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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
EMD Serono
ClinicalTrials.gov Identifier:
NCT01207648
First received: September 21, 2010
Last updated: August 9, 2013
Last verified: August 2013
  Purpose

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.


Condition Intervention
Multiple Sclerosis
Drug: Rebif®

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Retrospective
Official Title: Retrospective Cohort Study of Rebif® Use in Pediatric MS Patients

Resource links provided by NLM:


Further study details as provided by EMD Serono:

Primary Outcome Measures:
  • Number of Participants With Pre-specified Medical Events [ Time Frame: 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. ] [ Designated as safety issue: Yes ]
    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.

  • Number of Participants With Serious Medical Events, and Non-serious Medical Events (Reported by the Investigator as Related to Rebif®) [ Time Frame: 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. ] [ Designated as safety issue: Yes ]
    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.

  • Number of Participants With Abnormal Laboratory Parameters [ Time Frame: 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. ] [ Designated as safety issue: Yes ]
    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.


Secondary Outcome Measures:
  • Annualized Medically Confirmed Clinical Relapses Rate Prior to Rebif® Initiation and During Rebif® Treatment [ Time Frame: 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. ] [ Designated as safety issue: No ]
    Medically confirmed clinical relapses were defined as the emergence of new neurological symptoms that occurred more than 30 days after a previous attack and persisted for more than or equal to 24 hours in the absence of known inter-current illness. Annualized relapse rate was defined as number of attacks per year.

  • Time to First Medically Confirmed Clinical Relapse Post-Rebif® Initiation [ Time Frame: 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. ] [ Designated as safety issue: No ]
    Medically confirmed clinical relapses were defined as the emergence of new neurological symptoms that occurred more than 30 days after a previous attack and persisted for more than or equal to 24 hours in the absence of known inter-current illness.


Enrollment: 307
Study Start Date: July 2010
Study Completion Date: July 2011
Primary Completion Date: July 2011 (Final data collection date for primary outcome measure)
Groups/Cohorts Assigned Interventions
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®
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)

  Eligibility

Ages Eligible for Study:   up to 18 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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.

Criteria

Inclusion Criteria:

  • 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

Exclusion Criteria:

No exclusion criteria are applied

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01207648

Locations
United States, Alabama
Research Site
Birmingham, Alabama, United States
United States, California
Research Site
San Francisco, California, United States
United States, Massachusetts
Research Site
Boston, Massachusetts, United States
United States, New York
Research Site
Buffalo, New York, United States
Research Site
Rochester, New York, United States
Research Site
Stoney Brook, New York, United States
Argentina
Research Site
Buenos Aires, Argentina
Canada
Research Site
Toronto, Canada
France
Research Site
Le Kremlin Bicêtre Cedex, France
Italy
Research Site
Bari, Italy
Research Site
Catania, Italy
Research Site
Gallarate, Italy
Research Site
Milan, Italy
Research Site
Rome, Italy
Research Site
Torino, Italy
Russian Federation
Research Site
Moscow, Russian Federation
Tunisia
Research Site
Tunis, Tunisia
Venezuela
Research Site
Maracaibo, Venezuela
Sponsors and Collaborators
EMD Serono
Investigators
Study Director: Medical Responsible EMD Serono Inc., a subsidiary of Merck KGaA, Darmstadt, Germany
  More Information

No publications provided

Responsible Party: EMD Serono
ClinicalTrials.gov Identifier: NCT01207648     History of Changes
Other Study ID Numbers: EMR 200136-024
Study First Received: September 21, 2010
Results First Received: April 8, 2013
Last Updated: August 9, 2013
Health Authority: Argentina: Human Research Bioethics Committee
Canada: Ethics Review Committee
France: French Data Protection Authority and National Consultative Ethics Committee for Health and Life Sciences
Italy: Ethics Committee
Russia: Ethics Committee
Tunisia: Ethics Committee
United States: Institutional Review Board
Venezeula: Ethics Committee

Keywords provided by EMD Serono:
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

Additional relevant MeSH terms:
Multiple Sclerosis
Sclerosis
Demyelinating Autoimmune Diseases, CNS
Autoimmune Diseases of the Nervous System
Nervous System Diseases
Demyelinating Diseases
Autoimmune Diseases
Immune System Diseases
Pathologic Processes
Interferon beta 1a
Physiological Effects of Drugs
Adjuvants, Immunologic
Immunologic Factors
Pharmacologic Actions
Antineoplastic Agents
Therapeutic Uses

ClinicalTrials.gov processed this record on July 22, 2014