NL-OMON51808撤回2 期
A randomized open-label Phase 2a study to assess the pharmacokinetics and pharmacodynamic parameters of PXL770 after 12 weeks of treatment in male subjects with adrenomyeloneuropathy (AMN) - PXL770-011 / START770
Poxel S.A.0 个研究点目标入组 14 人开始时间: 待定最近更新:
适应症
试验速览
- 阶段
- 2 期
- 状态
- 撤回
- 发起方
- 入组人数
- 14
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 99（—）
入选标准
- •1. Male subjects with either a confirmed diagnosis of AMN by genetic testing
- •(mutation in the ATP binding cassette subfamily D (ABCD1 gene)) or a family
- •history of X-linked adrenoleukodystrophy (ALD) together with an elevation in
- •VLCFA obtained from overnight fasting plasma sample at Screening Visit (V1).
- •2. Age: >= 18 to <= 65 years at informed consent signature.
- •3. Normal brain magnetic resonance imaging (MRI) or brain MRI showing
- •non-specific abnormalities that can be observed in AMN subjects without signs
- •of cerebral form of ALD (C-ALD). MRI must be performed within 6 months prior to
- •V2. If there is no available brain MRI within this period, a brain MRI must be
- •performed before V2.
- •4. Appropriate steroid/adrenal hormone replacement if adrenal insufficiency is
- •5. Subjects with female partners of childbearing potential must agree to remain
- •sexually abstinent or use condoms during the treatment phase until 2 weeks
- •after the last IP intake. In addition, subjects must be willing to stop sperm
- •donation during this time.
- •6. Capable of providing written informed consent. Subjects must have given
- •written informed consent before any study-related activities are carried out.
排除标准
- •Target disease exclusions
- •1. Any progressive neurological disease other than AMN.
- •2. Arrested or progressing C-ALD as defined by cerebral lesions (except for
- •non-specific abnormalities that can be observed in AMN subjects).
- •3. Prior receipt of an allogeneic hematopoietic stem cell transplant or gene
- •Medical history and concurrent disease exclusions
- •Cardiovascular diseases
- •4. Any uncontrolled cardiovascular disorder in addition to those listed in
- •Exclusion Criteria #5, 6 and 7 that prevents subject*s participation in the
- •study per Investigator*s judgement.
- •5. Unstable arrhythmia or clinically significant arrhythmia diagnosed during
- •the Screening Period, long QT syndrome, short QT syndrome, history of
- •drug-induced Torsade de Pointes.
- •6. Uncontrolled high blood pressure (BP): diastolic BP >= 100 mmHg or systolic
- •BP >= 160 mmHg with or without antihypertensive treatment at V1.
- •7. Any of the following disease within 6 months prior to V2:
- •- Myocardial infarction
- •- Unstable congestive heart failure
- •- Heart failure Class III or IV according to the New York Heart Association
- •- Coronary revascularization (coronary artery bypass graft (CABG) /
- •percutaneous transluminal coronary angioplasty (PTCA))
- •- Unstable angina
- •- Transient ischemic attack, stroke or cerebrovascular disease.
- •Other diseases
- •8. Estimated glomerular filtration rate (eGFR)
- •<= 60 mL/min/1.73m² at V1 calculated by the chronic kidney disease -
- •epidemiology collaboration (CKD-EPI) formula.
- •9. Active malignancy or malignancy with a complete remission date within 2
- •years prior to V2 (except for treated basal cell carcinoma or treated squamous
- •cell carcinoma of the skin).
- •10. Uncontrolled hepatic disorder (aspartate aminotransferase (AST) or alanine
- •aminotransferase (ALT) > 2 x the upper limit of normal (ULN) at V1).
- •11. Type 1 diabetes mellitus.
- •12. Type 2 diabetes mellitus (T2DM) if not on stable treatment (i.e., same
- •doses and same drug(s)) for at least 6 months prior to V1 or uncontrolled T2DM
- •(glycated hemoglobin (HbA1c) > 7.5% at V1).
- •13. Uncontrolled hypothyroidism (thyroid stimulating hormone (TSH) > 2 x ULN at
- •Other exclusion conditions
- •14. Contra-indications for MRI procedure (e.g., the presence of paramagnetic
- •materials in the body, such as aneurysm clips, pacemakers, intraocular metal or
- •cochlear implants including allergies to anesthetics or contrast agents and
- •claustrophobia).
- •15. Positive screen at V1 for hepatitis B surface antigen (HbsAg), antibody to
- •the hepatitis C virus (Anti-HCV) with detected circulating ribonucleic acid
- •(RNA), antibodies to human immunodeficiency (Anti-HIV) 1 and 2 virus.
- •16. Any excessive alcohol intake (>= 14 units of alcohol/week) within 1 year
- •prior to V2, where a unit of alcohol equals approximately 250 mL of beer, 100
- •mL of wine, or 35 mL of spirits.
- •17. Any recent drug abuse (< 6 months prior to V2) or medically uncontrolled
- •current drug dependence per Investigator*s judgement (e.g.: opiate,
- 另有 6 项未显示
研究者
相似试验
进行中（未招募）
1 期
Trial on patients with adrenomyeloneuropathy to assess PXL770's behaviour, a new potential medicine, in the bodyEUCTR2021-006223-18-DEPoxel S.A.24
进行中（未招募）
不适用
Randomized, open-label phase II study evaluating the safety and efficacy of Cetuximab plus modified FOLFOX - 6 versus modified FOLFOX - 6 alone as 2nd line therapy after progression on the 1st line treatment with Cetuximab + FOLFIRI in patients with metastatic colorectal cancer KRAS wild-type – TAGUS Study - TAGUS StudyEUCTR2008-008282-36-PTGrupo de Investigação do Cancro Digestivo
招募中
不适用
A randomized open-label phase 2 study to evaluate the efficacy between only radiotherapy versus combination therapy with radiotherapy and temozolomideNeoplasmsKCT0000700Asan Medical Center104
进行中（未招募）
1 期
Randomised, open-label phase II study to compare the safety and efficacy of lapatinib plus trastuzumab or lapatinib plus capecitabine in trastuzumab-resistant HER2-overexpressing metastatic breast cancerEUCTR2012-000598-22-DEBerufsverband der niedergelassenen gynäkologischen Onkologen in Deutschland e.V.; BNGO e.V.100
进行中（未招募）
不适用
A randomized, open-label phase II. study evaluating the efficacy and safety of FOLFOX6+Cetuximab versus FOLFIRI+Cetuximab as first line therapy in patients with metastatic colorectal cancerfirst line therapy in patients with metastatic cancerEUCTR2004-002391-42-CZCECOG (Central European Cooperative Oncology Group)150