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Intravenous Immunoglobulin for Unverricht-Lundborg Disease.

NCT ID: NCT03351569

Last Updated: 2017-11-27

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Clinical Phase

PHASE3

Total Enrollment

1 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-12-09

Study Completion Date

2017-12-30

Brief Summary

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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

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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

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Unverricht-Lundborg Disease

Keywords

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Unverricht-Lundborg disease progressive myoclonus epilepsy pharmacoresistant epilepsy

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

CROSSOVER

sigle patient trial
Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors
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.

Study Groups

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Immunoglobulin

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

Group Type EXPERIMENTAL

Intravenous immunoglobulin

Intervention Type DRUG

Intravenous drip.

Saline solution

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

Group Type PLACEBO_COMPARATOR

Intravenous immunoglobulin

Intervention Type DRUG

Intravenous drip.

Interventions

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Intravenous immunoglobulin

Intravenous drip.

Intervention Type DRUG

Other Intervention Names

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Venital

Eligibility Criteria

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Inclusion Criteria

* Malattia di Unverricht-Lundborg (genetic diagnosis)

Exclusion Criteria

* Contraindications to intravenous immunoglobulin
Minimum Eligible Age

18 Years

Maximum Eligible Age

25 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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Azienda Socio Sanitaria Territoriale di Mantova

OTHER

Sponsor Role lead

Responsible Party

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Alfonso Ciccone

MD

Responsibility Role PRINCIPAL_INVESTIGATOR

References

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Lillie EO, Patay B, Diamant J, Issell B, Topol EJ, Schork NJ. The n-of-1 clinical trial: the ultimate strategy for individualizing medicine? Per Med. 2011 Mar;8(2):161-173. doi: 10.2217/pme.11.7.

Reference Type BACKGROUND
PMID: 21695041 (View on PubMed)

Guatelli JC, Gingeras TR, Richman DD. Alternative splice acceptor utilization during human immunodeficiency virus type 1 infection of cultured cells. J Virol. 1990 Sep;64(9):4093-8. doi: 10.1128/JVI.64.9.4093-4098.1990.

Reference Type BACKGROUND
PMID: 2384914 (View on PubMed)

Other Identifiers

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2017-002147-15

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

200200

Identifier Type: -

Identifier Source: org_study_id