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

Sponsor

St. Marianna University School of Medicine (Other)

Overall Status

Unknown status

CT.gov ID

NCT01343355

Collaborator

(none)

Enrollment

Location

Duration (Months)

1.1

Patients Per Site Per Month

Study Details

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.

Condition or Disease	Intervention/Treatment	Phase
HTLV-I-Associated Myelopathy	Drug: Tamibarotene	Phase 2/Phase 3

Study Design

Study Type:

Interventional

Anticipated Enrollment :

15 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

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 Start Date :

Jan 1, 2011

Anticipated Primary Completion Date :

Mar 1, 2012

Anticipated Study Completion Date :

Mar 1, 2012

Outcome Measures

Primary Outcome Measures

Change in Soluble IL-2 Receptor level in peripheral blood [0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks]
Change in HTLV-I viral load in peripheral blood [0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks]
Change in T cell population in peripheral blood [0,12, 24, 28 and 32 weeks]
Change in cerebrospinal fluid examination [baseline and after the treatment defined as from 24 to 32 weeks]

Secondary Outcome Measures

Change in Osame's Motor Disability Score for HAM patients [0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks]
Change in The Expanded Disability Status Scale (EDSS) [0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks]
Change in timed 10m walk [0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks]
Change in Manual Muscle Testing and vibratory perception of the lower limbs [0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks]
Change in Modified Ashworth Scale [0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks]
Change in Urination function and defecation score [0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks]

Eligibility Criteria

Criteria

Ages Eligible for Study:

30 Years to 75 Years

Sexes Eligible for Study:

All

Accepts 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.