Retrospective Cohort Study of Rebif® Use in Pediatric Multiple Sclerosis (MS) Subjects (REPLAY)

NCT01207648 | Completed Results

• 1 other identifier: EMR 200136-024
• Study Type: observational
• Target: 307
• Locations: 8 countries
• Sites: 18

Brief Summary

The aim of this retrospective study is to review and describe safety, tolerability and efficacy of Rebif® (subcutaneous interferon \[IFN\]-beta-1a) in children and adolescents, using information already recorded in medical records. The study duration is 13 July 2010 (first data collected) to 13 July 2011 (last data collected). In this study, Data of the subjects evaluated between 1997 and 2009 was observed.

Conditions

Multiple Sclerosis

Keywords

Interferon beta 1a; Autoimmune Diseases; Demyelinating Diseases; Immune System Diseases; Immunologic Factors; Nervous System Diseases; Physiological Effects of Drugs; Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Retrospective Cohort Study

Outcome Measures

Primary Outcomes (3)

• Number of Participants With Pre-specified Medical Events

  These pre-specified medical events categories were evaluated: injections site reactions, flu-like symptoms, hepatic disorders, blood cell disorders, allergic reactions, epilepsy and convulsive disorders, thyroid dysfunction, autoimmune diseases, bone/epiphyseal and cartilage disorders, serious infections, malignancies. Each category defined by group of events which best fit the medical concept either using a standard medical dictionary for regulatory activities (MedDRA) Query (SMQ) e.g., Malignancies was defined by the SMQ Malignancies (narrow scope) containing more than 1800 different preferred terms (PTs) (including procedures and lab tests) or using a customized query, e.g., Serious infections was defined by all PTs assessed as serious in System Organ Class (SOC) Infections and Infestation. Participants may be represented in more than once in a category (Participants could have reported several medicals events pertaining to a specific category) as well as in more than one category.

  Start of observation period (first medical record available on site) up to last medical record available on site or the end of the observation period (31 December 2009), whichever occurred first.

• Number of Participants With Serious Medical Events, and Non-serious Medical Events (Reported by the Investigator as Related to Rebif®)

  Medical events in the retrospective study are equivalent to adverse events in a prospective clinical study. A medical event was defined as any untoward medical occurrence in the form of signs, symptoms, abnormal laboratory findings, or diseases that emerges or worsens relative to baseline during a clinical study with an investigational medicinal product (IMP), regardless of causal relationship and even if no IMP has been administered. Serious medical event: A medical event that resulted in death; was life threatening; resulted in persistent/significant disability/incapacity; resulted in/prolonged an existing in-patient hospitalization; was a congenital anomaly/birth defect; or was a medically important condition. Participants may be represented in more than one category as participant who had experienced serious medical event may also had experienced non-serious medical event reported by the Investigator as related to Rebif®, so in that case it will be counted in both the categories.

  Start of observation period (first medical record available on site) up to last medical record available on site or the end of the observation period (31 December 2009), whichever occurred first.

• Number of Participants With Abnormal Laboratory Parameters

  Laboratory parameters assessed for abnormality were: total white blood cell count (Neutrophils, Lymphocytes, Leukocytes, Monocytes, Eosinophils and Basophils), differential hematogram (Hematocrit, Erythrocytes, Hemoglobin, and Platelet), aspartate aminotransferase (AST), alanine aminotransferase (ALT), and thyroid tests (including Triiodothyronine, Thyroxine, Thyroperoxidase Antibody and Thyroid-Stimulating Hormone). Due to the retrospective nature of the study, laboratory data should be interpreted with caution as data were not collected according to a specific time schedule and the time on study per participant was not standardized.

  Start of observation period (first medical record available on site) up to last medical record available on site or the end of the observation period (31 December 2009), whichever occurred first.

Secondary Outcomes (2)

• Annualized Medically Confirmed Clinical Relapses Rate Prior to Rebif® Initiation and During Rebif® Treatment

  Start of observation period (first medical record available on site) up to last medical record available on site or the end of the observation period (31 December 2009), whichever occurred first.

• Time to First Medically Confirmed Clinical Relapse Post-Rebif® Initiation

  Start of observation period (first medical record available on site) up to last medical record available on site or the end of the observation period (31 December 2009), whichever occurred first.

Study Arms (1)

Retrospective Cohort

Pediatric participants including both children (aged less than 12 years) and adolescents (aged 12 to less than 18 years) who were exposed to Rebif® for treatment of demyelinating events were observed in this retrospective cohort study. In this study, medical records of participants evaluated between 1997 to 2009 were reviewed. The observation period started with the first medical record available on site till last medical record available on site or the end of the observation period (31 December 2009), whichever occurred first.

Drug: Rebif®

Interventions

Rebif® DRUG

This is an retrospective cohort study in Pediatric participants including both children (aged less than 12 years) and adolescents (aged 12 to less than 18 years) who were exposed to Rebif® for treatment of demyelinating events (Dose regimen as per investigator's decision)

Retrospective Cohort

Eligibility Criteria

• Age: Up to 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

Every eligible subject at participating centers: subjects who have received one or more injections of Rebif® for treatment of a demyelinating event before the age of 18 and before the 30th June 2009.

You may qualify if:

• Received one or more injections of Rebif® for treatment of a demyelinating event
• Be younger than 18 years of age at time of Rebif® treatment initiation
• Rebif® therapy must have been initiated before June 30, 2009

Sponsors & Collaborators

• EMD Serono: lead

Study Sites (18)

Research Site

Birmingham, Alabama, United States

Location

Research Site

San Francisco, California, United States

Location

Research Site

Boston, Massachusetts, United States

Location

Research Site

Buffalo, New York, United States

Location

Research Site

Rochester, New York, United States

Location

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Stoney Brook, New York, United States

Location

Research Site

Buenos Aires, Argentina

Location

Research Site

Toronto, Canada

Location

Research Site

Le Kremlin-Bicêtre, France

Location

Research Site

Bari, Italy

Location

Research Site

Catania, Italy

Location

Research Site

Gallarate, Italy

Location

Research Site

Milan, Italy

Location

Research Site

Rome, Italy

Location

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Torino, Italy

Location

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Moscow, Russia

Location

Research Site

Tunis, Tunisia

Location

Research Site

Maracaibo, Venezuela

Location

MeSH Terms

Conditions

Multiple Sclerosis; Autoimmune Diseases; Demyelinating Diseases; Immune System Diseases; Nervous System Diseases; Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System

Interventions

Interferon beta-1a

Condition Hierarchy (Ancestors)

Leukoencephalopathies; Brain Diseases; Central Nervous System Diseases

Intervention Hierarchy (Ancestors)

Interferon-beta; Interferon Type I; Interferons; Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors

Results Point of Contact

• Title: Merck KGaA Communication Center
• Organization: Merck Serono, a division of Merck KGaA

service@merckgroup.com

+49-6151-72-5200

Study Officials

• Medical Responsible

  EMD Serono Inc., a subsidiary of Merck KGaA, Darmstadt, Germany

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 21, 2010
• First Posted: September 23, 2010
• Study Start: July 1, 2010
• Primary Completion: July 1, 2011
• Study Completion: July 1, 2011
• Last Updated: May 15, 2015
• Results First Posted: May 30, 2013

Record last verified: 2015-04

Locations

FR (1); RU (1); TU (1); AR (1); US (6); CA (1); IT (6); VE (1)