EUCTR2021-000903-19-ES进行中（未招募）1 期
A Phase 1/2 First-in-human, 2-part Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Single Ascending Doses (Part 1: Open-label) and Repeat Doses (Part 2: Randomized, Double-blind, Placebo-controlled) of UX053 in Patients with GSD III
ltragenyx Pharmaceutical Inc.0 个研究点目标入组 30 人开始时间: 2021年7月20日最近更新:
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 30
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •1. Confirmed diagnosis of Glycogen Storage Disease type III (GSD III) (all subtypes) based on pathogenic mutations in the amylo-a-1,6-glucosidase 4-alpha-glucanotransferase (AGL) gene on both alleles or glycogen debranching enzyme (GDE) deficiency based on biopsy of liver, muscle, or fibroblasts
- •2. History of any of the following:
- •a. Severe hypoglycemia, defined as neuroglycopenia (eg, altered mental status, seizure, dizziness, slurred speech, blurry vision, abnormal behavior, perioral paresthesia, requiring intervention by a caregiver) or blood glucose < 54 mg/dL (3 mmol/L) within the last year
- •b. = 2 incidents of symptomatic hypoglycemia (defined as blood glucose < 70 mg/dL [3.9 mmol/L] if measured at the time of symptoms) within the last year, despite nutrition management
- •c. Ongoing liver injury, defined as alanine aminotransferase (ALT) > 2.5x the upper limit of normal (ULN) within the last year
- •3. ALT = 5x ULN during the 3 months prior to the Baseline Visit
- •4. Males or females = 18 years of age
- •5. After nutrition optimization (if necessary) for subjects in the repeat dose (RD) Period, during the beginning of the Screening Period, willing and able to maintain nutritional intake consistent with the nutrition guidelines based on expert recommendations for the remainder of the study
- •6. Willing and able to provide access to medical records surrounding medical treatment that occurred prior to enrollment
- •7. Willing and able to provide written informed consent, or in the case of adult subjects with cognitive limitation, provide written assent (if required) and written informed consent by a legally authorized representative after the nature of the study has been explained and prior to any test procedures or assessments
- •8. Females of childbearing potential must have a negative pregnancy test at Screening (ISV) and be willing to have additional pregnancy tests during the study. Subjects of childbearing potential or fertile males who are sexually active with partners of child-bearing potential must consent to use a highly effective contraceptive method, as described in Appendix 4 of the Protocol, from the Period following the signing of the informed consent through 30 days after last dose of study drug
- •Are the trial subjects under 18? no
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 27
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 3
排除标准
- •1. History of liver transplant, including hepatocyte cell therapy/transplant, or active listing for liver transplant
- •2. History of cirrhosis, or presence of any of the following:
- •a. Total bilirubin = 1.3 mg/dL and international normalized ratio (INR) = 1.3
- •b. Evidence of portal hypertension, including, but not limited to the following symptoms splenomegaly, ascites, thrombocytopenia, esophageal varices, or history of hepatic encephalopathy
- •c. Model for End Stage Liver Disease (MELD) score > 12
- •3. Current Hepatitis B or C infection or history of chronic Hepatitis B or C infection
- •4. Severe renal impairment defined as a glomerular filtration rate (GFR) = 29 mL/min
- •5. Any prior history of hepatocellular carcinoma or presence of liver adenoma > 5 cm at the longest diameter or > 3 cm and = 5 cm in size that has an annual growth rate of = 0.5 cm per year
- •6. Current or history of malignancies in the 3 years prior to the Screening Visit (ISV for RD)
- •7. Hospitalizations related to GSD III disease between the Screening (ISV for RD) and Baseline Visit
- •8. Known history of human immunodeficiency virus infection
- •9. Presence or history of any hypersensitivity reactions requiring medical evaluation and management (including injection/infusion associated reactions, such as lymphadenopathy) to UX053, its excipients, or any drug products that contain polysorbate or PEG. This may include mRNA-based vaccines that contain PEG or polysorbate
- •10. Significant cardiac disease, including heart failure with New York Heart Association (NYHA) Function Capacity III or IV or Objective Assessment C or D, unstable angina, or ejection fraction (EF) < 35%, or uncontrolled arrhythmia or resistant hypertension. Asymptomatic cardiomyopathy and left ventricular hypertrophy (LVH) are allowed
- •11. Presence or history of any co-morbid condition or abnormal labs that, in the view of the Investigator, places the subject’s safety at risk; places the subject at high risk of poor treatment compliance or not completing the study; or would significantly affect the interpretation of study results
- •12. Poorly controlled diabetes, defined as the presence of any of the following:
- •a. Hemoglobin A1C > 8%
- •b. History of diabetic nephropathy, neuropathy, or retinopathy
- •c. History of diabetic ketoacidosis during the past year
- •13. Poorly controlled hypothyroidism, based on the judgement of the Investigator or Ultragenyx, whichever is most conservative
- •14. History of chronic coagulopathy, thrombophilia, or disorder of complement activation
- •15. Use of concomitant medications that alter PT/INR, including warfarin and direct oral anticoagulants (eg, rivaroxaban, apixaban, and edoxaban). Patients who receive medications that affect platelet function, such as aspirin or clopidogrel, are allowed, unless they have comorbidities that in the judgment of the Investigator place them at undue risk to participate in the study.
- •16. Current treatment with long-term immunosuppressive medications. This includes subjects with autoimmune conditions managed with immunosuppressive medications and solid organ transplant recipients.
- •17. Active tuberculosis requiring treatment in the past 3 years
- •18. Symptomatic COVID-19 infection
- •19. History of active alcohol and/or drug abuse that in the Investigator’s assessment would impair the subject’s ability to comply with the protocol
- •20. Receipt of only 1 of 2 planned doses of a COVID-19 vaccine. Subjects who have not received a COVID-19 vaccine, and th
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