Intravenous Immunoglobulin for Unverricht-Lundborg Disease.

Azienda Socio Sanitaria Territoriale di Mantova

Status and phase: Unknown; Phase 3

Conditions: Unverricht-Lundborg Disease

Treatments: Drug: Intravenous immunoglobulin

Study type: Interventional

Funder types: Other

Identifiers

NCT03351569

200200

2017-002147-15 (EudraCT Number)

Details and patient eligibility

About

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

Full 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.

Enrollment: 1 patient

Sex: Male

Ages: 18 to 25 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Malattia di Unverricht-Lundborg (genetic diagnosis)

Exclusion criteria

• Contraindications to intravenous immunoglobulin

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Crossover Assignment

Masking: Quadruple Blind

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1 participants in 2 patient groups, including a placebo group

Immunoglobulin

Experimental group

Description:

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

Treatment:

Drug: Intravenous immunoglobulin

Saline solution

Placebo Comparator group

Description:

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

Treatment:

Drug: Intravenous immunoglobulin