EUCTR2021-005331-23-ES进行中（未招募）1 期
A Phase 2/3, Randomized, Double-Blind, Placebo-Controlled Study of RAD011 (Cannabidiol Oral Solution) for the Treatment of Patients with Prader-Willi Syndrome
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 220
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 性别
- All
入选标准
- •1. Presence of a parent/legal guardian that is able to consent for their participation. Parent/caregiver/legal guardian can complete the required assessments throughout the study. Patient consent/assent will be obtained if the patient is 8 years of age or older and has the mental capacity to understand and sign a written consent/assent form and/or give verbal assent
- •2. Males and females between:
- •a. Phase 2: 12 and 65 years of age (inclusive) at time of consent/assent
- •b. Phase 3: 8 and 65 years of age (inclusive) at time of consent/assent
- •3. Documentation of genetically confirmed PWS diagnosis
- •4. If a caregiver helps in completing the HQ-CT or other questionnaires, the same caregiver is available to complete the questionnaire throughout the duration of the study
- •5. 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 up to 4 weeks after the last dose of RAD011 or placebo:
- •a. Double barrier method (i.e., condom plus occlusive cap (diaphragm or cervical/vault caps), condom or occlusive cap plus spermicide
- •b. Hormonal methods such as oral, implantable, injectable, vaginal ring, or transdermal contraceptives at a stable dose for a minimum of 1 full cycle (based on the patient’s usual menstrual cycle period) before IP administration
- •c. Intrauterine device (IUD) or intrauterine hormone-releasing system (IUS)
- •d. If not using hormonal contraceptives or IUD or IUS, then all male partners throughout the study must have been vasectomized and have received medical assessment of the surgical success
- •6. If male, is vasectomized and has received medical assessment of the surgical success or agrees to use an approved method of contraception (double barrier method as described in Inclusion Criterion 5, female partner’s use of an IUD or IUS [in place at least 12 weeks prior to dosing], oral contraceptives or female partner who is surgically sterile or 2 years postmenopausal) and agrees to use this method for 4 weeks after last administration of RAD011 or placebo
- •7. If receiving growth hormone (GH), psychotropic therapy, metabolic treatments that could affect appetite (including metformin), and other treatment including thyroid hormone, must be on the same medication and stable dose for at least 90 days prior to consent/assent
- •8. Any non-medical interventions (eg, counseling, behavior modification) should be stable for at least 90 days prior to consent/assent
- •9. HQ-CT eligibility automatically calculated in the electronic data capture (EDC) system:
- •i. Patients with a mean HQ-CT score =13 (from Visit 1 and Visit 2) will be eligible to continue in the Tolerability Period
- •i. Patients with a mean HQ-CT score =13 (from Visit 3, Visit 4 and Visit 5) will be eligible for randomization, and
- •ii. Patients with a decrease in HQ-CT score (from Tolerability Period to the Screening Period) =7 will be eligible for randomization
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 163
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 28
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1. Hypersensitivity or intolerance to CBD, or any other excipients used in the RAD011 preparation
- •2. Known use of cannabis or cannabinoid containing products (including topical products) within 90 days prior to consent/assent
- •3. History of chronic liver disease, such as cirrhosis or chronic hepatitis due to any cause
- •4. Positive urine test for drugs of abuse, including tetrahydrocannabinol (THC), or known history of drug, alcohol, or substance abuse
- •5. Use of prescription or over-the-counter weight loss agents within 90 days prior to consent/assent
- •6. Implementation of new food restrictions or environmental restrictions within 90 days of consent/assent
- •7. Any significant comorbid condition or disease:
- •a. Respiratory disease, heart disease, or psychiatric disorder which in the opinion of the Investigator would preclude the patient from participating or
- •b. Uncontrolled type 1 or type 2 diabetes as determined by the Investigator or
- •c. Clinically significant ECG abnormalities or other evidence of clinically significant heart disease as determined by the Investigator or
- •d. Uncontrolled sleep apnea as determined by the Investigator or
- •e. Neutropenia with neutrophil counts <1,000 /microL (grade 3 or grade 4 neutropenia as defined by CTCAE v5.0) or
- •f. History or presence of gastrointestinal disorders or any other condition known to interfere with the absorption, distribution, metabolism, or excretion of drugs taken orally as determined by the Investigator
- •8. QT interval corrected for heart rate (HR) according to Fridericia's formula (QTcF) meeting the following criteria:
- •a. For males and females aged 8 to <20 years: QTcF >439 msec
- •b. For males aged 20 to 65 years: QTcF >450 msec
- •c. For females aged 20 to 65 years; QTcF >470 msec
- •9. At consent/assent, patients with age-matched hypertensive levels of systolic and/or diastolic blood pressure may be excluded at the Investigator’s discretion if deemed to be in the best interest of the patient.
- •10. Pregnant (determined by a positive serum pregnancy test) or lactating female
- •11. Unwillingness or inability to follow the procedures outlined in the protocol
- •12. Estimated glomerular filtration rate <30 mL/min/1.73m2 or protein/creatinine ratio =0.4
- •13. Bilirubin >1.5 × upper limit of normal (ULN), or aspartate aminotransferase (AST), alanine aminotransferase (ALT) >3 × ULN, or international normalized ratio (INR) >1.5 × ULN
- •14. Patient 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
- •15. Other genetic, hormonal, chromosomal, cognitive, or behavioral impairment
- •16. If living in a group home, patient spends less than 25 waking hours with their caregiver per week
- •17. Significant risk of committing suicide based on history, routine psychiatric examination, or based on the Investigator's judgment
- •18. Participation in any other study involving an investigational product or device within 4 weeks or 5 half-lives (whichever is longer) of consent/assent or longer as required by local regulations
研究者
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