A Phase 2 interventional study of Cannabidiol Oral Solution in Spasms, Infantile, sponsored by INSYS Therapeutics Inc. Terminated at 4 sites in United States. Open to participants aged 6 Months to 36 Months. Per ClinicalTrials.gov, last updated 2018-09-19.
Sponsored by INSYS Therapeutics Inc · Phase 2, Interventional, and Treatment
Infantile Spasms (IS) is a diagnosis described as a fairly rare and terrible form of epilepsy that usually strikes children in the first year of life. There is a great need for safe and effective therapies in the treatment of IS. This need is even more important for infants and toddlers still sick after being treated with medicine that is already available.
This is a multi-center study to evaluate the efficacy and safety of Cannabidiol Oral Solution (CBD) in the treatment of children aged 6 months through 36 months with a diagnosis of infantile spasms who have not responded to first line therapies.
The overall study duration is expected to be 64 weeks for those subjects who respond to CBD treatment. The maximum possible study duration for each patient is approximately 64 weeks, however a subject will be deemed to have completed the study after 58 weeks.
A protocol amendment in May 2016 created two parts to this trial: Part A (the extended treatment period) and Part B (the safety treatment period), whose objectives are as follows:
Primary Part A: To evaluate the efficacy of Cannabidiol Oral Solution in treating refractory infantile spasms (IS).
Secondary:
Part A:
Part B:
47 studies on the registry are indexed under Spasms, Infantile; 8 are open to participants now.
This study's enrollment of 9 is below the median of 40 across 33 interventional studies indexed under Spasms, Infantile.
Browse Spasms, Infantile studies →INSYS Therapeutics Inc is the lead sponsor of 45 studies on the registry; none are open to participants now.
Of its 5 completed or terminated interventional studies of FDA-regulated products, 3 (60%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Signs, symptoms or history of any condition that, per protocol or in the opinion of the investigator, might compromise:
During the Safety Treatment and Follow-up Periods, subjects are not to receive the following:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
The dose of Cannabidiol Oral Solution will begin at 20 mg/kg/day \[10 mg/kg twice per day (BID)\], will be adjusted at any time if the investigator feels the safety or well-being of the participant is at risk, and will be titrated up or down according to protocol-stipulated parameters and at the investigator's discretion after Day 14 to enhance efficacy. Dose will not exceed 40 mg/kg/day.
Drug: Cannabidiol Oral Solution
20 or 40 mg/kg/day BID
Part A: Percentage of Participants Who Are Considered Complete Responders at Day 14
Complete response was defined as complete resolution of spasms and hypsarrythmia (if present at baseline) confirmed by video-electroencephalogram (EEG) at Day 14.
Time frame: Day 14
Part B: Percentage of Participants Experiencing Adverse Events (AEs), Treatment-Emergent AEs (TEAEs), and Serious Adverse Events (SAEs)
Time frame: Up to Week 64
Part A: Percentage of Participants With Absence of Infantile Spasms at Day 14
Time frame: Day 14
Part A: Percentage of Participants With Absence of Hypsarrhythmia at Day 14
Time frame: Day 14
Part A: Median Reduction in Seizure-burden Comparing Video-EEG at Baseline to Repeat Video-EEG at Day 14
Time frame: Baseline, Day 14
Part A: Parent Impression of Efficacy and Tolerability of Study Drug
Parent impression of efficacy and tolerability, as measured by Clinical Global Impression-Global Improvement Scale (CGI-I), was summarized by visit and status of response (Complete/Partial and No Response) at Visit 3 (Day 14), Visit 4 (Week 4), Visit 5 (Week 8), Visit 6 (Week 10), and end of study. The CGI-I was also analyzed in a continuous scale, as follows: 1 = Very much improved, 2 = Much improved, 3 = Minimally improved, 4 = No change, 5 = Minimally worse, 6 = Much worse, and 7 = Very much worse
Time frame: Visit 3 (Day 14), Visit 4 (Week 4), Visit 5 (Week 8), Visit 6 (Week 10), and end of study.
Part A: Percentage of Participants With a Partial Response to Treatment
Partial response was defined as a substantive change in background EEG or reduction in spasms on video EEG obtained at Day 14.
Time frame: Day 14
Part A: Percentage of Complete Responders With Relapse
Complete response was defined as complete resolution of spasms and hypsarrythmia (if present at baseline) confirmed by video-EEG at Day 14.
Time frame: Day 14
Part A: Time to Complete Responder Relapse
Complete response was defined as complete resolution of spasms and hypsarrythmia (if present at baseline) confirmed by video-EEG at Day 14.
Time frame: Day 14
Part B: Parent Impression of Efficacy and Tolerability of Study Drug as Measured by the Change in Clinical Global Impression of Improvement Assessment (CGI-I), Responses at Every Visit Throughout Part B
Time frame: Up to Week 64
Part B: Investigator Impression of Efficacy and Tolerability of Study Drug as Measured by the Change in CGI-I Responses at Every Visit Throughout Part B
Time frame: Up to Week 64
Part B: Median Reduction in Seizure-burden Comparing Seizure Diaries Throughout Part B.
