Clinical trial NCT05240612: A Clinical Pharmacological Study of MT-3921 in Subjects With HTLV-1 Associated Myelopathy (HAM)
Official title: A Clinical Pharmacological Study of MT-3921 in Subjects With Human T-cell Leukemia Virus Type 1 (HTLV-1)-Associated Myelopathy (HAM)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: HTLV-1-Associated Myelopathy (HAM)
Lead sponsor: Mitsubishi Tanabe Pharma Corporation
Sponsor class: INDUSTRY
First posted: 2022-02-15
Enrollment: 15
Sex: ALL
Minimum age (years): 20
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The purposes of this study is to assess the safety, tolerability, and pharmacokinetics of MT-3921 in subjects with Human T-cell Leukemia Virus Type 1 (HTLV-1)-Associated Myelopathy(HAM).

Subjects meeting eligibility criteria will enter the 6-month double-blind period. Subjects will be randomized in a 2:1 ratio to receive MT-3921 or placebo in a double blind manner.


Eligibility criteria: Inclusion Criteria:

Additional screening criteria check may apply for qualification:

* Subjects aged 20 years or older on the day of consent
* Subjects with a confirmed diagnosis of HAM according to the diagnostic algorithm of Practical Guideline for HAM 2019 on the day of consent
* Subjects with an Osame's motor disability score (OMDS) of ≥4 and ≤6 at Screening and on the first day of the Treatment period (predose)
* Subjects with no change in OMDS for at least 3 months before the day of consent
* Subjects with a CSF concentration neopterin of ≥6 pmol/mL at Screening
* Subjects on maintenance oral steroid therapy who have continued to receive the same dose equivalent to ≤10 mg/day of prednisolone for at least 3 months before the day of consent

Exclusion Criteria:

Additional screening criteria check may apply for qualification:

* Subjects who have a history of anaphylaxis or clinically significant allergic reactions due to administration of antibody products
* Subjects exhibiting or with a history of malignant tumor.
* Subjects with adult T-cell leukemia/lymphoma (ATL) or those with positive HTLV-1 clonality test (Southern blot) at screening and suspected of having ATL
* Subjects with spinal cord compression lesions, such as cervical spine disease, disc herniation, and ossification of the yellow ligament
* Subjects with psychiatric disorders, epileptic seizures, or dementia.
* Subjects with suicide attempts or suicidal ideation corresponding to item 4 or 5 on the Columbia Suicide Rating Scale (C-SSRS) at screening and/or on the first day of the Treatment period (predose)
* Subjects exhibiting or with a history of hepatitis B virus (HBV), hepatitis C virus (HCV), or human immunodeficiency virus (HIV) infection.
* Subjects with the novel Coronavirus disease 2019 (COVID-19)
* Subjects with severe illness
* Male or female subjects of childbearing potential who do not agree to use a contraceptive measure from the day of consent to 16 weeks after the last dose of the investigational medical product
* Female subjects who are pregnant, lactating, or may be pregnant
* Subjects who have received anti-repulsive guidance molecule (RGM) a antibody containing this investigational medical product
* Subjects who participated in other clinical studies (including clinical trials) and received drugs (including investigational medical products) or therapy within 12 weeks before the day of consent

Primary outcomes:
measure: Percentage of subjects with adverse events
timeFrame: 36 weeks
measure: Percentage of subjects with adverse reactions
timeFrame: 36 weeks
measure: Serum concentrations of MT-3921
timeFrame: PK samples will be collected at predose, 1.5 hours, 2, 4, 8, 12, 20 (predose and 1.5 hours), 24, 36 weeks post-dose.
measure: CSF concentrations of MT-3921
timeFrame: PK samples will be collected at 2, 4, 12, 24 weeks post-dose.
Secondary outcomes:
Other outcomes:
Locations:
city: Kawasaki-shi
country: Japan
facility: St. Marianna University Hospital
state: Kanagawa
Study officials:
affiliation: Mitsubishi Tanabe Pharma Corporation
name: General Manager
role: STUDY_DIRECTOR
References: