEye DiseasesEye Diseases, HereditaryGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

ColloidsComplex MixturesDosage FormsPharmaceutical Preparations

Study Officials

  • Rubens Belfort, MD

    Federal University of São Paulo UNIFESP

    STUDY CHAIR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Full Professor of Ophthalmology

Study Record Dates

First Submitted

March 7, 2016

First Posted

September 16, 2016

Study Start

August 1, 2015

Primary Completion

January 1, 2019

Study Completion

June 1, 2019

Last Updated

July 22, 2020

Record last verified: 2017-08

Data Sharing

IPD Sharing
Will share

scientific publication in a peer review journal

Locations