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Register:	ClinicalTrials.gov
Last refreshed on:	12 July 2021
Main ID:	NCT01469832
Date of registration:	08/11/2011
Prospective Registration:	Yes
Primary sponsor:	Astellas Institute for Regenerative Medicine
Public title:	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)
Scientific title:	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)
Date of first enrolment:	December 13, 2011
Target sample size:	12
Recruitment status:	Completed
URL:	https://clinicaltrials.gov/show/NCT01469832
Study type:	Interventional
Study design:	Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
Phase:	Phase 1/Phase 2

Countries of recruitment
United Kingdom

Contacts

Name:	Medical Director
Address:
Telephone:
Email:
Affiliation:	Astellas Institute for Regenerative Medicine

Key inclusion & exclusion criteria

Inclusion Criteria:

- Adult male or female over 18 years of age. Clinical diagnosis of SMD If known, the
patient"s genotype will be recorded in the medical history, if unknown, patient will
allow for the submission of a sample for genotyping.

Independently verified clinical findings consistent with SMD. The visual acuity of the eye
to receive the transplant will be no better than 20/400. The visual acuity of the eye in
the better vision cohort to receive the transplant will be no better than 20/100.

The visual acuity of the eye that is not to receive the transplant will be no better than
20/400 for the worse vision patients and no worse than 20/100 for the better vision
patients.

Electrophysiological findings consistent with SMD . Medically suitable to undergo
vitrectomy and subretinal injection. Medically suitable for general anaesthesia or waking
sedation, if needed.

Medically suitable for transplantation of an embryonic stem cell line:

- Normal serum chemistry (sequential multi-channel analyzer 20 [SMA-20]) and hematology
(complete blood count [CBC], prothrombin time [PT], and activated partial
thromboplastin time [aPTT]) screening tests.

- Negative urine screen for drugs of abuse.

- Negative human immunodeficiency virus (HIV), hepatitis B (HBV), and hepatitis C (HCV)
serologies.

- No history of malignancy, with the exception of successfully treated basal cell or
squamous cell carcinoma of the skin.

- Negative cancer screening within previous 6 months:

complete history & physical examination; dermatological screening exam for malignant
lesions; negative fecal occult blood test negative chest roentgenogram (CXR); normal CBC &
manual differential; negative urinalysis (U/A);normal thyroid exam and thyroid panel; if
male, normal testicular examination; if over age 40, digital rectal examination (DRE) and
prostate specific antigen (PSA); if female, normal pelvic examination with Papanicolaou
smear; and if female, normal clinical breast exam and if 50 years of age or older, negative
mammogram.

If female and of childbearing potential, willing to use an effective form of birth control
during the study.

If male, willing to use barrier and spermicide contraception during the study. Willing to
defer all future blood, blood component or tissue donation. Able to understand and willing
to sign the informed consent.

Exclusion Criteria:

- History of malignancy, with the exception of successfully treated basal cell or
squamous cell carcinoma of the skin.

History of myocardial infarction in previous 12 months. History of diabetes mellitus. Any
immunodeficiency. Any current immunosuppressive therapy other than intermittent or low dose
corticosteroids.

Serologic evidence of infection with Hepatitis B, Hepatitis C, or HIV. Current
participation in any other clinical trial. Participation within previous 6 months in any
clinical trial of a drug by ocular or systemic administration.

Any other sight-threatening ocular disease. Any chronic ocular medications. Any history of
retinal vascular disease (compromised blood-retinal barrier. Glaucoma. Uveitis or other
intraocular inflammatory disease. Significant lens opacities or other media opacity. Ocular
lens removal within previous 3 months

Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Stargardt's Macular Dystrophy

Intervention(s)

Biological: MA09-hRPE

Primary Outcome(s)

safety and tolerance of transplantation [Time Frame: 12 months]

Secondary Outcome(s)

Evidence of successful engraftment [Time Frame: 12 months]

Secondary ID(s)

ACT hESC-RPE SMD 01 EU

2011-000054-34

7316-CL-0003

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Ethics review

Results

Results available:
Date Posted:
Date Completed:
URL:

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