A Study to Evaluate Efficacy, Safety, and Tolerability of Alemtuzumab in Pediatric Patients With RRMS With Disease Activity on Prior DMT
- Conditions
- Multiple Sclerosis
- Interventions
- Drug: Beta-InterferonDrug: CeterizineDrug: DiphenydramineDrug: Other H1 antagonist
- Registration Number
- NCT03368664
- Lead Sponsor
- Genzyme, a Sanofi Company
- 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.
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 16
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Alemtuzumab Acyclovir - 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 Alemtuzumab Beta-Interferon - 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 Alemtuzumab Prednisolone - 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 Alemtuzumab Glatiramer acetate - 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 Alemtuzumab Ceterizine - 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 Alemtuzumab Other H1 antagonist - 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 Alemtuzumab Alemtuzumab GZ402673 - 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 Alemtuzumab Diphenydramine - 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 Alemtuzumab Methylprednisolone - 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 Alemtuzumab Ranitidine - 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 Alemtuzumab Paracetamol - 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 Alemtuzumab Dexchlorpheniramine - 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
- Primary Outcome Measures
Name Time Method Brain Magnetic Resonance Imaging (MRI) Assessment: Number of New or Enlarged T2 Lesions Per MRI Scan Period 1: Month -4 up to Month 0, Period 2: Month 4 to Month 8 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.
- Secondary Outcome Measures
Name Time Method Change From Baseline in Expanded Disability Status Scale (EDSS) Score at Months 4 and 8 Baseline, Months 4 and 8 EDSS is an ordinal scale in half-point increments that qualifies disability in participants with MS. It consists of 8 ordinal rating scales assessing seven functional systems (pyramidal, cerebellar, brainstem, sensory, bowel and bladder, visual, cerebral, and other). EDSS total score ranges from 0 (normal neurological examination) to 10 (death due to MS), where higher scores indicated worst outcomes.
Area Under the Plasma Concentration-Time Curve (AUC) of Alemtuzumab Up to 5 years Data for this outcome measure will be reported at the time of anticipated last participant last visit results posting (December 2026).
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 Number of participants with at least one new or enlarged T2 lesions per MRI scan was reported in this outcome measure. Number of new or enlarged T2 lesions per scan was defined as the total number of new or enlarged T2 lesion that occurred during treatment period divided by the total number of scans performed during treatment period.
Change From Baseline in Cognition Test Scores of Brief Visuospatial Memory Test - Revised (BVMT-R) Up to 5 years Data for this outcome measure will be reported at the time of anticipated last participant last visit results posting (December 2026).
Maximum Serum Concentration Observed (Cmax) of Alemtuzumab Up to 5 years Data for this outcome measure will be reported at the time of anticipated last participant last visit results posting (December 2026).
Terminal Half-life (T1/2z) of Alemtuzumab Up to 5 years Data for this outcome measure will be reported at the time of anticipated last participant last visit results posting (December 2026).
Number of Participants With Treatment-emergent Adverse Events (TEAE) and Treatment-emergent Serious Adverse Events (TESAE) Up to 5 years Data for this outcome measure will be reported at the time of anticipated last participant last visit results posting (December 2026).
Annualized Relapse Rate (ARR) Up to 5 years Data for this outcome measure will be reported at the time of anticipated last participant last visit results posting (December 2026).
Change From Baseline in Pediatric Quality of Life in Neurological Disorders (NeuroQoL) Questionnaire Score Up to 5 years Data for this outcome measure will be reported at the time of anticipated last participant last visit results posting (December 2026).
Time to Reach Maximum Observed Plasma Concentration (Tmax) of Alemtuzumab Up to 5 years Data for this outcome measure will be reported at the time of anticipated last participant last visit results posting (December 2026).
Area Under the Concentration-time Curve From Time 0 to the Time of the Last Quantifiable Concentration (AUC0-last) of Alemtuzumab Up to 5 years Data for this outcome measure will be reported at the time of anticipated last participant last visit results posting (December 2026).
Assessment of Lymphocyte Phenotyping Up to 5 years Data for this outcome measure will be reported at the time of anticipated last participant last visit results posting (December 2026).
Change From Baseline in Cognition Test Scores of Symbol Digit Modality Test (SDMT) Up to 5 years Data for this outcome measure will be reported at the time of anticipated last participant last visit results posting (December 2026).
Change From Baseline in Quality of Life (QoL) Measures of Pediatric Quality of Life (PedsQL) Questionnaire Score Up to 5 years Data for this outcome measure will be reported at the time of anticipated last participant last visit results posting (December 2026).
Serum Concentrations of Alemtuzumab Over Time Up to 5 years Data for this outcome measure will be reported at the time of anticipated last participant last visit results posting (December 2026).
Percentage of Participants With Incidence of Antidrug Antibodies (ADA) Up to 5 years Data for this outcome measure will be reported at the time of anticipated last participant last visit results posting (December 2026).
Trial Locations
- Locations (21)
Investigational Site Number :7920002
🇹🇷Ankara, Turkey
Investigational Site Number :7920001
🇹🇷Ankara, Turkey
Investigational Site Number :2500002
🇫🇷Strasbourg, France
Investigational Site Number :8260002
🇬🇧London, London, City Of, United Kingdom
Investigational Site Number :7920004
🇹🇷Istanbul, Turkey
Investigational Site Number :2500001
🇫🇷Le Kremlin Bicetre, France
Investigational Site Number :6160002
🇵🇱Poznan, Wielkopolskie, Poland
Investigational Site Number :0400001
🇦🇹Wien, Austria
Investigational Site Number :6430002
🇷🇺St-Petersburg, Russian Federation
Investigational Site Number :6430001
🇷🇺Moscow, Russian Federation
Investigational Site Number :5280001
🇳🇱Rotterdam, Netherlands
Investigational Site Number :6160003
🇵🇱Lodz, Lódzkie, Poland
Investigational Site Number :0560001
🇧🇪Gent, Belgium
Investigational Site Number :6160001
🇵🇱Warszawa, Poland
Investigational Site Number :6430005
🇷🇺Saint-Petersburg, Russian Federation
Investigational Site Number :6430004
🇷🇺Moscow, Russian Federation
Investigational Site Number :6200001
🇵🇹Coimbra, Portugal
Investigational Site Number :3800005
🇮🇹Cagliari, Italy
Investigational Site Number :3800001
🇮🇹Milano, Italy
Investigational Site Number :3800004
🇮🇹Napoli, Italy
Investigational Site Number :7920003
🇹🇷Istanbul, Turkey