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

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

Sponsors

Lead Sponsor: Erydel

Source Erydel
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.

Detailed Description

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-02-2015, and there will be no de novo enrollment of new patients.

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.

Overall Status Recruiting
Start Date June 12, 2018
Completion Date August 2020
Primary Completion Date August 2020
Phase Phase 3
Study Type Interventional
Primary Outcome
Measure Time Frame
Long term safety/tolerability of EDS-EP: # of pts with treatment related Adverse Events (CTCAE4.0) 1 year
Long term safety/tolerability of EDS-EP: lab parameters according to clinical practice such as hematology, biochemistry, urinalysis, HbA1c (haemoglobin A1c or glycated haemoglobin test), CD4+ lymphocytes count, CRP (C reactive protein) 1 year
Long term safety/tolerability of EDS-EP: 12-lead ECG (Electrocardiography) 1 year
Long term safety/tolerability of EDS-EP: physical and neurological exam 1 year
Long term safety/tolerability of EDS-EP: Columbia Suicide Severity Rating Scale (CSSRS) 1 year
Long term safety/tolerability of EDS-EP: Bone Mineral Density 1 year
Long term safety/tolerability of EDS-EP: Tanner staging 1 year
Long term safety/tolerability of EDS-EP: sterility test 1 year
Secondary Outcome
Measure Time Frame
To evaluate the long-term effect of EDS-EP on health related Quality of Life (EQ-5D-5L scale: EuroQol 5 dimension 5 level scale) 1 year
Enrollment 50
Condition
Intervention

Intervention Type: Combination Product

Intervention Name: EryDex System

Description: EryDex System is a combination product that is used to load dexamethasone sodium phosphate (DSP) into autologous erythrocytes (EDS) creating the EDS-end product which is infused into the patients

Arm Group Label: active drug

Eligibility

Criteria:

Inclusion Criteria:

- 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.

- 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:

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.

Exclusion Criteria:

General

1. Females that are

1. pregnant, or are breast-feeding (for EU countries only);

2. 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.

Gender: All

Minimum Age: 6 Years

Maximum Age: N/A

Healthy Volunteers: No

Overall Contact

Last Name: Irene Maccabruni, BS

Phone: +393458415028

Email: [email protected]

Location
Facility: Status: Contact:
Ataxia Center and HD Center of Excellence - UCLA | Los Angeles, California, 90095, United States Recruiting Susan Perlman, MD
Division of Pediatric Allergy and Immunology - Johns Hopkins Hospital | Baltimore, Maryland, 21287-3923, United States Recruiting Howard M. Lederman, M.D., Ph.D.
Cincinnati Children's Hospital Medical Center | Cincinnati, Ohio, 45229, United States Recruiting Steve Wu, MD
Department of Pediatrics Division of Child and Adolescent Neurology Mitochondrial Clinic - University of Texas Medical School | Houston, Texas, 77030, United States Recruiting Mary Kay Koenig, MD
Department of Neurology Royal Children's Hospital | Parkville, Victoria, 3052, Australia Recruiting Monique M Ryan, MD
Laboratory of Pediatric Immunology UZ Leuven | Leuven, 3000, Belgium Recruiting Isabelle Meyts, MD
Klinik für Kinder- und Jugendmedizin, Allergologie, pneumologie und Mukoviszidose, Universitätsklinikum Frankfurt | Frankfurt, 60590, Germany Recruiting Stefan Zielen, MD
National Institute of Mental Health and Neurosciences (NIMHANS) Department of Neurology | Bangalore, 560 029, India Recruiting Ravi Yadav, MD
Vijaya Health Centre Department of Neurology | Chennai, 600 026, India Recruiting Suresh Kumar, MD
Nizam's Institute of Medical Sciences Department of Neurology | Hyderabad, 500 082, India Recruiting Rupam Borgohain, MD
Jaslok Hospital and Research Center Department of Pediatric Neurology | Mumbai, 400 026, India Recruiting Anaita Udwadia-Hegde, MD
P.D. Hinduja National Hospital and Medical Research Centre Department of Pediatric Neurology | Mumbai, 400016, India Not yet recruiting Vrajesh Udani, MD
All India Institute of Medical Sciences Child Neurology Division Department of Pediatrics | New Delhi, 110 029, India Not yet recruiting Sheffali Gulati, MD
Department of Clinical Immunology - The Children's Memorial Health Institute | Warsaw, 04-730, Poland Recruiting Barbara Pietrucha, MD
Servicio de Neurolgia Pediatrica, Hospital Materno-Infantil La Paz | Madrid, 28046, Spain Recruiting Samuel Ignacio Pascual, MD
Location Countries

Australia

Belgium

Germany

India

Poland

Spain

United States

Verification Date

January 2020

Responsible Party

Type: Sponsor

Condition Browse
Number Of Arms 1
Arm Group

Label: active drug

Type: Experimental

Description: ~14-22 mg dexamethasone sodium phosphate (DSP)

Acronym OLE-IEDAT
Patient Data Undecided
Study Design Info

Allocation: N/A

Intervention Model: Single Group Assignment

Intervention Model Description: Open-label, Long-term, Extension Treatment

Primary Purpose: Treatment

Masking: None (Open Label)

Source: ClinicalTrials.gov