Open-label, Long-term, Extension Treatment using Intra-Erythrocyte Dexamethasone Sodium Phosphate in Patients with Ataxia Telangiectasia Who Participated in the IEDAT-02-2015 Study
试验速览
- 阶段
- 3 期
- 状态
- Other
- 发起方
- EryDel SpA
- 入组人数
- 50
- 试验地点
- 7
- 主要终点
- To monitor and evaluate the long-term safety and tolerability of EDS-EP in AT patients.
研究概览
简要总结
This is an international (North America, Europe, Africa, Asia and Australia), multi-center, prospective, open-label treatment study, designed to continue to provide the study medication to all patients who completed 12 months of treatment (including those treated with placebo) in the IEDAT-02-2015 trial, completed the study assessments, do not present safety contraindication to continuation of treatment, and provided informed consent. The study aims to collect information on the long-term safety and efficacy of the trial treatment. Patients meeting all selection criteria will receive monthly infusions of EDS-EP (dose range of ~14-22 mg DSP/infusion). If this dose of EDS-EP is not tolerated, the patient should be discontinued from the study. During the study, long-term efficacy assessments will be performed every 6 months, while safety parameters will be assessed at each monthly visit. The ICARS, EQ-5D-5L and the CGI-C/S will be administered by a site rater.
All patients enrolled in this study will have participated in Study IEDAT-022015, and there will be no de novo enrollment of new patients. It is estimated that a maximum of 50 patients from the prior study will enter this study.
研究设计
- 研究类型
- Interventional
- 分配方式
- Not Applicable
- 盲法
- Open Label
入排标准
- 年龄范围
- 6.00 Year(s) 至 99.00 Year(s)（—）
- 性别
- All
入选标准
- •Patient completed the double-blind period in the IEDAT-02-2015 trial and must have completed the final (Visit 15/Month 12) efficacy assessments of IEDAT-02-
- •Patient tolerated the study medication, without any evidence of steroid adverse events, or treatment-related severe/ serious adverse events.
- •Body weight > 15 kg.
- •The patient and his/her 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 patient must provide assent to participate in the study.
- •Patient does not present safety contraindication for continuation of treatment, as determined by the Principal Investigator (PI) according to the procedures described below.
- •Procedure for selecting patients for further treatment in IEDAT-03-2018 ï‚· The Principal Investigator will ask all patients who meet the above requirements, and determine their interest in continuing to receive treatment with the study medication in a new protocol.
- •The Principal Investigator will then determine the eligibility of the patients on the basis of his/her clinical judgement of patients’ status and their safety.
排除标准
- •Females that are a.
- •pregnant, or are breast-feeding (for EU countries only); b.
- •of childbearing potential, pregnant, or are breast-feeding (for US and Rest of World countries).
- •Females of childbearing potential using adequate birth control, as determined by their Health Care Provider, will be eligible.
- •A disability that may prevent the patient from completing all study requirements.
- •Current participation in a clinical study with another investigational drug.
- •Medical History and Current Status
- •CD4+ lymphocytes count <400/mm3 (for patients 6 years of age) or <200/mm3 (for patients >6 years).
- •Current neoplastic disease.
- •Severe impairment of the immunological system.
- •Uncontrolled diabetes.
- •Patients with diabetes that has been stabilized (i.e. no hypoglycemic or hyperglycemic episodes in the past 3 months) will be eligible.
- •Any other severe, unstable, or serious disease or condition that in the Investigator’s opinion would put the patient at risk for imminent lifethreatening morbidity, need for hospitalization, or mortality.
- •Eligibility of patients with abnormal laboratory test values will be determined by the Investigator.
- •Confirmed hemoglobinopathies, e.g. hemoglobin C disease, sickle cell anemia, or thalassemia.
- •Moderate or severe renal and/or hepatic impairment.
- •Patients who experienced moderate/ severe steroid side effects, or moderate/ severe adverse events associated with the study medication administered in the IEDAT-02 study.
- •Prior/Concomitant Medication
- •Treatment with inhaled or intranasal steroids for asthma or allergies, as well as use of topical steroids will be permitted.
- •Requires any other concomitant medication prohibited by the protocol.
- •Use of any drug that is a strong inducer/inhibitor of CYP3A4.
结局指标
主要结局
To monitor and evaluate the long-term safety and tolerability of EDS-EP in AT patients.
时间窗: 12 months
次要结局
- To monitor and evaluate the long-term safety and tolerability of EDS-EP in AT patients.(To evaluate the long term effect of EDS-EP in treating CNS symptoms as measured by the “Modified†International Cooperative Ataxia Rating Scale (mICARS), and Clinical Global Impression of severity and change (CGI-S/C).)