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Efficacy and Safety of Tamibarotene(AM80H) for HTLV-1 Associated Myelopathy/ Tropical Spastic Paraparesis (HAM/TSP)

Primary Purpose

HTLV-I-Associated Myelopathy

Status

Unknown status

Phase

Phase 2

Locations

Japan

Study Type

Interventional

Intervention

Tamibarotene

About this trial

This is an interventional treatment trial for HTLV-I-Associated Myelopathy focused on measuring Tropical Spastic Paraparesis, HAM, TSP

Eligibility Criteria

30 Years - 75 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Patients who have been diagnosed as HAM according to the WHO criteria
Patients who are positive for HTLV-I antibody in the spinal fluid
Patients, if female, who are not pregnant or breastfeeding, either agreed to take contraceptive measures during and two years after the treatment, or sterile
Patients, if male, who agreed to take contraceptive measures during and six months after the treatment
Patients who have been informed and understood the contents of the study and consented to participate in the signed form.

Exclusion Criteria:

Patients who has a rapid progress in the symptoms defined as an increase of two or more in Osame's Motor Disability Score for HAM patients in the past one year.
Patients of hyperlipidemia (serum triglyceride higher than 400 mg/dL)
Patients who were administered new or increased dose of corticosteroid in the past 8 weeks before the intervention
Patients who received steroid pulse therapy in the past 8 weeks before the intervention
Patients who were administered new or increased dose of immunosuppressant in the past 8 weeks before the intervention
Patients with a history of serious drug allergy
Patients with significant complication such as malignancy, severe heart failure, and other serious diseases.
Patients who were in the past administered etretinate.

Sites / Locations

Iseikai Medical Corporation, Shoyo Kashiwadai Hospital

Outcomes

Primary Outcome Measures

Change in Soluble IL-2 Receptor level in peripheral blood

Change in HTLV-I viral load in peripheral blood

Change in T cell population in peripheral blood

Change in cerebrospinal fluid examination

Secondary Outcome Measures

Change in Osame's Motor Disability Score for HAM patients

Change in The Expanded Disability Status Scale (EDSS)

Change in timed 10m walk

Change in Manual Muscle Testing and vibratory perception of the lower limbs

Change in Modified Ashworth Scale

Change in Urination function and defecation score

Full Information

NCT ID

NCT01343355

First Posted

April 25, 2011

Last Updated

July 21, 2011

Sponsor

St. Marianna University School of Medicine

1. Study Identification

Unique Protocol Identification Number

NCT01343355

Brief Title

Efficacy and Safety of Tamibarotene(AM80H) for HTLV-1 Associated Myelopathy/ Tropical Spastic Paraparesis (HAM/TSP)

Official Title

Open-Label, Exploratory Study of the Efficacy and Safety of Tamibarotene(AM80H) for HTLV-1 Associated Myelopathy/ Tropical Spastic Paraparesis (HAM/TSP)

Study Type

Interventional

2. Study Status

Record Verification Date

July 2011

Overall Recruitment Status

Unknown status

Study Start Date

January 2011 (undefined)

Primary Completion Date

March 2012 (Anticipated)

Study Completion Date

March 2012 (Anticipated)

3. Sponsor/Collaborators

Name of the Sponsor

St. Marianna University School of Medicine

4. Oversight

Data Monitoring Committee

5. Study Description

Brief Summary

An open-label, non-randomised, uncontrolled, proof-of-concept study of patients with HTLV-I-associated myelopathy/Tropical Spastic Paraparesis (HAM/TSP). Participants will receive oral administration of tamibarotene in the amount of 2 mg daily over a period of 12 weeks, then 4mg daily for another 12 weeks. The patients will be followed up for further 8 weeks. Efficacy will be monitored by measuring clinical scores including motor and urination function, HTLV-1 proviral load, immunological parameters, and markers in the spinal fluid. Safety will be evaluated at the same time.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

HTLV-I-Associated Myelopathy

Keywords

Tropical Spastic Paraparesis, HAM, TSP

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2, Phase 3

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

15 (Anticipated)

8. Arms, Groups, and Interventions

Intervention Type

Drug

Intervention Name(s)

Tamibarotene

Intervention Description

Oral administration of tamibarotene 2 mg daily over a period of 12 weeks, then 4mg daily for another 12 weeks.

Primary Outcome Measure Information:

Title

Change in Soluble IL-2 Receptor level in peripheral blood

Time Frame

0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks

Title

Change in HTLV-I viral load in peripheral blood

Time Frame

0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks

Title

Change in T cell population in peripheral blood

Time Frame

0,12, 24, 28 and 32 weeks

Title

Change in cerebrospinal fluid examination

Time Frame

baseline and after the treatment defined as from 24 to 32 weeks

Secondary Outcome Measure Information:

Title

Change in Osame's Motor Disability Score for HAM patients

Time Frame

0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks

Title

Change in The Expanded Disability Status Scale (EDSS)

Time Frame

0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks

Title

Change in timed 10m walk

Time Frame

0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks

Title

Change in Manual Muscle Testing and vibratory perception of the lower limbs

Time Frame

0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks

Title

Change in Modified Ashworth Scale

Time Frame

0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks

Title

Change in Urination function and defecation score

Time Frame

0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks

10. Eligibility

Sex

All

Minimum Age & Unit of Time

30 Years

Maximum Age & Unit of Time

75 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Patients who have been diagnosed as HAM according to the WHO criteria Patients who are positive for HTLV-I antibody in the spinal fluid Patients, if female, who are not pregnant or breastfeeding, either agreed to take contraceptive measures during and two years after the treatment, or sterile Patients, if male, who agreed to take contraceptive measures during and six months after the treatment Patients who have been informed and understood the contents of the study and consented to participate in the signed form. Exclusion Criteria: Patients who has a rapid progress in the symptoms defined as an increase of two or more in Osame's Motor Disability Score for HAM patients in the past one year. Patients of hyperlipidemia (serum triglyceride higher than 400 mg/dL) Patients who were administered new or increased dose of corticosteroid in the past 8 weeks before the intervention Patients who received steroid pulse therapy in the past 8 weeks before the intervention Patients who were administered new or increased dose of immunosuppressant in the past 8 weeks before the intervention Patients with a history of serious drug allergy Patients with significant complication such as malignancy, severe heart failure, and other serious diseases. Patients who were in the past administered etretinate.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Yoshihisa Yamano, MD

Organizational Affiliation

St. Marianna University School of Medicine

Official's Role

Principal Investigator

Facility Information:

Facility Name

Iseikai Medical Corporation, Shoyo Kashiwadai Hospital

City

Kanagawa

ZIP/Postal Code

243-0402

Country

Japan

12. IPD Sharing Statement

Links:

URL

http://www.htlv1joho.org/

Description

HTLV-1 Information for Japanese

Learn more about this trial

Efficacy and Safety of Tamibarotene(AM80H) for HTLV-1 Associated Myelopathy/ Tropical Spastic Paraparesis (HAM/TSP)

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