Fingolimod for Pediatric Multiple Sclerosis

Recruiting at 73 trial locations

Overseen By Novartis Pharmaceuticals

Age: < 18

Sex: Any

Trial Phase: Phase 3

Sponsor: Novartis Pharmaceuticals

Disqualifiers: Progressive MS, Chronic immune disease, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Breakthrough Therapy This drug has been fast-tracked for approval by the FDA given its high promise

Compensation: Varies

Number of Visits: Unknown

240 Participants Needed

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)

Do 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.

Do I need to stop my current medications for the trial?

The trial information does not specify if you need to stop taking your current medications. However, there is a mention of 'washout periods' for certain medications before a specific visit, which suggests that some medications might need to be paused. It's best to discuss your current medications with the trial coordinators.

What 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. ^{1 2 3 4 5}

What data supports the effectiveness of the drug Fingolimod for treating pediatric multiple sclerosis?

Fingolimod has been shown to be effective in reducing relapse rates in children with relapsing-remitting multiple sclerosis, as demonstrated by the PARADIGMS trial, which reported an 82% reduction in relapse rate compared to interferon beta-1a. Additionally, real-world cases have shown clinical stability and minimal disease activity in pediatric patients who switched to Fingolimod after other treatments. ^{1 2 3 4 5}

What 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. ^{1 2 4 6 7}

Is fingolimod safe for children with multiple sclerosis?

Fingolimod, also known as Gilenya, has been approved for use in children with relapsing multiple sclerosis and has a well-characterized safety profile. Common side effects include fatigue, stomach issues, headache, and respiratory infections, while rare but serious side effects can include heart rhythm problems and eye issues. It is important for patients to be monitored by healthcare professionals to manage these potential side effects. ^{1 2 4 6 7}

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. ^{1 2 3 4 8}

How is the drug Fingolimod unique for treating pediatric multiple sclerosis?

Fingolimod is unique because it is the first oral drug approved specifically for pediatric multiple sclerosis, offering a convenient alternative to injectable treatments like interferon beta-1a. It works by preventing immune cells from leaving the lymph nodes, which helps reduce the frequency of relapses in children with this condition. ^{1 2 3 4 8}

Research Team

Novartis Pharmaceuticals

Principal Investigator

Novartis Pharmaceuticals

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

Central review (including initial MRI report) of the diagnosis of pediatric MS will be required for all newly recruited patients

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.

Exclusion Criteria

I am not taking any medications or have conditions that would exclude me from this study.

I do not have severe heart disease or major issues on my heart test.

I have severe kidney problems.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Core Phase - Double-Blind Treatment

Participants receive either daily oral fingolimod or weekly intramuscular interferon beta-1a for 24 months

24 months

Regular visits as per study protocol

Extension Phase

Participants who complete the Core Phase receive open-label fingolimod for 60 months

60 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

6 months

Treatment Details

Interventions

• Fingolimod
• Interferon beta-1a

Trial Overview 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.

Participant Groups

Group I: Fingolimod-Younger Cohort Experimental Treatment 1 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: Fingolimod Experimental Treatment 2 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-1a Active Control 2 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

Who Is Running the Clinical Trial?

Novartis Pharmaceuticals

Lead Sponsor

Trials

2,963

Recruited

4,275,000+

Founded

1996

Headquarters

Basel, Switzerland

Known For

Precision medicine

Top Products

Gleevec, Cosentyx, Entresto, Kisqali

Dr. Vas Narasimhan

Novartis Pharmaceuticals

Chief Executive Officer since 2018

MD from Harvard Medical School

Dr. Shreeram Aradhye

Novartis Pharmaceuticals

Chief Medical Officer since 2021

MD

Findings from Research

Fingolimod (Gilenya®) has been approved for treating relapsing-remitting multiple sclerosis (MS) in children, with real-world data from two cases showing its effectiveness over more than 2 years of treatment.

The findings support the use of fingolimod as a viable therapeutic option for pediatric patients with active relapsing MS, highlighting its potential benefits in managing this condition.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Fingolimod as an effective therapeutic strategy for pediatric relapsing-remitting multiple sclerosis: two case reports. Zanetta, C., Filippi, M., Moiola, L.[2021]

A 14-year-old girl with pediatric-onset multiple sclerosis (POMS) switched from interferon β-1a to fingolimod after 12 months due to worsening symptoms, and she subsequently achieved clinical stability and minimal radiologic activity.

