Save

Safety and Efficacy of Fingolimod in Pediatric Patients With Multiple Sclerosis

PHASE3RECRUITING

To evaluate the safety and efficacy of fingolimod vs. interferon beta-1a i. m.

in pediatric patients with multiple sclerosis (MS).

Simpliy with AI

Study details:

The study is divided into a Core Phase, which includes the Double-Blind Treatment Period, and an Extension Phase in which all patients will be treated with fingolimod. The Core Phase is a 24-month, double-blind, randomized, active-controlled, parallel-group multicenter study phase to evaluate the efficacy and safety of fingolimod compared to IFN β-1a in children/adolescent patients aged 10-17 years old with MS. The Extension Phase is a 60-month (5 year) study phase for patients who complete the Core Phase of the study and meet all inclusion/exclusion criteria and for patients who will be recruited in the younger cohort to participate in the Extension Phase.

The 'younger cohort' refers to the population of pediatric patients fulfilling 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).

The recruitment of the younger cohort (up to 25 patients) was requested as a post- approval health authority commitment.

Simplify with AI

Eligibility criteria

Researchers look for people who fit a certain description, called eligibility criteria. See if you qualify.

Inclusion criteria

diagnosis of multiple sclerosis

at least one MS relapse during the previous year or two MS relapses in the previous 2 years or evidence of Gd enhancing lesions on MRI within 6 months EDSS score of 0 to 5.5, inclusive

All newly recruited patients' that enroll directly into the Extension Phase must fulfill the local country health authority product label approved for pediatric age group for inclusion criteria

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

Exclusion criteria

patients with progressive MS

patients with an active, chronic disease of the immune system other than MS

patients meeting the definition of ADEM

patients with severe cardiac disease or significant findings on the screening ECG

patients with severe renal insufficiency

Premature discontinuation of the study drug during the Core Phase due to: an adverse event, serious adverse event, laboratory abnormality, other conditions leading to permanent study drug discontinuation due to safety reasons

Patients with known new events or concomitant medications (washout periods required prior to Visit 15) that would exclude them from the Core Phase exclusion criteria

All newly recruited patients in the younger cohort that enroll directly into the Extension Phase must fulfill the exclusion criteria for the core phase

Simplify with AI

Eligibility

Age eligible for study : 10 and older

Healthy volunteers accepted : No

Gender eligible for study: All

Things to know

Study dates

Study start: 2013-07-26

Primary completion: 2017-07-14

Study completion finish: 2029-07-12

Study type

TREATMENT

Phase

PHASE3

Trial ID

NCT01892722

Intervention or treatment

DRUG: Interferon beta-1a

DRUG: Fingolimod

DRUG: Placebo capsule

DRUG: Placebo i.m. injection

Conditions

• Multiple Sclerosis

Condition: Multiple Sclerosis
DRUG: Interferon beta-1a and other drugs
Parkville, Victoria, Australia
Sponsor: Novartis Pharmaceuticals

You may be eligible to participate in this trial based on your search. Check eligibility by answering some questions.

Are you running this trial? If you're a clinic or sponsor, you can claim this study. Claim or learn more.

Find a site

Closest Location:

Novartis Investigative Site

Research sites nearby

Select from list below to view details:

Novartis Investigative Site
Parkville, Victoria, Australia

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Participant Group/Arm	Intervention/Treatment
EXPERIMENTAL: Fingolimod 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	DRUG: Fingolimod Administrated orally once daily: 0.5 mg capsule for patients over 40 kg or 0.25 mg capsule for patients 40 kg or less.
ACTIVE_COMPARATOR: Interferon beta-1a 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	DRUG: Interferon beta-1a Administration once weekly via i.m. injections.
EXPERIMENTAL: Fingolimod-Younger Cohort 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)	DRUG: Fingolimod Administrated orally once daily: 0.5 mg capsule for patients over 40 kg or 0.25 mg capsule for patients 40 kg or less.

What is the study measuring?

Primary outcome

Primary Outcome Measure	Primary Outcome Description	Primary Outcome Time Frame
Frequency of Relapses in Patients Treated for up to 24 Months	Frequency of relapses assessed by the annualized relapse rate (ARR). The ARR is defined as the average number of confirmed relapses per year (total number of confirmed relapses divided by the total days in the study multiplied by 365.25).	24 months

Secondary outcome

Secondary Outcome Measure	Secondary Outcome Description	Secondary Outcome Time Frame
New/Newly Enlarged T2 Lesions	Annualized rate of the number of new/newly enlarged T2 lesions up to Month 24	24 months
Time to First Relapse	Time to first relapse was determined.	24 months
Proportion of Patients Relapse-free	Proportion of patients relapse-free was determined	24 months
T1 Gd- Enhancing Lesions	Number of T1 Gd-enhancing lesions per scan up to Month 24	24 months
Pharmacokinetics (Cavg) of Fingolimod-P	Cavg (average drug concentration over the dose interval) will be evaluated.	24 months
Pharmacokinetic/Pharmacodynamic Relationship for Fingolimod-P to Lymphocyte Levels	Population PK/PD modeling approaches were used to relate the individual fingolimod-P concentrations to lymphocyte counts.	24 months

Frequently Asked Questions

Please note: some questions and answers are submitted by anonymous patients or using AI, and have not been verified by Clinrol

No questions submitted. Be the first to ask a question!

You may be eligible to participate in this trial based on your search.Apply for study

Are you running this trial? If you're a clinic or sponsor, you can claim this study.Claim this trial

References

Clinical Trials Gov: Safety and Efficacy of Fingolimod in Pediatric Patients With Multiple Sclerosis