Lemkids
Research type
Research Study
Full 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)
IRAS ID
222188
Contact name
Carlo Incerti
Contact email
Sponsor organisation
Genzyme Corporation
Eudract number
2016-003100-30
Duration of Study in the UK
7 years, 5 months, days
Research summary
Multiple Sclerosis (MS) is a neurological disease which commonly affects patients between 20 to 40 years of age. However, pediatric MS, defined as onset of MS before the age of 16, is increasingly recognised and is thought to account for approximately 5 percent of cases.
Multiple Sclerosis in children and differences from the common adult form of MS to a large extent are understudied. There are no approved disease modifying drugs for pediatric MS and the effects of drugs in children have not been formally evaluated in clinical trials.
Alemtuzumab has demonstrated advantages in adult relapsing forms of MS patients in reducing frequency of relapse, rate, disease progression, and MRI activity.
The objectives of the proposed clinical pediatric development program are to assess the efficacy, safety/tolerability,and pharmacokinetics (PK) of alemtuzumab in children and adolescents 10 to 17 years of age with remitting relapsing multiple sclerosis (RRMS).LAY SUMMARY OF STUDY RESULTS:
The LemKids study tested alemtuzumab (Lemtrada®), an infusion treatment, in children and teenagers aged 10-17 with relapsing-remitting multiple sclerosis (RRMS) whose disease was still active despite previous treatments.Sixteen young patients participated. The study compared brain scans before and after switching to alemtuzumab. Results showed a significant reduction in new brain lesions after alemtuzumab treatment (7 lesions) compared to previous treatments (178 lesions).
Common side effects included skin reactions like hives (55%), infections (82%), and infusion-related reactions such as headache and nausea. Two participants developed thyroid problems, and two became pregnant during the study. Most side effects were mild to moderate.
The study was stopped early due to operational reasons and new safety concerns. The small number of participants limits how much we can conclude from these results.
Overall, alemtuzumab showed promise in reducing disease activity in young MS patients, with a safety profile similar to that seen in adults.
REC name
West Midlands - Coventry & Warwickshire Research Ethics Committee
REC reference
17/WM/0184
Date of REC Opinion
5 Jun 2017
REC opinion
Favourable Opinion