Trial record 4 of 12 for:
"Stargardt disease"
Safety and Tolerability of MA09-hRPE Cells in Patients With Stargardt's Macular Dystrophy(SMD)
This study is currently recruiting participants.
Verified August 2012 by CHA Bio & Diostech
Sponsor:
CHA Bio & Diostech
Information provided by (Responsible Party):
CHA Bio & Diostech
ClinicalTrials.gov Identifier:
NCT01625559
First received: June 18, 2012
Last updated: October 22, 2012
Last verified: August 2012
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Purpose
The purpose of this study is:
- To evaluate the safety and tolerability of RPE cellular therapy in patients with SMD Group
- When-MA09-hRPE cell transplantation to evaluate the safety of surgical procedures.
- In future studies intended to assess the number of transplanted hRPE cells.
- In the past, MA09-hRPE cell therapy used in the study was to evaluate the validity of the potential.
- Homologous retinal pigment epithelial cells derived from embryonic stem cells, future studies of drugs that are used in representing the potential validity to evaluate the optimal dose.
| Condition | Intervention | Phase |
|---|---|---|
|
Stargardt's Macular Dystrophy |
Biological: MA09-hRPE |
Phase 1 |
| Study Type: | Interventional |
| Study Design: | Endpoint Classification: Safety Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | A Phase I, Open-Label, 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) |
Resource links provided by NLM:
Genetics Home Reference related topics:
age-related macular degeneration
X-linked juvenile retinoschisis
MedlinePlus related topics:
Macular Degeneration
U.S. FDA Resources
Further study details as provided by CHA Bio & Diostech:
Primary Outcome Measures:
- safety and tolerance of transplantation [ Time Frame: 18 months ] [ Designated as safety issue: Yes ]
The transplantation of hESC-derived RPE cells MA09-hRPE will be considered safe and tolerated in the absence of:
- Any grade 2 (NCI grading system) or greater adverse event related to the cell product
- Any evidence that the cells are contaminated with an infectious agent
- Any evidence that the cells show tumorigenic potential
Secondary Outcome Measures:
- Evidence of successful engraftment [ Time Frame: 18 months ] [ Designated as safety issue: No ]
Evidence of successful engraftmentEvidence of successful engraftment will consist of:
Structural evidence (OCT imaging, fluorescein angiography, autofluorescense photography, slit-lamp examination with fundus photography) that cells have been implanted in the correct location Electroretinographic evidence (mfERG) showing enhanced activity in the implant location
| Estimated Enrollment: | 3 |
| Study Start Date: | September 2012 |
| Estimated Study Completion Date: | October 2014 |
| Estimated Primary Completion Date: | August 2014 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
Experimental: 50,000 cells
Biological: MA09-hRPE Cellular therapy
|
Biological: MA09-hRPE
MA09-hRPE: 50,000 cells
|
Eligibility| Ages Eligible for Study: | 20 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- Adult male or female over 20 years of age.
- Clinical diagnosis of advanced SMD.
- The visual acuity of the eye to receive the transplant will be no better than hand movement.
- The visual acuity of the eye that is not to receive the transplant will be no better than 24 (20/320) Early Treatment of Diabetic Retinopathy Study (ETDRS) letters.
Exclusion Criteria:
- History of malignancy.
- History of myocardial infarction in previous 12 months.
- History of diabetes mellitus.
- Any immunodeficiency.
- Any current immunosuppressive therapy other than intermittent or low dose cortico steroids.
- 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
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01625559
Contacts
| Contact: Hunhee Kang | +82 2 3468 2894 | hunheekang@chamc.co.kr |
Locations
| Korea, Republic of | |
| CHA Bundang Medical Center | Recruiting |
| Seongnam-si, Gyeonggi-do, Korea, Republic of, 463-712 | |
| Contact: Wonkyung Song, MD. PhD 82-31-780-5479 | |
| Principal Investigator: Wonkyung Song, MD. PhD | |
Sponsors and Collaborators
CHA Bio & Diostech
Investigators
| Principal Investigator: | Wonkyung Song, MD. PhD. | CHA Bundang Medical Center |
More Information
No publications provided
| Responsible Party: | CHA Bio & Diostech |
| ClinicalTrials.gov Identifier: | NCT01625559 History of Changes |
| Other Study ID Numbers: | CHA_CTP_0903 |
| Study First Received: | June 18, 2012 |
| Last Updated: | October 22, 2012 |
| Health Authority: | Korea: Food and Drug Administration |
Additional relevant MeSH terms:
|
Macular Degeneration Retinal Degeneration Retinal Diseases Eye Diseases |
ClinicalTrials.gov processed this record on May 23, 2013