Trial record 2 of 19 for:    stargardt's macular dystrophy

Long Term Follow Up of Sub-retinal Transplantation of hESC Derived RPE Cells in Stargardt Macular Dystrophy Patients

This study is enrolling participants by invitation only.
Sponsor:
Information provided by (Responsible Party):
Ocata Therapeutics
ClinicalTrials.gov Identifier:
NCT02445612
First received: May 13, 2015
Last updated: May 14, 2015
Last verified: May 2015
  Purpose

This is the long term follow-up of a safety and tolerability trial to evaluate the effect of subretinal injection of human embryonic stem cell derived retinal pigment epithelium cells in patients with Stargardt's Macular Dystrophy (SMD).


Condition Intervention
Stargardt's Macular Dystrophy
Biological: MA09-hRPE

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Official Title: Long Term Follow Up to a Phase I/II, Open-Label, Multi-Center, Prospective Study to Determine the Safety and Tolerability of Sub-retinal Transplantation of Human Embryonic Stem Cell Derived Retinal Pigmented Epithelial (MA09-hRPE) Cells in Patients With Stargardt's Macular Dystrophy (SMD)

Resource links provided by NLM:


Further study details as provided by Ocata Therapeutics:

Primary Outcome Measures:
  • The safety and tolerance of transplantation of hESC-derived RPE cells MA09-hRPE [ Time Frame: 15 years ] [ Designated as safety issue: Yes ]
    • Any grade 2 (NCI grading system) or greater adverse event related to the cell product
    • Any evidence that the cells are contaminated with an infectious agent
    • Any evidence that the cells show tumorigenic potential


Secondary Outcome Measures:
  • Evidence of Successful Engraftment of MA09-hRPE Cells [ Time Frame: 15 years ] [ Designated as safety issue: No ]
    • Structural evidence (OCT imaging, fluorescein angiography, autofluorescense photography, slit-lamp examination with fundus photography) that cells have been implanted in the correct location
    • Electroretinographic evidence (mfERG) showing enhanced activity in the implant location

  • Evidence of Rejection of MA09-hRPE Cells [ Time Frame: 15 years ] [ Designated as safety issue: No ]
    • Structural (imaging) evidence that implanted MA09-hRPE cells are no longer in the correct location or the presence of vascular leakage


Estimated Enrollment: 13
Study Start Date: July 2012
Estimated Study Completion Date: December 2030
Estimated Primary Completion Date: December 2029 (Final data collection date for primary outcome measure)
Groups/Cohorts Assigned Interventions
Experimental: MA09-hRPE
Sub-retinal transplantation of MA09-hRPE cells
Biological: MA09-hRPE
Cohort 1 50,000 cells Cohort 2 100,000 cells Cohort 2a Better Vision 100,000 cells Cohort 3 150,000 cells Cohort 4 200,000 cells

Detailed Description:

This study is a long term, non interventional follow up to the phase I/II, open-label, non randomized, sequential, multi-center trial (referred to as the core protocol) in which a maximum of 13 SMD patients were treated. Ten poor vision subjects(20/400) were transplanted with sequential doses of hESC-RPE cells, starting at a dose of 50,000 hESC-RPE cells transplanted (three subjects), 100,000 hESC-RPE cells transplanted (three subjects), 150,000 hESC-RPE cells transplanted (three subjects) and increasing to a maximum dose of 200,000 hESC-RPE cells transplanted(one subject). Three better vision subjects(20/100) were transplanted with a dose of 100,000 hESC-RPE cells.

Eligible patients will be those who sign a consent form for the extension study, and who fulfill all inclusion/exclusion criteria.

The first visit of this extension protocol will correspond to the last visit of the core protocol, and will take place at 12 months post cell implantation.

During the first 2 years after completion of the core protocol, i.e. from Year 1 to Year 2 and from Year 2 to Year 3 post transplantation, the patients will be evaluated at approximately 3 month intervals; during years three thru five patients will be evaluated at approximately six month intervals. During years 6-15, patients will either be required to return to the study center or provide the name of an ophthalmologist in their locality who would provide an evaluation of the patient's vision and any abnormal findings and report these results to the study site annually.

Follow up will include obtaining information about visual acuity, ophthalmological findings and adverse events (AEs), specifically including cancer, neurological, autoimmune or hematological disorders that developed or worsened since the last visit.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population

The study population is by invitation only for previous participants in the core protocol.

Criteria

Inclusion Criteria:

  • Must have met the eligibility of the core protocol.
  • Able to understand and willing to sign the informed consent to participate in the follow-up.
  • Treated with hESC-RPE cell transplant in the core protocol.
  • Previous participation in study.

Exclusion Criteria:

  • Unwilling or unable to participate in the study
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02445612

Locations
United States, California
Jules Stein Eye Institute, UCLA School of Medicine
Los Angeles, California, United States, 90095
United States, Florida
Bascom Palmer Eye institute
Miami, Florida, United States, 33136
United States, Pennsylvania
Wills Eye Institute-Mid Atlantic Retina
Philadelphia, Pennsylvania, United States, 19107
Sponsors and Collaborators
Ocata Therapeutics
Investigators
Principal Investigator: Steven Schwartz, MD Jules Stein Eye Institute, UCLA School of Medicine
Principal Investigator: Carl D Regillo, MD Wills Eye Institute-Mid Atlantic Retina
Principal Investigator: Byron L Lam University of Miami
  More Information

Publications:
Responsible Party: Ocata Therapeutics
ClinicalTrials.gov Identifier: NCT02445612     History of Changes
Other Study ID Numbers: ACT MA09-hRPE 001(SMD)LTFU
Study First Received: May 13, 2015
Last Updated: May 14, 2015
Health Authority: United States: Food and Drug Administration

Keywords provided by Ocata Therapeutics:
SMD
long term follow-up
fundus flavimaculatus
juvenile macular dystrophy

Additional relevant MeSH terms:
Macular Degeneration
Eye Diseases
Retinal Degeneration
Retinal Diseases

ClinicalTrials.gov processed this record on July 01, 2015