Clinical trial NCT02844933: Cannabidiol Oral Solution for the Treatment of Patients With Prader-Willi Syndrome
Official title: A Randomized, Double-Blind, Placebo-Controlled, Phase 2 Study to Assess the Efficacy, Safety, and Tolerability of Cannabidiol Oral Solution for the Treatment of Patients With Prader-Willi Syndrome
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Prader-Willi Syndrome
Lead sponsor: Radius Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2016-07-26
Enrollment: 7
Sex: ALL
Minimum age (years): 8
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The primary objective of this study is to assess the efficacy of cannabidiol oral solution on hyperphagia-related behavior in patients with Prader-Willi Syndrome (PWS). The secondary objectives of this study are to assess the efficacy, safety and tolerability, impact on quality of life, and impact on physical activity of cannabidiol oral solution in patients with PWS.


Eligibility criteria: Inclusion Criteria:

* Participant and/or parent(s)/caregiver(s) fully comprehend the informed consent form and assent form, understand all study procedures, and can communicate satisfactorily with the investigator and study coordinator, in accordance with applicable laws, regulations, and local requirements.
* Participants with a genetically confirmed diagnosis of Prader-Willi Syndrome using standard deoxyribonucleic acid methylation test or fluorescent in situ hybridization. Documentation of genetically confirmed diagnosis of Prader-Willi Syndrome is acceptable.
* A score of ≥10 on the Hyperphagia Questionnaire for Clinical Trials (HQ-CT).
* A caregiver is available to complete the HQ-CT.
* If female, is either not of childbearing potential (defined as premenarchal or surgically sterile \[bilateral tubal ligation, bilateral oophorectomy, or hysterectomy\]) or practicing one of the following medically acceptable methods of birth control:

  1. Hormonal methods such as oral, implantable, injectable, vaginal ring, or transdermal contraceptives for a minimum of 1 full cycle (based on the participant's usual menstrual cycle period) before study drug administration.
  2. Total abstinence from sexual intercourse since the last menses before study drug administration.
  3. Intrauterine device.
  4. Double-barrier method (condoms, sponge, or diaphragm with spermicidal jellies or cream).
* Adequate renal function, defined as serum creatinine ≤ 1.5\*upper limit of normal (ULN) and urine protein/creatinine ratio ≤0.4. The Investigator may deem the participant eligible if he or she judges the laboratory values to be not clinically significant.
* Adequate hepatic function, defined as total bilirubin ≤ 1.5\*ULN and aspartate aminotransferase and alanine aminotransferase levels ≤ 3\*ULN.
* Growth hormone treatment will be permitted if doses have been stable for at least 1 month prior to screening.
* Psychotropic treatment will be permitted and should be stable at least 6 weeks prior to screening.
* Any other treatment including thyroid hormones should be stable for at least 6 weeks prior to screening.

Exclusion Criteria:

* Known use of cannabis or cannabinoid-containing products for 4 weeks prior to baseline.
* History of chronic liver diseases, such as cirrhosis or chronic hepatitis due to any cause, or suspected alcohol abuse.
* Use of weight loss agents or drugs known to affect appetite (including glucagon-like peptide-1 \[GLP-1\] analogs) within 2 months prior to screening.
* Uncontrolled Type I and Type II diabetes.
* Currently taking concomitant medication that are strong-moderate inhibitors/inducers/sensitive substrates with a narrow therapeutic index for CYP2C19 or CYP3A.
* Co-morbid condition or disease (such as respiratory disease, heart disease, or psychiatric disorder) diagnosed less than 1 month prior to screening.
* History or presence of gastrointestinal or any other condition known to interfere with the absorption, distribution, metabolism, or excretion of drugs as determined by the Investigator.
* Participants who have participated in any other trials involving an investigational product or device within 30 days of screening or longer as required by local regulations.
* Clinically significant abnormalities on electrocardiogram at screening or other evidence of heart disease as determined by the Investigator.
* Has screening systolic blood pressure ≥160 millimeters of mercury (mmHg) and diastolic blood pressure \>100 mm Hg (may be repeated 1 additional time after 5 minutes rest to verify). Participants with hypertensive levels lower than those specified may be excluded at the Investigator's discretion if deemed to be in the best interest of the participant.
* Currently taking felbamate.
* Uncontrolled sleep apnea.
* Pregnant or lactating female.
* History of hypersensitivity to drugs with a similar chemical structure or class as cannabidiol.
* Unwillingness or inability to follow the procedures outlined in the protocol.
* Participant judged by the investigator or sponsor (or designee) as unable to comply with the treatment protocol, including appropriate supportive care, follow-up and research tests.
* Positive drug screen, including tetrahydrocannabinol, at time of screening.
* Creatinine clearance test of \< 30 milliliters/minute (mL/min).

Primary outcomes:
measure: Change From Baseline in Hyperphagia Behavior as Measured by the Hyperphagia Questionnaire for Clinical Trials (HQ-CT)
timeFrame: Baseline, Week 13
Secondary outcomes:
measure: Change From Baseline In Total Body Weight
timeFrame: Baseline, Week 13
measure: Responder Rate From Baseline Through Study Completion
timeFrame: Baseline up to Week 13
measure: Change In Patient Global Impression Of Change (PGI-C) Questionnaire
timeFrame: Week 3, Week 13
measure: Change From Baseline In The Three Factor Eating Questionnaire - 18-item Version (TFEQ-R18)
timeFrame: Baseline, Week 13
measure: Change From Baseline In Quality Of Life [Patient-Reported Outcomes Measurement Information System (PROMIS) Life Satisfaction And Positive Affect Questionnaires]
timeFrame: Baseline, Week 13
measure: Change From Baseline In Physical Activity (PROMIS Physical Activity And Fatigue Questionnaires)
timeFrame: Baseline, Week 13
Other outcomes:
Locations:
city: Tucson
country: United States
facility: University of Arizona
state: Arizona
city: San Diego
country: United States
facility: Rady Children's, UC San Diego
state: California
city: Iowa City
country: United States
facility: University of Iowa
state: Iowa
city: Kansas City
country: United States
facility: The University of Kansas , Medical Center
state: Kansas
city: Baltimore
country: United States
facility: Johns Hopkins University
state: Maryland
city: Tulsa
country: United States
facility: The University of Oklahoma Health Sciences Center
state: Oklahoma
city: Tacoma
country: United States
facility: Institute for Research and Innovation | MultiCare Health System
state: Washington
Study officials:
affiliation: INSYS Therapeutics Inc
name: Ahmed Elkashef, MD
role: STUDY_DIRECTOR
References: