Primary objective:To evaluate the efficacy, safety and tolerability of alemtuzumab intravenously (IV) in paediatric patients from 10 to
ID
Source
Brief title
Condition
- Demyelinating disorders
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
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
* Number 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)
* Annualized relapse rate
* Assessment of cognition test scores through Brief Visuospatial Memory Test
* Assessment of generic pediatric QoL measures
* Assessment of PK parameters
* Assessment of PD parameter
* Number of patients with adverse events
* Assessment of development of anti-alemtuzumab antibodies
Background summary
Multiple sclerosis (MS) is a demyelinating disease of the central nervous
system (CNS) that affects approximately 2.5 million people worldwide. MS
repesents the leading cause of neurologic disability in young and middle-aged
adults. Multiple sclerosis is typically considered to be a disease of young
adults. However, pediatric MS, defined as onset of MS before the age of 16, is
increasingly recognized and accounts for approximately 5 percent of the cases.
Study objective
Primary objective:
To evaluate the efficacy, safety and tolerability of alemtuzumab intravenously
(IV) in paediatric patients from 10 to <18 years of age with relapsing
remitting multiple sclerosis (RRMS) who have disease activity on prior disease
modifying treatmetn (DMT).
Secondary objective:
To assess the pharmacokinetics (PK), pharmacodynamics (PD), antidrug antibody
formation, and potential effects of alemtuzumab on other multiple sclerosis
(MS) disease characteristics such as cognition and quality of life (QoL).
Study design
A phase III, multi-center, open-label, single-arm study, before and after
switch of DMT to alemtuzumab.
Intervention
Two doses of intravenous (IV) infusion of 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.
Study burden and risks
Risks and burdens related to blood collection, study procedures and possible
adverse events of study medication.
Paasheuvelweg 25
Amsterdam 1105BP
NL
Paasheuvelweg 25
Amsterdam 1105BP
NL
Listed location countries
Age
Inclusion criteria
-Patients with RRMS aged from 10 years to less than 18 years at study entry are
eligible. Patients must meet the criteria of diagnosis of MS as defined by the
International Pediatric Multiple Sclerosis Study Group (IPMSSG) criteria for
pediatric MS and the criteria of 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 MS attacks and at least 1 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 MS DMTs.
Exclusion criteria
-Any prior exposure to alemtuzumab.
-Any progressive or nonrelapsing form of MS.
-Treatment with natalizumab, daclizumab, fingolimod, methotrexate,
azathioprine, cyclosporine, or mycophenolate mofetil in the last 6 months, or
as 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 accelerated elimination procedure as per local teriflunomide
label.
-Previous treatment with mitoxantrone, cyclophosphamide, cladribine, rituximab,
ocrelizumab, leflunomide or any cytotoxic therapy.
-CD4+, CD8+, or CD19+ absolute cell count in blood at screening below lower
limit of normal (LLN).
-Prior documented history of thrombocytopenia, or platelet count at screening
Design
Recruitment
Medical products/devices used
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| Other | 2016-003100-30 |
| EudraCT | EUCTR2016-003100-30-NL |
| CCMO | NL60747.078.17 |