Clinical trial NCT02941991: 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)
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)
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Stargardt's Macular Dystrophy
Keywords: Stargardt's Macular Dystrophy
Retinal Diseases
Macular Degeneration
fundus flavimaculatus
hESC-RPE
ASP7316
juvenile macular dystrophy
SMD
Lead sponsor: Astellas Pharma Inc
Sponsor class: INDUSTRY
First posted: 2016-10-21
Enrollment: 12
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: The study population is by invitation only from previous participants in the core protocol.

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.

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.

Eligibility criteria: 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

Primary outcomes:
measure: Safety assessed by Adverse Events (AEs) of special interest in regards to the investigational product
timeFrame: 4 years
Secondary outcomes:
measure: Incidence of graft failure or rejection
timeFrame: 4 years
measure: Number of patients with changes in ocular examinations or images
timeFrame: 4 years
Other outcomes:
Locations:
city: London
country: United Kingdom
facility: Moorefields Eye Hospital NHS Foundation Trust
city: Newcastle upon Tyne
country: United Kingdom
facility: Newcastle on Tyne NHS Foundation Trust
Study officials:
affiliation: Astellas Institute for Regenerative Medicine
name: Medical Director
role: STUDY_DIRECTOR
References: