CTIS2023-506028-10-00进行中（未招募）1 期
A Phase 1/2, Randomized, Double-blind, Placebo-controlled Single- and Multiple-dose Escalation Study Evaluating the Safety, Tolerability,Pharmacokinetics, and Pharmacodynamics of VX-670 in Adult Subjects with Myotonic Dystrophy Type 1 - VX23-670-001
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 36
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 64（—）
- 性别
- All
入选标准
- •3. Body mass index (BMI) <35.0 kg/m2, inclusive, and a total body weight >40 kg., 4. Subjects (male and female) between the ages of 18 to 64 years, inclusive. Women of childbearing potential may be enrolled as allowed by local regulatory guidance., 5. Documented clinical diagnosis of DM1 with age of onset >1 year of age and documented positive genetic test for DM1 in the subject with CTG repeat of at least 100., 6. Part B: Ambulatory, defined as having the ability to complete 10-meter walk/run timed test unassisted (e.g., without the use of a cane or walker) or with the use of a brace or other orthotic device only., 7. Part B: Evidence of myotonia, defined by HOT of =2 seconds., 8. Part B: Evidence of weakness, defined by percent predicted QMT of hand grip of 20 to 80%, 9. Left ventricular ejection fraction (LVEF) >55% within the past 3 months.
排除标准
- •1. History of any illness or any clinical condition that, in the opinion of the investigator or the subject’s general practitioner, might confound the results of or participation in the study or pose an additional risk in administering study drug to the subject. This may include, but is not limited to, history of relevant drug or food allergies; history of cardiovascular or central nervous system disease (other than DM1); history or presence of clinically significant pathology; history of mental disease; significant intellectual or behavioral disability; and history of cancer, except for squamous cell skin cancer, basal cell skin cancer, and Stage 0 cervical carcinoma in situ (all 3 diagnosed = 3 years ago with no recurrence in the past 3 years)., 11. Clinically significant liver disease, even if intermittent., 12. History of cardiac anomalies., 13. Clinically significant kidney disease., 14. Exposure to any other investigational nucleic acid, cell and genetic therapies, including siRNA, ASO, mRNA within 6 months before Day 1 or 5 half-lives of investigational agent (confirmed at Screening), whichever is longer., 15. Exposure to any other investigational drugs or devices within 1 month before Day 1, 16. Part B: Any contraindication to a muscle biopsy in the opinion of the investigator including but not limited to: a limb injury, bleeding diathesis, ascites, or significant atrophy of the tibialis anterior muscles., 17. Thyroid dysfunction that is untreated (if on thyroid hormone replacement therapy, need to have adequate and stable replacement over the previous 6 months)., 18. Diabetes that is uncontrolled, in the opinion of the Investigator., 2. History of febrile illness or other acute illness that has not fully resolved within 14 days before the first dose of study drug., 3. Median QTcF of triplicate standard 12-lead ECGs >450 msec at Screening., 4. For female subjects: Females who are currently breastfeeding or pregnant or planning to become pregnant during the study or within 180 days after the last dose of study drug. a. For male subjects: Male subjects with a female partner who is pregnant, nursing, or planning to become pregnant during the study or within 180 days after the last dose of study drug., 5. Blood donation (of approximately 1 pint [500 mL] or more) within 56 days before the first dose of study drug., 6. Use of the substances, activities, or devices within the specified duration before the first study drug dose., 7. A screen positive for hepatitis B surface antigen (HBsAg), hepatitis C virus (HCV) antibody, or antibodies against human immunodeficiency virus 1 or 2 (HIV1 and HIV2 Abs)., 8. Hypersensitivity to any component of the investigational drug product or placebo, 10. Abnormal and clinically significant values for clinical chemistry, hematology, coagulation, or urinalysis parameters at Screening unless explained by disease or are benign in nature (such as Gilbert’s disease).
研究者
相似试验
进行中（未招募）
1 期
A Phase 1 and 2 study of multiple increasing doses to evaluate the ability of MEDI0382 to produce the intended result and the safety of effects of MEDI0382 in overweight and obese subjects with Type 2 Diabetes.Type 2 Diabetes in Overweight and Obese PatientsMedDRA version: 19.0Level: PTClassification code 10067585Term: Type 2 diabetes mellitusSystem Organ Class: 10027433 - Metabolism and nutrition disordersEUCTR2014-003716-36-DEMedImmune Limited, a wholly owned subsidiary of AstraZeneca107
进行中（未招募）
1 期
A 2-Part Safety and Tolerability Study of ALN-AAT02 in Healthy Volunteers and Patients with ZZ Type Alpha-1 Antitrypsin Deficiency Liver DiseaseZZ Type Alpha-1 Antitrypsin Deficiency Liver DiseaseMedDRA version: 20.1Level: PTClassification code 10001806Term: Alpha-1 anti-trypsin deficiencySystem Organ Class: 10010331 - Congenital, familial and genetic disordersEUCTR2018-001362-41-GBAlnylam Pharmaceuticals, Inc.96
进行中（未招募）
1 期
A Phase 1/2, Randomized, Double-Blind, Placebo-Controlled, Parallel-Arm Study of the Safety and Efficacy of LX3305, a Sphingosine-1-Phosphate Lyase Inhibitor, for Treatment of Darier’s Disease or Hailey-Hailey DiseaseDarier’s Disease or Hailey-Hailey DiseaseMedDRA version: 20.0Level: LLTClassification code 10011860Term: Darier's diseaseSystem Organ Class: 100000004850MedDRA version: 20.1Level: LLTClassification code 10019029Term: Hailey-Hailey diseaseSystem Organ Class: 100000004850EUCTR2018-000373-80-FRDermecular Therapeutics, Inc.33
进行中（未招募）
1 期
This global, multicenter, Phase 1/2, randomized, double-blind, placebo-controlled study will evaluate the efficacy and safety of adding TL-895 treatment to standard available therapy (SAT) in subjects with cancer hospitalized for COVID-19.EUCTR2020-002259-39-HUTelios Pharma, Inc.146
进行中（未招募）
1 期
A Phase 1/2 study of S-268019 in Japanese adult participantsJPRN-jRCT2051200092agata Tsutae300