Infantile SpasmPhase 3NCT03421496
A Study to Assess Cannabidiol Oral Solution With Vigabatrin as Initial Therapy in Participants With Infantile Spasms
A Phase 3 study testing Cannabidiol Oral Solution and Vigabatrin in people with Infantile Spasm, run by Radius Pharmaceuticals, Inc. at 5 sites in United States.
Trial summary
The primary purpose of this study was to evaluate the efficacy, safety, and tolerability of Cannabidiol Oral Solution (CBD) as adjunctive therapy with vigabatrin as initial therapy, compared to vigabatrin alone in the treatment of infants newly diagnosed with Infantile Spasms (IS). Read more about this study
- Who is it for?
- Ages 1 month to 24 months
- Conditions the study is for
- Infantile Spasm
- What is being tested?
- Cannabidiol Oral Solution and Vigabatrin
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 2 participants
- Where
- 5 sitesUnited States
- Study dates
- 5 Sep 2018to 29 May 2019
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 1 month to 24 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Parent(s)/caregiver(s) fully comprehends and signs the informed consent form, understands all study procedures, and can communicate satisfactorily with the Investigator and study coordinator, in accordance with applicable laws, regulations, and local requirements.
- Clinical diagnosis of Infantile Spasms, confirmed by video-EEG (including at least one cluster of electroclinical spasms [≥3 in any 10-minute epoch] and hypsarrythmia) obtained during the Screening Period and read by a central reader.
- General good health (defined as the absence of any clinically relevant abnormalities as determined by the Investigator) based on physical and neurological examinations, medical history, and clinical laboratory values completed during the Screening Visit).
- In the opinion of the investigator, the parent(s)/caregiver(s) is (are) willing and able to comply with the study procedures and visit schedules.
Who cannot join
- Is considered by the investigator, for any reason (including, but not limited to, the risks described as precautions, warnings, and contraindications in the current version of the Investigator's Brochure for Cannabidiol Oral Solution) to be an unsuitable candidate to receive the study drug.
- Known or suspected allergy to cannabidiol.
- History of an allergic reaction or a known or suspected sensitivity to any substance that is contained in the investigational product formulation.
- Use of any cannabidiol/cannabis product within 30 days of study entry.
- Participant is diagnosed or suspected of having tuberous sclerosis.
- Participant has received treatment with either vigabatrin, ACTH, or high-dose steroids previously.
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- Previous or concomitant therapy with felbamate, clobazam, valproic acid, or the ketogenic diet.
- Participant currently on any disallowed CYP3A4-related medication (phenytoin, fluvoxamine, carbamazepine, and St. John's Wort).
- Previously received any investigational drug or device or investigational therapy within 30 days before Screening.
- Clinically significant abnormal laboratory values, including: liver function tests (LFTs) such as albumin, direct bilirubin, total bilirubin, aspartate aminotransferase (AST), and alanine aminotransferase (ALT) ≥3 times the upper limit of normal (ULN). The investigator may deem the participant eligible if he or she judges the laboratory values to be not clinically significant.
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
United States5 sites
Akron Children's Hospital
Akron, Ohio, 44308
Nicklaus Children's Hospital
Miami, Florida, 33155
Oregon Health & Science University
Portland, Oregon, 97239
Beaumont Children's Hospital
Royal Oak, Michigan, 48073
Institute for Research and Innovation | MultiCare Health System
Tacoma, Washington, 98405
About this study
Official title: A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Assess the Efficacy, Safety, and Tolerability of Cannabidiol Oral Solution as Adjunctive Therapy With Vigabatrin as Initial Therapy in Patients With Infantile Spasms
The primary purpose of this study was to evaluate the efficacy, safety, and tolerability of Cannabidiol Oral Solution (CBD) as adjunctive therapy with vigabatrin as initial therapy, compared to vigabatrin alone in the treatment of infants newly diagnosed with Infantile Spasms (IS).
Read the detailed description
This was a randomized, double-blind, placebo-controlled, parallel-group study in which participants were randomized in a 1:1 ratio to 1 of 2 treatment groups. During the Initial Treatment Period, participants received either vigabatrin plus CBD or vigabatrin plus matching placebo and were dosed approximately every 12 hours, with a meal. This study was comprised of five periods: Screening, Initial Treatment, Extended Treatment, Taper, and Follow up Periods, with a maximum duration of approximately 140 days.
