UMIN-CTR Clinical Trial

Unique ID issued by UMIN UMIN000011929
Receipt number R000013279
Scientific Title A Study of transplantation of autologous induced pluripotent stem cell (iPSC) derived retinal pigment epithelium (RPE) cell sheet in subjects with exudative age related macular degeneration
Date of disclosure of the study information 2013/10/02
Last modified on 2019/04/05 11:12:03

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Basic information

Public title

A Study of transplantation of autologous induced pluripotent stem cell (iPSC) derived retinal pigment epithelium (RPE) cell sheet in subjects with exudative age related macular degeneration

Acronym

Transplantation of autologous iPSC derived RPE cell sheet in subject with exudative AMD

Scientific Title

A Study of transplantation of autologous induced pluripotent stem cell (iPSC) derived retinal pigment epithelium (RPE) cell sheet in subjects with exudative age related macular degeneration

Scientific Title:Acronym

Transplantation of autologous iPSC derived RPE cell sheet in subject with exudative AMD

Region

Japan


Condition

Condition

Exudative age related macular degeneration

Classification by specialty

Ophthalmology

Classification by malignancy

Others

Genomic information

NO


Objectives

Narrative objectives1

In this trial of retinal pigment epithelium (RPE) replacement, the aim will be to evaluate the safety and feasibility/efficacy of treating subjects with exudative age-related macular degeneration (AMD) by transplanting autologous iPSC derived RPE sheets

Basic objectives2

Safety

Basic objectives -Others


Trial characteristics_1

Exploratory

Trial characteristics_2


Developmental phase



Assessment

Primary outcomes

Safety of the proposed treatment protocol: incidence and severity of adverse events related to the use of autologous iPSC derived RPE sheet.
<Adverse events related to iPSC derived RPE sheet>
1 Poor survival of the graft, immune rejection
2 Tumor formation
<Adverse events related to surgical procedure>
1. Retinal detachment, choroidal hemorrhage, vitreous hemorrhage
2 Retinal tear and retinal detachment

Key secondary outcomes



Base

Study type

Interventional


Study design

Basic design

Parallel

Randomization

Non-randomized

Randomization unit


Blinding

Open -no one is blinded

Control

Uncontrolled

Stratification


Dynamic allocation


Institution consideration


Blocking


Concealment



Intervention

No. of arms

2

Purpose of intervention

Treatment

Type of intervention

Device,equipment Maneuver Other

Interventions/Control_1

The size of the RPE sheet used in the first 3 cases will be 1.3 mm x 3 mm

Interventions/Control_2

In the 3 following cases, the use of RPE sheets of a larger size, or of in multiple number with the same size, will be allowed. In case of using multiple sheets, maximum of 3 sheets will be used.

Interventions/Control_3


Interventions/Control_4


Interventions/Control_5


Interventions/Control_6


Interventions/Control_7


Interventions/Control_8


Interventions/Control_9


Interventions/Control_10



Eligibility

Age-lower limit

50 years-old <=

Age-upper limit


Not applicable

Gender

Male and Female

Key inclusion criteria

1) Diagnosis of exudative AMD including non-typical subtypes in at least one eye.
2) Male and female subjects aged 50 year and older at the time of acquired consent
3) Presence of subfoveal CNV, fibrotic scar, or RPE tear related to exudative AMD
4) The visual acuity of less than 0.3 but better than hand motion.
5) Treatment history of standard anti-VEGF therapy with limited effects such as poor response or persistent recurrence (minimum treatment with anti-VEGF induction therapy followed by at least one additional injection)
6) The mean Central 4-degree retinal sensitivity of less than 5 db on MicroPerimeter1 (MP-1) examination
7) Able to understand and willing to sign the informed consent

Key exclusion criteria

1) Presence of ocular infection
2) Presence of other retinal diseases such as diabetic retinopathy, hypertensive retinopathy, retinal vessel occlusions.
3) Presence of optic nerve atrophy
4) Presence of glaucoma of uncontrolled ocular pressure
5) Presence of significant hepatic failure (with either AST or ALT higher than 100 IU/L)
6) Presence of renal failure
7) Negative serum tests for human immunodeficiency virus (HIV), hepatitis B (HBV), hepatitis C (HCV) or syphilis.
8)No history of allergic reaction to antibiotics or bovine serum
9) No medical requirements for a continuous treatment of anti-coagulants or anti-platelets
10) Medically suitable for general anesthesia
11) No presence of or history of malignancy in past 5 years
12) No history of allergic reaction to ICG or Fluorescein
13) Pregnant females; breastfeeding females; and females of childbearing potential.
14) No participation in other clinical trials that involve the last one month before informed consent is obtained
15) Medically judged inadequate by the docters responsible for this study

Target sample size

6


Research contact person

Name of lead principal investigator

1st name
Middle name
Last name Masayo Takahashi

Organization

RIKEN

Division name

Laboratory for Retinal Regeneration

Zip code


Address

2-2-3 Minatojima-minamimachi, Chuo-ku, Kobe, Hyogo

TEL

078-306-3305

Email

retinalab@cdb.riken.jp


Public contact

Name of contact person

1st name
Middle name
Last name Yasuhiko Hirami

Organization

Kobe City Medical Center General Hospital

Division name

Department of Ophthalmology

Zip code


Address

2-1-1 Minatojima-minamimachi, Chuo-ku, Kobe, Hyogo

TEL

078-302-4321

Homepage URL

http://www.riken-ibri.jp/AMD/

Email

hirami@fbri.org


Sponsor or person

Institute

RIKEN

Institute

Department

Personal name



Funding Source

Organization

AMED
MHLW

Organization

Division

Category of Funding Organization

Japanese Governmental office

Nationality of Funding Organization

Japan


Other related organizations

Co-sponsor

Foundation for Biomedical Research and Innovation
Kobe City Medical Center general Hospital

Name of secondary funder(s)



IRB Contact (For public release)

Organization


Address


Tel


Email



Secondary IDs

Secondary IDs

NO

Study ID_1


Org. issuing International ID_1


Study ID_2


Org. issuing International ID_2


IND to MHLW



Institutions

Institutions

先端医療センター病院(兵庫県)
理化学研究所 多細胞システム形成研究センター(兵庫県)


Other administrative information

Date of disclosure of the study information

2013 Year 10 Month 02 Day


Related information

URL releasing protocol


Publication of results

Partially published


Result

URL related to results and publications

https://www.nejm.org/doi/full/10.1056/NEJMoa1608368

Number of participants that the trial has enrolled

1

Results


Results date posted


Results Delayed


Results Delay Reason


Date of the first journal publication of results


Baseline Characteristics


Participant flow


Adverse events


Outcome measures


Plan to share IPD


IPD sharing Plan description



Progress

Recruitment status

Completed

Date of protocol fixation

2013 Year 07 Month 29 Day

Date of IRB

2013 Year 07 Month 29 Day

Anticipated trial start date

2013 Year 10 Month 02 Day

Last follow-up date

2019 Year 02 Month 28 Day

Date of closure to data entry


Date trial data considered complete


Date analysis concluded



Other

Other related information

The trial was halted after two patients because of Japan's Regenerative Medicine Law of 2014


Management information

Registered date

2013 Year 10 Month 01 Day

Last modified on

2019 Year 04 Month 05 Day



Link to view the page

Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000013279