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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03368664
Recruitment Status : Recruiting
First Posted : December 11, 2017
Last Update Posted : November 19, 2019
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )

Brief Summary:

Primary Objective:

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

Condition or disease Intervention/treatment Phase
Multiple Sclerosis 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 Phase 3

Detailed Description:
The duration of study per patient will be approximately 5 years and 5 months.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 50 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Multi-center, Open-label, Single-arm, Before and After Switch Study to Evaluate the Efficacy, Safety and Tolerability of Alemtuzumab in Paediatric Patients With Relapsing Remitting Multiple Sclerosis (RRMS) With Disease Activity on Prior Disease Modifying Therapy (DMT)
Actual Study Start Date : October 24, 2017
Estimated Primary Completion Date : July 2026
Estimated Study Completion Date : July 2026

Resource links provided by the National Library of Medicine

Drug Information available for: Alemtuzumab

Arm Intervention/treatment
Experimental: 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
Drug: Alemtuzumab GZ402673
Pharmaceutical form:solution Route of administration: intravenous
Other Name: Lemtrada

Drug: Glatiramer acetate
Pharmaceutical form:solution Route of administration: subcutaneous
Other Name: Copaxone

Drug: Beta-Interferon
Pharmaceutical form:solution Route of administration: subcutaneous / intramuscular

Drug: Methylprednisolone
Pharmaceutical form:solution Route of administration: intravenous

Drug: Ranitidine
Pharmaceutical form:tablet Route of administration: oral

Drug: Ceterizine
Pharmaceutical form:tablet Route of administration: oral

Drug: Dexchlorpheniramine
Pharmaceutical form:tablet Route of administration: oral

Drug: Paracetamol
Pharmaceutical form:tablet Route of administration: oral

Drug: Acyclovir
Pharmaceutical form:tablet Route of administration: oral

Drug: Prednisolone
Pharmaceutical form:tablet Route of administration: oral

Drug: Diphenydramine
Pharmaceutical form:solution Route of administration: intravenous

Drug: Other H1 antagonist
Pharmaceutical form:solution Route of administration: intravenous

Drug: Other H1 antagonist
Pharmaceutical form:tablet/pill Route of administration: oral

Primary Outcome Measures :
  1. Number of new or enlarging T2 lesions - [ Time Frame: month -4 to month 0 (Period 1) and month 4 to month 8 (Period 2) - ]
    The number of new or enlarging T2 lesions on brain MRI, during continuation of prior Disease Modifying Therapies (DMTs) (Period 1) compared to an equal period after the first course of alemtuzumab treatment (Period 2). -

Secondary Outcome Measures :
  1. Number of patients with new or enlarging T2 lesions [ Time Frame: Month -4 to month 0 (Period 1) and month 4 to month 8 (Period 2) ]
    The proportion of patients with new or enlarging T2 lesions during continuation of prior DMT (Period 1) compared to an equal period after the first course of alemtuzumab treatment (Period 2)

  2. Change in Expanded Disability Status Scale (EDSS) [ Time Frame: Month 4 to month 8 (Period 2) ]
    Percentages of stable/improved/worsened since the end of Period 1

  3. Annualized relapse rate (ARR) [ Time Frame: At Year 2 ]
    ARR at Year 2

  4. Assessment of cognition test scores [ Time Frame: Baseline to over 2 years ]
    Change from baseline in cognition test scores of Brief Visuospatial Memory Test - Revised (BVMT-R) over 2 years

  5. Assessment of cognition test scores [ Time Frame: Baseline to over 2 years ]
    Change from baseline in cognition test scores of Symbol Digit Modality Test (SDMT) over 2 years

  6. Assessment of generic pediatric Quality of Life (QoL) measures [ Time Frame: Baseline to over 2 years ]
    Change from baseline in QoL measures of PedsQL questionnaire score over 2 years

  7. Assessment of generic pediatric Quality of Life (QoL) measures [ Time Frame: Baseline to over 2 years ]
    Change from baseline in QoL measures of pediatric NeuroQoL questionnaire score over 2 years

  8. Assessment of pharmacokiinetic (PK) parameter: maximum concentration (Cmax) [ Time Frame: 2 years ]
    To evaluate maximum serum concentration observed

  9. Assessment of PK parameter: time to Cmax (tmax) [ Time Frame: 2 years ]
    To evaluate time to reach Cmax

  10. Assessment of PK parameter: area under plasma concentration (AUC) [ Time Frame: 2 years ]
    To evaluate area under the cumulative serum concentration versus time curve

  11. Assessment of pharmacodynamic (PD) parameter: lymphocyte phenotyping [ Time Frame: Until Month 60 ]
    Lymphocyte phenotyping will be assessed at screening until M24/at EOTP; annually in Safety Monitoring Phase.

  12. Safety: Adverse Events (AE) [ Time Frame: Baseline to over 2 years ]
    Adverse Events reported at each visit

  13. Anti-drug Antibody (ADA) [ Time Frame: Visit 1 ]
    Assessment of development of antialemtuzumab antibodies at baseline

Information from the National Library of Medicine

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, Learn About Clinical Studies.