- Conditions
- Infantile Spasm
- Being tested
- Cannabidiol Oral Solution, Placebo, Vigabatrin
- Keywords
- Infantile spasm, Vigabatrin, Cannabidiol oral solution
- Medical subject headings
- Spasms, Infantile
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Cannabidiol Oral SolutionDrug
An oral solution containing pharmaceutical grade cannabidiol (nonplant-based).
PlaceboDrug
Matching oral solution
VigabatrinDrug
Also called Sabril
Powder suspension
| Study group | Role | What they receive |
|---|---|---|
| CBD with Vigabatrin | Experimental | Participants received up to 40 milligrams per kilogram per day (mg/kg/day) divided twice daily (BID) of CBD with food. Participants also received up to 150 mg/kg/day BID of vigabatrin with food. Receives: Drug: Cannabidiol Oral Solution; Drug: Vigabatrin |
| Placebo with Vigabatrin | Placebo comparator | Participants received a matching placebo to CBD with food, and also received up to 150 mg/kg/day BID of vigabatrin with food. Receives: Drug: Placebo; Drug: Vigabatrin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Participants Considered Complete Responders
Complete response is defined as complete resolution of spasms and hypsarrhythmia confirmed by 24-hour video-electroencephalogram (EEG).
Measured: Up to Day 15
Secondary outcomes (6)
Percentage of Participants With Resolution of Infantile Spasms
Resolution of IS was assessed by 24-hour video-EEG.
Measured: Up to Day 15
Percentage of Participants With Resolution of Hypsarrhythmia
Resolution of hypsarrhythmia was assessed by 24-hour video-EEG.
Measured: Up to Day 15
Investigator Impression of Efficacy and Tolerability of Study Drug Clinical Global Impression- Global Improvement (CGI-I)
Investigators will use the CGI-I scale, which is a 7-point scale that requires the clinician to assess how much the participant's illness has improved or worsened relative to a baseline state at the beginning of the intervention and is rated as: 1=very much improved; 2=much improved; 3=minimally improved; 4=no change; 5=minimally worse; 6=much worse; 7=very much worse. Higher scores indicated worse condition.
Measured: Day 15
Percentage of Participants With Increase in Number of Spasm-Free Days Between Day 1 and Day 15
Increase in spasm-free days will be determined by seizure diary entries.
Measured: Up to Day 15
Percentage of Participants With Complete Response During the Initial Treatment Period Who Relapse During the Extended Treatment Period
Relapse during the extended treatment period will be confirmed by video-EEG following parent report of relapse.
Measured: Up to Day 75
Time to Relapse During the Extended Treatment Period
Measured: Up to Day 75
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Feb 2018
- Study start5 Sep 2018
- Primary completion29 May 2019
- Study completion29 May 2019
Why it stopped: The study was terminated due to slow enrollment and failure to identify adequate patients that met entry criteria.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Infantile SpasmStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- Radius Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03421496
- Sponsor's protocol ID
- INS011-16-082
- Status last verified
- May 2023
- First submitted
- 26 Jan 2018
- Last updated
- 7 Jun 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Jun 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Assess the Efficacy, Safety, and Tolerability of Cannabidiol Oral Solution as Adjunctive Therapy With Vigabatrin as Initial Therapy in Patients With Infantile Spasms
- ClinicalTrials.gov ID
- NCT03421496
- Acronym / sponsor ID
- INS011-16-082
- Conditions
- Infantile Spasm
- Keywords
- Infantile spasm, Vigabatrin, Cannabidiol oral solution
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: The study was terminated due to slow enrollment and failure to identify adequate patients that met entry criteria.
- Enrolment
- 2 participants (actual)
- Lead sponsor
- Radius Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 5 Sep 2018 (actual)
- Primary completion
- 29 May 2019 (actual)
- Study completion
- 29 May 2019 (actual)
- First posted
- 5 Feb 2018 (actual)
- Results
- Posted 7 Jun 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The primary purpose of this study was to evaluate the efficacy, safety, and tolerability of Cannabidiol Oral Solution (CBD) as adjunctive therapy with vigabatrin as initial therapy, compared to vigabatrin alone in the treatment of infants newly diagnosed with Infantile Spasms (IS).
