EUCTR2020-004184-12-PT进行中（未招募）1 期
A Phase 3, Randomized, Double-blind, Placebo-controlled Study of Adeno-associated Virus Serotype 8-mediated Gene Transfer of Glucose-6-phosphatase in Patients With Glycogen Storage Disease Type Ia.EMA Decision number on PIP: EMA/PDCO/154270/2021
ltragenyx Pharmaceutical Inc.0 个研究点目标入组 50 人开始时间: 2021年9月29日最近更新:
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 50
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. Male and female patients = 8 years of age at time of informed consent or assent.
- •2. Subject has a diagnosis of GSDIa confirmed by deficient enzymatic activity (on liver biopsy), or by molecular testing of G6PC gene revealing 2 pathogenic mutations. In the case where only a single pathogenic mutation is identified, clinical diagnosis is compatible with GSDIa and absence of characteristic features of GSDIb (ie, chronic neutropenia, inflammatory bowel disease).
- •3. Subject is currently receiving a therapeutic regimen of cornstarch (or equivalent) and clinically stable as evidenced by no more than a 10% weekly change in cornstarch (or equivalent) regimen; no more than 15% variation in weekly average euglycemia (60-120 mg/dL) assessed by CGM and no hospitalization for hypoglycemia during the 4-week period preceding randomization and dosing.
- •4. Subject is willing and able to comply with study procedures and requirements, including periodic inpatient hospitalization or admission in a research facility; CFC studies; frequent blood collection; wearing a CGM device for the duration of the study (and excluding the use of any non-study CGM or flash glucose device); performing capillary glucose measurements using a study-approved glucometer (and excluding the use of any other glucometer); completing an eDiary routinely throughout the study to track daily cornstarch, dietary intake, and reason for performing SMBG; and completing patient-reported questionnaires. If < 18 years (or as required by region), has a parent or legal guardian willing and able to assist with study requirements.
- •5. From the period following informed consent through the duration of participation in the study, female subjects of childbearing potential and fertile male subjects must consent to use highly effective contraception as defined by the Food and Drug Administration (FDA) and Clinical Trial Facilitation Group Recommendations Related to Contraception and Pregnancy Testing in Clinical Trials (Version 1.1 dated 21 Sep 2020). Female subjects must agree not to become pregnant and male subjects must agree not father a child or donate sperm for at least 48 weeks after the last dose of IP if they decide to withdraw early from the study.
- •6. Subject is willing and able to provide written informed consent after the study has been explained and before any study-related procedures are performed. If < 18 years (or as required by region), willing and able to provide written assent and have a parent or legal guardian willing and able to provide written informed consent after the study has been explained and before any study-related procedures are performed.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 10
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 36
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 4
排除标准
- •1. Detectable pre-existing antibodies to the AAV8 capsid.
- •2. History of liver transplant, including hepatocyte cell therapy/transplant.
- •3. History of liver disease as evidenced by any of the following: portal hypertension, ascites, splenomegaly, esophageal varices, hepatic encephalopathy, or a liver biopsy with evidence of stage III fibrosis.
- •4. Presence of liver adenoma > 5 cm in size or presence of liver adenoma > 3 cm and = 5 cm in size with a documented annual growth rate of = 0.5 cm per year.
- •5. Significant hepatic inflammation or cirrhosis as evidenced by imaging or any of the following laboratory abnormalities:
- •- ALT or aspartate aminotransferase > 2.5 × the ULN
- •- Total bilirubin > 2.0 × ULN (except if patient has documented Gilbert’s syndrome)
- •- Alkaline phosphatase > 2.5 × ULN
- •6.Non-fasting triglycerides = 1000 mg/dL. For the purposes of this study, non-fasting refers to the longest fasting period that each individual subject is able to tolerate. Depending on the meal and cornstarch schedule, the blood draw could occur in the morning before breakfast or before the first dose of cornstarch.
- •7. Presence or history of hepatitis B virus infection, hepatitis C virus infection, or both.
- •8. Human immunodeficiency virus infection AND any of the following: CD4+ cell count < 350 cells/mm3, change in antiretroviral therapy regimen within 6 months before baseline, or plasma viral load > 200 copies/mL on 2 separate occasions as measured by polymerase chain reaction.
- •9. Presence or history of any disease or condition that, in the Investigator’s opinion, would interfere with the subject’s safety or ability to participate in the study or would significantly affect interpretation of study results. This includes any intercurrent febrile or nonfebrile illness including common viral infections, epidemic influenza, and coronavirus disease 2019 (COVID-19) until full clinical recovery.
- •10. Female subjects of childbearing potential who have a positive serum pregnancy test at screening or a positive urine pregnancy test at baseline or who are unwilling to have additional pregnancy tests during the study. Females considered not of childbearing potential include those who have not experienced menarche, are postmenopausal (defined as having no menses for at least 12 months without an alternative medical cause), or are permanently sterile due to having total hysterectomy, bilateral salpingectomy, or bilateral oophorectomy.
- •11. Pregnant, breastfeeding, or planning to become pregnant (self or partner) at any time during the study.
- •12. Presence or history of any hypersensitivity to the excipients of DTX401 or placebo or to prednisolone, or inability to swallow capsules that, in the judgment of the Investigator, places the subject at increased risk for adverse effects.
- •13. Current or previous participation in another gene transfer study.
- •14. Use of any IP or investigational medical device within 3 months preceding screening or planning to use at any time during the study.
- •15. History of illicit drug use within 60 days prior to screening or positive results from a 9-panel urine drug screen during the Screening Period completed at 2 time points at least 4 weeks apart. Positive results that are due to a prescribed medication may be allowed if not impacting glycemic control and liver function and after agreement with the Sponsor. For the purposes of this protocol, the use of recreational cannabis products is not allowed, even if legal in the region where the pati
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