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An Observational Study in Adult Patients With Non-dystrophic Myotonic Disorders

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04616807
Recruitment Status : Recruiting
First Posted : November 5, 2020
Last Update Posted : February 26, 2021
Sponsor:
Information provided by (Responsible Party):
Lupin Ltd.

Study Description
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Brief Summary:
This is a non-interventional, prospective, observational, multicentre study to evaluate the long-term safety and effectiveness of Namuscla in adult patients with NDM.

Condition or disease Intervention/treatment
Myotonic Dystrophy Drug: Mexiletine

Detailed Description:

This is a non-interventional, prospective, observational, multicentre study to evaluate the long-term safety and effectiveness of Namuscla in adult patients with NDM. Namuscla should be prescribed as per the approved Summary of Product Characteristics (SmPC).

Adult patients with non-dystrophic myotonic disorders who have been prescribed Namuscla by the treating physician, and who meet the eligibility criteria will be enrolled in this study.

This includes:

  • Patients newly initiated on Namuscla for the treatment of NDM (newly exposed)
  • Patients already on Namuscla/ mexiletine at enrolment - For patients receiving mexiletine other than Namuscla, only those who switch to Namuscla will be included in the study.

Patients already being treated with Namuscla/ mexiletine at the time of enrolment will be considered for enrolment provided they meet the eligibility criteria.

The study will be initiated at specialized centres for the treatment of myotonic disorders ("reference centres") in the United Kingdom (UK), France, and Germany, depending on availability of Namuscla in the specific country. Depending on the enrolment and marketing status (availability) of Namuscla in other countries in the EU, inclusion of additional sites in other countries will be considered.

The study population will comprise patients who are diagnosed with non-dystrophic myotonic disorders and considered suitable candidates for the treatment by Namuscla by the investigators according to the approved SmPC. Patients will be enrolled over an approximate 2-year enrolment period and will be followed-up on-treatment for up to 3 years. Each enrolled patient will be observed for 3 years or until discontinuation (if discontinued early).

For all enrolled patients, the baseline would be the latest data available at the enrolment visit.

For the patients already on Namuscla, cumulative data (data related previous exposure as well as current data) will be collected for adverse events (AEs) on Namuscla treatment.

No drug will be supplied for this study; patients will receive medicines through local standard practices. All evaluations and investigations during the study will be performed according to the routine clinical practices and discretion of the treating physician.

Study Design
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Study Type : Observational
Estimated Enrollment : 50 participants
Observational Model: Other
Time Perspective: Prospective
Official Title: An Observational Study to Describe the Long-term Safety and Effectiveness of Namuscla in the Symptomatic Management of Myotonia in Adult Patients With Non-dystrophic Myotonic Disorders
Actual Study Start Date : December 17, 2020
Estimated Primary Completion Date : November 19, 2025
Estimated Study Completion Date : February 24, 2026

Resource links provided by the National Library of Medicine


Groups and Cohorts
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Intervention Details:
  • Drug: Mexiletine
    Observational Study
    Other Name: Namuscla ™

Outcome Measures
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Primary Outcome Measures :
  1. Primary Outcome 1 Proportion of patients with treatment-emergent AEs [ Time Frame: Approximately 3 years ]
    Proportion of patients with treatment-emergent AEs ([TEAEs], including SAEs) from study enrolment to 6, 12, 24 and 36 months on Namuscla

  2. Primary Outcome 2 Proportion of patients requiring dose reduction or treatment discontinuation [ Time Frame: Approximately 3 years ]
    Proportion of patients requiring dose reduction or treatment discontinuation due to AEs (including SAEs).


Secondary Outcome Measures :
  1. Secondary Outcome Proportion of patients with AEs /SAEs/ Adverse Event of Special Interest (AESI) [ Time Frame: Approximately 3 years ]
    Proportion of patients with AEs /SAEs/ Adverse Event of Special Interest (AESI) from study enrolment to 6, 12, 24, and 36 months


Eligibility Criteria
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Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Sampling Method:   Probability Sample
Study Population
The study will target the enrolment of 50 patients who are treated with Namuscla.
Criteria

Inclusion Criteria:

  1. Adult, male or female patients with non-dystrophic myotonic disorders planned to be started on Namuscla according to the approved SmPC
  2. Patients already receiving Namuscla/mexiletine for the treatment of NDM; (for patients on mexiletine other than Namuscla, only those who switch to Namuscla will be enrolled).
  3. Patients who understand and are willing to provide informed consent.

