A Safety Surveillance Study in Subjects With Macular Degenerative Disease Treated With Human Embryonic Stem Cell-derived Retinal Pigment Epithelial Cell Therapy

Macular Degenerative Disease Clinical Trial

Official title:

A Safety Surveillance Study of Events of Special Interest Occurring in Subjects With Macular Degenerative Disease Treated With Human Embryonic Stem Cell-derived Retinal Pigment Epithelial Cell Therapy

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

• NCT number: NCT03167203
• Other study ID #: 7316-CL-0007
• Secondary ID: 2016-005245-23RP
• Status: Enrolling by invitation
• Phase: Phase 1/Phase 2
• Start date: January 8, 2018
• Est. completion date: March 31, 2029

Study information

• Verified date: June 2024
• Source: Astellas Pharma Inc
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The purpose of this study is to is to evaluate the occurrence of late onset (i.e., greater than 5 years after treatment) adverse events of special interest (AESI) in participants who have received sub-retinal transplant of human embryonic stem cell derived - retinal pigment epithelial (hESC-RPE) cells in an AIRM-sponsored clinical trial. The events of special interest are adverse events (AEs) that are presumed to have a potential causal relationship to the hESC-RPE cells.

Description:

Participants will be contacted by the Patient Outreach Center (POC) on an annual basis to complete a questionnaire about the occurrence of AESI, as defined in the Outcome Measure section.

The questionnaire will be completed directly by the participant (via a secure web based platform) or will be administered via telephone by a POC staff member. In the event of the occurrence of a AESI, study participant will be encouraged to contact the POC as soon as possible, rather than wait for the annual questionnaire completion. Follow up data is inclusive of the 1 year in the core trial, 4 years in the long term follow up trial and 10 years in this long term safety follow up. Participation for United Kingdom participants will be life-long.

Recruitment information / eligibility

• Status: Enrolling by invitation
• Enrollment: 36
• Est. completion date: March 31, 2029
• Est. primary completion date: March 31, 2029
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• Participant was treated with hESC-RPE cell therapy in an Astellas Institute for Regenerative Medicine (AIRM) sponsored clinical trial in macular degenerative disease.

• Participant is able to understand.

Exclusion Criteria:

• There are no exclusion criteria.

Related Conditions & MeSH terms

• Macular Degenerative Disease

Intervention

Biological:
• Human Embryonic Stem Cell-Derived Retinal Pigment Epithelial Cells
Participants who have received sub-retinal transplant of hESC-RPE cells in an AIRM sponsored clinical trial

Locations

Country	Name	City	State
United Kingdom	Site GB44001	London
United States	Private Practice	Kansas City	Missouri

Sponsors (1)

Lead Sponsor	Collaborator
Astellas Institute for Regenerative Medicine

Countries where clinical trial is conducted

United States,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of adverse events (AEs) that are ophthalmologic, neurologic, infectious or hematologic	Adverse events (AEs) will be coded using Medical Dictionary for Regulatory Activities (MedDRA). Participant self-reported via an annual questionnaire of adverse events that are ophthalmologic, neurologic, infectious or hematologic.	Up to 15 Years
Primary	Number of new diagnoses of an immune-mediated disorder	Any new diagnosis of an immune-mediated disorder will be participant self-reported via an annual questionnaire.	Up to 15 Years
Primary	Incidents of new cancer, irrespective of prior history	Any new cancer, irrespective of prior history, will be participant self-reported via an annual questionnaire.	Up to 15 Years
Primary	Incidents of hESC-RPE cell proliferation	Occurrence of human embryonic stem cell derived - retinal pigment epithelial (hESC-RPE) cell proliferation will be participant self-reported via an annual questionnaire.	Up to 15 Years
Primary	Incidents of ectopic tissue (RPE or non-RPE) formation	Occurrence of ectopic tissue (retinal pigment epithelial [RPE] or non-RPE) will be participant self-reported via an annual questionnaire	Up to 15 Years
Primary	Number of participant reported pregnancies or pregnancy of participant's partner	Occurrence of pregnancy will be participant self-reported via an annual questionnaire	Up to 15 Years
Primary	Number of reported pregnancy outcome(s)	Occurrence of pregnancy outcomes will be participant self-reported via an annual questionnaire	Up to 15 Years
Primary	All cause death	All causes of death will be collected via an annual questionnaire (through participant-designated secondary contacts)	Up to 15 Years