UMIN-CTR Clinical Trial

Basic information
Public title	A Phase 3 Multicenter, Randomized, Double-Blind and Placebo-Controlled Study, and Open Study of KW-0761 in Patients With HTLV-1 Associated Myelopathy (HAM)
Acronym	A Phase 3 Study of KW-0761 in Subjects With HTLV-1 Associated Myelopathy (HAM)
Scientific Title	A Phase 3 Multicenter, Randomized, Double-Blind and Placebo-Controlled Study, and Open Study of KW-0761 in Patients With HTLV-1 Associated Myelopathy (HAM)
Scientific Title:Acronym	A Phase 3 Study of KW-0761 in Subjects With HTLV-1 Associated Myelopathy (HAM)
Region	Japan

Condition
Condition	HTLV-1 Associated Myelopathy (HAM)
Classification by specialty	Neurology
Classification by malignancy	Others
Genomic information	YES

Objectives
Narrative objectives1	The objective of this study is to assess the efficacy and safety of KW-0761 in subjects with HTLV-1 associated myelopathy (HAM).
Basic objectives2	Efficacy
Basic objectives -Others
Trial characteristics_1	Confirmatory
Trial characteristics_2	Explanatory
Developmental phase	Phase III

Assessment
Primary outcomes	Improvement in Osame's motor disability score
Key secondary outcomes	<Efficacy> - HTLV-1 proviral load in peripheral blood and 10 m walking time at each measuring point - Evaluation of muscle spasticity (Modified Ashworth Scale) - Evaluation of Clinical Global Impression(CGI-I,VAS) - Evaluation of Urinary dysfunction(OABSS,I-PSS) - Evaluation of sensory dysfunction(Numbness in the lower limbs (VAS), Pain in the lower limbs (VAS)) - Neopterine Concentration in CSF <Safety> - Adverse events

Base
Study type	Interventional

Study design
Basic design	Parallel
Randomization	Randomized
Randomization unit	Individual
Blinding	Double blind -all involved are blinded
Control	Placebo
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment

Intervention
No. of arms	2
Purpose of intervention	Treatment
Type of intervention	Medicine
Interventions/Control_1	KW-0761 0.3 mg/kg IV
Interventions/Control_2	Placebo (saline)
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	20 years-old <=
Age-upper limit	Not applicable
Gender	Male and Female
Key inclusion criteria	1) Voluntary written informed consent to participate in the study 2) Diagnosis as HAM according to the second edition of HAM Treatment Manual 3) At least 1-year history of HAM 4) Ongoing medication*1 for HAM, with no changes in 3 months before enrollment; or inadequate response or intolerance to prior medication,*2 which must have been discontinued for at least 3 months before enrollment. Subjects on maintenance therapy with steroids must have been receiving =< 10 mg/day prednisolone equivalent continuously for at least 3 months before enrollment. *1 Steroids, salazosulfapyridine, or >= 1.5 g/day vitamin C *2 Steroids, IFN-alpha, salazosulfapyridine, or >= 1.5 g/day vitamin C 5) No change in the degree of motor dysfunction for at least 3 months before the date of screening, as judged by the investigator or subinvestigator 6) A OMDS of >=3 at screening and able to walk >=10 m at screening (use of a single cane or double canes is allowed)
Key exclusion criteria	1) Any of the following significant concomitant diseases: Type 1 diabetes mellitus, Poorly controlled type 2 diabetes mellitus (HbA1c (NGSP) > 8.5%), Congestive heart failure (Class II to IV of the New York Heart Association Functional Classification), Myocardial infarction within 1 year before enrollment, Unstable angina within 1 year before enrollment, Poorly controlled hypertension (systolic blood pressure > 150 mm Hg and diastolic blood pressure > 90 mm Hg at screening), Sever chronic lung disease requiring oxygen therapy, Multiple sclerosis or any other demyelinating disease, Epilepsy requiring treatment with antiepileptics (with the exception of epilepsy controlled by antiepileptics, with no occurrence of seizures for at least 3 years before informed consent), and Active malignancy (including ATL); or onset of malignancy or previous treatment for malignancy (with the exception of resected or surgically cured intraepithelial carcinoma of the uterine cervix, basal cell carcinoma of the skin, squamous cell carcinoma of the skin, or ductal breast carcinoma) within 5 years before informed consent 2) Active infection 3) Concurrent spinal cord compression lesion (e.g., cervical spine diseases, disk herniation, or ossification of the ligamentum flavum) , with the exception of conditions that would not affect efficacy evaluation in the study, as judged by the investigator or subinvestigator 4) Concurrent dementia 5) Concurrent psychiatric disorder, with the exception of conditions that would not affect obtaining informed consent or efficacy evaluation in the study, as judged by the investigator or subinvestigator 6) History of or current alcohol or drug dependence 7) Planned surgery during the study period 8) Any other conditions unsuitable for participation in the study in the opinion of the investigator or subinvestigator
Target sample size	52

Research contact person
Name of lead principal investigator	1st name Yoshihisa Middle name Last name Yamano
Organization	St. Marianna University School of Medicine
Division name	Institute of Medical Science
Zip code	216-8512
Address	2-16-1 Sugao, Miyamae-ku, Kawasaki, Kanagawa, 216-8512 Japan
TEL	044-977-8111
Email	yyamano@marianna-u.ac.jp

Public contact
Name of contact person	1st name - Middle name Last name Clinical trial information contact
Organization	Kyowa Kirin Co., Ltd.
Division name	R&D Division, R&D Planning Departmen
Zip code	100-0004
Address	1-9-2 Otemachi, Chiyoda-ku, Tokyo
TEL	03-5205-7200
Homepage URL
Email	clinical.info.jp@kyowakirin.com

Sponsor
Institute	Kyowa Kirin Co., Ltd.
Institute
Department

Funding Source
Organization	None
Organization
Division
Category of Funding Organization	Self funding
Nationality of Funding Organization

Other related organizations
Co-sponsor
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	東北大学病院（宮城県）、聖マリアンナ医科大学病院（神奈川県）、名古屋大学病院（愛知県）、京都府立医科大学病院（京都府）、関西医科大学病院（大阪府）、愛媛大学病院（愛媛県）、福岡大学病院（福岡県）、藤元総合病院（宮崎県）、鹿児島大学病院（鹿児島県）、国立沖縄病院（沖縄県）、琉球大学病院（沖縄県）、熊本大学病院（熊本県）、産業医科大学病院（福岡県）、鹿児島市立病院（鹿児島県）

Other administrative information
Date of disclosure of the study information	2017 Year 11 Month 13 Day

Related information
URL releasing protocol
Publication of results	Unpublished

Result
URL related to results and publications
Number of participants that the trial has enrolled	67
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	Terminated
Date of protocol fixation	2017 Year 04 Month 14 Day
Date of IRB	2017 Year 05 Month 22 Day
Anticipated trial start date	2017 Year 06 Month 02 Day
Last follow-up date	2021 Year 08 Month 31 Day
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

Other
Other related information

Management information
Registered date	2017 Year 11 Month 10 Day
Last modified on	2022 Year 04 Month 07 Day

Link to view the page
URL(English)	https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000034172