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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT02941991
Other study ID # 7316-CL-0006
Secondary ID 2012-002827-14AC
Status Completed
Phase
First received
Last updated
Start date January 16, 2013
Est. completion date October 2, 2019

Study information

Verified date December 2020
Source Astellas Pharma Inc
Contact n/a
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial 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.


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.


Recruitment information / eligibility

Status Completed
Enrollment 12
Est. completion date October 2, 2019
Est. primary completion date October 2, 2019
Accepts healthy volunteers No
Gender All
Age group 18 Years and older
Eligibility 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

Study Design


Related Conditions & MeSH terms


Intervention

Biological:
hESC-RPE
sub-retinal injection

Locations

Country Name City State
United Kingdom Moorefields Eye Hospital NHS Foundation Trust London
United Kingdom Newcastle on Tyne NHS Foundation Trust Newcastle upon Tyne

Sponsors (1)

Lead Sponsor Collaborator
Astellas Institute for Regenerative Medicine

Country where clinical trial is conducted

United Kingdom, 

Outcome

Type Measure Description Time frame Safety issue
Primary Safety assessed by Adverse Events (AEs) of special interest in regards to the investigational product 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. 4 years
Secondary Incidence of graft failure or rejection 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). 4 years
Secondary Number of patients with changes in ocular examinations or images 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. 4 years
See also
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Active, not recruiting NCT02749734 - Clinical Study of Subretinal Transplantation of Human Embryo Stem Cell Derived Retinal Pigment Epitheliums in Treatment of Macular Degeneration Diseases Phase 1/Phase 2
Completed NCT01345006 - Sub-retinal Transplantation of hESC Derived RPE(MA09-hRPE)Cells in Patients With Stargardt's Macular Dystrophy Phase 1/Phase 2
Completed NCT02445612 - Long Term Follow Up of Sub-retinal Transplantation of hESC Derived RPE Cells in Stargardt Macular Dystrophy Patients
Active, not recruiting NCT03364153 - Zimura Compared to Sham in Patients With Autosomal Recessive Stargardt Disease (STGD1) Phase 2
Completed NCT01469832 - 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) Phase 1/Phase 2