CTIS2023-506200-24-00进行中（未招募）1 期
A Phase 3, Randomized, Double-blind, Placebo-controlled, Parallel-group Study of Carbetocin Nasal Spray for the Treatment of Hyperphagia in Prader-Willi Syndrome - ACP-101-302
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 170
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
- 年龄范围
- 0 至 64（—）
- 性别
- All
入选标准
- •Informed consent prior to the conduct of any study procedures is required as follows: a. For subjects who are minors: written informed consent will be obtained from the LAR. The subject should provide written or oral assent if deemed able by the Investigator, and according to local regulations. b. For subjects who are of legal age of consent: written informed consent will be obtained from the subject if deemed able by the Investigator. If the subject is deemed not able to provide consent, the subject should provide written or verbal assent if deemed able by the Investigator and a written informed consent will be obtained from the subject’s LAR according to local regulations. c. The subject’s caregiver provides written consent to participate as an informant in study assessments. The caregiver may or may not be the LAR., Caregiver agrees to request and provide medical records to the investigative site., Has been on a stable dose of any allowed chronic concomitant medications for at least 3 months prior to the Screening visit. Adjustments in growth hormone dose =10% or medication changes that are not clinically significant in the judgment of the Investigator (i.e., having no reasonable possibility of causing changes in mood, behavior, or appetite, or otherwise affecting study endpoints) are allowed. If the medication was discontinued, the discontinuation occurred at least 2 weeks or 5 half-lives (whichever is greater) prior to Screening., If the subject is female, she must not be pregnant or breastfeeding. Subjects of childbearing potential must abstain from sexual activity for the duration of the study and for at least 30 days thereafter. If a subject is sexually active or becomes sexually active during the study, she must use a highly effective contraceptive method per Clinical Trials Facilitation and Coordination Group (CTFG) recommendations (Appendix A). The contraceptive method should be used for at least 1 month prior to Baseline, throughout the study, and for at least 30 days thereafter., If the subject is male and sexually active, he must use a condom (even if vasectomized) from the time of Screening until 90 days after the last dose of study drug. The male subject’s female partner must use a highly effective contraceptive method per CTFG recommendations (Appendix A). The female partner’s contraceptive method should be used for at least 1 month prior to Baseline, throughout the study, and for at least 30 days thereafter; OR the male subject must not have a female partner of childbearing potential. Subject must also agree not to donate sperm from the time of Screening until 90 days after the last dose of study drug., Is a male or female 5 to 30 years of age, inclusive, at Screening., Has Prader-Willi syndrome with a documented disease-causing mutation., Has increased appetite with decreased satiety accompanied by food seeking (consistent with PWS Nutritional Phase 3)., Has an HQ-CT total score of =13 at Screening and Baseline., Has a CGI-S score for hyperphagia in PWS of =4 at Screening and Baseline., Lives with a caregiver who understands and is willing and able to adhere to study-related procedures and is willing to participate in all study visits. a. The subject must be under the caregiver’s consistent care and observation during the study when not attending school or day programs. b. The caregiver should be a family member of the subject or someone whose association with the subject is the equivalent of a family relation. OR The
排除标准
- •Is genetically diagnosed with Schaaf-Yang syndrome or another genetic, hormonal, or chromosomal cognitive impairment besides PWS., Has a history of suicide attempt or inpatient psychiatric hospitalization., Has new food-related interventions, including environment or dietary restrictions, within 1 month of the Screening visit., Has an active upper respiratory infection at the Screening visit or the Baseline visit., Has participated in an interventional research study involving another investigational medication or device in the 6 months prior to the Screening visit., Has a history of or current abuse of/dependence on alcohol or illicit drugs., Has a clinically significant abnormal laboratory value at Screening. Laboratory testing may be repeated during the Screening period with agreement of the Medical Monitor., Has serum potassium below the normal range (according to the central laboratory) at Screening. Serum potassium may be repeated during the Screening period with the agreement of the Medical Monitor., Has a clinically significant thyroid function test result at Screening (as measured by thyroid stimulating hormone [TSH] and reflex free thyroxine [T4]). If TSH is abnormal and the reflex free T4 is normal, the subject may be randomized., Has clinically significant abnormality in vital signs at Screening or Baseline., Has any of the following: a. QTcF interval of >450 ms at Screening or Baseline (before dosing) b. History of a risk factor for torsades de pointes (e.g., heart failure or family history of long QT syndrome) c. History of clinically significant QT prolongation that is deemed to put the subject at increased risk of clinically significant QT prolongation d. Has any other clinically significant finding on ECG at Screening or Baseline (before dosing), Has another nasal disorder that may affect deposition of intranasal medication., Has a positive pregnancy test at Screening., Is an employee or is a family member of an employee of Acadia Pharmaceuticals Inc., Is judged by the Investigator or the Medical Monitor to be inappropriate for the study for any reason., Has any clinically significant cardiovascular disorder, renal, hepatic, gastrointestinal, or respiratory disease, including severe asthma., Has a history of, or current, cerebrovascular disease, brain trauma, epilepsy, or frequent migraines. A history of febrile seizures is not exclusionary., Has significant, uncorrected visual or uncorrected hearing impairment., Has had major surgery within 1 month of the Screening visit or planning to have surgery during the study., Has had nasal surgery within 1 month of Screening visit or planning to have nasal surgery during the study., Is unwilling to abstain from nasal saline, other nasal irrigation, and other intranasal medications during the Screening period and through the treatment period of the study., Has had more than three episodes of sinusitis in the 12 months prior to the Baseline visit., Has known hypersensitivity to any component of study drug., In France only: Is under court protection, not affiliated to a social security system, or a protected adult under French law (Art. L. 1121-6, Art. L. 1121-8, Art. L. 1121-8-1)., Has been diagnosed with cancer (except managed basal cell carcinoma or squamous cell carcinoma of the skin)., Has had clinically significant irritability or agitation, requiring initiation of antipsychotic medication, within the 6 months prior to the Screening visit., Has used prostaglandins, prostagla
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