Intravenous Immunoglobulin for Unverricht-Lundborg Disease.

Phase 3

• Conditions: Unverricht-Lundborg Disease
• Interventions: Drug: Intravenous immunoglobulin

• Registration Number: NCT03351569
• Lead Sponsor: Azienda Socio Sanitaria Territoriale di Mantova

Brief 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.

Study Timeline

• Study Start: 2015-12-09
• Primary Completion: 2016-12-06
• Study First Submitted: 2017-10-27
• Status Verified: 2017-11
• Study First Submitted QC: 2017-11-21
• Last Update Submitted: 2017-11-22
• Study First Posted: 2017-11-24
• Last Update Posted: 2017-11-27
• Study Completion: 2017-12-30

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: Male
• Target Recruitment: 1

Inclusion Criteria

• Malattia di Unverricht-Lundborg (genetic diagnosis)

Exclusion Criteria

• Contraindications to intravenous immunoglobulin

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: CROSSOVER

Arm && Interventions

Group	Intervention	Description
Saline solution	Intravenous immunoglobulin	Intravenous saline solution 500 ml (five 100 ml bottles), in 3 hours, once a month for one year.
Immunoglobulin	Intravenous immunoglobulin	Intravenous immunoglobulin 25 grams (five 100 ml bottles, 5g/100ml), in 3 hours, once a month for one year.

Primary Outcome Measures

Name	Time	Method
Improvement of at least 20% of the action myoclonus at one year, measured with section 4 (Action Myoclonus) of the Unified Myoclonus Rating Scale.	monthly for one year	The range for Action Myoclonus Score is 0 (best) - 160 (worst, , i.e. more severe involuntary movements). Percent change = 100 X (Placebo UMRS4 - Treatment UMRS4) / Placebo UMRS4).

Secondary Outcome Measures

Name	Time	Method
Unified Myoclonus Rating Scale (UMRS) overall score improvement.	monthly for one year	The total value of the UMRS (range from 0 - best - to 365 - worst) is composed of the sum of 6 sections: (1) Patient Questionnaire (range 0-48), (2) Myoclonus at rest (range 0-108), (3) Stimulus Sensitivity (range 0-17), (4) Myoclonus with Action (range 0-160), (5) Functional Tests (0-28), (6) Global Disability Score (range 0-4).
Patient's preference	one year	The program was to discuss the patient's analysis data with the patient himself and to let him decide in three possible ways: (1) to continue the trial, (2) to continue treatment with immunoglobulins, (3) to suspend the treatment.The choice number 2 is considered a favorable outcome.