2023-509077-23-00招募中3 期
A Multi-center, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Neurological Effects of EryDex on subjects with Ataxia-Telangiectasia.
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 67
- 试验地点
- 12
- 主要终点
- Primary Efficacy Endpoint= To evaluate the effect of EryDex on CNS symptoms, as measured by the change of the RmICARS from baseline to Visit 9 compared to placebo in A-T (6- to 9-year-old participants primary analysis population).
研究概览
简要总结
Primary Efficacy Objective: To evaluate the effect of eDSP on CNS symptoms, as measured by the change in the RmICARS from baseline to Visit 9 compared to placebo in A-T (6- to 9-year-old participants primary analysis population).
研究设计
- 分配方式
- Randomized
- 主要目的
- Multi-center, randomized, double-blind, placebo-controlled, Phase 3 study
- 盲法
- Double (Analyst, Subject, Monitor, Investigator)
入排标准
- 年龄范围
- 0 years 至 64 years（18-64 Years, 0-17 Years）
- 接受健康志愿者
- 是
入选标准
- •Participants meets clinical criteria for diagnosis of A-T. The neurological signs of A-T (incoordination of the head and eyes in lateral gaze deflection, gait ataxia associated with an inappropriately narrow base) must be documented. Such signs of A-T illustrate the body systems in which changes shall be confirmed, but the listed signs are examples and other changes in those systems may be observed and documented to confirm the diagnosis of A-T.
- •Participant is in autonomous gait or is helped by periodic use of a support (i.e., ICARS score for Item 1 – Walking Capacities between 0 and 4 included).
- •Participant is at least 6 years of age (Dose 1 must be on or after date of 6th birthday), of either sex.
- •Genetic confirmation of A-T.
- •Body weight ≥15 kg.
- •The participant and parent/caregiver (if below the age of consent), or a legal representative, has provided written informed consent to participate. If consent is provided solely by the caregiver in accordance with local regulations, the participant must provide assent to participate in the trial, to the extent possible.
排除标准
- •General - A disability that may prevent the participant from completing all trial requirements.
- •Medical History and Current Status - Any clinically significant abnormality on standard laboratory examinations (haematology, biochemistry, urinalysis) at screening that remains abnormal on repeat testing, if considered as a possible sign of a clinical condition putting the participant at risk if enrolled. Eligibility of participants with abnormal laboratory test values will be determined by the Investigator in consultation with the Medical Monitor.
- •Medical History and Current Status - Participant with an early morning plasma cortisol level below 3-5 μg/dL (depending on assay), or participant exhibits signs or symptoms of adrenal insufficiency, with an early morning plasma cortisol level below 10 μg/dL, and fails the ACTH stimulation test at screening.
- •Medical History and Current Status - Confirmed hemoglobinopathies (e.g., haemoglobin C disease, sickle cell anemia, hereditary spherocytosis, or thalassemia).
- •Medical History and Current Status - Current chronic or acute significant renal and/or hepatic impairment that in Investigator’s opinion will impact participant participation in the trial.
- •Medical History and Current Status - Participants with suicidal ideation
- •Medical History and Current Status - Females whoare pregnant or breast feeding. Females of childbearing potential using an adequate birth control method, as determined by their healthcare provider, will be eligible. For further details on the adequate contraceptive measures
- •Prior/Concomitant Medication - Any previous oral or parenteral steroid use within 6 weeks before Baseline. Treatment with inhaled or intranasal steroids for asthma or allergies, as well as use of topical steroids will be permitted.
- •Prior/Concomitant Medication - Chronic condition or prior allergic reaction representing a contraindication to the use of dexamethasone or other steroid drugs.
- •Prior/Concomitant Medication - Has participated in any other trial with an investigational drug and received a dose within 30 days or at least 5 half-lives (whichever is greater) prior to the Screening visit.
- •Prior/Concomitant Medication - Has participated in a previous trial with EryDex treatment
- •General - Current participation in another clinical trial. Participation in observational, non-interventional studies is allowed with approval by the Medical Monitor as long as trial investigational endpoint raters can remain blinded to the assessments from other studies, and as long as the other trial participation does not interfere with participation in this trial.
- •Prior/Concomitant Medication - Requires any concomitant medication prohibited by the protocol
- •Medical History and Current Status - Immune impairment that includes CD4+ lymphocytes count <400/mm3 (for participants less than 7 years old) or <150/mm3 (for participants ≥7 years old). In presence of oral infections, like oral candidiasis, documented at screening or recurrent as per medical history documentation, the limit increases to <200/mm3 (for participants ≥7 years old).
- •Medical History and Current Status - History of severe impairment of the immunological system such that steroid treatment would be contraindicated.
- •Medical History and Current Status - Loss/removal of 250 mL or more of blood within the past 4 weeks prior to screening.
- •Medical History and Current Status - Current neoplastic disease or previous neoplastic disease not in remission for at least 2 years.
- •Medical History and Current Status - Severe or unstable pulmonary disease that impacts participant participation in the trial, in the opinion of the Investigators.
- •Medical History and Current Status - Uncontrolled diabetes. Participants with diabetes that has been stabilized (i.e., no hypoglycaemic or hyperglycaemic episodes in the past 3 months) will be eligible.
- •Medical History and Current Status - Any other severe, unstable, or serious disease or condition that in the Investigator’s opinion would put the participant at risk for imminent life-threatening morbidity, need for hospitalization, or mortality.
结局指标
主要结局
Primary Efficacy Endpoint= To evaluate the effect of EryDex on CNS symptoms, as measured by the change of the RmICARS from baseline to Visit 9 compared to placebo in A-T (6- to 9-year-old participants primary analysis population).
Primary Efficacy Endpoint= To evaluate the effect of EryDex on CNS symptoms, as measured by the change of the RmICARS from baseline to Visit 9 compared to placebo in A-T (6- to 9-year-old participants primary analysis population).
次要结局
- Key Secondary Efficacy Endpoint = To evaluate the overall clinical effect of EryDex, compared to placebo, in A-T (6- to 9-year-old participants primary analysis population), based on CGI-S from baseline to Visit 9 (Day 168)
- Other Secondary Efficacy Endpoint = CGI-C from baseline to Visit 9 (Day 168)
研究者
Dirk Thye
Scientific
Quince Therapeutics S.p.A.
研究点 (12)
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