ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 1 of 1 for:    lemkids
Previous Study | Return to List | Next Study

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.
ClinicalTrials.gov Identifier: NCT03368664
Recruitment Status : Recruiting
First Posted : December 11, 2017
Last Update Posted : May 16, 2018
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )

Brief Summary:

Primary Objective:

To evaluate the efficacy, safety, and tolerability of alemtuzumab (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 Phase 3

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

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 Pediatric 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 : March 2025
Estimated Study Completion Date : March 2025

Resource links provided by the National Library of Medicine

Drug Information available for: Alemtuzumab
U.S. FDA Resources

Arm Intervention/treatment
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.
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



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

  3. 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 year

  4. 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

  5. Assesment 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

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

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

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

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

  10. Assessment of PD parameter: lymphocyte phenotyping - 10 [ Time Frame: until Month 60 ]
    Lymphocyte phenotyping will be assessed at screening until M24/at EOTP; annually in Safety Monitoring Phase



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.


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

Inclusion criteria :

  • Patients with Relapsing Remitting Multiple Sclerosis (RRMS) aged from 10 years to less than 18 years at study entry are eligible. Patients should 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 McDonald criteria 2010.
  • Signed 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 interferon-beta (IFNB) or glatiramer acetate (GA) after having been on that therapy for at least 6 months
  • 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.
  • 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 reliable and effective contraceptive method as defined for contraception in the Informed Consent form (ICF) 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 ClinicalTrials.gov identifier (NCT number): NCT03368664


Contacts
Contact: For site information, send an email with site number to Contact-Us@sanofi.com

  Show 31 Study Locations
Sponsors and Collaborators
Genzyme, a Sanofi Company
Investigators
Study Director: Clinical Sciences & Operations Sanofi

Responsible Party: Genzyme, a Sanofi Company
ClinicalTrials.gov Identifier: NCT03368664     History of Changes
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: May 16, 2018
Last Verified: May 2018
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: https://www.clinicalstudydatarequest.com/
URL: http://

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:
Sclerosis
Multiple Sclerosis
Multiple Sclerosis, Relapsing-Remitting
Pathologic Processes
Demyelinating Autoimmune Diseases, CNS
Autoimmune Diseases of the Nervous System
Nervous System Diseases
Demyelinating Diseases
Autoimmune Diseases
Immune System Diseases
Alemtuzumab
Interferons
Prednisolone
Methylprednisolone Hemisuccinate
Interferon-beta
Acyclovir
Acetaminophen
Methylprednisolone
Prednisolone acetate
Methylprednisolone acetate
Prednisolone hemisuccinate
Prednisolone phosphate
Ranitidine
Glatiramer Acetate
Dexchlorpheniramine
Antineoplastic Agents
Antiviral Agents
Anti-Infective Agents
Analgesics, Non-Narcotic
Analgesics