A Study to Assess the Safety, Tolerability and Preliminary Efficacy of ASP0367 (MA-0211) in Pediatric Male Participants with Duchenne Muscular Dystrophy (DMD), Trial ID 0367-CL-0102

试验摘要

The primary purpose of this study is to evaluate the safety and tolerability of ASP0367.

This study will also evaluate the pharmacokinetics, pharmacodynamics and efficacy on muscle function of ASP0367.

研究文件

Clinical Study Report

Available Language(s): English

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Plain Language Summary

Available Language(s): English, English, English

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Protocol

Available Language(s): English

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Children's Hospital of The King's Daughters

Norfolk, VA, 美国, 23507

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Children's Hospital of Richmond at VCU

Richmond, VA, 美国, 23219

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Sacramento, CA, 美国, 95817

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Kansas city, KS, 美国, 66160

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UMass Memorial Medical Center

Worcester, MA, 美国, 01655

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University of Minnesota

Minneapolis, MN, 美国, 55455

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试验信息

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参与者年龄

8 - 16 Years

疾病

杜氏肌肉营养不良症（DMD）

参与者性别

男性

试验开展时间

Feb 2021 - Mar 2022

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入选标准

Subject has a diagnosis of Duchenne muscular dystrophy (DMD) (confirmed by Central Genetic Counselor) defined as a clinical picture consistent with typical DMD and 1 of the following:
- Dystrophin immunofluorescence and/or Western blot showing severe dystrophin deficiency consistent with the diagnosis of DMD.
- Identifiable mutation within the DMD gene (deletion/duplication of 1 or more exons), where reading frame can be predicted as “out-of-frame”
- Complete dystrophin gene sequencing showing an alteration (point mutation, duplication or other) that is expected to preclude production of the functional dystrophin protein (i.e., nonsense mutation or deletion/duplication leading to a downstream stop codon).
A male subject of reproductive potential (Tanner Stage 2 and above) must agree to do either of the following from screening throughout the study until 30 days after the last dose of the investigational product (IP):
- Abstain from sexual intercourse, OR
- If having heterosexual intercourse, must use a condom and their female partners who are of childbearing potential must use a highly effective contraception method.
Subject has been on a stable regimen of corticosteroids for 6 months prior to the time of enrollment (at baseline).
Subject has been on stable cardiac therapy for 3 months prior to the time of enrollment (at baseline), if used, which may include prophylactic angiotensin-converting enzyme inhibitors (ACE), angiotensin II receptor blocker (ARB), aldosterone receptor antagonists (e.g., spironolactone, eplerenone), and/or beta-blocker therapy or a combination therapy thereof.
Subject is unable to complete the 10 meter run/walk in <6 seconds at screening.
Subject has a PUL 2.0 entry item A score of 4, 5 or 6 at screening.
Subject and subject’s parent(s) or legal guardian agrees not to participate in another interventional study while participating in the present study.
For those subjects receiving exon-skipping therapy, the subject has been on a stable dose regimen with a single commercially-available product for at least 6 months prior to randomization at baseline.

排除标准

Subject has had an acute illness (i.e., upper respiratory or viral infection) within 4 weeks prior to study enrollment (at baseline), which precludes participation.
Subject has a cardiac ejection fraction < 55% on echocardiogram at screening.
Subject has a mean QT interval from triplicate electrocardiogram (ECG) using Fridericia’s correction (QTcF) of > 450 msec at screening. If the mean QTcF exceeds the limits stated above, 1 additional triplicate ECG can be taken and utilized at screening.
Subject has cardiac troponin I (cTnI) above the upper limit of normal (ULN) (or cardiac troponin T [cTnT] above the ULN if cTnI is not available immediately) at screening.
Subject has used coenzyme Q10 (CoQ10), metformin, idebenone, carnitine, or other mitochondrial focused supplements or drugs within 4 weeks prior to randomization at baseline. In addition, subject has used any peroxisome proliferator-activated receptors (PPAR) ligands such as fibrates and thiazolidinediones 4 weeks prior to randomization at baseline.
Subject has a known or suspected hypersensitivity to ASP0367, or any components of the formulation used.
Subject has inadequate renal function, as defined by serum Cystatin C > 2 x ULN at screening.
Subject who has any of the following liver function tests elevated: gamma-glutamyl transferase [GGT] and/or total bilirubin [TBL]) > 1.5 x ULN at screening.
Subject who has a positive test result for hepatitis A virus (HAV) antibodies (immunoglobulin M [IgM]), hepatitis B surface (HBs) antigen, hepatitis C virus (HCV) antibody or human immunodeficiency virus (HIV) antigen/antibody at screening.
Subject has mental conditions such as schizophrenia, bipolar disorder or major depressive disorder.
Subject has a history of suicide attempt, suicidal behavior or has any suicidal ideation within 1 year prior to screening that meets criteria at a level of 4 or 5 by using the Columbia Suicide Severity Rating Scale (C-SSRS) or who is at significant risk to commit suicide, as assessed at screening or at baseline.
Subject has severe behavioral or cognitive problems that preclude participation in the study.
Subject has any condition, which makes the subject unsuitable for study participation.
Subject is taking any other investigational therapy currently or has taken any other investigational therapy within 3 months prior to the time of enrollment (at baseline).
Subject and parent/guardian are unwilling and unable to comply with scheduled visits, IP administration plan and study procedures.
Subject has had clinical signs and symptoms consistent with coronavirus (SARSCoV-2) infection or who has tested positive within 2 months prior to randomization at baseline.
Subject whose parent(s) and/or caregiver(s) have increased risk of coronavirus (SARS-CoV-2) exposure from work history (e.g., nursing home, meat processing facility and correctional facility) or recent travel history unless the subject’s parent(s) and/or caregiver(s) have been appropriately vaccinated with one of the COVID-19 vaccines.

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临床试验是否只针对晚期癌症患者？

虽然某些临床试验可能侧重于更晚期的癌症，但许多试验对处于不同癌症阶段的患者开放。每项研究都有关于参与资格的规定。例如，只有特定年龄段的患者或患有特定类型肿瘤的患者才能参与。

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A Study to Assess the Safety, Tolerability and Preliminary Efficacy of ASP0367 (MA-0211) in Pediatric Male Participants with Duchenne Muscular Dystrophy (DMD)

试验摘要

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研究文件

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