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

NCT02829827 | Terminated Phase 2 DMC

• 2 other identifiers: EP00782016-002107-26
• Study Type: interventional
• Target: 3
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of the study is to evaluate the safety and tolerability, the pharmacokinetics and the efficacy of radiprodil in abolishing clinical spasms in subjects with drug-resistant infantile spasms

Conditions

Infantile Spasms (IS)

Keywords

Radiprodil; Infantile spasms; IS; Infantile; Spasms; Epilepsy; Paediatrics; Drug-resistant; West Syndrome; Hypsarrhythmia

Outcome Measures

Primary Outcomes (4)

• Percentage of subjects with clinical response on Day 14 of treatment with the maintenance dose of radiprodil

  Clinical response is defined as no spasms on Day 14 of treatment with the maintenance dose of radiprodil. This is the primary efficacy variable for Part A.

  Day 14, counting from the first day of radiprodil at maintenance dose

• Estimates of exposure generated from a population-Pharmacokinetic modelling

  This is a primary variable for Part A.

  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

• Percentage of subjects with electro-clinical response on Day 14 of treatment with the maintenance dose of radiprodil

  Electro-clinical response is defined as no spasms and resolution of hypsarrythmia on Day 14 of treatment with the maintenance dose of radiprodil. This is the primary efficacy variable for Part B.

  Day 14, counting from the first day of radiprodil at maintenance dose

• Incidence of Adverse Events (AEs) during the study

  An AE is any untoward medical occurrence in a subject or trial subject that is administered a drug or biologic (medicinal product) or that is using a medical device. The event does not necessarily have a causal relationship with that treatment or usage. This is a primary variable for all parts.

  From Baseline (Day -1) to the end of the Post-treatment Period (28 days post last dosing)

Secondary Outcomes (14)

• Percentage of subjects with electro-clinical response on Day 14 of treatment with the maintenance dose of radiprodil

  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

  Day 14, counting from Day 14 of treatment with the maintenance dose of radiprodil

• Estimates of exposure generated from a population-Pharmacokinetic modelling

  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.

• Time to cessation of spasms

  During the first 14 days of treatment with radiprodil

• Percentage of responders with clinical relapse

  12 months, counting from Day 14 of treatment with the maintenance dose of radiprodil

Study Arms (1)

Radiprodil

EXPERIMENTAL

Each subject will enter an individualized dose titration schedule.

Drug: Radiprodil

Interventions

Radiprodil DRUG

Radiprodil at individualized doses.

Radiprodil

Eligibility Criteria

• Age: 2 Months - 14 Months
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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:

Sponsors & Collaborators

• UCB Biopharma S.P.R.L.: lead

Study Sites (1)

Ep0078 401

Paris, France

Location

MeSH Terms

Conditions

Spasms, Infantile; Spasm; Epilepsy

Interventions

radiprodil

Condition Hierarchy (Ancestors)

Epilepsy, Generalized; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Epileptic Syndromes; Neuromuscular Manifestations; Neurologic Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Study Officials

• UCB Cares

  +1 8445992273 (UCB)

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 8, 2016
• First Posted: July 12, 2016
• Study Start: December 4, 2017
• Primary Completion: October 2, 2018
• Study Completion: October 2, 2018
• Last Updated: September 17, 2019

Record last verified: 2019-09

Locations

FR (1)