Intravenous Immunoglobulin for Unverricht-Lundborg Disease.

NCT03351569 | Unknown Phase 3 DMC

• 2 other identifiers: 2002002017-002147-15
• Study Type: interventional
• Target: 1
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Unverricht-Lundborg Disease

Keywords

Unverricht-Lundborg disease; progressive myoclonus epilepsy; pharmacoresistant epilepsy

Outcome Measures

Primary Outcomes (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.

  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 Outcomes (2)

• Unified Myoclonus Rating Scale (UMRS) overall score improvement.

  monthly for one year

• Patient's preference

  one year

Study Arms (2)

Immunoglobulin

EXPERIMENTAL

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

Drug: Intravenous immunoglobulin

Saline solution

PLACEBO COMPARATOR

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

Drug: Intravenous immunoglobulin

Interventions

Intravenous immunoglobulin DRUG

Intravenous drip.

Also known as: Venital

Immunoglobulin; Saline solution

Eligibility Criteria

• Age: 18 Years - 25 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• Malattia di Unverricht-Lundborg (genetic diagnosis)

You may not qualify if:

• Contraindications to intravenous immunoglobulin

Sponsors & Collaborators

• Azienda Socio Sanitaria Territoriale di Mantova: lead

Related Publications (2)

• 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.

  PMID: 21695041 BACKGROUND

• 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.

  PMID: 2384914 BACKGROUND

MeSH Terms

Conditions

Unverricht-Lundborg Syndrome; Myoclonic Epilepsies, Progressive

Interventions

Immunoglobulins, Intravenous

Condition Hierarchy (Ancestors)

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; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Immunoglobulin G; Immunoglobulin Isotypes; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: 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.
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: MD

Model Details: sigle patient trial

Study Record Dates

• First Submitted: October 27, 2017
• First Posted: November 24, 2017
• Study Start: December 9, 2015
• Primary Completion: December 6, 2016
• Study Completion: December 30, 2017
• Last Updated: November 27, 2017

Record last verified: 2017-11

Data Sharing

• IPD Sharing: Will share