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Trial record 10 of 27 for:    "X-linked infantile spasm syndrome"

A Study to Assess the Efficacy, Safety, and Tolerability of Cannabidiol Oral Solution With Vigabatrin as Initial Therapy in Participants With Infantile Spasms

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ClinicalTrials.gov Identifier: NCT03421496
Recruitment Status : Recruiting
First Posted : February 5, 2018
Last Update Posted : September 13, 2018
Sponsor:
Information provided by (Responsible Party):
INSYS Therapeutics Inc

Brief Summary:
The primary objective of this study is to evaluate the efficacy of Cannabidiol Oral Solution as adjunctive therapy with vigabatrin as initial therapy in treating participants with Infantile Spasms. The secondary objectives for this study are to evaluate the continued efficacy of Cannabidiol Oral Solution after the 14-day treatment with vigabatrin or vigabatrin plus Cannabidiol Oral Solution is complete and to evaluate the safety and tolerability of Cannabidiol Oral Solution as adjunctive therapy with vigabatrin as initial therapy in treating participants with infantile spasms.

Condition or disease Intervention/treatment Phase
Infantile Spasm Drug: Cannabidiol Oral Solution Drug: Placebo Drug: Vigabatrin Phase 3

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 190 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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
Actual Study Start Date : September 5, 2018
Estimated Primary Completion Date : December 2019
Estimated Study Completion Date : December 2019


Arm Intervention/treatment
Experimental: Cannabidiol Oral Solution (CBD)

Cannabidiol Oral Solution, up to 40 milligrams per kilogram per day (mg/kg/day), participants will be dosed approximately every 12 hours with food.

Participants will also be taking vigabatrin, up to 150 mg/kg/day, divided twice daily with food.

Drug: Cannabidiol Oral Solution
An oral solution containing pharmaceutical grade cannabidiol (nonplant-based).

Drug: Vigabatrin
Powder suspension
Other Name: Sabril

Placebo Comparator: Placebo

Matching CBD placebo, up to 40 mg/kg/day, participants will be dosed approximately every 12 hours with food.

Participants will also be taking vigabatrin, up to 150 mg/kg/day, divided twice daily with food.

Drug: Placebo
Matching oral solution

Drug: Vigabatrin
Powder suspension
Other Name: Sabril




Primary Outcome Measures :
  1. Percentage of Participants Considered Complete Responders [ Time Frame: Visit 1 (Day -14 to -1 of Screening Period) up through Visit 6 (Day 15 of Initial Treatment Period) ]
    Complete response is defined as complete resolution of spasms and hypsarrhythmia confirmed by 24-hour video-electroencephalogram (EEG) at Visit 5.


Secondary Outcome Measures :
  1. Percentage of Participants with Resolution of Infantile Spasms [ Time Frame: Visit 1 (Day -14 to -1 of Screening Period) up through Visit 6 (Day 15 of Initial Treatment Period ]
    Resolution of infantile spasms will be assessed by 24-hour video-EEG on Visit 5.

  2. Percentage of Participants with Resolution of Hypsarrhythmia [ Time Frame: Visit 1 (Day -14 to -1 of Screening Period) up through Visit 6 (Day 15 of Initial Treatment Period) ]
    Resolution of hypsarrhythmia will be assessed by 24-hour video-EEG on Visit 5.

  3. Investigator Impression of Efficacy and Tolerability of Study Drug Clinical Global Impression- Global Improvement (CGI-I) at Visit 6 [ Time Frame: Visit 6 (Day 15 of Initial Treatment Period) ]
    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.

  4. Increase in Number of Spasm-Free Days Between Day 1 and Day 15 [ Time Frame: Visit 2 (Day 1 of Initial Treatment Period) up through Visit 6 (Day 15 of Initial Treatment Period) ]
    Increase in spasm-free days will be determined by seizure diary entries.

  5. Participants with Complete Response During Initial Treatment Period: Percentage of Participants Who Relapse During the Extended Treatment Period [ Time Frame: Visit 6 (Day 15 of Initial Treatment Period) up through Visit 9 (Week 13 of Extended Treatment Period) ]
    Relapse during the extended treatment period will be confirmed by video-EEG following parent report of relapse, and time to relapse.



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Ages Eligible for Study:   1 Month to 24 Months   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. 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.
  2. Clinical diagnosis of Infantile Spasms and hypsarrythmia, confirmed by a 9-hour video-EEG obtained during screening Period and read by the central reader.
  3. 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 (Visit 1).
  4. 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.

Exclusion Criteria:

  1. 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.
  2. Known or suspected allergy to cannabidiol.
  3. History of an allergic reaction or a known or suspected sensitivity to any substance that is contained in the investigational product formulation.
  4. Use of any cannabidiol/cannabis product within 30 days of study entry.
  5. Patient is diagnosed or suspected of having tuberous sclerosis.
  6. Patient has received treatment with either vigabatrin, ACTH, or high-dose steroids previously.
  7. Previous or concomitant therapy with felbamate, clobazam, valproic acid, or the ketogenic diet.
  8. Patient currently on any disallowed CYP3A4-related medication listed in Appendix 1 (phenytoin, fluvoxamine, carbamazepine, and St. John's Wort).
  9. Previously received any investigational drug or device or investigational therapy within 30 days before Screening.
  10. 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 patient eligible if he or she judges the laboratory values to be not clinically significant.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03421496


Contacts
Contact: Clinical Operations (602) 910-2617 InsysCBD@insysrx.com

Locations
United States, Florida
Nicklaus Children's Hospital Recruiting
Miami, Florida, United States, 33155
Contact: Research Coordinator    786-624-3611    Abel.Ortega@Nicklaushealth.org   
United States, Michigan
Beaumont Children's Hospital Recruiting
Royal Oak, Michigan, United States, 48073
Contact: Research Coordinator    248-551-3302    Elizabeth.Kring@beaumont.org   
United States, Oregon
Oregon Health & Science University Recruiting
Portland, Oregon, United States, 97239
Contact: Research Coordinator    503-418-8297    mccarbry@ohsu.edu   
United States, Washington
Institute for Research and Innovation | MultiCare Health System Recruiting
Tacoma, Washington, United States, 98405
Contact: Research Coordinator    253-403-9348    ccanorro@multicare.org   
Sponsors and Collaborators
INSYS Therapeutics Inc
Investigators
Study Director: Giovanni DeCastro INSYS Therapeutics Inc

Responsible Party: INSYS Therapeutics Inc
ClinicalTrials.gov Identifier: NCT03421496     History of Changes
Other Study ID Numbers: INS011-16-082
First Posted: February 5, 2018    Key Record Dates
Last Update Posted: September 13, 2018
Last Verified: September 2018

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Spasm
Spasms, Infantile
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Epilepsy, Generalized
Epilepsy
Brain Diseases
Central Nervous System Diseases
Pharmaceutical Solutions
Vigabatrin
Anticonvulsants
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
GABA Agents
Neurotransmitter Agents
Physiological Effects of Drugs