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Ages Eligible for Study:   10 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion criteria :

  • Patients with Relapsing Remitting Multiple Sclerosis (RRMS) aged from 10 years to less than 18 years at study entry are eligible. Patients must meet the criteria of diagnosis of Multiple Sclerosis as defined by the International Pediatric Multiple Sclerosis Study Group (IPMSSG) criteria for pediatric Multiple Sclerosis (MS) and the criteria of Multiple Sclerosis (MS) based on 2010 McDonald criteria.
  • Signed written informed consent/assent obtained from patient and patient'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 Multiple Sclerosis (MS) attacks and at least 1 Multiple Sclerosis (MS) attack (relapse) in the last year during treatment with a beta interferon therapy (IFNB) or glatiramer acetate (GA) after having been on that therapy for at least 6 months, and is 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 Multiple Sclerosis Disease Modifying Therapies (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. Patients who were treated with a comparator agent approved for screening inclusion (INF or GA) may be considered for this trial.
  • Patient 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 patient or any condition that could make the patient potentially non-compliant to the study procedures in the opinion of the Investigator.
  • Mental condition rendering the patient 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 patient 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 patients or those who plan 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 patients only).
  • Female patients who have commenced menstruating (ie, 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 have residual immune suppression from these or other MS treatments.
  • Treatment with teriflunomide in the last 12 months except if the patient 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 has not been approved at any dose or for any indication). Use of an investigational medication that was subsequently licensed and nonstandard use of a licensed medication (eg, using a dose other than the dose that is stated in the licensed product labeling or using a licensed therapy for an alternative indication) is not exclusionary. Prior treatment with herbal medications or nutritional supplements is 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.
  • Patients 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 patient safety or interfere with the interpretation of study results, eg, current peptic ulcer disease, or other conditions that may 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 patient's ability to understand the patient 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 (eg, dysfibrinogenemia, factor IX deficiency, hemophilia, Von Willebrand's disease, disseminated intravascular coagulation [DIC], 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, patient is at high risk for infection (eg, 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 patient's potential participation in a clinical trial.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03368664

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Contact: Trial Transparency email recommended (Toll free number for US & Canada) 800-633-1610 ext 1 then #

Hide Hide 36 study locations
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Investigational Site Number 0400001 Recruiting
Wien, Austria, 1090
Investigational Site Number 0560001 Recruiting
Gent, Belgium, 9000
Investigational Site Number 1000001 Recruiting
Sofia, Bulgaria, 1113
Investigational Site Number 2030001 Recruiting
Praha 5 - Motol, Czechia, 15006
Investigational Site Number 2500001 Recruiting
Le Kremlin Bicetre, France, 94270
Investigational Site Number 2500002 Recruiting
Strasbourg Cedex, France, 67091
Investigational Site Number 2760001 Terminated
Göttingen, Germany, 37075
Investigational Site Number 3000001 Recruiting
Athens, Greece, 115 28
Investigational Site Number 3000002 Recruiting
Thessaloniki, Greece, 54642
Investigational Site Number 3800005 Recruiting
Cagliari, Italy, 09126
Investigational Site Number 3800002 Recruiting
Gallarate, Italy, 21013
Investigational Site Number 3800001 Recruiting
Milano, Italy, 20132
Investigational Site Number 3800004 Recruiting
Napoli, Italy, 80131
Investigational Site Number 3800003 Recruiting
Orbassano, Italy, 10043
Investigational Site Number 5280001 Recruiting
Rotterdam, Netherlands, 3015 CN
Investigational Site Number 5780002 Recruiting
Stavanger, Norway, 4011
Investigational Site Number 6160003 Recruiting
Lodz, Poland, 93-338
Investigational Site Number 6160002 Recruiting
Poznan, Poland, 60-355
Investigational Site Number 6160001 Recruiting
Warszawa, Poland, 04-730
Investigational Site Number 6200001 Recruiting
Coimbra, Portugal, 3000-075
Russian Federation
Investigational Site Number 6430001 Recruiting
Moscow, Russian Federation, 119602
Investigational Site Number 6430004 Recruiting
Moscow, Russian Federation, 129110
Investigational Site Number 6430003 Recruiting
Novosibirsk, Russian Federation, 630087
Investigational Site Number 6430005 Recruiting
Saint-Petersburg, Russian Federation, 197022
Investigational Site Number 6430002 Recruiting
St-Petersburg, Russian Federation, 197110
Investigational Site Number 7240005 Recruiting
Barcelona, Spain, 08035
Investigational Site Number 7240004 Recruiting
Madrid, Spain, 28006
Investigational Site Number 7240001 Recruiting
Murcia, Spain, 30120
Investigational Site Number 7240002 Recruiting
Pozuelo De Alarcón, Spain, 28223
Investigational Site Number 7240003 Recruiting
Sevilla, Spain, 41071
Investigational Site Number 7920002 Recruiting
Ankara, Turkey, 06100
Investigational Site Number 7920001 Recruiting
Ankara, Turkey, 06500
Investigational Site Number 7920003 Recruiting
Istanbul, Turkey, 34390
Investigational Site Number 7920004 Recruiting
Istanbul, Turkey
United Kingdom
Investigational Site Number 8260001 Recruiting
Birmingham, United Kingdom, B4 6NH
Investigational Site Number 8260002 Recruiting
London, United Kingdom, WC1N 3JH
Sponsors and Collaborators
Genzyme, a Sanofi Company
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Study Director: Clinical Sciences & Operations Sanofi

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Responsible Party: Genzyme, a Sanofi Company Identifier: NCT03368664    
Other Study ID Numbers: EFC13429
2016-003100-30 ( EudraCT Number )
U1111-1180-6352 ( Other Identifier: UTN )
First Posted: December 11, 2017    Key Record Dates
Last Update Posted: November 19, 2019
Last Verified: November 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at:

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes
Additional relevant MeSH terms:
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Multiple Sclerosis
Pathologic Processes
Demyelinating Autoimmune Diseases, CNS
Autoimmune Diseases of the Nervous System
Nervous System Diseases
Demyelinating Diseases
Autoimmune Diseases
Immune System Diseases
Methylprednisolone Acetate
Methylprednisolone Hemisuccinate
Prednisolone acetate
Glatiramer Acetate
Prednisolone hemisuccinate
Prednisolone phosphate
Antineoplastic Agents
Antiviral Agents
Anti-Infective Agents
Analgesics, Non-Narcotic