Fingolimod for Pediatric Multiple Sclerosis

Not currently recruiting at 96 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the safety and effectiveness of two treatments, fingolimod and interferon beta-1a, for children with multiple sclerosis (MS), a condition where the immune system attacks the protective covering of nerves. Fingolimod is taken as a daily pill, while interferon beta-1a is administered as a weekly injection. The trial compares how these treatments reduce MS symptoms in children. Children who have experienced at least one MS flare-up in the past year or have specific MRI findings might be suitable for this study. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to potentially groundbreaking treatments for 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.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research shows that fingolimod is generally well-tolerated in children with relapsing forms of multiple sclerosis (MS). One study found it effective and safe for children aged 10 to 18, with a daily dose of 0.5 mg for those weighing over 40 kg. While side effects can occur, they are usually mild. Common ones include headaches, flu-like symptoms, and back pain.

The FDA has approved fingolimod for treating MS in both adults and children, supporting its safety. However, individual experiences can vary, and some people might experience different or more severe side effects.

Research indicates that interferon beta-1a is also generally safe, though it can cause flu-like symptoms after injections. These symptoms usually decrease over time. Both treatments have been used for MS, and their safety is well established. Always consult a healthcare provider about potential risks and benefits before joining a trial. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for MS?

Fingolimod is unique because it's taken as an oral capsule, offering a more convenient alternative to the regular injections required by many existing multiple sclerosis treatments, like interferon beta-1a. This oral administration can be especially beneficial for children, simplifying their treatment routine. Moreover, fingolimod works by targeting specific receptors on immune cells, helping to prevent these cells from attacking the nervous system — a different approach compared to standard therapies. Researchers are excited about fingolimod because it has shown promise in providing effective disease management with potentially fewer side effects, improving the quality of life for pediatric patients.

What evidence suggests that this trial's treatments could be effective for pediatric multiple sclerosis?

Research has shown that fingolimod, a treatment in this trial, effectively treats multiple sclerosis in children. One study found that fingolimod reduced relapses more effectively than interferon beta-1a, another treatment option in this trial. Another study showed that twice as many children on fingolimod experienced significant improvements in their quality of life compared to those on interferon beta-1a. Fingolimod works by adjusting the immune system, helping to prevent attacks on the nervous system. These findings suggest that fingolimod is a promising option for children with multiple sclerosis. ^{2 6 7 8 9}

Who Is on the Research Team?

Novartis Pharmaceuticals

Principal Investigator

Novartis Pharmaceuticals

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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

Published Research Related to This Trial

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]

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®) 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]

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]

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 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]

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 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]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10170592/

Narrative review based on fingolimod therapy in pediatric MS A recent study from Turkey found higher rates: 13% of the patients were under fingolimod treatment, amounting to 38% of the nDMD users.

2. tascenso.com tascenso.com/hcp/what-is-tascenso-odt/bioequivalence/

Efficacy and Safety TASCENSO ODT is a sphingosine 1-phosphate receptor modulator indicated for the treatment of relapsing forms of multiple sclerosis (MS).

3. clinicaltrials.gov clinicaltrials.gov/study/NCT01892722

Safety and Efficacy of Fingolimod in Pediatric Patients With ... Safety and Efficacy of Fingolimod in Pediatric Patients With Multiple Sclerosis. ClinicalTrials.gov ID NCT01892722. Sponsor Novartis Pharmaceuticals.

4. nejm.org nejm.org/doi/full/10.1056/NEJMoa1800149

Trial of Fingolimod versus Interferon Beta-1a in Pediatric ... This double-blind, active-comparator trial showed superior efficacy of fingolimod over interferon beta-1a in reducing relapses in children and ...

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC8883212/

Effect of fingolimod on health-related quality of life in ... Overall, a twofold higher proportion of patients achieved clinically meaningful improvement in the PedsQL Total Scale Score with fingolimod versus IFN β-1a per ...

6. fda.gov fda.gov/media/181806/download

Gilenya Pediatric Postmarketing Safety Review This review evaluates FDA Adverse Event Reporting System (FAERS) reports for Gilenya. (fingolimod) capsules and Tascenso ODT (fingolimod) ...

7. accessdata.fda.gov accessdata.fda.gov/drugsatfda_docs/label/2022/214962s000lbl.pdf

TASCENSO ODT (fingolimod) - accessdata.fda.gov Pediatric Use. Safety and effectiveness of fingolimod for the treatment of relapsing forms of multiple sclerosis in pediatric patients 10 to less than. 18 ...

8. mayoclinic.org mayoclinic.org/drugs-supplements/fingolimod-oral-route/description/drg-20074478

Fingolimod (oral route) - Side effects & dosage For multiple sclerosis: Adults and children 10 years of age and older weighing more than 40 kilograms (kg)—0.5 milligrams (mg) once a day.

9. tascenso.com tascenso.com/patient/what-is-tascenso-odt/odt/

Orally Disintegrating Tablet (ODT) TASCENSO ODT is a prescription medicine used to treat relapsing forms of multiple sclerosis (MS), to include clinically isolated syndrome, relapsing-remitting ...