Fingolimod for Pediatric Multiple Sclerosis
Recruiting in Palo Alto (17 mi)
+71 other locations
Age: < 18
Sex: Any
Travel: May be covered
Time Reimbursement: Varies
Trial Phase: Phase 3
Waitlist Available
Sponsor: Novartis Pharmaceuticals
No Placebo Group
Pivotal Trial (Near Approval)
Prior Safety Data
Breakthrough Therapy
Trial Summary
What is the purpose of this trial?To evaluate the safety and efficacy of fingolimod vs. interferon beta-1a i.m. in pediatric patients with multiple sclerosis (MS)
Is the drug Fingolimod a promising treatment for children with multiple sclerosis?
Yes, Fingolimod is a promising drug for children with multiple sclerosis. It has been shown to effectively reduce relapse rates and improve stability in pediatric patients, and it is the only drug approved by major health agencies for this condition.
15678What safety data is available for Fingolimod in treating pediatric multiple sclerosis?
Fingolimod, also known as Gilenya, is approved by the FDA and EMA for treating pediatric relapsing-remitting multiple sclerosis. Safety data from clinical trials and observational studies indicate that common side effects include fatigue, gastrointestinal disturbances, headache, and upper respiratory tract infections. Serious but rare adverse events can include atrioventricular block, symptomatic bradycardia, herpetic viral infections, and macular edema. The safety profile is well-characterized, and side effects are manageable with patient monitoring.
34578What data supports the idea that Fingolimod for Pediatric Multiple Sclerosis is an effective drug?
The available research shows that Fingolimod is effective for treating Pediatric Multiple Sclerosis. One study reported that children treated with Fingolimod experienced clinical stability and minimal new disease activity on brain scans. Another study highlighted that Fingolimod reduced the relapse rate by 82% compared to another drug, interferon β-1a. These findings suggest that Fingolimod is a strong option for managing the condition in children.
25678Do I have to stop taking my current medications for this trial?
The trial protocol does not specify if you must stop taking your current medications. However, there is a mention of washout periods required for certain concomitant medications before Visit 15, so you might need to stop some medications temporarily.
Eligibility Criteria
This trial is for children with multiple sclerosis (MS) who've had at least one MS relapse in the past year or two in the last two years, or recent MRI evidence of active lesions. They should be able to perform daily activities with minimal assistance (EDSS score 0-5.5).Inclusion Criteria
I have been diagnosed with multiple sclerosis.
I have had at least one MS flare-up in the last year or two in the last two years, or recent MRI shows active MS.
My disability score is between 0 and 5.5.
I completed the initial part of the study, with or without the study drug.
Exclusion Criteria
I do not have severe heart disease or major issues on my heart test.
I have severe kidney problems.
I stopped taking the study drug early due to a serious side effect or lab result.
My multiple sclerosis is getting worse.
I have been diagnosed with ADEM.
I meet the exclusion criteria for the initial phase of the study.
I have a chronic immune system disease other than MS.
Participant Groups
The study compares the safety and effectiveness of a drug called Fingolimod against Interferon beta-1a in pediatric MS patients. Some participants will receive Fingolimod while others will get Interferon beta-1a injections; there's also a placebo group.
3Treatment groups
Experimental Treatment
Active Control
Group I: Fingolimod-Younger CohortExperimental Treatment1 Intervention
The 'younger cohort' refers to the new pediatric patients to be recruited in the extension phase who fulfill any single one or a combination of the following criteria: being ≤12 years of age, or weighing ≤40 kg, or being prepubertal (i.e. pubertal status of Tanner stage \<2)
Group II: FingolimodExperimental Treatment2 Interventions
Fingolimod was administered orally once daily at a dose of either 0.5 mg or 0.25 mg (depending on patient's body weight) with the aim to achieve systemic exposure in range of that in adults at the licensed 0.5 mg dose. Participants in this arm during core continued into extension and received open-label treatment
Group III: Interferon beta-1aActive Control2 Interventions
An intramuscular (IM) injection of Interferon beta-1a was administered once weekly during core phase. Participants switched to receive open-label fingolimod in extension phase
Fingolimod is already approved in European Union, United States, Canada for the following indications:
🇪🇺 Approved in European Union as Gilenya for:
- Relapsing forms of multiple sclerosis
🇺🇸 Approved in United States as Gilenya for:
- Relapsing forms of multiple sclerosis
🇨🇦 Approved in Canada as Gilenya for:
- Relapsing forms of multiple sclerosis
Find A Clinic Near You
Research locations nearbySelect from list below to view details:
Novartis Investigative SiteLos Angeles, CA
UAB Childrens Hospital Harbor Center Neurology DeptBirmingham, AL
Childrens Hospital Los AngelesLos Angeles, CA
UCSFSan Francisco, CA
More Trial Locations
Loading ...
Who is running the clinical trial?
Novartis PharmaceuticalsLead Sponsor
References
Fingolimod for multiple sclerosis. [2015]Fingolimod (Gilenya--Novartis) is a new treatment for patients with highly active relapsing-remitting multiple sclerosis. It is the first oral disease-modifying drug for the treatment of patients with multiple sclerosis. Here we assess the place of this drug.
[The comparative study of efficacy and tolerability of intramuscular introduction of beta-interferon-1a in adults and adolescents with remitting multiple sclerosis]. [2016]The experience of the treatment of patients with remitting multiple sclerosis (MS) with intramuscular introduction of beta-interferon-1a (avonex) is presented. Seventeen children and adolescents, aged from 11 to 18 years, and 55 adults, aged over 55 years, were treated for at least one-year period. Results revealed a significant reduction of exacerbations in both groups (from 1.35 to 0.06 in average in adolescents and from 0.86 to 0.17 in adults). The changes were accompanied by the stabilization of MS severity index: EDSS scores have decreased in 23.6% of adults and in 17.6% of adolescents. In both groups, good tolerability of the treatment was noted. There was a low probability of side-effects with the exception of increased frequency of a flu-like syndrome (47% cases) in patients younger than 18 years that demands special attention from children neurologists. The high efficacy and good tolerability and safety profile of beta-interferon-1a give grounds for administering this drug to children and adolescents with MS.
Fingolimod for the treatment of relapsing multiple sclerosis. [2015]Fingolimod 0.5 mg (Gilenya(™), Novartis Pharmaceuticals Corporation, FL, USA) is the first once-daily oral therapy approved for relapsing forms of multiple sclerosis (MS) in the USA and for rapidly evolving severe MS or highly active disease despite IFN-β treatment in Europe. An extensive clinical development program has established fingolimod as an effective therapy that reduces relapses by approximately half compared with placebo or intramuscular IFN-β1a. Over 2 years of postmarketing experience in >63,000 MS patients (with >73,000 patient-years of exposure) across the world has contributed to a well-characterized safety profile for fingolimod, and its side effects are manageable through patient monitoring. This article discusses the unique mechanisms of action of fingolimod in the immune and nervous systems, the key data underlying its efficacy and safety profile and perspectives on the role of fingolimod in current and future treatment strategies for MS.
Overview and safety of fingolimod hydrochloride use in patients with multiple sclerosis. [2015]Fingolimod (Gilenya®, FTY720) is an oral sphingosine-1-phosphate analogue that was approved by the FDA in 2010 for the treatment of relapsing forms of multiple sclerosis (MS). Fingolimod's mechanism of action is primarily related to lymphocyte sequestration in primary and secondary lymphoid tissues. Phase III trials demonstrated a reduction in annualized relapse rate and MRI progression in fingolimod-treated subjects compared with both placebo and IFN-β-treated subjects. Frequent adverse effects include fatigue, gastrointestinal disturbance, headache and upper respiratory tract infection. More serious, but rare, adverse events associated with fingolimod include atrioventricular block, symptomatic bradycardia, herpetic viral infections and macular edema.
