UMIN-CTR Clinical Trial

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

HTLV-1 Associated Myelopathy (HAM)

Classification by specialty

Neurology

Classification by malignancy

Others

Genomic information

YES

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

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

Study type

Interventional

Basic design

Parallel

Randomization

Randomized

Randomization unit

Individual

Blinding

Double blind -all involved are blinded

Control

Placebo

Stratification

Dynamic allocation

Institution consideration

Blocking

Concealment

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

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

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

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

Institute

Kyowa Kirin Co., Ltd.

Institute

Department

Personal name

Organization

None

Organization

Division

Category of Funding Organization

Self funding

Nationality of Funding Organization

Co-sponsor

Name of secondary funder(s)

Organization

-

Address

-

Tel

-

Email

-

Secondary IDs

NO

Study ID_1

Org. issuing International ID_1

Study ID_2

Org. issuing International ID_2

IND to MHLW

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）

Date of disclosure of the study information

2017

Year

11

Month

13

Day

URL releasing protocol

Publication of results

Unpublished

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

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 related information

Registered date

2017

Year

11

Month

10

Day

Last modified on

2022

Year

04

Month

07

Day

Link to view the page

Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000034172