CTIS2022-501398-38-00招募中1 期
CALIBRATE: A Phase 3, Randomized, Open-Label Study Evaluating the Efficacy and Safety of Encaleret Compared to Standard of Care in Participants with Autosomal Dominant Hypocalcemia Type 1 (ADH1) - CLTX-305-302
适应症
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 60
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
- 18 至 65+（—）
- 性别
- All
入选标准
- •1. Participant must be at least 18 years of age, at the time of signing the informed consent., 10. Postmenopausal females and females not of childbearing potential may participate in this study without use of contraception: a) Females are considered postmenopausal if they have had 12 months of natural (spontaneous) amenorrhea without an alternative medical cause and must be confirmed with plasma FSH. b) Females are considered not of childbearing potential if they have had surgical bilateral oophorectomy (with or without hysterectomy) or hysterectomy. In the case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow up hormone level assessment, shall she be considered not of childbearing potential.., 11. Females of childbearing potential, defined as all females physiologically capable of becoming pregnant, must use highly effective methods of contraception starting at Screening and for 3 months following the last dose of encaleret. Contraceptive use by males and females should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies., 12. Capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the ICF and in this protocol., 2. Participants must have a documented pathogenic or likely pathogenic activating variant, or variant of uncertain significance, of the CASR gene associated with biochemical findings of hypoparathyroidism either present at Screening or a documented history of both: a) cCa<8.5 mg/dL (2.1 mmol/L), b) iPTH<40 pg/mL (4.2 pmol/L). Note: A genetic analysis report for CASR will be acceptable. In the absence of any documentation for the CASR variant, participants must be willing to undergo CASR variant analysis. Participants who have undergone an allogeneic bone marrow transplant must provide CASR variant analysis report performed prior to the bone marrow transplant Participants who have received a packed red blood cell transfusion must wait 2 weeks following the transfusion before undergoing CASR variant analysis, and participants who have received a whole blood transfusion must wait 4 weeks following the transfusion before undergoing CASR variant analysis., 3. Participants must have a documented history of symptoms or signs of ADH1. Symptoms of ADH1 include agitation, anxiety, back pain, brain fog, bronchospasm, blepharospasm, cardiac failure, cognitive impairment, difficulty focusing/concentration, esophageal spasm, facial spasm, headaches, hypoesthesia (including facial and oral), irritability, laryngospasm, muscle cramping, muscle fatigue, muscle spasms, muscle tightness, muscle twitching, musculoskeletal pain, musculoskeletal stiffness, myalgia, nephrolithiasis, nervousness, paresthesia (including oral), polydipsia, polyuria, seizure, tetany, throat tightness, or tremor. Signs of ADH1 include basal ganglia or other cerebral calcification, Chvostek sign, hypercalciuria, nephrocalcinosis, papilledema, premature cataracts, prolonged QT interval on ECG, pseudotumor cerebri, or Trousseau sign, 4. Participants treated with thiazide diuretics must discontinue thiazides for at least 14 days prior to SoC Optimization Visit 1 through Week 24 (Period 3). When the thiazide is being used as an antihypertensive, alternative therapy will be prescribed by the Investigator as needed., 5. Participants treated with phosphate binders must discontinue the phosphate binders
排除标准
- •1. History of hypocalcemic seizure within the past 3 months preceding Screening., 3. History of the following within 5 years of screening: cancer except thyroid cancer, basal cell skin cancer or squamous cell skin cancer, patients with skeletal malignancies or bone metastases or irradiation (radiotherapy) to the skeleton., 4. History of renal transplantation., 5. Pregnant or nursing (lactating) women, where pregnancy is confirmed by a positive beta-human chorionic gonadotropin (ß-hCG) laboratory test., 6. History of treatment with PTH 1-84 or 1-34 within the 2 months preceding Screening and requiring SoC doses exceeding >1.2× their pre-PTH treatment total daily doses or bone turnover markers, CTx and P1NP, > upper limit of normal for sex, age (men only) and menopausal status (women only)., 7. Received any investigational medicinal product other than encaleret within 30 days or 5 half-lives, whichever is longer, prior to the first day on study, or are in follow-up for another interventional clinical study during Screening. If the half-life of an investigational medicinal product is unknown, then 30 days prior to Screening.., 8. Blood 25-OH Vitamin D level <25 ng/mL If participant has a blood 25-OH Vitamin D level <25 ng/mL at the Screening Visit, they will be prescribed cholecalciferol or ergocalciferol supplementation per the Investigator. Once the 25-OH Vitamin D level is >25 ng/mL, the participant will be eligible to proceed with SoC Optimization (if still within Screening window of up to 84 days total) or re-screen for the study., 9. Estimated glomerular filtration rate <30 mL/minute/1.73 m2 using CKD-EPIcr_R. See Appendix C., 17. Presence or history of any disease or condition (eg, drug or alcohol dependency) that, in the view of the Investigator, would affect the participant’s safety or places the participant at high risk of poor treatment compliance or of not completing the study., 10. 12-lead resting ECG with clinically important abnormalities except for asymptomatic QTc prolongation clinically ascribed to hypocalcemia., 11. Participants with positive Hepatitis B surface antigen (HBbsAg), Hepatitis A immunoglobulin M (IgM), or human immunodeficiency virus (HIV) viral serology test at the Screening Visit. Participants who are in complete remission from Hepatitis C virus (HCV) as evidenced by sensitive assay = 12 weeks after completion of HCV therapy may participate in the study., 12. Male or female participants planning to conceive a child prior to the LTE., 13. Hypersensitivity to any active substance or excipient of encaleret. See Section 7.2.1 for list of active ingredients and excipients., 14. Any current disease that might affect calcium metabolism or calcium-phosphate homeostasis other than hypoparathyroidism., 15. Current treatment with cardiac glycosides (because hypercalcemia of any cause may predispose to digitalis toxicity)., 16. Patients with rare hereditary problems of fructose intolerance, glucose galactose malabsorption or sucrase-isomaltase insufficiency., 2. History of thyroid or parathyroid surgery.
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