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CTRI Number  CTRI/2019/02/017732 [Registered on: 20/02/2019] Trial Registered Prospectively
Last Modified On: 18/01/2023
Post Graduate Thesis  No 
Type of Trial  Interventional 
Type of Study   Drug 
Study Design  Other 
Public Title of Study   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 
Scientific Title of Study   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 
Trial Acronym  OLE-IEDAT  
Secondary IDs if Any  
Secondary ID  Identifier 
IEDAT-03-2018  Protocol Number 
 
Details of Principal Investigator or overall Trial Coordinator (multi-center study)  
Name  Shiv Issar 
Designation  Head, Clinical and RA 
Affiliation  CliniRx Tangent Research India Pvt Ltd  
Address  Patriot House, 4th Floor
3 BSZ Marg, New Delhi
Central
DELHI
110002
India 
Phone    
Fax    
Email  shiv.issar@clinirx.com  
 
Details of Contact Person
Scientific Query  
Name  Shiv Issar 
Designation  Head, Clinical and RA 
Affiliation  CliniRx Tangent Research India Pvt Ltd  
Address  Patriot House, 4th Floor
3 BSZ Marg, New Delhi
Central
DELHI
110002
India 
Phone    
Fax    
Email  shiv.issar@clinirx.com  
 
Details of Contact Person
Public Query  
Name  Shiv Issar 
Designation  Head, Clinical and RA 
Affiliation  CliniRx Tangent Research India Pvt Ltd  
Address  Patriot House, 4th Floor
3 BSZ Marg, New Delhi
Central
DELHI
110002
India 
Phone    
Fax    
Email  shiv.issar@clinirx.com  
 
Source of Monetary or Material Support  
EryDel SpA 
 
Primary Sponsor  
Name  EryDel SpA  
Address  Via Meucci 3, 20091 Bresso, Italy  
Type of Sponsor  Pharmaceutical industry-Global 
 
Details of Secondary Sponsor  
Name  Address 
CliniRx Tangent Research India Pvt Ltd   Patriot House, 4th Floor, 3 BSZ Marg, New Delhi-110002  
 
Countries of Recruitment     Australia
Belgium
Germany
India
Israel
Italy
Norway
Poland
Spain
United Kingdom
United States of America  
Sites of Study  
No of Sites = 7  
Name of Principal Investigator  Name of Site  Site Address  Phone/Fax/Email 
Dr Sheffali Gulati  All India Institute of Medical Sciences  Department of Child Neurology, Ansari Nagar, New Delhi-110 029
South
DELHI 		 9868397532

sheffaligulati@gmail.com 
Dr Vinayan KP  Amrita Institute of Medical Sciences   Department of Pediatric Neurology, Ponekkara, Kochi, Kerala 682041
Ernakulam
KERALA 		 9447800303

drvinayan@gmail.com 
Dr Anaita U Hegde  Jaslok Hospital and Research centre, Department of Neurology  Department of Neurology, 15-Dr. Deshmukh Marg, Pedder Road, Mumbai-400 026 Mumbai
Mumbai
MAHARASHTRA 		 9820186155

docanaita@rediffmail.com 
Dr Ravi Yadav  National Institute of Mental Health and Neurosciences  Department of Neurology, Hosur Road, Bangalore-560 029 Bangalore
Bangalore
KARNATAKA 		 9742379536

docravi20@yahoo.com 
Dr Rupam Borgohain  Nizams Institute of Medical Sciences  Department of Neurology, Ground Floor, Millenium Block, Punjagutta, Hyderabad-500 082
Hyderabad
TELANGANA 		 9866191476

b_rupam@hotmail.com 
Dr Vrajesh Udani  P.D. Hinduja National Hospital and Medical Research Centre, Department of Pediatric Neurology  Department of Pediatric Neurology, Veer Savarkar Marg, Mahim, Mumbai, Maharashtra 400016
Mumbai
MAHARASHTRA 		 9819596661