Review Of The Safety, Efficacy And Tolerability Of Fingolimod In The Treatment Of Pediatric Patients With Relapsing-Remitting Forms Of Multiple Sclerosis (RRMS). [2020]Pediatric-onset multiple sclerosis (POMS) is an immune-mediated, demyelinating, neurodegenerative disease that accounts for 3-5% of all multiple sclerosis (MS) cases. Although evidence suggests that it has similar risk factors and disease pathophysiology as adult-onset MS (AOMS), there are distinctive features in disease characteristics and patient demographics of POMS that require unique therapeutic development and treatment considerations. Gilenya® (Novartis International AG, Basel, Switzerland) (fingolimod) is a sphingosine-1-phosphate (S1P) receptor modulator that prevents lymphocytic outflow from peripheral lymph nodes. It has demonstrated efficacy in AOMS. In POMS, there have been three observational studies and one pivotal clinical trial evaluating the efficacy, safety, and tolerability of fingolimod. Currently, fingolimod is the only Food and Drug Administration and European Medicines Agency approved disease-modifying therapy to treat POMS. This review will critically evaluate the available evidence of fingolimod in the treatment of POMS in detail, as well as discussing its treatment implications.
Two-year follow-up during fingolimod treatment in a pediatric multiple sclerosis patient still active on first-line treatment. [2021]Treatment of pediatric multiple sclerosis (MS) has been increasingly debated in the last few years due to limited knowledge of treatment strategies and therapeutic options. When MS develops at a young age, it usually has a very inflammatory disease course, with many relapses and disease activity as seen in magnetic resonance imaging (MRI). Therefore, treatment with immunomodulatory drugs may be beneficial in these patients. However, limited data are available to date on the treatment of pediatric MS. Although observational, prospective, and retrospective studies provide some information on its treatment course, only one clinical trial in pediatric patients has been published, the PARADIGMS trial, which showed an 82% reduction in relapse rate with fingolimod (0.5 mg/day) versus interferon β-1a (30 μg once weekly intramuscularly). Here, we present the case of a pediatric patient with MS (age of onset, 13 years), who was initially treated with interferon β-1a for 2 years and subsequently switched to fingolimod, owing to clinical and radiological activity despite treatment with interferon β-1a.
Efficacy of fingolimod after switching from interferon β-1a in an adolescent with multiple sclerosis: case report. [2021]Pediatric-onset multiple sclerosis (POMS) accounts for approximately 2-10% of all cases of multiple sclerosis (MS) and is associated with higher levels of disease activity than adult-onset MS, including higher rates of clinical relapse and a greater incidence of new T2 lesions on magnetic resonance imaging (MRI). First-line therapy for POMS usually includes interferon β or glatiramer acetate; however, there is limited evidence from randomized trials regarding the safety and efficacy of these disease-modifying drugs in pediatric patients. Fingolimod represents a second-line therapy option for relapsing-remitting MS in pediatric patients. Here, we report the case of a 14-year-old girl with a diagnosis of POMS who started interferon β-1a as first-line therapy and then switched to fingolimod after 12 months due to radiologic progression and clinical relapse. The patient subsequently experienced clinical stability and showed minimal radiologic activity on follow-up MRI. Our case demonstrates the real-world clinical effectiveness and safety of fingolimod in pediatric MS and is in line with the results of previous randomized and observational studies.
Fingolimod as an effective therapeutic strategy for pediatric relapsing-remitting multiple sclerosis: two case reports. [2021]Approximately 3-10% of patients with multiple sclerosis (MS) have onset during childhood. Pediatric MS is characterized by a relapsing-remitting course and a high relapse rate. In 2010, fingolimod (Gilenya®) was approved in the USA for the treatment of relapsing-remitting MS in adults. In 2018, both the United States Food and Drug Administration and the European Medicines Agency expanded the approved indications of fingolimod to include its use in children with relapsing MS, and the drug was approved in Italy for this indication in September 2020. We describe two cases of children with relapsing-remitting MS who were treated with fingolimod at IRCCS Ospedale San Raffaele Multiple Sclerosis Center (Milan, Italy) for more than 2 years. Our real-world data confirm that fingolimod is an effective therapeutic strategy for children with relapsing MS, and its use could be considered in pediatric patients with active disease.