UMIN-CTR Clinical Trial

Basic information
Public title	Phase IIb clinical trial of steroid therapy in patients with HAM
Acronym	HAMLET-P
Scientific Title	Phase IIb clinical trial of steroid therapy in patients with HAM
Scientific Title:Acronym	HAMLET-P
Region	Japan

Condition
Condition	HTLV-1-Associated-Myelopathy Tropical Spastic Paraparesis (HAM/TSP)
Classification by specialty	Neurology
Classification by malignancy	Others
Genomic information	NO

Objectives
Narrative objectives1	To evaluate the effectiveness of the Methylprednisolone therapy to the Prednisolone oral treatment in rapid progressive patients with HAM/TSP. To evaluate the safety of the Methylprednisolone therapy and the Prednisolone therapy in rapid progressive patients with HAM/TSP. To evaluate the effectiveness of the Prednisolone therapy in rapid progressive patients with HAM/TSP.
Basic objectives2	Safety,Efficacy
Basic objectives -Others
Trial characteristics_1	Confirmatory
Trial characteristics_2
Developmental phase	Phase II,III

Assessment
Primary outcomes	Efficacy:Presence or absence of 30% or more improvement in a walking time of 10-meter walk test or one grade or more improvement in OMDS at 15 days compared with baseline level.
Key secondary outcomes	Efficacy: -A walking time of 10-meter walk test. 1. Presence or absence of 30% or more improvement in a walking time of 10-meter walk test. 2. An area under the curve from at 15 days and 29 days (4 weeks) compared with baseline level. 3. A variation at 15 days and 29 days (4 weeks) compared with baseline level. -A walking distance for 2 or 6 minutes. 1. An area under the curve from at 15 days and 29 days (4 weeks) compared with baseline level. 2. A variation at 15 days and 29 days (4 weeks) compared with baseline level. -OMDS. Presence or absence of one grade or more improvement in OMDS at 15 days compared with baseline level. -A neopterin concentration in cerebrospinal fluid. A variation at 15 days compared with baseline -A ratio of subjects treated the Methylprednisolone pulse therapy from 29 days (4weeks) to 169 days (24 weeks).

Base
Study type	Interventional

Study design
Basic design	Parallel
Randomization	Randomized
Randomization unit	Individual
Blinding	Open -but assessor(s) are blinded
Control	Dose comparison
Stratification	NO
Dynamic allocation	NO
Institution consideration	Institution is not considered as adjustment factor.
Blocking	YES
Concealment	Central registration

Intervention
No. of arms	2
Purpose of intervention	Treatment
Type of intervention	Medicine
Interventions/Control_1	Following to administrate Methylprednisolone 1000mg/day intravenously slowly for consecutive three days, Prednisolone is administered orally for a maximum dose in 0.5mg/kg/day, then decreased gradually and terminated. There is a case to maintain the dose of Prednisolone by the degree of a progress of underlying disease.
Interventions/Control_2	Prednisolone is administered orally for a maximum dose in 0.5mg/kg/day, then decreased gradually and terminated. There is a case to maintain the dose of Prednisolone by the degree of a progress of underlying disease.
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	Not applicable
Gender	Male and Female
Key inclusion criteria	(1) The patient who has diagnosed with HAM based on the Belem criterion. (2) The patient over 18 years of age at the day of giving informed consent. (3) The patient who can walk more than 10 meters regardless of the necessity of walking aids at the day of giving informed consent. However, the use of the walk aid is allowed it less than two sticks. The use of the walker is possible. (4) The patient that the main organ function is maintained. (To confirm by the most recent inspection data within 28 days of enrollment.) 1. Neutrophils: more than 1500/mm3 2. PLT: more than 100,000/mm3 3. Hb : more than 9.0 g/dL 4. AST: within 3 times of the upper limit of the institution criteria 5. ALT: within 3 times of the upper limit of the institution criteria 6. Serum Cre: within 3 times of the upper limit of the institution criteria 7. HbA1c: within 6.5 percent (5) The patient or the legally acceptable representative gives of his/her own free will consent to participate this study. (6) The patient that can come to the hospital for a treatment and a follow-up along the schedule of study.
Key exclusion criteria	(1)Patients who have received corticosteroids or other treatment targeted to HAM within 12 weeks prior to giving informed consent (3)Patients who have undergone invasive surgeries requiring general anesthesia within 24 weeks prior to giving informed consent (4)Patients who have participated in other treatment studies within 16 weeks prior to giving informed consent (5)Patients who have undergone live or attenuated/inactivated vaccinations within four weeks of giving informed consent, or who plan to be vaccinated during the course of the study. (6)Patients taking the ascorbic acid more than 1.5g/day, prosultiamine, or pentosan polysulfate within two weeks of giving informed consent. (7)Patients with a history of acute myocardial infarction (8)Patients with a history of tuberculosis or with active tuberculosis (9)Patients with serious complicating conditions (10)Patients with uncontrolled hypertension (11)Patients with uncontrolled electrolyte imbalance (12)Patients with thrombosis (13)Patients with a history of cancer with complications (14)Patients with peptic ulcer (15)Patients with ATL (16)Patients with poorly controlled eye disease (17)Patients with a history of steroid-induced glaucoma (18)Pregnant or breastfeeding women or patients who may become pregnant or withhold assent to prevention of conception by taking appropriate approach such as condom in cooperation with their partners during the study period (19)Patients in whom assessment with the walk test is difficult or the symptoms can be worsened by the walk test (20)Patients with neurological deficits or findings on MRI suggesting it due to disorders other than HAM
Target sample size	8

Research contact person
Name of lead principal investigator	1st name Middle name Last name Yoshihisa Yamano M.D.,Ph.D.
Organization	St. Marianna University School of Medicine Hospital
Division name	Department of Neurology
Zip code
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 Ushitani,Kuwahara
Organization	HAMLET-P Coordinating Center
Division name	Clinical Research Data Center,St. Marianna University School of Medicine
Zip code
Address	2-16-1 Sugao, Miyamae-ku, Kawasaki, Kanagawa, 216-8511 Japan
TEL	044-977-8111(6191)
Homepage URL
Email	mariadc_ham@marianna-u.ac.jp

Sponsor
Institute	St. Marianna University School of Medicine Hospital
Institute
Department

Funding Source
Organization	The Research on Measures for Intractable Diseases Project of Japan Agency for Medical Research and Development
Organization
Division
Category of Funding Organization	Japanese Governmental office
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	2016/08/03

Institutions
Institutions	聖マリアンナ医科大学病院（神奈川県）

Other administrative information
Date of disclosure of the study information	2016 Year 08 Month 31 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
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	2016 Year 07 Month 04 Day
Date of IRB	2016 Year 07 Month 14 Day
Anticipated trial start date	2016 Year 08 Month 31 Day
Last follow-up date	2020 Year 03 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	2016 Year 08 Month 28 Day
Last modified on	2022 Year 03 Month 04 Day

Link to view the page
URL(English)	https://center6.umin.ac.jp/cgi-open-bin/icdr_e/ctr_view.cgi?recptno=R000027409