ISRCTN12014920已完成2 期
A Phase II open-label pilot study to evaluate the safety and efficacy of subcutaneously administered ELX-02 in patients with Alport syndrome with Col4A5 and Col4A3/4 nonsense mutation
Eloxx Pharmaceuticals (United States)0 个研究点目标入组 3 人开始时间: 2022年5月5日最近更新:
适应症
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 3
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. Evidence of signed and dated informed consent/assent document(s) indicating that the participant (and/or their parent/legal guardian) has been informed of all pertinent aspects of the trial
- •2. Understands, and is willing and able to comply with all scheduled visits, treatment plan, laboratory tests, and other study procedures
- •3. Male and female participants
- •4. Aged between 6 and 30 years
- •5. A confirmed diagnosis of X-linked or autosomal recessive Alport syndrome with a documented nonsense mutation of Col4A5 in a male or nonsense mutation of Col4A3 or Col4A4 (male or female)
- •6. The nonsense mutation should be UAG or UGA
- •7. eGFR >60 ml/min/1.73 m² (based on Chronic Kidney Disease Epidemiology Collaboration [CKD-EPI] for ages =18 years and Schwartz formula for participants <18 years)
- •8. Urinary protein based on two spot urine collections [urine protein/creatinine ratio (UPCR) =500 mg/g]
- •9. Stable regimen of ACEi/ARB for 4 weeks before screening (unless there is a contraindication)
- •10. Must be willing to abstain from strenuous exercise during the 48 h prior to study visits
- •11. Females of childbearing potential and males capable of fathering a child must meet the contraception requirements (outlined in protocol section 6.3)
- •12. Non-lactating females
- •13. Females on hormone replacement therapy (estrogen or progesterone) or contraceptive therapy must be stabilized on a product and dose for at least 30 days prior to Screening
- •14. Have not received systemic medications with the potential to impair renal function on a frequent basis (e.g., nonsteroidal anti-inflammatory drugs [NSAIDs]) or with ototoxic potential (e.g., quinine or salicylates), or any injectable aminoglycosides for a period of at least 14 days prior
- •15. Negative human immunodeficiency virus (HIV), hepatitis B surface antigen (HBsAg), and hepatitis C virus antigen (HCV Ag) serology tests at Screening
- •16. No history of alcohol or drug abuse within the 6 months prior to Screening
- •17. Body Mass Index (BMI) of 19.0 to 30.0 kg/m² (inclusive). Participants with a lower BMI may be entered into the study at the discretion of the investigator following consultation with the Sponsor
排除标准
- •1. Participants who are investigational site staff members directly involved in the conduct of the study and their family members, site staff members otherwise supervised by the Investigator, or participants who are Eloxx Pharmaceuticals employees directly involved in the conduct of the study
- •2. Participation in clinical study including administration of any investigational drug or device in the last 30 days or 5 half-lives (whichever is longer) prior to investigational product dosing in the current study
- •3. Use of prohibited medications as defined in (protocol) section 6.1 within the specified windows
- •4. History of any comorbidity which in the opinion of the investigator might confound the study or pose an additional risk in administering the study drug to the participant
- •5. History of any organ transplantation
- •6. Mutation consistent with autosomal dominant Alport syndrome
- •7. Liver disease characterized by cirrhosis or portal hypertension. Participants with alanine aminotransferase (ALT), aspartate aminotransferase (AST), and/or a total bilirubin 3.0 times the upper limit of normal (ULN) will be excluded
- •8. History of congestive heart failure diagnosed clinically or with documented left ventricular ejection fraction (LVEF) =40%
- •9. Evidence or history of clinically relevant psychiatric condition
- •10. A positive urine drug screen (amphetamines, benzodiazepines, cocaine and opiates) at Screening
- •11. Screening supine 12-lead electrocardiogram (ECG) demonstrating any clinically significant findings as judged by the Investigator
- •12. Participants with any abnormalities in clinical laboratory tests at Screening, considered by the study Investigator as clinically relevant
- •13. Major surgery within 180 days (6 months) of Screening
- •14. History of dialysis
- •15. Known allergy to any aminoglycoside
- •16. Participants with any acute medical situation unresolved within 14 days of first dose that is considered of significance by the Investigator
- •17. Participants with >10 dB change in threshold between audiometric Initial test to baseline test for the frequencies from 0.5 -12.5 kHz
- •18. Dizziness Handicap Inventory (DHI) score at screening =16 for adults, and active dizziness reported for pediatric participants.
研究者
相似试验
进行中（未招募）
不适用
A Phase II, Open Label Pilot Study to Evaluate the Safety and Efficacy of A Bioresorbable Subcutaneous Implant of CUV1647 in Patients with Solar Urticaria (SU) - Phase II SU Pilot StudySolar Urticaria (SU)MedDRA version: 9.1Level: LLTClassification code 10041307Term: Solar urticariaEUCTR2007-001068-55-GBClinuvel Pharmaceuticals Limited
进行中（未招募）
1 期
A dose escalation trial to assess the safety and tolerability of multiple doses of the LAVA-051 antibody in patients with chronic lymphocytic leukemia, multiple myeloma or acute myeloid leukemiaCD1d-positive relapsed/refractory chronic lymphocytic leukemia (CLL), multiple myeloma (MM), or acute myeloid leukemia (AML)EUCTR2020-004583-26-ESAVA Therapeutics B.V.102
招募中
1 期
A dose escalation trial to assess the safety and tolerability of multiple doses of the LAVA-051 antibody in patients with chronic lymphocytic leukemia, multiple myeloma or acute myeloid leukemiaRelapsed/refractory chronic lymphocytic leukemia (CLL), multiple myeloma (MM), or acute myeloid leukemia (AML)EUCTR2020-004583-26-FRAVA Therapeutics B.V.102
进行中（未招募）
1 期
A dose escalation trial to assess the safety and tolerability of multiple doses of the LAVA-051 antibody in patients with chronic lymphocytic leukemia, multiple myeloma or acute myeloid leukemiaRelapsed/refractory chronic lymphocytic leukemia (CLL), multiple myeloma (MM), or acute myeloid leukemia (AML)EUCTR2020-004583-26-NLAVA Therapeutics N.V.114
招募中
不适用
A Phase 1 and 2a open-label study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, immunogenicity and antitumor activity of LAVA-051 in patients with relapsed or refractory chronic lymphocytic leukemia, multiple myeloma or acute myeloid leukemiamyeloma10024324leukemiaNL-OMON51940AVA Therapeutics BV20