UMIN-CTR Clinical Trial

Basic information
Official scientific title of the study	An investigator initiated phase II clinical trial of Tamibarotene (AM80G) for chronic graft-versus-host disease
Title of the study (Brief title)	A phase II trial of Tamibarotene administration for Chronic GVHD
Region	Japan

Condition
Condition	Recipients refractory or resistant chronic GVHD to therapy with corticosteroid
Classification by specialty	Hematology and clinical oncology
Classification by malignancy	Others
Genomic information	NO

Objectives
Narrative objectives1	To evaluate the safety and efficacy of Tamibarotene (AM80G) for the patients of steroid-refractory chronic GVHD
Basic objectives2	Safety,Efficacy
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase

Assessment
Primary outcomes	Probability of failure-free survival (FFS) and complete/partial response at 24 weeks
Key secondary outcomes	1. Probability of FFS and complete/partial response at 12 weeks 2.Probability of FFS and overall survival rate at 1 year 3.Proportion of patients who are FFS and have at least 50% reduction in baseline steroid dose at 24 weeks

Base
Study type	Interventional

Study design
Basic design	Single arm
Randomization	Non-randomized
Randomization unit
Blinding	Open -no one is blinded
Control	Uncontrolled
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment

Intervention
No. of arms	1
Purpose of intervention	Treatment
Type of intervention	Medicine
Interventions/Control_1	Patients were orally administered Tamibarotene (AM80G) (4 mg/day) daily for 24 weeks.
Interventions/Control_2
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	18 years-old <=
Age-upper limit	85 years-old >
Gender	Male and Female
Key inclusion criteria	Patients diagnosed as chronic GVHD according to diagnostic criteria proposed by National Institutes of Health Consensus Development Project on Criteria for Clinical Trials in Chronic GVHD. 1. Aged 18 to 84 years at consent 2. Patients with Karnofsky Performance Scale of 60 or more 3. Patient or guardian willing and able to provide informed consent 4. Steroid-refractory or steroid-resistant chronic GVHD defined as: Inability to begin prednisone therapy at a dose of greater than 0.5 mg/kg/day or equivalent dose if on another steroid. (1) Progression of chronic GVHD while being treated with prednisone therapy at a dose of greater than 1 mg/kg/day for 2 weeks. (2) Failure to improve after 4 weeks while being treated with prednisone therapy at a dose of greater than 0.5 mg/kg/day for at least 2 weeks. (3) Flare of chronic GVHD while tapering of steroids. (4) steroid-dependent chronic GVHD received prednisone therapy at a dose of 10 mg/day or equivalent dose if on another steroid. 5. Stated willingness to use contraception during the study and through 2 years for wowen and 6 months for men after the final dose of study treatment.
Key exclusion criteria	1. Mental illness which needs psychiatric medication or are judged by physician as being unsuitable 2. Active, uncontrolled infection (not responding to antibiotics, antifungal or antiviral therapies) 3. Patients with severe liver dysfunction (CTCAE ver.4.0 Grade 3 or more, excepting liver GVHD) 4. Patients with previous experience with Am80 or other retinoid in the past 12 weeks 5. Patients who are allergic or hypersensitive to Am80 or other retinoid. 6. Patients who are pregnant, patients who have possibility of being pregnant, or patients who are breastfeeding. 7. Pulmonary: DLCO <40% or FEV1% <40% 8. Patients with life expectancy of less than 6 months. 9. Patients who are enrolled in other clinical studies in the past 3 months. 10. Other patients who are judged by physician as being unsuitable
Target sample size	18

Research contact person
Name of lead principal investigator	Yoshinobu Maeda
Organization	Okayama University Hospital
Division name	Department of Hematology and Oncology
Address	2-5-1 Shikata-cho, Kita-ku, Okayama
TEL	086-235-7227
Email	yosmaeda@md.okayama-u.ac.jp

Public contact
Name of contact person	Hisakazu Nishimori
Organization	Okayama University Hospital
Division name	Department of Hematology and Oncology
Address	2-5-1 Shikata-cho, Kita-ku, Okayama
TEL	086-235-7227
Homepage URL
Email	n-mori@md.okayama-u.ac.jp

Sponsor
Institute	Okayama University Hospital
Institute
Department

Funding Source
Organization	AMED
Organization
Division
Category of Funding Organization	Japanese Governmental office
Nationality of Funding Organization

Other related organizations
Co-sponsor
Name of secondary funｄer(s)

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	岡山大学病院（岡山県）、安城更生病院（愛知県）、大阪市立大学病院（大阪府）、都立駒込病院（東京都）、九州大学病院（福岡県）、自治医科大学附属病院（栃木県）

Other administrative information
Date of disclosure of the study information	2015 Year 12 Month 27 Day

Progress
Recruitment status	Terminated
Date of protocol fixation	2013 Year 04 Month 22 Day
Anticipated trial start date	2013 Year 10 Month 01 Day
Last follow-up date
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

Related information
URL releasing protocol
Publication of results	Unpublished
URL releasing results
Results
Other related information

Management information
Registered date	2015 Year 12 Month 27 Day
Last modified on	2017 Year 02 Month 28 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000023213