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

Study Description

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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

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Study Type :	Interventional (Clinical Trial)
Estimated Enrollment :	15 participants
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 :	January 2011
Estimated Primary Completion Date :	March 2012
Estimated Study Completion Date :	March 2012

Arms and Interventions

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Intervention Details:

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

Outcome Measures

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Primary Outcome Measures :

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

Secondary Outcome Measures :

1. Change in Osame's Motor Disability Score for HAM patients [ Time Frame: 0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks ]
2. Change in The Expanded Disability Status Scale (EDSS) [ Time Frame: 0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks ]
3. Change in timed 10m walk [ Time Frame: 0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks ]
4. 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 ]
5. Change in Modified Ashworth Scale [ Time Frame: 0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks ]
6. Change in Urination function and defecation score [ Time Frame: 0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks ]

Eligibility Criteria

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Ages Eligible for Study:	30 Years to 75 Years (Adult, Senior)
Sexes Eligible for Study:	All
Accepts Healthy Volunteers:	No

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.

Contacts and Locations

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Locations

Japan
Iseikai Medical Corporation, Shoyo Kashiwadai Hospital
Kanagawa, Japan, 243-0402

Sponsors and Collaborators

St. Marianna University School of Medicine

Investigators

Principal Investigator:	Yoshihisa Yamano, MD	St. Marianna University School of Medicine

More Information

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Additional Information:

HTLV-1 Information for Japanese 

Responsible Party:	Yoshihisa Yamano, MD, associate professor, Department of Molecular Medical Science, Institute of Medical Science
ClinicalTrials.gov Identifier:	NCT01343355 History of Changes
Other Study ID Numbers:	AM80H-01
First Posted:	April 28, 2011
Last Update Posted:	July 22, 2011
Last Verified:	July 2011

Keywords provided by St. Marianna University School of Medicine:

Tropical Spastic Paraparesis; HAM; TSP

Additional relevant MeSH terms:

Muscle Spasticity; Spinal Cord Diseases; Bone Marrow Diseases; Paraparesis; Paraparesis, Spastic; Paraparesis, Tropical Spastic; Muscular Diseases; Musculoskeletal Diseases; Muscle Hypertonia; Neuromuscular Manifestations; Neurologic Manifestations; Nervous System Diseases	Signs and Symptoms; Central Nervous System Diseases; Hematologic Diseases; Paresis; HTLV-I Infections; Deltaretrovirus Infections; Retroviridae Infections; RNA Virus Infections; Virus Diseases; Myelitis; Central Nervous System Infections