Clinical trial NCT01343355: 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
Overall status: UNKNOWN
Phase: PHASE2
PHASE3
Conditions: HTLV-I-Associated Myelopathy
Keywords: Tropical Spastic Paraparesis
HAM
TSP
Lead sponsor: St. Marianna University School of Medicine
Sponsor class: OTHER
First posted: 2011-04-28
Enrollment: 15
Sex: ALL
Minimum age (years): 30
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


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.

Primary outcomes:
measure: Change in Soluble IL-2 Receptor level in peripheral blood
timeFrame: 0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks
measure: Change in HTLV-I viral load in peripheral blood
timeFrame: 0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks
measure: Change in T cell population in peripheral blood
timeFrame: 0,12, 24, 28 and 32 weeks
measure: Change in cerebrospinal fluid examination
timeFrame: baseline and after the treatment defined as from 24 to 32 weeks
Secondary outcomes:
measure: Change in Osame's Motor Disability Score for HAM patients
timeFrame: 0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks
measure: Change in The Expanded Disability Status Scale (EDSS)
timeFrame: 0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks
measure: Change in timed 10m walk
timeFrame: 0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks
measure: Change in Manual Muscle Testing and vibratory perception of the lower limbs
timeFrame: 0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks
measure: Change in Modified Ashworth Scale
timeFrame: 0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks
measure: Change in Urination function and defecation score
timeFrame: 0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks
Other outcomes:
Locations:
city: Kanagawa
country: Japan
facility: Iseikai Medical Corporation, Shoyo Kashiwadai Hospital
Study officials:
affiliation: St. Marianna University School of Medicine
name: Yoshihisa Yamano, MD
role: PRINCIPAL_INVESTIGATOR
References: