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

NCT03368664 | Terminated Phase 3 Results DMC FDA Drug

• 3 other identifiers: EFC13429U1111-1180-63522016-003100-30
• Study Type: interventional
• Target: 16
• Locations: 7 countries
• Sites: 16

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).

Conditions

Multiple Sclerosis

Outcome Measures

Primary Outcomes (1)

• Brain Magnetic Resonance Imaging (MRI) Assessment: Number of New or Enlarged T2 Lesions Per MRI Scan

  Number of new or enlarged T2 lesions per scan was defined as the total number of new or enlarged T2 lesion that occurred during a specified period divided by the total number of scans performed during that specified period.

  Period 1: Month -4 up to Month 0, Period 2: Month 4 to Month 8

Secondary Outcomes (16)

• Brain Magnetic Resonance Imaging (MRI) Assessment: Number of Participants With New or Enlarged T2 Lesions Per MRI Scan

  Period 1: Month -4 up to Month 0, Period 2: Month 4 to Month 8

• Change From Baseline in Expanded Disability Status Scale (EDSS) Score at Months 4 and 8

  Baseline, Months 4 and 8

• Number of Participants With Treatment-emergent Adverse Events (TEAE) and Treatment-emergent Serious Adverse Events (TESAE)

  Up to 5 years

• Annualized Relapse Rate (ARR)

  Up to 5 years

• Change From Baseline in Cognition Test Scores of Brief Visuospatial Memory Test - Revised (BVMT-R)

  Up to 5 years

Study Arms (1)

Alemtuzumab

EXPERIMENTAL

\- 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

Drug: Alemtuzumab GZ402673; Drug: Glatiramer acetate; Drug: Beta-Interferon; Drug: Methylprednisolone; Drug: Ranitidine; Drug: Ceterizine; Drug: Dexchlorpheniramine; Drug: Paracetamol; Drug: Acyclovir; Drug: Prednisolone; Drug: Diphenydramine; Drug: Other H1 antagonist

Interventions

Alemtuzumab GZ402673 DRUG

Pharmaceutical form: solution, Route of administration: IV

Also known as: Lemtrada

Alemtuzumab

Glatiramer acetate DRUG

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

Also known as: Copaxone

Alemtuzumab

Beta-Interferon DRUG

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

Alemtuzumab

Methylprednisolone DRUG

Pharmaceutical form: solution, Route of administration: IV

Alemtuzumab

Ranitidine DRUG

Pharmaceutical form: tablet, Route of administration: oral

Alemtuzumab

Ceterizine DRUG

Pharmaceutical form: tablet, Route of administration: oral

Alemtuzumab

Dexchlorpheniramine DRUG

Pharmaceutical form: tablet, Route of administration: oral

Alemtuzumab

Paracetamol DRUG

Pharmaceutical form: tablet, Route of administration: oral

Alemtuzumab

Acyclovir DRUG

Pharmaceutical form: tablet, Route of administration: oral

Alemtuzumab

Prednisolone DRUG

Pharmaceutical form: tablet, Route of administration: oral

Alemtuzumab

Diphenydramine DRUG

Pharmaceutical form: solution, Route of administration: IV

Alemtuzumab

Other H1 antagonist DRUG

Pharmaceutical form: solution, Route of administration: IV

Alemtuzumab

Eligibility Criteria

• Age: 10 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• Any progressive or non-relapsing forms of MS.
• Conditions/situations such as:
• Impossibility to meet specific protocol requirements.
• 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).

Sponsors & Collaborators

• Genzyme, a Sanofi Company: lead

Study Sites (16)

Investigational Site Number : 0400001

Vienna, 1090, Austria

Location

Investigational Site Number : 2500001

Le Kremlin-Bicêtre, 94270, France

Location

Investigational Site Number : 2500002

Strasbourg, 67091, France

Location

Investigational Site Number : 3800004

Naples, Napoli, 80131, Italy

Location

Investigational Site Number : 3800005

Cagliari, 09126, Italy

Location

Investigational Site Number : 3800001

Milan, 20132, Italy

Location

Investigational Site Number : 6160002

Poznan, Greater Poland Voivodeship, 60-355, Poland

Location

Investigational Site Number : 6160003

Lodz, Lódzkie, 93-338, Poland

Location

Investigational Site Number : 6160001

Warsaw, 04-730, Poland

Location

Investigational Site Number : 6430001

Moscow, 119602, Russia

Location

Investigational Site Number : 6430004

Moscow, 129110, Russia

Location

Investigational Site Number : 6430005

Saint Petersburg, 197022, Russia

Location

Investigational Site Number : 6430002

Saint Petersburg, 197110, Russia

Location

Investigational Site Number : 7920001

Ankara, 06500, Turkey (Türkiye)

Location

Investigational Site Number : 7920003

Istanbul, 34390, Turkey (Türkiye)

Location

Investigational Site Number : 8260002

London, London, City of, WC1N 3JH, United Kingdom

Location

MeSH Terms

Conditions

Multiple Sclerosis

Interventions

Alemtuzumab; Glatiramer Acetate; Interferon-beta; Methylprednisolone; Ranitidine; ceterizine hydrochloride; dexchlorpheniramine; Acetaminophen; Acyclovir; Prednisolone; aminophylline, cycloclenbutrerol, diphenydramine, phenobarbital drug combination

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Antibodies, Monoclonal, Humanized; Antibodies, Monoclonal; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins; Peptides; Interferon Type I; Interferons; Cytokines; Intercellular Signaling Peptides and Proteins; Biological Factors; Pregnadienetriols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Furans; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Acetanilides; Anilides; Amides; Organic Chemicals; Aniline Compounds; Amines; Guanine; Hypoxanthines; Purinones; Purines; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring

Results Point of Contact

• Title: Trial Transparency Team
• Organization: Sanofi

Contact-US@sanofi.com

800-633-1610

Study Officials

• Clinical Sciences & Operations

  Sanofi

  STUDY DIRECTOR

Publication Agreements

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

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 2, 2017
• First Posted: December 11, 2017
• Study Start: October 24, 2017
• Primary Completion: May 4, 2021
• Study Completion: September 8, 2025
• Last Updated: November 26, 2025
• Results First Posted: July 12, 2022

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will share

Locations

AU (1); FR (2); IT (3); GB (1); PL (3); RU (4); TU (2)