Detailed description
This was a randomized, double-blind, placebo-controlled, parallel-group study in which participants were randomized in a 1:1 ratio to 1 of 2 treatment groups. During the Initial Treatment Period, participants received either vigabatrin plus CBD or vigabatrin plus matching placebo and were dosed approximately every 12 hours, with a meal. This study was comprised of five periods: Screening, Initial Treatment, Extended Treatment, Taper, and Follow up Periods, with a maximum duration of approximately 140 days.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| CBD with Vigabatrin | Experimental | Drug: Cannabidiol Oral Solution, Drug: VigabatrinParticipants received up to 40 milligrams per kilogram per day (mg/kg/day) divided twice daily (BID) of CBD with food. Participants also received up to 150 mg/kg/day BID of vigabatrin with food. |
| Placebo with Vigabatrin | Placebo comparator | Drug: Placebo, Drug: VigabatrinParticipants received a matching placebo to CBD with food, and also received up to 150 mg/kg/day BID of vigabatrin with food. |
Who may qualify
- Ages
- 1 month to 24 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- Parent(s)/caregiver(s) fully comprehends and signs the informed consent form, understands all study procedures, and can communicate satisfactorily with the Investigator and study coordinator, in accordance with applicable laws, regulations, and local requirements.
- Clinical diagnosis of Infantile Spasms, confirmed by video-EEG (including at least one cluster of electroclinical spasms [≥3 in any 10-minute epoch] and hypsarrythmia) obtained during the Screening Period and read by a central reader.
- General good health (defined as the absence of any clinically relevant abnormalities as determined by the Investigator) based on physical and neurological examinations, medical history, and clinical laboratory values completed during the Screening Visit).
- In the opinion of the investigator, the parent(s)/caregiver(s) is (are) willing and able to comply with the study procedures and visit schedules.
Key exclusion criteria (10 listed)
- Is considered by the investigator, for any reason (including, but not limited to, the risks described as precautions, warnings, and contraindications in the current version of the Investigator's Brochure for Cannabidiol Oral Solution) to be an unsuitable candidate to receive the study drug.
- Known or suspected allergy to cannabidiol.
- History of an allergic reaction or a known or suspected sensitivity to any substance that is contained in the investigational product formulation.
- Use of any cannabidiol/cannabis product within 30 days of study entry.
- Participant is diagnosed or suspected of having tuberous sclerosis.
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 |
|---|---|---|
| Cannabidiol Oral SolutionAn oral solution containing pharmaceutical grade cannabidiol (nonplant-based). | Drug | CBD with Vigabatrin |
| PlaceboMatching oral solution | Drug | Placebo with Vigabatrin |
| VigabatrinAlso: SabrilPowder suspension | Drug | CBD with Vigabatrin, Placebo with Vigabatrin |
Primary outcome
Percentage of Participants Considered Complete Responders
Time frame: Up to Day 15
Complete response is defined as complete resolution of spasms and hypsarrhythmia confirmed by 24-hour video-electroencephalogram (EEG).
6 secondary outcomes
Percentage of Participants With Resolution of Infantile Spasms
Time frame: Up to Day 15
Percentage of Participants With Resolution of Hypsarrhythmia
Time frame: Up to Day 15
Investigator Impression of Efficacy and Tolerability of Study Drug Clinical Global Impression- Global Improvement (CGI-I)
Time frame: Day 15
Percentage of Participants With Increase in Number of Spasm-Free Days Between Day 1 and Day 15
Time frame: Up to Day 15
Percentage of Participants With Complete Response During the Initial Treatment Period Who Relapse During the Extended Treatment Period
Time frame: Up to Day 75
Time to Relapse During the Extended Treatment Period
Time frame: Up to Day 75
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Jun 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
The record lists no contact. Use the official record to reach the sponsor.
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Cannabidiol Oral Solution and Vigabatrin in people with Infantile Spasm, run by Radius Pharmaceuticals, Inc. at 5 sites in United States.
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 4 inclusion and 10 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 7 Jun 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States. 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. Some participants in this study may receive a placebo. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 7 Jun 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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 Participants Considered Complete Responders (time frame: Up to Day 15). 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 7 Jun 2023.