vrajesh.udani@gmail.com 
Dr Suresh Kumar  Vijaya Health Centre  Department of Neurology, Room No.192, OPD Block, No. 323, N.S.K. Salai, Vadapalani, Chennai-600 026
Chennai
TAMIL NADU 		 9841183019

doc_suresh@yahoo.com 
 
Details of Ethics Committee  
No of Ethics Committees= 7  
Name of Committee  Approval Status 
Ethics Committee, All India Institute of Medical Sciences, Room # 102, 1st Floor, Old OT Building, Ansari Nagar, New Delhi-110029   Submittted/Under Review 
Ethics Committee, Jaslok Hospital & Research Centre, 15, Dr. S. Deshmukh Marg, Pedder Road, Mumbai  Approved 
Instituional Ethics Committee, Administrative Office, Vijaya Health Centre, 175, NSK Salai, Vadapalani, Chennai-600026.  Submittted/Under Review 
Institutional Ethics Committee, Nizam’s Institute of Medical Sciences, Punjagutta, Hyderabad – 500 082- Telangana  Submittted/Under Review 
Institutional Ethics Committee, Amrita Institute of Medical and Research Centre, AIMS – Ponekkara Post, Kochi, Kerala – 682 041  Submittted/Under Review 
Institutional Ethics Committee, P.D. Hinduja National Hospital & Medical Research Centre, Veer Savarkar Marg, Mumbai-400 016  Submittted/Under Review 
NIMHANS Human Ethics Committee, NIMHANS, Bangalore-560029  Submittted/Under Review 
 
Regulatory Clearance Status from DCGI  
Status 
Approved/Obtained 
 
Health Condition / Problems Studied  
Health Type  Condition 
Patients  (1) ICD-10 Condition: Q079||Congenital malformation of nervoussystem, unspecified,  
 
Intervention / Comparator Agent  
Type  Name  Details 
Intervention  EDS-EP dose range of approx 14-22 mg  EDS-EP dose range of approx 14-22 mg DSP/infusion Frequency: Once in a month Route: IV  
Comparator Agent  EDS-EP dose range of approx 14-22 mg  EDS-EP dose range of approx 14-22 mg DSP/infusion Frequency: Once in a month Route: IV 
 
Inclusion Criteria  
Age From  6.00 Year(s)
Age To  99.00 Year(s)
Gender  Both 
Details  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 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.  
 
ExclusionCriteria 
Details  General 1. 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. 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 lifethreatening 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.  
 
Method of Generating Random Sequence   Not Applicable 
Method of Concealment   Not Applicable 
Blinding/Masking   Open Label 
Primary Outcome  
Outcome  TimePoints 
To monitor and evaluate the long-term safety and tolerability of EDS-EP in AT patients.   12 months 
 
Secondary Outcome  
Outcome  TimePoints 
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).

To evaluate the long-term effect of EDS-EP on health related Quality of Life (QoL; EQ-5D-5L scale).  		 12 months 
 
Target Sample Size   Total Sample Size="50"
Sample Size from India="12" 
Final Enrollment numbers achieved (Total)= "Applicable only for Completed/Terminated trials"
Final Enrollment numbers achieved (India)="Applicable only for Completed/Terminated trials" 
Phase of Trial   Phase 3 
Date of First Enrollment (India)   25/02/2019 
Date of Study Completion (India) Applicable only for Completed/Terminated trials 
Date of First Enrollment (Global)  18/01/2019 
Date of Study Completion (Global) Applicable only for Completed/Terminated trials 
Estimated Duration of Trial   Years="18"
Months="0"
Days="0" 
Recruitment Status of Trial (Global)
Modification(s)  		 Completed 
Recruitment Status of Trial (India)  Other (Terminated) 
Publication Details
Modification(s)  		 None 
Individual Participant Data (IPD) Sharing Statement

Will individual participant data (IPD) be shared publicly (including data dictionaries)?  

Response - NO
Brief Summary   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.


 
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