Spinal and Bulbar Muscular Atrophy
Conditions
Brief summary
The purpose of this survey is to evaluate the long-term safety and efficacy of leuprorelin acetate injection kit 11.25 mg in patients with spinal and bulbar muscular atrophy (SBMA) in the routine clinical setting.
Detailed description
The drug being tested in this survey is called leuprorelin acetate injection kit 11.25 mg. This injection kit is being tested to treat people who have SBMA. This survey is an observational (non-interventional) study and will look at the long-term safety and efficacy of the leuprorelin acetate injection kit 11.25 mg in the routine clinical setting. The planned number of observed patients will be approximately 300. This multi-center observational trial will be conducted in Japan.
Interventions
DRUGLeuprorelin Acetate
Leuprorelin Acetate Injection Kit
Sponsors
Takeda
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
* All SBMA patients who have been confirmed as receiving the drug
Exclusion criteria
* None
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants who had One or More Adverse Drug Reactions | Up to 1 Year | Adverse drug reaction refers to adverse events related to administered drug. Percentage of participants who have the adverse drug reactions that occurred between initiation of treatment with the drug and 1 year after the start of treatment with the drug (or 3 months after the last dose of the drug if the treatment was discontinued within the first four doses) will be reported. |
| Percentage of Participants who had One or More Serious Adverse Events | Up to 1 Year | Percentage of participants who have the serious adverse events that occurred between initiation of treatment with the drug and 1 year after the start of treatment with the drug (or 3 months after the last dose of the drug if the treatment was discontinued within the first four doses) will be reported. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants without Death Event | At final assessment point (up to 8 years) | Percentage of participants without events of death at final assessment point (up to 8 years from initiation of treatment) will be reported. |
| Percentage of Participants without Pneumonia Requiring Hospitalization Event | At final assessment point (up to 8 years) | Percentage of participants without events of pneumonia requiring hospitalization at final assessment point (up to 8 years from initiation of treatment) will be reported. |
| Percentage of Participants without Composite Events of Death and Pneumonia Requiring Hospitalization | At final assessment point (up to 8 years) | Percentage of participants without composite events of death and pneumonia requiring hospitalization at final assessment point (up to 8 years from initiation of treatment) will be reported. |
| Percentage of Participants without Dysphagia Events | At final assessment point (up to 8 years) | Percentage of participants without events of dysphagia at final assessment point (up to 8 years from initiation of treatment) will be reported. |
Countries
Japan
Contacts
STUDY_DIRECTORStudy Director
Takeda
Outcome results
None listed