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

NCT ID: NCT01343355

Last Updated: 2011-07-22

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: PHASE2/PHASE3
• Total Enrollment: 15 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2011-01-31
• Study Completion Date: 2012-03-31

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.

Conditions

HTLV-I-Associated Myelopathy

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Interventions

Tamibarotene

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

Intervention Type: DRUG

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.

• Minimum Eligible Age: 30 Years
• Maximum Eligible Age: 75 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

St. Marianna University School of Medicine

OTHER

Sponsor Role: lead

Responsible Party

Department of Molecular Medical Science, Institute of Medical Science

Principal Investigators

Yoshihisa Yamano, MD

Role: PRINCIPAL_INVESTIGATOR

St. Marianna University School of Medicine

Locations

Iseikai Medical Corporation, Shoyo Kashiwadai Hospital

Kanagawa, , Japan

Countries

Japan

Other Identifiers

AM80H-01

Identifier Type: -

Identifier Source: org_study_id