Multiple SclerosisREPLAYNCT01207648
Retrospective Cohort Study of Rebif® Use in Pediatric Multiple Sclerosis (MS) Subjects (REPLAY)
An observational study of Multiple Sclerosis, run by EMD Serono at 18 sites in 8 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages Up to 18 years
- Conditions the study is for
- Multiple Sclerosis
- What is being tested?
- Rebif®
- How many people
- 307 participants
- Where
- 18 sitesin 8 countries
- Study dates
- Jul 2010to Jul 2011
- Phase
- Observational
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
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.
Who may be able to join
- 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
Who cannot join
- No exclusion criteria are applied
Trial locations and contacts
18 sites in 8 countries.
18 sites in 8 countries
United States6 sites
Research Site
Birmingham, Alabama
Research Site
Boston, Massachusetts
Research Site
Buffalo, New York
Research Site
Rochester, New York
Research Site
San Francisco, California
Research Site
Stoney Brook, New York
Italy6 sites
Research Site
Bari
Research Site
Catania
Research Site
Gallarate
Research Site
Milan
Research Site
Rome
Research Site
Torino
About this study
Official title: Retrospective Cohort Study of Rebif® Use in Pediatric MS Patients
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
- Being tested
- Rebif®
- 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
- Medical subject headings
- Multiple Sclerosis, Autoimmune Diseases, Demyelinating Diseases, Immune System Diseases, Nervous System Diseases, Demyelinating Autoimmune Diseases, CNS, Autoimmune Diseases of the Nervous System
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
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)
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Rebif® |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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.
Measured: 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
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.
Measured: 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.
Measured: 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.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Sep 2010 (estimated)
- Study startJul 2010
- Primary completionJul 2011
- Study completionJul 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Multiple SclerosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 307 participants (actual)
Sponsor and investigators
- Lead sponsor
- EMD Serono
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Responsible, Study director, EMD Serono Inc., a subsidiary of Merck KGaA, Darmstadt, Germany
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01207648
- Sponsor's protocol ID
- EMR 200136-024
- Acronym
- REPLAY
- Status last verified
- Apr 2015
- First submitted
- 21 Sep 2010
- Last updated
- 15 May 2015
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 May 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Retrospective Cohort Study of Rebif® Use in Pediatric MS Patients
- ClinicalTrials.gov ID
- NCT01207648
- Acronym / sponsor ID
- REPLAY / EMR 200136-024
- 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
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 307 participants (actual)
- Lead sponsor
- EMD SeronoIndustry (pharma, biotech, devices)
- Study start
- Jul 2010
- Primary completion
- Jul 2011 (actual)
- Study completion
- Jul 2011 (actual)
- First posted
- 23 Sep 2010 (estimated)
- Results
- Posted 30 May 2013
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Retrospective Cohort | – | Drug: Rebif®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. |
Who may qualify
- Ages
- Up to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- 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.
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (1 listed)
- No exclusion criteria are applied
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 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) | Drug | Retrospective Cohort |
Primary outcome
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.
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.
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.
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.
2 secondary outcomes
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.
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.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 May 2015.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Responsible study director
EMD Serono Inc., a subsidiary of Merck KGaA, Darmstadt, Germany
18 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Multiple Sclerosis, run by EMD Serono at 18 sites in 8 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 15 May 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 18 sites in United States, Italy, Argentina, Canada, France and 3 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Yes. Results are posted in the registry (first posted 30 May 2013). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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.). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 May 2015.