Safety and Tolerability of MA09-hRPE Cells in Patients With Stargardt's Macular Dystrophy(SMD)

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
CHABiotech CO., Ltd
ClinicalTrials.gov Identifier:
NCT01625559
First received: June 18, 2012
Last updated: February 16, 2015
Last verified: August 2012

June 18, 2012
February 16, 2015
September 2012
March 2015   (final data collection date for primary outcome measure)
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:

  1. Any grade 2 (NCI grading system) or greater adverse event related to the cell product
  2. Any evidence that the cells are contaminated with an infectious agent
  3. Any evidence that the cells show tumorigenic potential
safety and tolerance of transplantation [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]
The safety and tolerance of transplantation of hESC-derived 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, or have tumorigenic potential, Adverse Event and Serious Adverse Event assessment, Serial vital signs and Clinical laboratory tests Direct ophthalmological monitoring Monitoring of RPE cells acceptance/integrity/rejection Monitoring of local and systemic infection or tumorigenic cell transformation
Complete list of historical versions of study NCT01625559 on ClinicalTrials.gov Archive Site
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

Evidence of successful engraftment [ Time Frame: 12months ] [ 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

Not Provided
Not Provided
 
Safety and Tolerability of MA09-hRPE Cells in Patients With Stargardt's Macular Dystrophy(SMD)
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)

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.
Not Provided
Interventional
Phase 1
Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Stargardt's Macular Dystrophy
Biological: MA09-hRPE
MA09-hRPE: 50,000 cells
Experimental: 50,000 cells
Biological: MA09-hRPE Cellular therapy
Intervention: Biological: MA09-hRPE
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Active, not recruiting
3
June 2015
March 2015   (final data collection date for primary outcome measure)

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
Both
20 Years and older
No
Contact information is only displayed when the study is recruiting subjects
Korea, Republic of
 
NCT01625559
CHA_CTP_0903
Yes
CHABiotech CO., Ltd
CHABiotech CO., Ltd
Not Provided
Principal Investigator: Wonkyung Song, MD. PhD. CHA Bundang Medical Center
CHABiotech CO., Ltd
August 2012

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP