A Study to Evaluate Efficacy, Safety, and Tolerability of Alemtuzumab in Pediatric Patients With RRMS With Disease Activity on Prior DMT

NCT ID: NCT03368664

Last Updated: 2025-11-26

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE3
• Total Enrollment: 16 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2017-10-24
• Study Completion Date: 2025-09-08

Brief Summary

Primary Objective:

To evaluate the efficacy, safety, and tolerability of alemtuzumab intravenously (IV) in pediatric participants from 10 to less than (<) 18 years of age with Relapsing Remitting Multiple Sclerosis (RRMS) who have disease activity on prior DMT.

Secondary Objective:

To assess the pharmacokinetics (PK), pharmacodynamics (PD), anti-drug antibody (ADA) formation, and potential effects of alemtuzumab on other multiple sclerosis (MS) disease characteristics such as cognition and quality of life (QoL).

Detailed Description

The duration of study per participant will be approximately 5 years and 5 months.

Conditions

Multiple Sclerosis

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Alemtuzumab

\- alemtuzumab - Dose 1 (initial course) of alemtuzumab will be administered intravenously on 5 consecutive days, followed by Dose 2 (second course) on 3 consecutive days administered 12 months after initial course. Pre-medications (methylprednisolone, prednisolone, H1 antagonist \[antihistamine\], H2 antagonist, paracetamol, acyclovir) will be administered prior alemtuzumab administration. - Type: Experimental

Group Type: EXPERIMENTAL

Alemtuzumab GZ402673

Intervention Type: DRUG

Pharmaceutical form: solution, Route of administration: IV

Glatiramer acetate

Intervention Type: DRUG

Pharmaceutical form: solution, Route of administration: subcutaneous (SC)

Beta-Interferon

Intervention Type: DRUG

Pharmaceutical form: solution, Route of administration: SC / intramuscular (IM)

Methylprednisolone

Intervention Type: DRUG

Pharmaceutical form: solution, Route of administration: IV

Ranitidine

Intervention Type: DRUG

Pharmaceutical form: tablet, Route of administration: oral

Ceterizine

Intervention Type: DRUG

Pharmaceutical form: tablet, Route of administration: oral

Dexchlorpheniramine

Intervention Type: DRUG

Pharmaceutical form: tablet, Route of administration: oral

Paracetamol

Intervention Type: DRUG

Pharmaceutical form: tablet, Route of administration: oral

Acyclovir

Intervention Type: DRUG

Pharmaceutical form: tablet, Route of administration: oral

Prednisolone

Intervention Type: DRUG

Pharmaceutical form: tablet, Route of administration: oral

Diphenydramine

Intervention Type: DRUG

Pharmaceutical form: solution, Route of administration: IV

Other H1 antagonist

Intervention Type: DRUG

Pharmaceutical form: solution, Route of administration: IV

Other H1 antagonist

Intervention Type: DRUG

Pharmaceutical form: tablet/pill, Route of administration: oral

Interventions

Alemtuzumab GZ402673

Pharmaceutical form: solution, Route of administration: IV

Intervention Type: DRUG

Glatiramer acetate

Pharmaceutical form: solution, Route of administration: subcutaneous (SC)

Intervention Type: DRUG

Beta-Interferon

Pharmaceutical form: solution, Route of administration: SC / intramuscular (IM)

Intervention Type: DRUG

Methylprednisolone

Pharmaceutical form: solution, Route of administration: IV

Intervention Type: DRUG

Ranitidine

Pharmaceutical form: tablet, Route of administration: oral

Intervention Type: DRUG

Ceterizine

Pharmaceutical form: tablet, Route of administration: oral

Intervention Type: DRUG

Dexchlorpheniramine

Pharmaceutical form: tablet, Route of administration: oral

Intervention Type: DRUG

Paracetamol

Pharmaceutical form: tablet, Route of administration: oral

Intervention Type: DRUG

Acyclovir

Pharmaceutical form: tablet, Route of administration: oral

Intervention Type: DRUG

Prednisolone

Pharmaceutical form: tablet, Route of administration: oral

Intervention Type: DRUG

Diphenydramine

Pharmaceutical form: solution, Route of administration: IV

Intervention Type: DRUG

Other H1 antagonist

Pharmaceutical form: solution, Route of administration: IV

Intervention Type: DRUG

Other H1 antagonist

Pharmaceutical form: tablet/pill, Route of administration: oral

Intervention Type: DRUG

Other Intervention Names

Lemtrada; Copaxone

Eligibility Criteria

Inclusion Criteria

• Participants with RRMS aged from 10 years to <18 years at study entry are eligible. Participants must meet the criteria of diagnosis of MS as defined by the International Pediatric MS Study Group (IPMSSG) criteria for pediatric MS and the criteria of MS based on 2010 McDonald criteria.
• Signed written informed consent/assent obtained from participant and participant's legal representative (parent or guardian) according to local regulations.
• Expanded Disability Status Scale (EDSS) score of 0.0 to 5.0 (inclusive) at screening.
• At least 2 recorded MS attacks and at least 1 MS attack (relapse) in the last year during treatment with a beta interferon therapy (IFNB) or glatiramer acetate (GA) after being on that therapy for at least 6 months, and was currently still taking the same therapy.
• At least 1 of the following:
• >=1 new or enlarging T2 hyperintense lesion or gadolinium enhancing lesion while on that same prior therapy (IFNB or GA), or
• Two or more relapses in the prior year, or
• Tried at least 2 MS DMTs.

Exclusion Criteria

• Any progressive or non-relapsing forms of MS.
• Conditions/situations such as:
• Impossibility to meet specific protocol requirements.
• Current participation in another interventional clinical study. Participants who are treated with a comparator agent approved for screening inclusion (INF or GA) may be considered for this trial.
• Participant is the Investigator or any Sub-Investigator, research assistant, pharmacist, study coordinator, other staff or relative thereof directly involved in the conduct of the protocol.
• Uncooperative participant or any condition that could make the participant potentially non-compliant to the study procedures in the opinion of the Investigator.
• Mental condition rendering the participant or parent/guardian unable to understand the nature, scope, and possible consequences of the study.
• Clinically relevant cardiovascular, hepatic, neurological, endocrine, or other major systemic disease making implementation of the protocol or interpretation of the study results difficult or that would put the participant at risk by participating in the study in the opinion of the Investigator.
• History of drug or alcohol abuse.
• History of known human immunodeficiency virus (HIV) positivity.
• Pregnant or breast-feeding female participants or those who had planned to become pregnant during the study.
• Unwilling to agree to use a highly effective contraceptive method when receiving a course of alemtuzumab treatment and for 4 months following that course of treatment (fertile participants only).
• Female participants who have commenced menstruating (i.e., are of childbearing potential) and are unwilling or unable to be tested for pregnancy.
• Previous treatment with alemtuzumab.
• Treatment with natalizumab, daclizumab, fingolimod, methotrexate, azathioprine, cyclosporine, or mycophenolate mofetil in the last 6 months prior to screening, or determined by the treating physician to had residual immune suppression from these or other MS treatments.
• Treatment with teriflunomide in the last 12 months except if the participant underwent the recommended elimination procedure as per Summary of Product Characteristics (SmPC).
• Previous treatment with mitoxantrone, cyclophosphamide, cladribine, rituximab, ocrelizumab, leflunomide, or any cytotoxic therapy.
• Previous treatment with any investigational medication (drug that had not been approved at any dose or for any indication). Use of an investigational medication that is subsequently licensed and nonstandard use of a licensed medication (e.g., using a dose other than the dose that is stated in the licensed product labeling or using a licensed therapy for an alternative indication) was not exclusionary. Prior treatment with herbal medications or nutritional supplements was also permitted.
• Intolerance of pulsed corticosteroids, especially a history of steroid psychosis.
• History of malignancy.
• Prior documented history of thrombocytopenia, or platelet count at screening < lower limits of normal (LLN).
• Any disability acquired from trauma or another illness that, in the opinion of the Investigator, could interfere with evaluation of disability due to MS.
• Participants with known Type 1 hypersensitivity or anaphylactic reactions to the active substances or any of the excipients, or intolerance of acyclovir or its therapeutic equivalent.
• Major systemic disease or other illness that would, in the opinion of the Investigator, compromise participant safety or interfere with the interpretation of study results, e.g., current peptic ulcer disease, or other conditions that might predispose to hemorrhage, immune cytopenias, rheumatoid arthritis, systemic lupus erythematosus, other connective tissue disorders, vasculitis, inflammatory bowel disease, severe psoriasis.
• Medical, psychiatric, cognitive, or other conditions that, in the Investigator's opinion, compromise the participant's ability to understand the participant information, to give informed consent, to comply with the trial protocol, or to complete the study.
• Major psychiatric disorder that is not adequately controlled by treatment in the opinion of the Investigator.
• Epileptic seizures that are not adequately controlled by treatment.
• Magnetic resonance imaging (MRI)-related conditions: conditions that could interfere with MRI acquisition and/or interpretation of MRI results (eg, claustrophobia, orthopedic implants/treatments, orthodontic treatments etc).
• Known bleeding disorder (e.g., dysfibrinogenemia, factor IX deficiency, hemophilia, Von Willebrand's disease, disseminated intravascular coagulation, fibrinogen deficiency, clotting factor deficiency).
• Prior history of invasive fungal infections.
• Active infection, eg, deep-tissue infection, that the Investigator considers sufficiently serious to preclude study participation.
• In the Investigator's opinion, participant is at high risk for infection (e.g., indwelling catheter, dysphagia with aspiration, decubitus ulcer, history of prior aspiration pneumonia or recurrent urinary tract infection).

The above information is not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.

• Minimum Eligible Age: 10 Years
• Maximum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Genzyme, a Sanofi Company

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Clinical Sciences & Operations

Role: STUDY_DIRECTOR

Sanofi

Locations

Investigational Site Number : 0400001

Vienna, , Austria

Investigational Site Number : 2500001

Le Kremlin-Bicêtre, , France

Investigational Site Number : 2500002

Strasbourg, , France

Investigational Site Number : 3800004

Naples, Napoli, Italy

Investigational Site Number : 3800005

Cagliari, , Italy

Investigational Site Number : 3800001

Milan, , Italy

Investigational Site Number : 6160002

Poznan, Greater Poland Voivodeship, Poland

Investigational Site Number : 6160003

Lodz, Lódzkie, Poland

Investigational Site Number : 6160001

Warsaw, , Poland

Investigational Site Number : 6430001

Moscow, , Russia

Investigational Site Number : 6430004

Moscow, , Russia

Investigational Site Number : 6430005

Saint Petersburg, , Russia

Investigational Site Number : 6430002

Saint Petersburg, , Russia

Investigational Site Number : 7920001

Ankara, , Turkey (Türkiye)

Investigational Site Number : 7920003

Istanbul, , Turkey (Türkiye)

Investigational Site Number : 8260002

London, London, City of, United Kingdom

Countries

Austria; France; Italy; Poland; Russia; Turkey (Türkiye); United Kingdom

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

U1111-1180-6352

Identifier Type: REGISTRY

Identifier Source: secondary_id

2016-003100-30

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

EFC13429

Identifier Type: -

Identifier Source: org_study_id