Exclusion Criteria:

  1. Patients who are enrolled or participating in any other clinical trial for an investigational product. -
  2. Hypersensitivity to mexiletine, or to any of the excipients of Namuscla, or hypersensitivity to any local anaesthetic
  3. Ventricular tachyarrhythmia
  4. Atrial tachyarrhythmia, fibrillation or flutter
  5. Complete heart block (ie, third-degree atrioventricular block) or any heart block susceptible to evolve to complete heart block (first-degree atrioventricular block with markedly prolonged PR interval (≥ 240 ms) and/or wide QRS complex (≥ 120 ms), second-degree atrioventricular block, bundle branch block, bifascicular and trifascicular block),
  6. Myocardial infarction (acute or past), or abnormal Q-waves
  7. Symptomatic coronary artery disease
  8. Heart failure with reduced ejection fraction <50%
  9. Sinus node dysfunction (including sinus rate < 50 bpm)
  10. Patients receiving drugs that can induce torsades de pointes
  11. Patients receiving medicinal products with narrow therapeutic index (ie, theophylline, tizanidine, digoxin, lithium, phenytoin or warfarin)
  12. Patients who are pregnant or lactating.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04616807


Contacts
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Contact: Nikki Adetoro 443-447-4534 NikkiAdetoro@lupin.com

Locations
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France
Hôpital Universitaire de La Pitié Salpêtrière Recruiting
Paris, Cedex, France, 13 75013
Contact: Savine Vicart, MD    33 (0) 1 42 16 16 91    savine.vicart@aphp.fr   
Principal Investigator: Savine Vicart, MD         
CHRU Lille Recruiting
Lille, France, 59000
Contact: Celine Tard, MD    33 (0) 3 20 44 59 62    CELINE.TARD@CHRU-LILLE.FR   
Principal Investigator: Celine Tard, MD         
Germany
St. Josef-Hospital Klinikum der Ruhr Universitaet Bochum Not yet recruiting
Bochum, North-Rhine Westphalia, Germany, 44791
Contact: Christiane Schneider-Gold, MD    +492345096439    Christiane.Schneider-Gold@ruhr-uni-bochum.de   
Principal Investigator: Christiane Schneider-Gold, MD         
Universitätsklinikum Ulm, Klinik für Neurologie Not yet recruiting
Ulm, Germany, 89081
Contact: Angela Rosenbohm, MD    0049 (0)731 177 5261    angela.rosenbohm@uni-ulm.de   
Principal Investigator: Angela Rosenbohm, MD         
United Kingdom
Institute of Neurology Not yet recruiting
London, England, United Kingdom, WC1N 3BG
Contact: Michael Hanna, MD       m.hanna@ucl.ac.uk   
Principal Investigator: Michael Hanna, MD         
Nottingham University Hospitals NHS Trust Not yet recruiting
Nottingham, England, United Kingdom, NG7 2UH
Contact: Saam Sedehizadeh, MD    0115 924 9924    Saam.Sedehizadeh@nuh.nhs.uk   
Principal Investigator: Saam Sedehizadeh, MD         
Sponsors and Collaborators
Lupin Ltd.
More Information
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Responsible Party: Lupin Ltd.
ClinicalTrials.gov Identifier: NCT04616807    
Other Study ID Numbers: LUP/MEX/2018/001
First Posted: November 5, 2020    Key Record Dates
Last Update Posted: February 26, 2021
Last Verified: February 2021

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Myotonic Dystrophy
Myotonic Disorders
Muscular Dystrophies
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Nervous System Diseases
 Neuromuscular Diseases
Genetic Diseases, Inborn
Mexiletine
Anti-Arrhythmia Agents
Voltage-Gated Sodium Channel Blockers
Sodium Channel Blockers
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action