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A Follow up Study to Determine the Safety and Tolerability of Sub-retinal Transplantation of Human Embryonic Stem Cell Derived Retinal Pigmented Epithelial (hESC-RPE) Cells in Patients With Stargardt's Macular Dystrophy (SMD)

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ClinicalTrials.gov Identifier: NCT02941991
Recruitment Status : Active, not recruiting
First Posted : October 21, 2016
Last Update Posted : December 28, 2017
Information provided by (Responsible Party):

Study Description
Brief Summary:
The purpose of this study is to evaluate the long term safety and tolerability of hESC-RPE cellular therapy in patients with advanced SMD from 1 to 5 years following the surgical procedure to implant the hESC-RPE cells.

Condition or disease Intervention/treatment
Stargardt's Macular Dystrophy Biological: hESC-RPE

Detailed Description:
This study is a long-term, follow- up of a Phase I/II, open-label, non-randomized, 4-cohort, dose escalation, multi-center clinical trial (referred to as the core trial or core protocol) in which a maximum of 12 SMD patients were transplanted with sequential doses of hESC-RPE cells, starting at a dose of 50,000 hESC-RPE cells transplanted and increasing to a maximum dose of 200,000 hESC-RPE cells transplanted. All patients who participate in the core protocol are eligible for participation in the follow-up protocol. 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. Informed consent for this follow-up protocol will be obtained at the first visit which will occur at the 12-month visit of the core protocol. Patients will be evaluated at 18, 24, 36, 48 and 60 months post-transplant (or more frequently as clinically indicated). Follow-up will include obtaining information about ophthalmological findings and events of special interest as defined in the Primary Outcome. At the last visit of this follow up study, whether at 60 months post-transplant or at early discontinuation, patients will be invited to participate in a life-long annual health survey, under a separate protocol, to further monitor long-term safety.

Study Design

Study Type : Observational
Actual Enrollment : 11 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Follow-up to 5 Years of 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 (hESC-RPE) Cells in Patients With Stargardt's Macular Dystrophy (SMD)
Actual Study Start Date : January 16, 2013
Estimated Primary Completion Date : December 2019
Estimated Study Completion Date : December 2019

Groups and Cohorts

Group/Cohort Intervention/treatment
uniocular subretinal injection of hESC-RPE cells
Cohort 1. 50,000 cells transplanted; Cohort 2. 100,000 cells transplanted; Cohort 3. 150,000 cells transplanted; Cohort 4. 200,000 cells transplanted
Biological: hESC-RPE
sub-retinal injection

Outcome Measures

Primary Outcome Measures :
  1. Safety assessed by Adverse Events (AEs) of special interest in regards to the investigational product [ Time Frame: 4 years ]
    This will include obtaining information about Serious Adverse Events (SAEs) that are neurologic, infectious, hematologic or fatal, any Adverse Event (AE) that causes the subject to withdraw from the study, any new diagnosis of an ocular or immune-mediated disorder, cancer (irrespective of prior history), ectopic or proliferative cell growth (Retinal pigment epithelium (RPE) or non-RPE) with adverse clinical consequence, unexpected, clinically significant AE possibly related to the cell transplant procedure or the investigational product (hESC-RPE cells), Pregnancy in a female subject or the partner of a male subject and pregnancy outcome.

Secondary Outcome Measures :
  1. Incidence of graft failure or rejection [ Time Frame: 4 years ]
    Evidence of graft failure or rejection may consist of: Presence of retinal edema, cystoid macular edema, retinal white dots, retinal hemorrhage, serous retinal detachment, sub-retinal exudates, sub-retinal fibrosis, ocular vascular and/or optic disc leakage, and elevated intraocular pressure or hypotony. Evidence of unanticipated and persistent or increasing non-infectious ocular inflammation (e.g., vasculitis, retinitis, choroiditis, vitritis, pars planitis, anterior segment inflammation/uveitis).

  2. Number of patients with changes in ocular examinations or images [ Time Frame: 4 years ]
    The number of patients with clinically significant absolute values or changes from baseline in Intra-ocular pressure (IOP) and Best Corrected Visual Acuity (BCVA) will be summarized.

Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
The study population is by invitation only from previous participants in the core protocol.

Inclusion Criteria:

  • Must have been treated with hESC-RPE cell transplant in the core protocol.
  • Able to understand and willing to sign the informed consent to participate in the follow-up study.

Exclusion Criteria:

  • There are no exclusion criteria
Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02941991

United Kingdom
Moorefields Eye Hospital NHS Foundation Trust
London, United Kingdom, EC1V2PD
Newcastle on Tyne NHS Foundation Trust
Newcastle upon Tyne, United Kingdom, NE7 7DN
Sponsors and Collaborators
Astellas Institute for Regenerative Medicine
Study Director: Medical Director Astellas Institute for Regenerative Medicine
More Information

Responsible Party: Astellas Institute for Regenerative Medicine
ClinicalTrials.gov Identifier: NCT02941991     History of Changes
Other Study ID Numbers: 7316-CL-0006
2012-002827-14 ( EudraCT Number )
ACT-hESC-RPE-SMD-01FU EU ( Other Identifier: Sponsor )
First Posted: October 21, 2016    Key Record Dates
Last Update Posted: December 28, 2017
Last Verified: December 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Keywords provided by Astellas Pharma Inc ( Astellas Institute for Regenerative Medicine ):
Stargardt's Macular Dystrophy
Retinal Diseases
Macular Degeneration
fundus flavimaculatus
juvenile macular dystrophy

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