EUCTR2018-000338-36-NO进行中（未招募）1 期
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 - OLE-IEDAT
EryDel S.p.A.0 个研究点目标入组 104 人开始时间: 2020年2月4日最近更新:
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 104
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •1.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-02-2015.
- •2.Patient tolerated the study medication, without any evidence of steroid adverse events, or treatment-related severe/ serious adverse events.
- •3.Body weight > 15 kg.
- •4.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.
- •5.Patient does not present safety contraindications for continuation of treatment, as determined by the Principal Investigator (PI) according to the procedures described below.
- •Procedure for selecting patients for further treatment:
- •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.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 139
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 16
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
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 will be eligible. A woman is considered of childbearing potential (WOCBP), i.e. fertile, following menarche and until becoming post-menopausal unless permanently sterile. Permanent sterilisation methods include hysterectomy, bilateral salpingectomy and bilateral oophorectomy. A postmenopausal state is defined as no menses for 12 months without an alternative medical cause. A high follicle stimulating hormone (FSH) level in the postmenopausal range may be used to confirm a post-menopausal state in women not using hormonal contraception or hormonal replacement therapy. However in the absence of 12 months of amenorrhea, a single FSH measurement is insufficient. See Section 11.7 Concomitant Medications for adequate methods of birth control.
- •2.A disability that may prevent the patient from completing all study requirements.
- •3.Current participation in a clinical study with another investigational drug.
- •Medical History and Current Status
- •4.CD4+ lymphocytes count <400/mm3 (for patients 6 years of age) or <200/mm3 (for patients >6 years).
- •5.Current neoplastic disease.
- •6.Severe impairment of the immunological system.
- •7.Severe or unstable pulmonary disease.
- •8.Uncontrolled diabetes.
- •Patients with diabetes that has been stabilized (i.e. no hypoglycemic or hyperglycemic episodes in the past 3 months) will be eligible.
- •9.Any other severe, unstable, or serious disease or condition that in the Investigator’s opinion would put the patient at risk for imminent life-threatening morbidity, need for hospitalization, or mortality.
- •10.Eligibility of patients with abnormal laboratory test values will be determined by the Investigator.
- •11.Confirmed hemoglobinopathies, e.g. hemoglobin C disease, sickle cell anemia, or thalassemia.
- •12.Moderate or severe renal and/or hepatic impairment.
- •13.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
- •14.Requires treatment with an oral or parenteral steroid. Treatment with inhaled or intranasal steroids for asthma or allergies, as well as use of topical steroids will be permitted.
- •15.Requires any other concomitant medication prohibited by the protocol.
- •16.Use of any drug that is a strong inducer/inhibitor of CYP3A4.
研究者
相似试验
进行中（未招募）
1 期
ot ApplicablePatient with neurological symptoms of Ataxia TelangiectasiaMedDRA version: 21.0Level: PTClassification code 10003594Term: Ataxia telangiectasiaSystem Organ Class: 10010331 - Congenital, familial and genetic disordersEUCTR2018-000338-36-DEEryDel S.p.A.104
Unknown
3 期
Open-label, Long-term, Extension Treatment using Intra-Erythrocyte Dexamethasone Sodium Phosphate in Patients with Ataxia Telangiectasia Who Participated in the IEDAT-02-2015 StudyCTRI/2019/02/017732EryDel SpA15
进行中（未招募）
1 期
ot ApplicableEUCTR2018-000338-36-BEEryDel S.p.A.51
进行中（未招募）
1 期
ot ApplicableEUCTR2018-000338-36-PLEryDel S.p.A.155
进行中（未招募）
1 期
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.eurological symptoms of Ataxia TelangiectasiaMedDRA version: 21.0Level: PTClassification code 10003594Term: Ataxia telangiectasiaSystem Organ Class: 10010331 - Congenital, familial and genetic disordersEUCTR2018-000338-36-ITERYDEL S.P.A.155