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

HTLV-I-Associated Myelopathy Clinical Trial

Official title:

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT01343355
• Other study ID #: AM80H-01
• Status: Active, not recruiting
• Phase: Phase 2/Phase 3
• First received: April 25, 2011
• Last updated: July 21, 2011
• Start date: January 2011
• Est. completion date: March 2012

Study information

• Verified date: July 2011
• Source: St. Marianna University School of Medicine
• Contact: n/a
• Is FDA regulated: No
• Health authority: Japan: Pharmaceuticals and Medical Devices Agency
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 15
• Est. completion date: March 2012
• Est. primary completion date: March 2012
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 30 Years to 75 Years

Eligibility

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.

Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Bone Marrow Diseases
• HTLV-I-Associated Myelopathy
• Paraparesis
• Paraparesis, Spastic

Intervention

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

Locations

Country	Name	City	State
Japan	Iseikai Medical Corporation, Shoyo Kashiwadai Hospital	Kanagawa

Sponsors (1)

Lead Sponsor	Collaborator
St. Marianna University School of Medicine

Country where clinical trial is conducted

Japan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in Soluble IL-2 Receptor level in peripheral blood		0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks	No
Primary	Change in HTLV-I viral load in peripheral blood		0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks	No
Primary	Change in T cell population in peripheral blood		0,12, 24, 28 and 32 weeks	No
Primary	Change in cerebrospinal fluid examination		baseline and after the treatment defined as from 24 to 32 weeks	No
Secondary	Change in Osame's Motor Disability Score for HAM patients		0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks	No
Secondary	Change in The Expanded Disability Status Scale (EDSS)		0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks	No
Secondary	Change in timed 10m walk		0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks	No
Secondary	Change in Manual Muscle Testing and vibratory perception of the lower limbs		0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks	No
Secondary	Change in Modified Ashworth Scale		0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks	No
Secondary	Change in Urination function and defecation score		0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks	No

External Links

•   HTLV-1 Information for Japanese