An observational study in Multiple Sclerosis, sponsored by EMD Serono. Completed at 18 sites in 8 countries. Open to participants aged Up to 18 Years. Per ClinicalTrials.gov, last updated 2015-05-15.
Sponsored by EMD Serono · Observational
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.
3,460 studies on the registry are indexed under Multiple Sclerosis; 661 are open to participants now.
This study's enrollment of 307 is above the median of 100 across 1,016 observational studies indexed under Multiple Sclerosis.
Browse Multiple Sclerosis studies →EMD Serono is the lead sponsor of 88 studies on the registry; none are open to participants now.
Counted across the registry records on this site, refreshed daily.
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.
Exclusion Criteria:
No exclusion criteria are applied
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)
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.
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.
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.
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.
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.
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.
Annualized Medically Confirmed Clinical Relapses Rate Prior to Rebif® Initiation and During Rebif® Treatment
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 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.
Time to First Medically Confirmed Clinical Relapse Post-Rebif® Initiation
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.
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.
| Milestone | Retrospective Cohort |
|---|---|
| Started | 307 |
| Completed | 307 |
| Not completed | 0 |
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.
| Participants | Retrospective Cohort |
|---|---|
| Injections site reactions | 85 |
| Flu-like symptoms | 75 |
| Hepatic disorders | 44 |
| Blood cell disorders | 14 |
| Allergic reactions | 5 |
| Epilepsy and convulsive disorders | 5 |
| Thyroid dysfunction | 3 |
| Autoimmune diseases | 2 |
| Bone/epiphyseal and cartilage disorders | 2 |
| Serious infections | 2 |
| Malignancies | 1 |
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.
| Participants | Retrospective Cohort |
|---|---|
| Serious medical events | 12 |
| Non-serious medical events | 184 |
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.
| Participants | Retrospective Cohort |
|---|---|
| Alanine Aminotransferase (n=195) | 74 |
| Aspartate Aminotransferase (n=194) | 59 |
| Basophils (n=190) | 4 |
| Eosinophils (n=190) | 13 |
| Erythrocytes (n=193) | 28 |
| Hematocrit (n=196) | 38 |
| Hemoglobin (n=196) | 33 |
| Leukocytes (n=200) | 45 |
| Lymphocytes (n=190) | 55 |
| Monocytes (n=189) | 18 |
| Neutrophils (n=192) | 46 |
| Platelet (n=196) | 13 |
| Thyroid-Stimulating Hormone (n=116) | 4 |
| Thyroperoxidase Antibody (n=71) | 2 |
| Thyroxine (n=99) | 3 |
| Triiodothyronine (n=80) | 4 |
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.
| Attacks per year | Retrospective Cohort |
|---|---|
| Prior to Rebif® initiation | 1.79 |
| During Rebif® treatment | 0.47 |
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.
| Months | Retrospective Cohort |
|---|---|
| Time to First Medically Confirmed Clinical Relapse Post-Rebif® Initiation | 19.5 (14.5 to 27.2) |
Collected over 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.. Non-serious events are listed at a 1% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Retrospective Cohort | — | 12/307 (3.9%) | 184/307 (59.9%) |
| Event | Retrospective Cohort |
|---|---|
| Injection site injuryGeneral disorders | 1/307 |
| Injection site necrosisGeneral disorders | 1/307 |
| IrritabilityGeneral disorders | 1/307 |
| Autoimmune hepatitisHepatobiliary disorders | 1/307 |
| CholelithiasisHepatobiliary disorders | 1/307 |
| Anaphylactic reactionImmune system disorders | 1/307 |
| HypersensitivityImmune system disorders | 1/307 |
| CellulitisInfections and infestations | 1/307 |
| Injection site cellulitisInfections and infestations | 1/307 |
| ConvulsionNervous system disorders | 1/307 |
| Event | Retrospective Cohort |
|---|---|
| Influenza like illnessGeneral disorders | 75/307 |
| Injection site erythemaGeneral disorders | 48/307 |
| Injection site painGeneral disorders | 34/307 |
| HeadacheNervous system disorders | 19/307 |
| Alanine aminotransferase increasedInvestigations | 13/307 |
| PyrexiaGeneral disorders | 9/307 |
| Hepatic enzyme increasedInvestigations | 9/307 |
| Liver function test abnormalInvestigations | 8/307 |
| Aspartate aminotransferase increasedInvestigations | 7/307 |
| LeukopeniaBlood and lymphatic system disorders | 7/307 |
| Age, Continuous(years) | Retrospective Cohort |
|---|---|
| Mean | 14.0 ± 3.0 |
| Sex: Female, Male(Participants) | Retrospective Cohort |
|---|---|
| Female | 190 |
| Male | 117 |
This study is completed, as verified in Apr 2015. You cannot join it, but the record below documents what was studied.
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