HTLV-1 Associated Myelopathy Clinical Trial
Official title:
A Phase 3 Multicenter, Randomized, Double-Blind and Placebo-Controlled Study, and Open Study of KW-0761 in Patients With HTLV-1 Associated Myelopathy (HAM)
| Verified date | April 2022 |
| Source | Kyowa Kirin Co., Ltd. |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Interventional |
The objective of this study is to assess the efficacy and safety of KW-0761 after intravenous injections in subjects with HTLV-1 associated myelopathy (HAM) in Japan.
| Status | Terminated |
| Enrollment | 66 |
| Est. completion date | August 31, 2021 |
| Est. primary completion date | June 6, 2019 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 20 Years and older |
| Eligibility | Inclusion Criteria: 1. Voluntary written informed consent to participate in the study 2. Diagnosis as HAM according to the second edition of HAM Treatment Manual 3. At least 1-year history of HAM 4. Ongoing medication*1 for HAM, with no changes in 3 months before enrollment; or inadequate response or intolerance to prior medication,*2 which must have been discontinued for at least 3 months before enrollment. Subjects on maintenance therapy with steroids must have been receiving = 10 mg/day prednisolone equivalent continuously for at least 3 months before enrollment. - 1 Steroids, salazosulfapyridine, or = 1.5 g/day vitamin C - 2 Steroids, Interferon-a, salazosulfapyridine, or = 1.5 g/day vitamin C 5. No change in the degree of motor dysfunction for at least 3 months before the date of screening, as judged by the investigator or subinvestigator 6. A OMDS of =3 at screening and able to walk =10 m at screening (use of a single cane or double canes is allowed) Exclusion Criteria: 1. Any of the following significant concomitant diseases: Type 1 diabetes mellitus, Poorly controlled type 2 diabetes mellitus (HbA1c (NGSP) > 8.5%), Congestive heart failure (Class II to IV of the New York Heart Association Functional Classification), Myocardial infarction within 1 year before enrollment, Unstable angina within 1 year before enrollment, Poorly controlled hypertension (systolic blood pressure > 150 mm Hg and diastolic blood pressure > 90 mm Hg at screening), Sever chronic lung disease requiring oxygen therapy, Multiple sclerosis or any other demyelinating disease, Epilepsy requiring treatment with antiepileptics (with the exception of epilepsy controlled by antiepileptics, with no occurrence of seizures for at least 3 years before informed consent), and Active malignancy (including ATL); or onset of malignancy or previous treatment for malignancy (with the exception of resected or surgically cured intraepithelial carcinoma of the uterine cervix, basal cell carcinoma of the skin, squamous cell carcinoma of the skin, or ductal breast carcinoma) within 5 years before informed consent 2. Active infection 3. Concurrent spinal cord compression lesion (e.g., cervical spine diseases, disk herniation, or ossification of the ligamentum flavum) , with the exception of conditions that would not affect efficacy evaluation in the study, as judged by the investigator or subinvestigator 4. Concurrent dementia 5. Concurrent psychiatric disorder, with the exception of conditions that would not affect obtaining informed consent or efficacy evaluation in the study, as judged by the investigator or subinvestigator 6. History of or current alcohol or drug dependence 7. Planned surgery during the study period 8. Any other conditions unsuitable for participation in the study in the opinion of the investigator or subinvestigator |
| Country | Name | City | State |
|---|---|---|---|
| Japan | Fukuoka University Hospital | Fukuoka | Fukuoka Prefecture |
| Japan | Kyushu University Hospital | Fukuoka | Fukuoka Prefecture |
| Japan | National Hospital Organization Okinawa National Hospital | Ginowan | Okinawa Prefecture |
| Japan | Kansai Medical University Hosipital | Hirakata | Osaka Prefecture |
| Japan | Kagoshima City Hospital | Kagoshima | Kagoshima Prefecture |
| Japan | Kagoshima University Hospital | Kagoshima | Kagoshima Prefecture |
| Japan | St. Marianna University School of Medicine Hospital | Kawasaki | Kanagawa Prefecture |
| Japan | Hospital of the University of Occupational and Environmental Health, Japan | Kitakyushu | Fukuoka |
| Japan | Kumamoto University Hospital | Kumamoto | Kumamoto Prefecture |
| Japan | University Hosipital, Kyoto Prefectural University of Medicine | Kyoto | |
| Japan | Fujimoto General Hospital | Miyakonojo | Miyazaki |
| Japan | Nagoya University Hosipital | Nagoya | Aichi |
| Japan | University of the Ryukyus Hospital | Nakagami | Okinawa Prefecture |
| Japan | Oita Prefectural Hospital | Oita | Oita Prefecture |
| Japan | Tohoku University Hosipital | Sendai | Miyagi Prefecture |
| Japan | Ehime University Hospital | Tone | Ehime |
| Lead Sponsor | Collaborator |
|---|---|
| Kyowa Kirin Co., Ltd. |
Japan,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Improvement in Osame's motor disability score | At week 4, 8 and 12 after second injection | ||
| Secondary | HTLV-1 Proviral load in peripheral blood | Pre-dose, 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 weeks post-dose | ||
| Secondary | Mean of twice 10 m walking time | Pre-dose, 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 weeks post-dose | ||
| Secondary | Modified Ashworth Scale | Pre-dose, 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 weeks post-dose | ||
| Secondary | Evaluation of Clinical Global Impression (CGI-I) | Pre-dose, 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 weeks post-dose | ||
| Secondary | Evaluation of Clinical Global Impression (VAS) | Pre-dose, 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 weeks post-dose | ||
| Secondary | Evaluation of Urinary dysfunction (OABSS) | Pre-dose, 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 weeks post-dose | ||
| Secondary | Evaluation of Urinary dysfunction (I-PSS) | Pre-dose, 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 weeks post-dose | ||
| Secondary | Evaluation of sensory dysfunction (numbness in the lower limbs (VAS)) | Pre-dose, 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 weeks post-dose | ||
| Secondary | Evaluation of sensory dysfunction (Pain in the lower limbs (VAS)) | Pre-dose, 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 weeks post-dose | ||
| Secondary | Neopterine Concentration in CSF | At week 12 |