Intravenous Immunoglobulin for Unverricht-Lundborg Disease.

Sponsor

Azienda Socio Sanitaria Territoriale di Mantova (Other)

Overall Status

Unknown status

CT.gov ID

NCT03351569

Collaborator

(none)

Enrollment

Arms

24.7

Anticipated Duration (Months)

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Unverricht-Lundborg Disease	Drug: Intravenous immunoglobulin	Phase 3

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 Design

Study Type:

Interventional

Actual Enrollment :

1 participants

Allocation:

Randomized

Intervention Model:

Crossover Assignment

Intervention Model Description:

sigle patient trialsigle patient trial

Masking:

Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Masking Description:

Drug and placebo had the same appearance and are wrapped in foil paper before being shown to the patient. Outcome evaluation is carried out by personnel not involved in the treatment at a distant site, one month after treatment.

Primary Purpose:

Treatment

Official Title:

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

Actual Study Start Date :

Dec 9, 2015

Actual Primary Completion Date :

Dec 6, 2016

Anticipated Study Completion Date :

Dec 30, 2017

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Immunoglobulin Intravenous immunoglobulin 25 grams (five 100 ml bottles, 5g/100ml), in 3 hours, once a month for one year.	Drug: Intravenous immunoglobulin Intravenous drip. Other Names: Venital
Placebo Comparator: Saline solution Intravenous saline solution 500 ml (five 100 ml bottles), in 3 hours, once a month for one year.	Drug: Intravenous immunoglobulin Intravenous drip. Other Names: Venital

Arm

Intervention/Treatment

Experimental: Immunoglobulin

Intravenous immunoglobulin 25 grams (five 100 ml bottles, 5g/100ml), in 3 hours, once a month for one year.

Drug: Intravenous immunoglobulin

Intravenous drip.

Other Names:

Venital

Placebo Comparator: Saline solution

Intravenous saline solution 500 ml (five 100 ml bottles), in 3 hours, once a month for one year.

Drug: Intravenous immunoglobulin

Intravenous drip.

Other Names:

Venital

Outcome Measures

Primary Outcome Measures

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

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.