Cannabidiol Oral Solution for the Treatment of Patients With Prader-Willi Syndrome

Prader-Willi Syndrome Clinical Trial

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT02844933
• Other study ID #: INS011-16-085
• Status: Terminated
• Phase: Phase 2
• Start date: May 9, 2018
• Est. completion date: July 31, 2019

Study information

• Verified date: July 2023
• Source: Radius Pharmaceuticals, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 7
• Est. completion date: July 31, 2019
• Est. primary completion date: July 17, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 8 Years to 17 Years

Eligibility

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).

Related Conditions & MeSH terms

• Prader-Willi Syndrome
• Syndrome

Intervention

Drug:
• Cannabidiol
Oral solution
• Placebo
Matching oral solution

Locations

Country	Name	City	State
United States	Johns Hopkins University	Baltimore	Maryland
United States	University of Iowa	Iowa City	Iowa
United States	The University of Kansas , Medical Center	Kansas City	Kansas
United States	Rady Children's, UC San Diego	San Diego	California
United States	Institute for Research and Innovation | MultiCare Health System	Tacoma	Washington
United States	University of Arizona	Tucson	Arizona
United States	The University of Oklahoma Health Sciences Center	Tulsa	Oklahoma

Sponsors (1)

Lead Sponsor	Collaborator
Radius Pharmaceuticals, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change From Baseline in Hyperphagia Behavior as Measured by the Hyperphagia Questionnaire for Clinical Trials (HQ-CT)	The HQ-CT measures hyperphagia by Prader-Willi syndrome (PWS)-specialized clinicians. The HQ-CT generates a score ranging from 0 to 36, where a higher score represents more severe abnormal food related behaviors.; The change from baseline was calculated from two time points as the value at the later time point minus the value at the earlier time point: value at Week 13 minus value at Baseline.	Baseline, Week 13
Secondary	Change From Baseline In Total Body Weight	Total body weight refers to participants' weight throughout the study. The change from baseline was calculated from two time points as the value at the later time point minus the value at the earlier time point: value at Week 13 minus value at Baseline.	Baseline, Week 13
Secondary	Responder Rate From Baseline Through Study Completion	A responder was defined as having a 6 or more points decrease in HQ-CT score.	Baseline up to Week 13
Secondary	Change In Patient Global Impression Of Change (PGI-C) Questionnaire	The change in PGI-C scores at Week 3, Week 9, and Week 13 of the participant are evaluated.	Week 3, Week 13
Secondary	Change From Baseline In The Three Factor Eating Questionnaire - 18-item Version (TFEQ-R18)	The change from baseline to Week 13 of the participant's TFEQ-R18 score is evaluated.	Baseline, Week 13
Secondary	Change From Baseline In Quality Of Life [Patient-Reported Outcomes Measurement Information System (PROMIS) Life Satisfaction And Positive Affect Questionnaires]	The change from baseline to Week 13 of the participant's quality of life (PROMIS life satisfaction and positive affect questionnaire) score is evaluated.	Baseline, Week 13
Secondary	Change From Baseline In Physical Activity (PROMIS Physical Activity And Fatigue Questionnaires)	The change from baseline to Week 13 of the participant's physical activity (PROMIS physical activity and fatigue questionnaires) score is evaluated.	Baseline, Week 13