This case supports the effectiveness and safety of fingolimod as a second-line therapy for pediatric MS, aligning with previous studies that suggest it may be beneficial for patients who do not respond adequately to first-line treatments.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy of fingolimod after switching from interferon β-1a in an adolescent with multiple sclerosis: case report. Amidei, A., Siciliano, G., Pasquali, L.[2021]

In pediatric multiple sclerosis, treatment with fingolimod has shown a significant reduction in relapse rates, with the PARADIGMS trial reporting an 82% decrease compared to interferon β-1a.

A case study of a 13-year-old patient demonstrated that switching from interferon β-1a to fingolimod was necessary due to ongoing disease activity, highlighting the need for effective immunomodulatory treatments in young patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Two-year follow-up during fingolimod treatment in a pediatric multiple sclerosis patient still active on first-line treatment. Immovilli, P., Rota, E., Morelli, N., et al.[2021]

Fingolimod (Gilenya®) is currently the only FDA and EMA approved treatment for pediatric-onset multiple sclerosis (POMS), which accounts for 3-5% of all MS cases, highlighting its significance in managing this condition.

The drug has shown efficacy in adult-onset MS and has been evaluated in POMS through three observational studies and one pivotal clinical trial, indicating its potential effectiveness and safety in younger patients with this disease.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Review Of The Safety, Efficacy And Tolerability Of Fingolimod In The Treatment Of Pediatric Patients With Relapsing-Remitting Forms Of Multiple Sclerosis (RRMS). Feng, J., Rensel, M.[2020]

In a study involving 17 children and adolescents and 55 adults treated with beta-interferon-1a for at least one year, there was a significant reduction in multiple sclerosis exacerbations, with adolescents experiencing a drop from 1.35 to 0.06 and adults from 0.86 to 0.17.

The treatment was well-tolerated overall, although 47% of younger patients experienced flu-like symptoms, indicating a need for careful monitoring in this age group.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[The comparative study of efficacy and tolerability of intramuscular introduction of beta-interferon-1a in adults and adolescents with remitting multiple sclerosis]. Boĭko, AN., Batysheva, TT., Bykova, OV., et al.[2016]

Fingolimod is an effective oral treatment for relapsing forms of multiple sclerosis, showing significant reductions in relapse rates and MRI progression compared to placebo and other treatments like IFN-β.

While generally well tolerated, fingolimod can cause common side effects such as fatigue and headaches, and rare but serious risks like heart issues and infections, necessitating careful patient monitoring.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Overview and safety of fingolimod hydrochloride use in patients with multiple sclerosis. Ward, MD., Jones, DE., Goldman, MD.[2015]

Fingolimod (0.5 mg) is the first oral therapy approved for relapsing forms of multiple sclerosis (MS), showing a significant reduction in relapses by about 50% compared to placebo and intramuscular IFN-β1a over extensive clinical trials.

With over 63,000 MS patients monitored for more than 73,000 patient-years, fingolimod has established a well-characterized safety profile, with manageable side effects through proper patient monitoring.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Fingolimod for the treatment of relapsing multiple sclerosis. Singer, BA.[2015]

Fingolimod (Gilenya) is the first oral disease-modifying treatment approved for patients with highly active relapsing-remitting multiple sclerosis, marking a significant advancement in MS therapy.

This new treatment offers a novel mechanism of action by modulating the immune system, which may improve patient outcomes compared to traditional therapies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Fingolimod for multiple sclerosis. [2015]

References

1. Italy pubmed.ncbi.nlm.nih.gov

Fingolimod as an effective therapeutic strategy for pediatric relapsing-remitting multiple sclerosis: two case reports. [2021]

2. Italy pubmed.ncbi.nlm.nih.gov

Efficacy of fingolimod after switching from interferon β-1a in an adolescent with multiple sclerosis: case report. [2021]

3. Italy pubmed.ncbi.nlm.nih.gov

Two-year follow-up during fingolimod treatment in a pediatric multiple sclerosis patient still active on first-line treatment. [2021]

4. New Zealand pubmed.ncbi.nlm.nih.gov

Review Of The Safety, Efficacy And Tolerability Of Fingolimod In The Treatment Of Pediatric Patients With Relapsing-Remitting Forms Of Multiple Sclerosis (RRMS). [2020]

5. Russia (Federation) pubmed.ncbi.nlm.nih.gov

[The comparative study of efficacy and tolerability of intramuscular introduction of beta-interferon-1a in adults and adolescents with remitting multiple sclerosis]. [2016]

6. England pubmed.ncbi.nlm.nih.gov

Overview and safety of fingolimod hydrochloride use in patients with multiple sclerosis. [2015]

7. England pubmed.ncbi.nlm.nih.gov

Fingolimod for the treatment of relapsing multiple sclerosis. [2015]

8. England pubmed.ncbi.nlm.nih.gov

Fingolimod for multiple sclerosis. [2015]