NCT03351569

Intravenous Immunoglobulin for Unverricht-Lundborg Disease.

Intravenous Immunoglobulin for Unverricht-Lundborg Disease: Single-patient Trial.

• Status: Unknown
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 1 (actual)
• Sponsor: Azienda Socio Sanitaria Territoriale di Mantova · Academic / Other
• Sex: Male
• Age: 18 Years – 25 Years
• Healthy volunteers: Not accepted

Summary

Single patient randomized double blind trial to assess whether intravenous immunoglobulin can improve the clinical outcome of a case suffering from Unverricht-Lundborg disease.

Detailed description

Single patient randomized double blind trial to assess whether intravenous immunoglobulin can improve the clinical outcome of a case suffering from Unverricht-Lundborg disease (clinical and genetic diagnosis). The patient was randomized to be treated with intravenous immunoglobulin or placebo 1:1 (crossover) once a month for at least one year. Main objective: improvement of the action myoclonus. Secondary objectives: Improvement in the overall score and in individual sections of the Unified Myoclonus Rating Scale at one year; patient preferences based on results at the end of the trial. The first analyst was scheduled at one year from the start of the trial. The program was to discuss the patient's analysis data and to let the patient decide in three possible ways: to continue the trial, to continue treatment with immunoglobulins, to suspend the treatment. Depending on the decision, it was planned to follow the patient throughout the year after the analysis, at least for one year.

Conditions

• Unverricht-Lundborg Disease

Interventions

Type	Name	Description
DRUG	Intravenous immunoglobulin	Intravenous drip.

Timeline

• Start date: 2015-12-09
• Primary completion: 2016-12-06
• Completion: 2017-12-30
• First posted: 2017-11-24
• Last updated: 2017-11-27