Lamotrigine as Treatment of Myotonia

NCT01939561 | Completed Phase 3 DMC

• 2 other identifiers: 2013-MY2013-003309-24
• Study Type: interventional
• Target: 27
• Locations: 1 country
• Sites: 1

Brief Summary

Myotonia is a functional limiting symptom where the muscle stiffens on action leading to arrest of movement. Pharmacological treatment may make the difference between a physically restricted and a normal life. Today, patients with myotonia are treated with Mexiletine a medications resulting in adverse events up to 40 % and which very expensive and difficult to obtain. Our clinic has, forced by the above problems related to Mexiletine, treated a few patients with the drug Lamotrigine with pronounced positive effect in all. Lamotrigine belongs to the same category of drugs as Mexiletine but has fewer and milder side effects. Based on the similarities of the 2 drugs in pharmacological action and the positive experiences investigators are convinced that Lamotrigine will show a positive effect if evaluated in a broader scale. Due to the advantages of Lamotrigine compared to Mexiletine investigators find it of outmost importance for patients that this drug is assessed formally to establish Lamotrigine as a treatment choice for myotonia. Investigators believe that this will potentially make a huge difference in life quality for persons with myotonia. Investigators aim at investigating the efficacy and tolerability of Lamotrigine in the treatment of myotonia in a randomized doublet blinded placebo controlled crossover study.

Conditions

Dystrophia Myotonica Type 1; Myotonia Congenita; Paramyotonia Congenita; Hyperkalemic Periodic Paralysis; Potassium-Aggravated Myotonia

Keywords

Myotonia; Lamotrigine; RCT; treatment

Outcome Measures

Primary Outcomes (1)

• change from baseline in Myotonia Behavior Scale (MBS)

  Self evaluated Myotonia at the verified scale MBS. Participant evaluate myotonia for 4-7 days.

  8 weeks

Secondary Outcomes (3)

• Change from baseline in evaluation of Myotonia

  8 weeks

• average in use of escape medicine

  8 weeks

• change from baseline in the SF-36 questionnaire

  8 weeks

Other Outcomes (2)

• Lamotrigine blood concentration

  8 weeks

• change in creatin kinase level from baseline

  8 weeks

Study Arms (2)

Lamotrigine

ACTIVE COMPARATOR

Participants are taken oral tablets Lamotrigine once daily. The dosis is escalating every other weeks, from 25 mg - 50 mg - 150 mg- 300mg during the period of 8 weeks.

Drug: Lamotrigine

Placebo

PLACEBO COMPARATOR

Participants are taken oral tablets placebo once daily. The dosis is escalating every other weeks, from 25 mg - 50 mg - 150 mg- 300 mg during the period of 8 weeks.

Drug: Placebo

Interventions

Lamotrigine DRUG

Also known as: ATC-code: N03AX09

Lamotrigine

Placebo DRUG

Placebo is tablets identically with the Lamotrigine tablets.

Placebo

Eligibility Criteria

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

You may qualify if:

• Clinical myotonia: Myotonia affecting patients daily life, such as chewing function, handshake, initiation of walking and running, or dropping objects. Patients in antimyotonic treatment.
• Gen-verified diagnosis: Myotonia Congenita, Paramyotonia Congenita, Potassium-aggravated Myotonia or Dystrophia Myotonica type 1.

You may not qualify if:

• In treatment with medicines affecting the study results, estimated by investigators.
• Participated in other drug-trials within 30 days prior to study start.
• Known intolerance or allergy to Lamotrigine.
• Significant renal or liver function, epilepsy, or long QT interval on the ECG.
• Pregnancy and breast-feeding.
• After the investigators discretion

Sponsors & Collaborators

• Grete Andersen, MD: lead

Study Sites (1)

Copenhagen Neuromuscular Center, department of Neurology, Rigshospitalet

Copenhagen, DK-2100, Denmark

Location

MeSH Terms

Conditions

Myotonic Dystrophy; Myotonia Congenita; Myotonic Disorders; Paralysis, Hyperkalemic Periodic; Potassium aggravated myotonia; Myotonia

Interventions

Lamotrigine

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Nervous System Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Paralyses, Familial Periodic; Metal Metabolism, Inborn Errors; Metabolism, Inborn Errors; Metabolic Diseases; Nutritional and Metabolic Diseases; Neuromuscular Manifestations; Neurologic Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Triazines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Study Officials

• Grete Andersen, MD

  Copenhagen Neuromuscular Center, Rigshospitalet, Denmark, Europe

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 3
• 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: MD

Study Record Dates

• First Submitted: August 23, 2013
• First Posted: September 11, 2013
• Study Start: November 1, 2013
• Primary Completion: November 1, 2015
• Study Completion: November 1, 2015
• Last Updated: April 25, 2016

Record last verified: 2016-04

Locations

DK (1)