Intravenous Immunoglobulin for Unverricht-Lundborg Disease.

Overview

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 Title of Study: “Intravenous Immunoglobulin for Unverricht-Lundborg Disease: Single-patient Trial.”

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: Randomized
    • Intervention Model: Crossover Assignment
    • Primary Purpose: Treatment
    • Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
  • Study Primary Completion Date: December 6, 2016

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.

Interventions

  • Drug: Intravenous immunoglobulin
    • Intravenous drip.

Arms, Groups and Cohorts

  • Experimental: Immunoglobulin
    • Intravenous immunoglobulin 25 grams (five 100 ml bottles, 5g/100ml), in 3 hours, once a month for one year.
  • Placebo Comparator: Saline solution
    • Intravenous saline solution 500 ml (five 100 ml bottles), in 3 hours, once a month for one year.

Clinical Trial Outcome Measures

Primary 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.
    • 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 Measures

  • 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).
  • 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.

Participating in This Clinical Trial

Inclusion Criteria

  • Malattia di Unverricht-Lundborg (genetic diagnosis) Exclusion Criteria:

  • Contraindications to intravenous immunoglobulin

Gender Eligibility: Male

Minimum Age: 18 Years

Maximum Age: 25 Years

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Azienda Socio Sanitaria Territoriale di Mantova
  • Provider of Information About this Clinical Study
    • Principal Investigator: Alfonso Ciccone, MD – Azienda Socio Sanitaria Territoriale di Mantova

References

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.

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.

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