Time frame: Up to Week 64
Part B: Percentage of Participants Who Have a Relapse of Spasms Based on Video-EEG
Time frame: Up to Week 64
Part B: Time to Relapse as Confirmed by Video-EEG
Time frame: Up to Week 64
| Milestone | Cannabidiol Oral Solution: 20 or 40 mg/kg/Day BID |
|---|---|
| Started | 9 |
| Completed | 7 |
| Not completed | 2 |
| Withdrew: Withdrawal by subject | 2 |
Complete response was defined as complete resolution of spasms and hypsarrythmia (if present at baseline) confirmed by video-electroencephalogram (EEG) at Day 14.
| Percentage of participants | Cannabidiol Oral Solution: 20 or 40 mg/kg/Day BID |
|---|---|
| Part A: Percentage of Participants Who Are Considered Complete Responders at Day 14 | 14.3 |
| Percentage of participants | Cannabidiol Oral Solution: 20 or 40 mg/kg/Day BID |
|---|---|
| AEs | 44.4 |
| TEAEs | 33.3 |
| SAEs | 0.0 |
| Percentage of participants | Cannabidiol Oral Solution: 20 or 40 mg/kg/Day BID |
|---|---|
| Part A: Percentage of Participants With Absence of Infantile Spasms at Day 14 | 42.9 |
| Percentage of participants | Cannabidiol Oral Solution: 20 or 40 mg/kg/Day BID |
|---|---|
| Part A: Percentage of Participants With Absence of Hypsarrhythmia at Day 14 | 42.9 |
| Number of spasms | Cannabidiol Oral Solution: 20 or 40 mg/kg/Day BID |
|---|---|
| Visit 1(Baseline) | 0.0 (0 to 3) |
| Visit 3 (Day 14) | 9.0 (8 to 10) |
Parent impression of efficacy and tolerability, as measured by Clinical Global Impression-Global Improvement Scale (CGI-I), was summarized by visit and status of response (Complete/Partial and No Response) at Visit 3 (Day 14), Visit 4 (Week 4), Visit 5 (Week 8), Visit 6 (Week 10), and end of study. The CGI-I was also analyzed in a continuous scale, as follows: 1 = Very much improved, 2 = Much improved, 3 = Minimally improved, 4 = No change, 5 = Minimally worse, 6 = Much worse, and 7 = Very much worse
| Units on a scale | Cannabidiol Oral Solution: 20 or 40 mg/kg/Day BID |
|---|---|
| Visit 3 (Day 14) | 3.0 ± 1.0 |
| Visit 4 (Week 4) | 1.0 ± NA |
| Visit 5 (Week 8) | 1.0 ± NA |
| Visit 6 (Week 10) | 1.0 ± NA |
| Early Discontinuation/ End of Study | 4.0 ± NA |
Partial response was defined as a substantive change in background EEG or reduction in spasms on video EEG obtained at Day 14.
| Percentage of participants | Cannabidiol Oral Solution: 20 or 40 mg/kg/Day BID |
|---|---|
| Part A: Percentage of Participants With a Partial Response to Treatment | 0 |
Complete response was defined as complete resolution of spasms and hypsarrythmia (if present at baseline) confirmed by video-EEG at Day 14.
| Percentage of participants | Cannabidiol Oral Solution: 20 or 40 mg/kg/Day BID |
|---|---|
| Part A: Percentage of Complete Responders With Relapse | 0 |
Complete response was defined as complete resolution of spasms and hypsarrythmia (if present at baseline) confirmed by video-EEG at Day 14.
| Days | Cannabidiol Oral Solution: 20 or 40 mg/kg/Day BID |
|---|---|
| Part A: Time to Complete Responder Relapse | NA |
No measurements were reported for this outcome.
No measurements were reported for this outcome.
No measurements were reported for this outcome.
No measurements were reported for this outcome.
No measurements were reported for this outcome.
Collected over Up to 64 weeks.. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Cannabidiol Oral Solution: 20-40 mg/kg/Day BID | 0/9 (0%) | 0/9 (0%) | 4/9 (44.4%) |
| Event | Cannabidiol Oral Solution: 20-40 mg/kg/Day BID |
|---|---|
| Infantile SpasmsNervous system disorders | 3/9 |
| InfectionInfections and infestations | 1/9 |
| InfluenzaInfections and infestations | 1/9 |
| IrritabilityPsychiatric disorders | 1/9 |
| SedationNervous system disorders | 1/9 |
| Upper Respiratory Tract InfectionInfections and infestations | 1/9 |
| Age, Continuous(Months) | Cannabidiol Oral Solution: 20 or 40 mg/kg/Day BID |
|---|---|
| Mean | 23.0 ± 7.11 |
| Sex: Female, Male(Participants) | Cannabidiol Oral Solution: 20 or 40 mg/kg/Day BID |
|---|---|
| Female | 6 |
| Male | 3 |
| Ethnicity (NIH/OMB)(Participants) | Cannabidiol Oral Solution: 20 or 40 mg/kg/Day BID |
|---|---|
| Hispanic or Latino | 2 |
| Not Hispanic or Latino | 7 |
| Unknown or Not Reported | 0 |
| Race (NIH/OMB)(Participants) | Cannabidiol Oral Solution: 20 or 40 mg/kg/Day BID |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 1 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 0 |
| White | 7 |
| More than one race | 1 |
| Unknown or Not Reported | 0 |
Ran from Jan 2016 to primary completion in Sep 2016, 7 months.
Results were first posted in Sep 2018, 2 years after primary completion.
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This study is terminated, as verified in Aug 2018. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Cannabidiol