A Phase 2 Study of Radiprodil in Subjects With Drug-resistant Infantile Spasms (IS)

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02829827
• Date of registration: 08/07/2016
• Prospective Registration: Yes
• Primary sponsor: UCB Biopharma S.P.R.L.
• Public title: A Phase 2 Study of Radiprodil in Subjects With Drug-resistant Infantile Spasms (IS)
• Scientific title: An Open-label Adaptive Study for the Assessment of Safety, Tolerability, Pharmacokinetics, and Efficacy of Multiple Doses of Radiprodil in Subjects With Drug-resistant Infantile Spasms
• Date of first enrolment: December 4, 2017
• Target sample size: 3
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT02829827
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Belgium, Bulgaria, France, Germany, United Kingdom

Contacts

• Name: UCB Cares
• Affiliation: [phone] (UCB)

Key inclusion & exclusion criteria

Inclusion Criteria:

Part A and B:

- Subject is male or female between 2 and 14 months of age

- The diagnosis of infantile spasms (IS)

- Subject has drug-resistant IS

Part C:

- Subject participated in EP0078 Part A and received 2 radiprodil treatment cycles

- Subject experienced a relapse of spasms during the down taper or within 5 half-lives
(3 days) discontinuation of radiprodil treatment in Cycle 2 of Part A

- Electroencephalogram (EEG) on baseline Part C is compatible with the diagnosis of
infantile spasms

Exclusion Criteria:

Part A and B:

- More than 6 months have passed since the diagnosis of Infantile Spasms (IS)

- Current treatment with cannabinoids

- Subject has hematocrit greater than 60

- Subject has any medical condition that, in the opinion of the Investigator, could
jeopardize or would compromise the subject's ability to participate in this study

- Subject has a history or current condition predisposing to respiratory dysfunction

- Current treatment with felbamate

- Current treatment with perampanel

- Ketogenic diet

- Clinically significant lab abnormalities

- Clinically significant abnormality on ECG that, in the opinion of the Investigator,
increases the safety risks of participating in the study

- Subject has a lethal or potentially lethal condition other than IS, with a significant
risk of death before 18 months of age such as non-ketotic hyperglycinemia

- Body weight is below 4 kg

- Known history of severe anaphylactic reaction secondary to medication intake or
serious blood dyscrasias

Part C:

- Subject experienced any acute tolerability issues in either treatment cycle in Part A
which the investigator and the sponsor medical monitor consider a risk for further
participation

- Subject met any withdrawal criteria in Part A

- Subject has experienced any adverse effects or developed any new medical conditions
since enrollment in Part A which the investigator considers could significantly
increase the safety risks of participating in Part C



Age minimum: 2 Months
Age maximum: 14 Months
Gender: All

Health Condition(s) or Problem(s) studied

Infantile Spasms (IS)

Intervention(s)

Drug: Radiprodil

Primary Outcome(s)

Incidence of Adverse Events (AEs) during the study [Time Frame: From Baseline (Day -1) to the end of the Post-treatment Period (28 days post last dosing)]

Percentage of subjects with electro-clinical response on Day 14 of treatment with the maintenance dose of radiprodil [Time Frame: Day 14, counting from the first day of radiprodil at maintenance dose]

Percentage of subjects with clinical response on Day 14 of treatment with the maintenance dose of radiprodil [Time Frame: Day 14, counting from the first day of radiprodil at maintenance dose]

Estimates of exposure generated from a population-Pharmacokinetic modelling [Time Frame: Samples will be taken at baseline (time during Day -14 to -1 prior to dosing) and 3, 4, 5 & 12hr after the 1st dose on Day 1 of radiprodil low, mid & high dose. Blood samples will be taken at same timepoints after 1st dose on Day 2 of radiprodil low dose]

Secondary Outcome(s)

Percentage of subjects with extended electro-clinical response [Time Frame: 28 days, counting from Day 14 (inclusive) of treatment with the maintenance dose of radiprodil]

Number of treatment cycles per subject [Time Frame: During Part C (Day -1 to Day 28 of the Maintenance Period)]

Percentage of subjects with extended clinical response [Time Frame: 28 days, counting from Day 14 (inclusive) of treatment with the maintenance dose of radiprodil]

Percentage of subjects with electro-clinical response on Day 14 of treatment with the maintenance dose of radiprodil [Time Frame: Day 14, counting from the first day of radiprodil at maintenance dose]

Percentage of electro-clinical responders with electro-clinical relapse [Time Frame: 12 months, counting from Day 14 of treatment with the maintenance dose of radiprodil]

Percentage of subjects with clinical response on Day 14 of treatment with the maintenance dose of radiprodil [Time Frame: Day 14, counting from Day 14 of treatment with the maintenance dose of radiprodil]

Time to clinical relapse from the first day of no witnessed spasms for each treatment cycle [Time Frame: From day of no witnessed spasms up to 42 months of age]

Percentage of responders with clinical relapse [Time Frame: 12 months, counting from Day 14 of treatment with the maintenance dose of radiprodil]

Time to clinical relapse from the day of spasm cessation [Time Frame: From day of spasms cessation up to 42 months of age]

Time to electro-clinical relapse from the day of spasm cessation [Time Frame: From day of spasms cessation up to 42 months of age]

Time to cessation of spasms [Time Frame: During the first 14 days of treatment with radiprodil]

Estimates of exposure generated from a population-Pharmacokinetic modelling [Time Frame: Pharmacokinetic samples will be collected on Day 1 of radiprodil low dose, mid dose and high dose. Additionally, blood samples will be taken after 1st dose on Day 2 of radiprodil low dose.]

Percentage of subjects with extended clinical response to each additional treatment cycle on Day 14 of treatment with the maintenance dose of radiprodil [Time Frame: 28 days, counting from Day 14 (inclusive) of maintenance dose]

Percentage of subjects with electro-clinical response to each additional treatment cycle on Day 14 of treatment with the maintenance dose of radiprodil [Time Frame: Day 14, counting from the first day of maintenance dose]

Secondary ID(s)

2016-002107-26

EP0078

Source(s) of Monetary Support

Please refer to primary and secondary sponsors