Infantile Spasms (IS)Phase 2NCT02829827
A Phase 2 Study of Radiprodil in Subjects With Drug-resistant Infantile Spasms (IS)
A Phase 2 study testing Radiprodil in people with Infantile Spasms (IS), run by UCB Biopharma S.P.R.L. at 1 site in France.
Trial 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 Read more about this study
- Who is it for?
- Ages 2 to 14 months
- Conditions the study is for
- Infantile Spasms (IS)
- What is being tested?
- Radiprodil
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- 1 siteFrance
- Study dates
- 4 Dec 2017to 2 Oct 2018
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 14 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
Who cannot join
- 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
Show all 18Show fewer
- 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
Trial locations and contacts
1 site in 1 country.
About this study
Official 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
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
Read the detailed description
The study is divided into 3 parts:
Part A - exploratory, Part B - confirmatory, Part C - open label extension
- Conditions
- Infantile Spasms (IS)
- Being tested
- Radiprodil
- Keywords
- Radiprodil, Infantile spasms, IS, Infantile, Spasms, Epilepsy, Paediatrics, Drug-resistant, West Syndrome, Hypsarrhythmia
- Medical subject headings
- Spasms, Infantile, Spasm, Epilepsy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RadiprodilDrug
Radiprodil at individualized doses.
| Study group | Role | What they receive |
|---|---|---|
| Radiprodil | Experimental | Each subject will enter an individualized dose titration schedule. Receives: Drug: Radiprodil |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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
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 secondary efficacy variable for Part A.
Measured: 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
Clinical response is defined as no spasms on Day 14 of treatment with the maintenance dose of radiprodil. This is the secondary efficacy variable for Part B.
Measured: Day 14, counting from Day 14 of treatment with the maintenance dose of radiprodil
Estimates of exposure generated from a population-Pharmacokinetic modelling
This is a secondary variable for Part B.
Measured: 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
Time to cessation of spasms for clinical responders on Day 14 of treatment with the maintenance dose of radiprodol. This is a secondary efficacy variable for parts A and B.
Measured: During the first 14 days of treatment with radiprodil
Percentage of responders with clinical relapse
The percentage of clinical responders on Day 14 of treatment with the maintenance dose of radiprodil with clinical relapse within 12 months. This is a secondary efficacy variable for parts A and B.
Measured: 12 months, counting from Day 14 of treatment with the maintenance dose of radiprodil
Time to clinical relapse from the day of spasm cessation
This is a secondary efficacy variable for parts A and B.
Measured: From day of spasms cessation up to 42 months of age
Percentage of electro-clinical responders with electro-clinical relapse
The percentage of electro-clinical responders on Day 14 of treatment with the maintenance dose of radiprodil with electro-clinical relapse within 12 months. This is a secondary efficacy variable for parts A and B.
Measured: 12 months, counting from Day 14 of treatment with the maintenance dose of radiprodil
Time to electro-clinical relapse from the day of spasm cessation
This is a secondary efficacy variable for parts A and B.
Measured: From day of spasms cessation up to 42 months of age
Percentage of subjects with extended clinical response
Extended clinical response is defined as no spasms for 28 consecutive days from Day 14 of treatment with the maintenance dose of radiprodil. This is a secondary efficacy variable for parts A and B.
Measured: 28 days, counting from Day 14 (inclusive) of treatment with the maintenance dose of radiprodil
Percentage of subjects with extended electro-clinical response
Extended electro-clinical response is defined as no spasms and resolution of hypsarrythmia for 28 consecutive days from Day 14 of treatment with the maintenance dose of radiprodil. This is a secondary efficacy variable for parts A and B.
Measured: 28 days, counting from Day 14 (inclusive) of treatment with the maintenance dose of radiprodil
Percentage of subjects with extended clinical response to each additional treatment cycle on Day 14 of treatment with the maintenance dose of radiprodil
Extended clinical response is defined as no spasms for 28 consecutive days from Day 14 of treatment with the maintenance dose of radiprodil. This is a secondary efficacy variable for part C.
Measured: 28 days, counting from Day 14 (inclusive) of maintenance dose
Number of treatment cycles per subject
This is a secondary variable for Part C.
Measured: During Part C (Day -1 to Day 28 of the Maintenance Period)
Percentage of subjects with electro-clinical response to each additional treatment cycle 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 a secondary efficacy variable for Part C.
Measured: Day 14, counting from the first day of maintenance dose
Time to clinical relapse from the first day of no witnessed spasms for each treatment cycle
This is a secondary efficacy variable for part C.
Measured: From day of no witnessed spasms up to 42 months of age
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Jul 2016 (estimated)
- Study start4 Dec 2017
- Primary completion2 Oct 2018
- Study completion2 Oct 2018
Why it stopped: After reviewing the feasibility and projected completion date of the study, UCB has made the decision to stop the study
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Infantile Spasms (IS)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Lead sponsor
- UCB Biopharma S.P.R.L.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- UCB Cares, Study director, +1 8445992273 (UCB)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02829827
- Sponsor's protocol ID
- EP0078
- Other IDs
- 2016-002107-26, 2016
- Status last verified
- Sep 2019
- First submitted
- 8 Jul 2016
- Last updated
- 17 Sep 2019
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Sep 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT02829827
- Acronym / sponsor ID
- EP0078
- Conditions
- Infantile Spasms (IS)
- Keywords
- Radiprodil, Infantile spasms, IS, Infantile, Spasms, Epilepsy, Paediatrics, Drug-resistant
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: After reviewing the feasibility and projected completion date of the study, UCB has made the decision to stop the study
- Enrolment
- 3 participants (actual)
- Lead sponsor
- UCB Biopharma S.P.R.L.Industry (pharma, biotech, devices)
- Study start
- 4 Dec 2017 (actual)
- Primary completion
- 2 Oct 2018 (actual)
- Study completion
- 2 Oct 2018 (actual)
- First posted
- 12 Jul 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Detailed description
The study is divided into 3 parts:
Part A - exploratory, Part B - confirmatory, Part C - open label extension
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Radiprodil | Experimental | Drug: RadiprodilEach subject will enter an individualized dose titration schedule. |
Who may qualify
- Ages
- 2 to 14 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (8 listed)
- Subject is male or female between 2 and 14 months of age
- The diagnosis of infantile spasms (IS)
- Subject has drug-resistant IS
- 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
Key exclusion criteria (18 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RadiprodilRadiprodil at individualized doses. | Drug | Radiprodil |
Primary outcome
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
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.
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
This is a primary variable for Part A.
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
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.
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)
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.
14 secondary outcomes
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 Day 14 of treatment with the maintenance dose of 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.
Time to cessation of spasms
Time frame: During the first 14 days of treatment with radiprodil
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
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
Time to electro-clinical relapse from the day of spasm cessation
Time frame: From day of spasms cessation up to 42 months of age
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 extended electro-clinical response
Time frame: 28 days, counting from Day 14 (inclusive) of treatment with the maintenance dose of radiprodil
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
Number of treatment cycles per subject
Time frame: During Part C (Day -1 to Day 28 of the Maintenance Period)
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
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
Results and safety
The study was stopped early (reason given: After reviewing the feasibility and projected completion date of the study, UCB has made the decision to stop the study). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Sep 2019.
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
UCB Cares study director
+1 8445992273 (UCB)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Radiprodil in people with Infantile Spasms (IS), run by UCB Biopharma S.P.R.L. at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 17 Sep 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: After reviewing the feasibility and projected completion date of the study, UCB has made the decision to stop the study). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Sep 2019.