Intravenous Immunoglobulin for Unverricht-Lundborg Disease.

Unverricht-Lundborg Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT03351569
• Other study ID #: 200200
• Secondary ID: 2017-002147-15
• Status: Active, not recruiting
• Phase: Phase 3
• First received: October 27, 2017
• Last updated: November 22, 2017
• Start date: December 9, 2015
• Est. completion date: December 30, 2017

Study information

• Verified date: November 2017
• Source: Azienda Socio Sanitaria Territoriale di Mantova
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 1
• Est. completion date: December 30, 2017
• Est. primary completion date: December 6, 2016
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 18 Years to 25 Years

Eligibility

Inclusion Criteria:

• Malattia di Unverricht-Lundborg (genetic diagnosis)

Exclusion Criteria:

• Contraindications to intravenous immunoglobulin

Related Conditions & MeSH terms

• Unverricht-Lundborg Disease
• Unverricht-Lundborg Syndrome

Intervention

Drug:
• Intravenous immunoglobulin
Intravenous drip.

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Azienda Socio Sanitaria Territoriale di Mantova

References & Publications (2)

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. — View Citation

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. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Improvement of at least 20% of the action myoclonus at one year, measured with section 4 (Action Myoclonus) of the Unified Myoclonus Rating Scale.	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).	monthly for one year
Secondary	Unified Myoclonus Rating Scale (UMRS) overall score improvement.	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).	monthly for one year
Secondary	Patient's preference	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.	one year