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Intravenous Immunoglobulin for Unverricht-Lundborg Disease.

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ClinicalTrials.gov Identifier: NCT03351569
Recruitment Status : Unknown
Verified November 2017 by Alfonso Ciccone, Azienda Socio Sanitaria Territoriale di Mantova.
Recruitment status was:  Active, not recruiting
First Posted : November 24, 2017
Last Update Posted : November 27, 2017
Sponsor:
Information provided by (Responsible Party):
Alfonso Ciccone, 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.

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 1 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Intervention Model Description: sigle 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 : December 9, 2015
Actual Primary Completion Date : December 6, 2016
Estimated Study Completion Date : December 30, 2017


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 Name: 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 Name: Venital




Primary Outcome Measures :
  1. Improvement of at least 20% of the action myoclonus at one year, measured with section 4 (Action Myoclonus) of the Unified Myoclonus Rating Scale. [ Time Frame: 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 :
  1. Unified Myoclonus Rating Scale (UMRS) overall score improvement. [ Time Frame: 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).

  2. Patient's preference [ Time Frame: 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.



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Ages Eligible for Study:   18 Years to 25 Years   (Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Malattia di Unverricht-Lundborg (genetic diagnosis)

Exclusion Criteria:

  • Contraindications to intravenous immunoglobulin

Publications:
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Responsible Party: Alfonso Ciccone, MD, Azienda Socio Sanitaria Territoriale di Mantova
ClinicalTrials.gov Identifier: NCT03351569    
Other Study ID Numbers: 200200
2017-002147-15 ( EudraCT Number )
First Posted: November 24, 2017    Key Record Dates
Last Update Posted: November 27, 2017
Last Verified: November 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: We planned to publish the results of this single patient trial.

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Alfonso Ciccone, Azienda Socio Sanitaria Territoriale di Mantova:
Unverricht-Lundborg disease
progressive myoclonus epilepsy
pharmacoresistant epilepsy
Additional relevant MeSH terms:
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Unverricht-Lundborg Syndrome
Myoclonic Epilepsies, Progressive
Epilepsies, Myoclonic
Epilepsy, Generalized
Epilepsy
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Epileptic Syndromes
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Immunoglobulins
Antibodies
Immunoglobulins, Intravenous
gamma-Globulins
Rho(D) Immune Globulin
Immunologic Factors
Physiological Effects of Drugs