Specified Drug-Use Survey of Leuprorelin Acetate Injection Kit 11.25 mg "All-Case Investigation: Spinal and Bulbar Muscular Atrophy (SBMA)"

NCT03555578 | Completed

• 2 other identifiers: Leuprorelin-5004jRCT1080223924
• Study Type: observational
• Target: 1,905
• Locations: 1 country
• Sites: 1

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.

Conditions

Spinal and Bulbar Muscular Atrophy

Outcome Measures

Primary Outcomes (2)

• Percentage of Participants who had One or More Adverse Drug Reactions

  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.

  Up to 1 Year

• Percentage of Participants who had One or More Serious Adverse Events

  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.

  Up to 1 Year

Secondary Outcomes (4)

• Percentage of Participants without Death Event

  At final assessment point (up to 8 years)

• Percentage of Participants without Pneumonia Requiring Hospitalization Event

  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)

• Percentage of Participants without Dysphagia Events

  At final assessment point (up to 8 years)

Study Arms (1)

Leuprorelin Acetate 11.25 mg

Leuprorelin Acetate Injection Kit 11.25 mg, every 12 weeks subcutaneously, for up to at most 8 years. Participants received interventions as part of routine medical care.

Drug: Leuprorelin Acetate

Interventions

Leuprorelin Acetate DRUG

Leuprorelin Acetate Injection Kit

Also known as: Leuplin SR for Injection Kit 11.25 mg

Leuprorelin Acetate 11.25 mg

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

SBMA patients treated with Leuprorelin Acetate Injection Kit 11.25 mg as part of routine medical care

You may qualify if:

• All SBMA patients who have been confirmed as receiving the drug

You may not qualify if:

• None

Sponsors & Collaborators

• Takeda: lead

Study Sites (1)

Takeda Selected Site

Tokyo, Japan

Location

Related Links

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MeSH Terms

Conditions

Bulbo-Spinal Atrophy, X-Linked

Interventions

Leuprolide

Condition Hierarchy (Ancestors)

Muscular Atrophy, Spinal; Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Motor Neuron Disease; Neuromuscular Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Gonadotropin-Releasing Hormone; Pituitary Hormone-Releasing Hormones; Hypothalamic Hormones; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Neuropeptides; Peptides; Amino Acids, Peptides, and Proteins; Oligopeptides; Nerve Tissue Proteins; Proteins

Study Officials

• Study Director

  Takeda

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 1, 2018
• First Posted: June 13, 2018
• Study Start: November 2, 2017
• Primary Completion: April 30, 2026
• Study Completion: April 30, 2026
• Last Updated: May 22, 2026

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• Access Criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.

Locations

JP (1)