HTLV-1 Associated MyelopathyPhase 3NCT03191526
A Study of KW-0761 in Subjects With HTLV-1 Associated Myelopathy (HAM)
A Phase 3 study testing KW-0761 0.3 mg/kg IV in people with HTLV-1 Associated Myelopathy, run by Kyowa Kirin Co., Ltd. at 16 sites in Japan.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 20 years and older
- Conditions the study is for
- HTLV-1 Associated Myelopathy
- What is being tested?
- KW-0761 0.3 mg/kg IV
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 66 participants
- Where
- 16 sitesJapan
- Study dates
- 22 May 2017to 31 Aug 2021
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 20 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Voluntary written informed consent to participate in the study
- Diagnosis as HAM according to the second edition of HAM Treatment Manual
- At least 1-year history of HAM
- 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-α, salazosulfapyridine, or ≥ 1.5 g/day vitamin C
- 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
- A OMDS of ≥3 at screening and able to walk ≥10 m at screening (use of a single cane or double canes is allowed)
Who cannot join
- 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
- Active infection
- 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
- Concurrent dementia
- 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
Show all 9Show fewer
- History of or current alcohol or drug dependence
- Planned surgery during the study period
- Any other conditions unsuitable for participation in the study in the opinion of the investigator or subinvestigator
Trial locations and contacts
16 sites in 1 country.
16 sites in 1 country
Japan16 sites
Fukuoka University Hospital
Fukuoka, Fukuoka
Kyushu University Hospital
Fukuoka, Fukuoka
National Hospital Organization Okinawa National Hospital
Ginowan, Okinawa
Kansai Medical University Hosipital
Hirakata, Osaka
Kagoshima City Hospital
Kagoshima, Kagoshima-ken
Kagoshima University Hospital
Kagoshima, Kagoshima-ken
St. Marianna University School of Medicine Hospital
Kawasaki, Kanagawa
Hospital of the University of Occupational and Environmental Health, Japan
Kitakyushu, Fukuoka
Kumamoto University Hospital
Kumamoto, Kumamoto
University Hosipital, Kyoto Prefectural University of Medicine
Kyoto
Fujimoto General Hospital
Miyakonojō, Miyazaki
Nagoya University Hosipital
Nagoya, Aichi-ken
University of the Ryukyus Hospital
Nakagami, Okinawa
Oita Prefectural Hospital
Ōita, Oita Prefecture
Tohoku University Hosipital
Sendai, Miyagi
Ehime University Hospital
Tone, Ehime
About this study
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)
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.
Read the detailed description
The effects of KW-0761 (0.3 mg/kg) on the Osame's motor disability score (OMDS) of subjects with HTLV-1 associated myelopathy (HAM).
- Conditions
- HTLV-1 Associated Myelopathy
- Being tested
- KW-0761 0.3 mg/kg IV, Placebo (saline)
- Medical subject headings
- Paraparesis, Tropical Spastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
KW-0761 0.3 mg/kg IVDrug
Intravenous injection every 12 weeks.
Placebo (saline)Drug
Intravenous injection every 12 weeks.
| Study group | Role | What they receive |
|---|---|---|
| KW-0761 0.3 mg/kg IV | Experimental | Intravenous injection every 12 weeks. Duration of double-blind treatment is going to be for 24 weeks and be followed by transitional period, which is for maximal 4 weeks. After that, duration of open label treatment is going to be conducted for 24 weeks. And an extension treatment will be continued until the approval or termination. Receives: Drug: KW-0761 0.3 mg/kg IV |
| Placebo (saline) | Placebo comparator | Intravenous injection every 12 weeks. Duration of double-blind treatment is going to be for 24 weeks and be followed by transitional period, which is for maximal 4 weeks. After that, duration of open label treatment is going to be conducted for 24 weeks. And an extension treatment will be continued until the approval or termination. Receives: Drug: Placebo (saline) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Improvement in Osame's motor disability score
Measured: At week 4, 8 and 12 after second injection
Secondary outcomes (10)
HTLV-1 Proviral load in peripheral blood
Measured: Pre-dose, 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 weeks post-dose
Mean of twice 10 m walking time
Measured: Pre-dose, 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 weeks post-dose
Modified Ashworth Scale
Measured: Pre-dose, 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 weeks post-dose
Evaluation of Clinical Global Impression (CGI-I)
Measured: Pre-dose, 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 weeks post-dose
Evaluation of Clinical Global Impression (VAS)
Measured: Pre-dose, 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 weeks post-dose
Evaluation of Urinary dysfunction (OABSS)
Measured: Pre-dose, 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 weeks post-dose
Evaluation of Urinary dysfunction (I-PSS)
Measured: Pre-dose, 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 weeks post-dose
Evaluation of sensory dysfunction (numbness in the lower limbs (VAS))
Measured: Pre-dose, 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 weeks post-dose
Evaluation of sensory dysfunction (Pain in the lower limbs (VAS))
Measured: Pre-dose, 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 weeks post-dose
Neopterine Concentration in CSF
Measured: At week 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Jun 2017
- Study start22 May 2017
- Primary completion6 Jun 2019
- Study completion31 Aug 2021
Why it stopped: Due to not achieving the primary outcome.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for HTLV-1 Associated MyelopathyStudies of the same condition, open and under way.
