Treatment of Myotonia - Lamotrigine Versus Namuscla

NCT05639257 | Unknown DMC

• 1 other identifier: 2021-003784-94
• Study Type: interventional
• Target: 32
• Locations: 1 country
• Sites: 1

Brief Summary

In this clinical study, the aim is to investigate whether there is a difference in treatment of myotonia using two drugs. A difference there can justify the significantly higher cost when treated by Namuscla versus Lamotrigine. According to the current corona pandemic, the investigators designed an app to use for data collection in the study. The app also ensures that patients who live far from the clinic more easily can participate.

Conditions

Non-Dystrophic Myotonia

Keywords

Becker myotonia; Thomsen myotonia; Paramyotonia; Hyperkalemic periodic paralysis

Outcome Measures

Primary Outcomes (1)

• Change in Myotonia Behavior Scale (MBS)

  Each participant chose one of six statements about how myotonia affect their daily living. MBS is register once daily in a week. 1 is no symptoms of myotonia, 6 is invalidating symptoms of myotonia. Measured without treatment and in the last week of treatment.

  baseline and week 8

Secondary Outcomes (6)

• Change in Eye-myotonia

  baseline and week 8

• Change in hand-myotonia

  baseline and week 8

• Change in time-up-and-go-test (TUG)

  baseline and week 8

• Change in Individualized Neuromuscular Quality of Life Questionnaire (INQoL)

  baseline and week 8

• Days with Side effects (SE)

  through study completion, in total 123 days

Study Arms (2)

Lamotrigine

ACTIVE COMPARATOR

An escalation phase of 28 days: \- tablet Lamotrigine 25 mg once daily in 14 days followed by 50 mg once daily in 14 days. A treatment phase of 30 days: \- tablet Lamotrigine 100 mg, once daily in 10 days, twice daily in 10 days, followed by third daily in 10 days.

Drug: Lamotrigine

Namuscla

ACTIVE COMPARATOR

A placebo phase of 28 days: \- tablet placebo 25 mg once daily in 14 days followed by 50 mg once daily in 14 days. A treatment phase of 30 days: \- tablet Namuscla 167 mg, once daily in 10 days, twice daily in 10 days, followed by third daily in 10 days.

Drug: Namuscla

Interventions

Lamotrigine DRUG

Lamotrigine tablets increasing dosis to maximal 300 mg daily

Lamotrigine

Namuscla DRUG

Namuscla tablets increasing dosis to maximal 501 mg daily

Also known as: mexiletine

Namuscla

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Capable adult women and men (age ≥ 18 years).
• Diagnosed with Myotonia Congenita (Becker and Thomsen type), Paramyotonia Congenita, or Hyperkalemic periodic paralysis.
• Myotonia under treatment or which significantly limits the daily activities (MBS\> 2).

You may not qualify if:

• Allergy to lamotrigine, mexiletine, or the inactive ingredients in trial medication.
• Disease, which is affected by trial medication such as heart disease (ischemia and arrhythmia), epilepsy, and significant renal or hepatic failure.
• Treatment that, in the opinion of the project manager, can affect the study result - medication with significant interactions with trial medication.
• In case of smoking, start or cessation during the study.
• Pregnant or breastfeeding during the study period. Fertile women with a positive pregnancy test at the time of entry into the trial, or who do not use safe contraception during the project period.

Sponsors & Collaborators

• Grete Andersen, MD: lead
• GCP-Copenhagen: collaborator
• Region Capital Denmark: collaborator
• Danish Region: collaborator
• Lupin Atlantis Holdings S.A.: collaborator
• ZiteLab: collaborator

Study Sites (1)

Copenhagen Neuromuscular Center, department of Neurology, Rigshospitalet

Copenhagen, DK-2100, Denmark

RECRUITING

MeSH Terms

Conditions

Paralysis, Hyperkalemic Periodic

Interventions

Lamotrigine; Mexiletine

Condition Hierarchy (Ancestors)

Paralyses, Familial Periodic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Metal Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Triazines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Propylamines; Amines; Organic Chemicals; Phenyl Ethers; Phenols; Benzene Derivatives; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons

Central Study Contacts

Grete Andersen

CONTACT

35453545; grete.andersen.01@regionh.dk

John Vissing

CONTACT

35453545; grete.andersen.01@regionh.dk

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: October 4, 2022
• First Posted: December 6, 2022
• Study Start: December 5, 2022
• Primary Completion: April 1, 2024
• Study Completion: April 1, 2024
• Last Updated: December 6, 2022

Record last verified: 2022-12

Data Sharing

• IPD Sharing: Will not share

Locations

DK (1)