Sub-retinal Transplantation of hESC Derived RPE(MA09-hRPE)Cells in Patients With Stargardt's Macular Dystrophy

• Register: ClinicalTrials.gov
• Last refreshed on: 12 July 2021
• Main ID: NCT01345006
• Date of registration: 28/04/2011
• Prospective Registration: Yes
• Primary sponsor: Astellas Institute for Regenerative Medicine
• Public title: Sub-retinal Transplantation of hESC Derived RPE(MA09-hRPE)Cells in Patients With Stargardt's Macular Dystrophy
• 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 (MA09-hRPE) Cells in Patients With Stargardt's Macular Dystrophy (SMD)
• Date of first enrolment: June 16, 2011
• Target sample size: 13
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01345006
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Other. Masking: None (Open Label).
• Phase: Phase 1/Phase 2

Countries of recruitment

United States

Contacts

• Name: Medical Director
• Affiliation: Astellas Institute for Regenerative Medicine

Key inclusion & exclusion criteria

Inclusion Criteria:

- Adult male or female over 18 years of age.

- Clinical diagnosis of advanced 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.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.

- Peripheral visual field constriction documented on standard kinetic visual field
testing.

- Electrophysiological findings consistent with SMD.

- Medically suitable to undergo vitrectomy and subretinal injection.

- Medically suitable for general anesthesia 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), 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 & if over age 50 years, negative colonoscopy within
previous 7 years;

- negative chest roentgenogram (CXR);

- normal CBC & manual differential;

- negative urinalysis (U/A);

- normal thyroid exam;

- 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 40 years of age or older, negative
mammogram.

- If female and of childbearing potential, willing to use two effective forms 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.

- If female, pregnancy or lactation.

- Any other medical condition, which, in the Investigator's judgment, will interfere
with the patient's ability to comply with the protocol, compromises patient safety, or
interferes with the interpretation of the study results.



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 Assessments [Time Frame: 12 months]

The safety and tolerance of transplantation of hESC-derived RPE cells MA09-hRPE [Time Frame: 12 months]

Secondary Outcome(s)

Evidence of successful engraftment [Time Frame: 12 months]

Secondary ID(s)

ACT SMD 01 MA09-hRPE

ACT MA09-hRPE SMD-001

7316-CL-0001

Source(s) of Monetary Support

Please refer to primary and secondary sponsors