- More trials run by Kyowa Kirin Co., Ltd.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 66 participants (actual)
Sponsor and investigators
- Lead sponsor
- Kyowa Kirin Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03191526
- Sponsor's protocol ID
- 0761HAM-001
- Status last verified
- Apr 2022
- First submitted
- 13 Jun 2017
- Last updated
- 14 Apr 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Sato T, Nagai M, Watanabe O, Misu T, Takenouchi N, Ohkubo R, Ishihara S, Tsuboi Y, Katsuno M, Nakagawa M, Matsushita T, Aso Y, Matsuura E, Tokashiki T, Mukaino A, Adachi H, Nakanishi K, Yamaguchi Y, Yamaguchi S, Yamano Y. Multicenter, randomized, double-blind, placebo-controlled phase 3 study of mogamulizumab with open-label extension study in a minimum number of patients with human T-cell leukemia virus type-1-associated myelopathy. J Neurol. 2024 Jun;271(6):3471-3485. doi: 10.1007/s00415-024-12239-x. Epub 2024 Mar 2.
PubMed 38430272
Study at a glance
From the registry record, last updated by the sponsor on 14 Apr 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT03191526
- Acronym / sponsor ID
- 0761HAM-001
- Conditions
- HTLV-1 Associated Myelopathy
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Due to not achieving the primary outcome.
- Enrolment
- 66 participants (actual)
- Lead sponsor
- Kyowa Kirin Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 22 May 2017 (actual)
- Primary completion
- 6 Jun 2019 (actual)
- Study completion
- 31 Aug 2021 (actual)
- First posted
- 19 Jun 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
The effects of KW-0761 (0.3 mg/kg) on the Osame's motor disability score (OMDS) of subjects with HTLV-1 associated myelopathy (HAM).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| KW-0761 0.3 mg/kg IV | Experimental | Drug: KW-0761 0.3 mg/kg IVIntravenous injection every 12 weeks. Duration of double-blind treatment is going to be for 24 weeks and be followed by transitional period, which is for maximal 4 weeks. After that, duration of open label treatment is going to be conducted for 24 weeks. And an extension treatment will be continued until the approval or termination. |
| Placebo (saline) | Placebo comparator | Drug: Placebo (saline)Intravenous injection every 12 weeks. Duration of double-blind treatment is going to be for 24 weeks and be followed by transitional period, which is for maximal 4 weeks. After that, duration of open label treatment is going to be conducted for 24 weeks. And an extension treatment will be continued until the approval or termination. |
Who may qualify
- Ages
- 20 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Voluntary written informed consent to participate in the study
- Diagnosis as HAM according to the second edition of HAM Treatment Manual
- At least 1-year history of HAM
- 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.
- 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
Key exclusion criteria (9 listed)
- 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
- Active infection
- 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
- Concurrent dementia
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| KW-0761 0.3 mg/kg IVIntravenous injection every 12 weeks. | Drug | KW-0761 0.3 mg/kg IV |
| Placebo (saline)Intravenous injection every 12 weeks. | Drug | Placebo (saline) |
Primary outcome
Improvement in Osame's motor disability score
Time frame: At week 4, 8 and 12 after second injection
10 secondary outcomes
HTLV-1 Proviral load in peripheral blood
Time frame: Pre-dose, 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 weeks post-dose
Mean of twice 10 m walking time
Time frame: Pre-dose, 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 weeks post-dose
Modified Ashworth Scale
Time frame: Pre-dose, 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 weeks post-dose
Evaluation of Clinical Global Impression (CGI-I)
Time frame: Pre-dose, 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 weeks post-dose
Evaluation of Clinical Global Impression (VAS)
Time frame: Pre-dose, 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 weeks post-dose
Evaluation of Urinary dysfunction (OABSS)
Time frame: Pre-dose, 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 weeks post-dose
Evaluation of Urinary dysfunction (I-PSS)
Time frame: Pre-dose, 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 weeks post-dose
Evaluation of sensory dysfunction (numbness in the lower limbs (VAS))
Time frame: Pre-dose, 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 weeks post-dose
Evaluation of sensory dysfunction (Pain in the lower limbs (VAS))
Time frame: Pre-dose, 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 weeks post-dose
Neopterine Concentration in CSF
Time frame: At week 12
Results and safety
The study was stopped early (reason given: Due to not achieving the primary outcome.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Apr 2022.
- Derived from this study: PubMed 38430272Sato T, Nagai M, Watanabe O, Misu T, Takenouchi N, Ohkubo R, Ishihara S, Tsuboi Y, Katsuno M, Nakagawa M, Matsushita T, Aso Y, Matsuura E, Tokashiki T, Mukaino A, Adachi H, Nakanishi K, Yamaguchi Y, Yamaguchi S, Yamano Y. Multicenter, randomized, double-blind, placebo-controlled phase 3 study of mogamulizumab with open-label extension study in a minimum number of patients with human T-cell leukemia virus type-1-associated myelopathy. J Neurol. 2024 Jun;271(6):3471-3485. doi: 10.1007/s00415-024-12239-x. Epub 2024 Mar 2.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
16 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing KW-0761 0.3 mg/kg IV in people with HTLV-1 Associated Myelopathy, run by Kyowa Kirin Co., Ltd. at 16 sites in Japan.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 14 Apr 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 16 sites in Japan. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Due to not achieving the primary outcome.). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Improvement in Osame's motor disability score (time frame: At week 4, 8 and 12 after second injection). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03191526, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Apr 2022.