EUCTR2023-001104-33-Outside-EU/EEA进行中（未招募）1 期
A Sequential, Two-Part Study to Evaluate the Clinical Benefit, Safety, Pharmacokinetics, and Pharmacodynamics of AT-007 in Pediatric Patients with Classic Galactosemia (CG)
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
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
- 性别
- All
入选标准
- •1. Male or non-pregnant, non-lactating female patient aged =2 to <18 years.
- •2. CG diagnosis confirmed by evidence of absent or significantly decreased (<1%) GALT activity in RBCs, or a historical record of diagnosis of CG (medical record or written communication by health care professional containing gene analysis and/or GALT enzyme activity).
- •3. No clinically significant abnormal laboratory value as determined by the Investigator during screening.
- •4. Patient’s vital signs (measured after 5 minutes rest in a seated position) at screening must be within the normal ranges for the specific age, as determined by the Investigator.
- •5. Patient may be on concomitant medications and dietary supplements; however, they must be on stable doses for at least 1 month prior to screening, unless approved by the Investigator.
- •6. Patient may have long-term complications of galactosemia, including mild neurological deficits such as ataxia, tremor, dysmetria and dystonia, mild cognitive impairment, and mild language difficulty, and primary ovarian insufficiency, that, in the opinion of the Investigator, do not interfere with the patient’s ability to participate in the study.
- •7. Patient need to be on a galactose restricted diet for =90 days prior to their enrollment in the study and remain and throughout the study.
- •8. Screening 12-lead ECG with normal sinus rhythm without pathological Q wave or clinically significant ST/T wave changes.
- •9. Willing and able to be confined to the clinical research unit (CRU) as required by the protocol.
- •10. Parent(s)/LAR(s) willing and able to consent voluntarily for patient to participate in this study by providing written informed consent prior to the start of any study-specific procedures (also, patient assent, if applicable).
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 47
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1. History or presence of clinically significant hematopoietic, hepatic (i.e. ALT or AST or total bilirubin > 1.5 x ULN), endocrine, metabolic, pulmonary, neurological, psychiatric, immunological, dermatological, or gastrointestinal diseases; conditions capable of altering the absorption, metabolism, or elimination of drugs; or conditions that constitute a risk factor when taking the study drug and/or impact the conduct or results of the study.
- •2. Evidence of diagnosis of any type of galactosemia other than CG. Subjects with Duarte variant galactosemia are not eligible.
- •3. Severe cognitive impairment defined as:
- •a. Bayley Scales of Infant and Toddler Development (Bayley-4) Development Quotient < 50, or Vineland Adaptive Behavior Scales (Vineland-3) Comprehensive Interview Form Standard Score < 50 for age 24 to =30-month old
- •b. Wechsler Preschool and Primary Scale of Intelligence (WPPSI) Full Scale Intelligence Quotient (FSIQ) score < 50, or Vineland-3 Comprehensive Interview Form Standard Score < 50 for age >30 months to < 7 years old (y.o)
- •c. Wechsler Intelligence Scale for Children (WISC) FSIQ score < 50, or Vineland-3 Comprehensive Interview Form Standard Score < 50 for age 7 to < 17 y.o
- •d. Wechsler Adult Intelligence Scale (WAIS) FSIQ score < 50, or Vineland-3 Comprehensive Interview Form Standard Score < 50 for age 17 y.o.
- •4. Complications of CG resulting in disability that, in the opinion of the Investigator (PI), may prevent the patient from completing all study requirements.
- •5. Impaired renal function or estimated glomerular filtration rate < 90 mL/min/1.73 m2 (Schwartz Formula).
- •6. History or presence of significant cardiovascular disorders including myocardial infarction, stroke, left ventricular hypertrophy, atrial fibrillation, valvular heart disease, or uncontrolled hypertension (sitting blood pressure >140/90 mmHg in children =13 to <18 y.o; >120/80 in children =7 to =12 y.o; >110/80 in children aged =2 to =6 y.o).
- •7. Clinically significant overweight or Body Mass Index > 97th percentile for the specific age.
- •8. Clinically significant underweight (in the opinion of the PI) and BMI < 3rd percentile for the specific age and gender.
- •9. Positive test for hepatitis B (HBV) surface antigen, hepatitis C (HCV) antibody, or human immunodeficiency virus (HIV) at screening or previous treatment for HBV, HCV, or HIV infection.
- •10. Abnormal findings on the screening 12-lead ECG, such as ST/T wave changes of ischemia, pathological Q wave changes, or any rhythm other than normal sinus rhythm.
- •11. Evidence of significant active hematological disease and/or cumulative blood donation of 1 unit (500 mL) or more including blood drawn during clinical studies in the last 90 days.
- •12. Pregnant, lactating, or not using/not willing to use appropriate means of contraception.
- •13. Any prior history of substance abuse or treatment for such.
- •14. History of clinically significant drug hypersensitivity reactions.
- •15. Immediate family members of Investigators and site personnel directly affiliated with this study (defined as a child or sibling, whether biological or legally adopted).
- •16. Any significant health problems unrelated to CG or any other condition that, in the opinion of the PI, precludes the patient from following and completing the protocol.
- •17. Unwilling to comply with a galactose-restricted diet.
- •18. Positive urine screen for drugs of abuse unless clinically indicated.
- •19. Participation in another clinical trial of a different IMP (rando
研究者
相似试验
进行中（未招募）
不适用
Evaluation of the clinical utility of the FebriDx® test for viral and bacterial upper respiratory infection in a UK A&E departmentISRCTN14380378niversity of Leeds908
招募中
不适用
A Multicentre Observational Study to Evaluate Clinical Outcomes of the G7 Acetabular SystemHip ArthrosisHip Wear10005944NL-OMON50332Biomet GSCC B.V.21
进行中（未招募）
不适用
A Multicenter, Double-Blind Study to Evaluate the Safety, Efficacy and Pharmacokinetics of the Human Anti-TNF Monoclonal Antibody Adalimumab in Pediatric Subjects with Moderate to Severe Crohn's DiseaseEUCTR2006-004814-41-NLAbbott GmbH & Co. KG184
进行中（未招募）
不适用
A Multicenter, Double-Blind Study to Evaluate the Safety, Efficacy and Pharmacokinetics of the Human Anti-TNF Monoclonal Antibody Adalimumab in Pediatric Subjects with Moderate to Severe Crohn's DiseaseModerate to Severe Crohn's DiseaseMedDRA version: 8.1Level: LLTClassification code 10011401Term: Crohn's diseaseEUCTR2006-004814-41-BEAbbott GmbH & Co. KG184
进行中（未招募）
1 期
A Multicenter, Double-Blind Study to Evaluate the Safety, Efficacy and Pharmacokinetics of the Human Anti-TNF Monoclonal Antibody Adalimumab in Pediatric Subjects with Moderate to Severe Crohn's DiseaseModerate to Severe Crohn's DiseaseMedDRA version: 8.1Level: LLTClassification code 10011401Term: Crohn's diseaseEUCTR2006-004814-41-FRAbbott GmbH & Co. KG192