NL-OMON56166招募中3 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 - DTX401-CL301 (GlucoGene)
ltragenyx Pharmaceutical Inc.0 个研究点目标入组 6 人开始时间: 待定最近更新:
适应症
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- 入组人数
- 6
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
- 2 至 99（—）
入选标准
- •1. Males and females >=*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 case of a single pathogenic mutation, 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) following international guidance/recommendations (Appendix 1) with
- •stable nutrition, glycemic, and clinical status as evidenced by:
- •a. no more than a 10% variation in weekly average daily cornstarch (or
- •equivalent) intake over the last 4 weeks.
- •b. no more than a 25% variation in weekly average daily non-cornstarch
- •carbohydrate over the last 4 weeks.
- •c. No more than 15% variation in weekly percentage of values in the target
- •blood glucose range (60-120 mg/dL) over the last 4 weeks as measured by CGM and
- •corroborated by SMBG. If adequate corroboration is not observed, this
- •assessment should be made by SMBG.
- •d. No hospitalization for hypoglycemia and no severe hypoglycemic event (SHE)
- •during the 4-week period preceding randomization and dosing (see Section 10.4.2
- •for more detail on SHE), notwithstanding events of hypoglycemia due to
- •unavoidable and unforeseeable events (eg, infection, trauma) that transiently
- •prevent the subject from tolerating enteral intake or acutely change the
- •subject's metabolic demands, provided that the subject quickly returns to their
- •prior physiologic state.
- •4. Subject is willing and able to comply with study procedures, requirements,
- •and study medication, 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
- •according to the protocol using a study approved glucometer (and excluding the
- •use of any other glucometer); completing an eDiary to track daily cornstarch,
- •diet intake, and reasons for doing SMBG routinely throughout the study as
- •required by the protocol; and completing patient-reported questionnaires.
- •Subject must strictly comply with prednisolone/placebo prednisolone
- •prescription including changes in prescription that may be implemented during
- •the study by the Investigator, if needed. (See Section 9.2, Prednisolone
- •Taper.) 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.
排除标准
- •Detectable pre-existing antibodies to the AAV8 capsid during Screening.
- •History of liver transplant, including hepatocyte cell therapy/transplant.
- •History of severe hepatic fibrosis or cirrhosis 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.
- •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.
- •Significant hepatic injury or dysfunction as evidenced by imaging or any of the
- •following laboratory abnormalities from 2 consecutive samples (collected at
- •least 4 weeks apart). Liver function tests may be repeated during Screening at
- •the Investigator's discretion; those with initially abnormal values may be
- •retested and the subject will qualify for this criterion if the most recent
- •results during Screening are within the allowed range:
- •- ALT or aspartate aminotransferase >*2.5*×*the ULN
- •- Total bilirubin >*ULN (unless the subject has Gilbert*s syndrome)
- •- Alkaline phosphatase >*ULN, with gamma-glutamyl transferase > ULN
- •Presence or history of hepatitis*B virus infection, hepatitis*C virus
- •infection, or both.
- •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.
- •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.
- •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 other viral illnesses, and coronavirus
- •disease 2019 (COVID-19) until full clinical recovery.
- •Female subjects of childbearing potential who have a positive pregnancy test
- •who are unwilling to use contraception, or are unwilling to have additional
- •pregnancy tests during the study.
- •Pregnant, breastfeeding, or planning to become pregnant (self or partner) at
- •any time during the study.
- •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
- •Current or previous participation in another gene transfer study.
- •Use of any IP or investigational medical device within 3*months preceding
- •screening or planning to use at any time during the study.
- •History of illicit drug use within 60 days prior to Screening or positive
- •results from a 9-panel urine drug screen prior to dosing and completed at 2
- •time points at least 4 weeks apart. Positive results that are due to a
- •prescribed medic
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