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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)

NCT ID: NCT02941991

Last Updated: 2020-12-10

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

12 participants

Study Classification

OBSERVATIONAL

Study Start Date

2013-01-16

Study Completion Date

2019-10-02

Brief Summary

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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.

Detailed Description

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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.

Conditions

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Stargardt's Macular Dystrophy

Keywords

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Stargardt's Macular Dystrophy Retinal Diseases Macular Degeneration fundus flavimaculatus hESC-RPE ASP7316 juvenile macular dystrophy SMD

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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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

hESC-RPE

Intervention Type BIOLOGICAL

sub-retinal injection

Interventions

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hESC-RPE

sub-retinal injection

Intervention Type BIOLOGICAL

Other Intervention Names

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ASP7316

Eligibility Criteria

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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.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Astellas Institute for Regenerative Medicine

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Medical Director

Role: STUDY_DIRECTOR

Astellas Institute for Regenerative Medicine

Locations

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Moorefields Eye Hospital NHS Foundation Trust

London, , United Kingdom

Site Status

Newcastle on Tyne NHS Foundation Trust

Newcastle upon Tyne, , United Kingdom

Site Status

Countries

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United Kingdom

Related Links

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https://astellasclinicalstudyresults.com/study.aspx?ID=394

Link to results on the Astellas Clinical Study Results website.

Other Identifiers

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2012-002827-14

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

ACT-hESC-RPE-SMD-01FU EU

Identifier Type: OTHER

Identifier Source: secondary_id

7316-CL-0006

Identifier Type: -

Identifier Source: org_study_id