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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)

Primary Purpose

Ataxia Telangiectasia

Status
Unknown status
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
EryDex System
Sponsored by
Erydel
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Ataxia Telangiectasia

Eligibility Criteria

6 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Ataxia Center and HD Center of Excellence - UCLA
  • Division of Pediatric Allergy and Immunology - Johns Hopkins Hospital
  • Cincinnati Children's Hospital Medical Center
  • Department of Pediatrics Division of Child and Adolescent Neurology Mitochondrial Clinic - University of Texas Medical School
  • Department of Neurology Royal Children's Hospital
  • Laboratory of Pediatric Immunology UZ Leuven
  • Klinik für Kinder- und Jugendmedizin, Allergologie, pneumologie und Mukoviszidose, Universitätsklinikum Frankfurt
  • National Institute of Mental Health and Neurosciences (NIMHANS) Department of Neurology
  • Vijaya Health Centre Department of Neurology
  • Nizam's Institute of Medical Sciences Department of Neurology
  • Jaslok Hospital and Research Center Department of Pediatric Neurology
  • P.D. Hinduja National Hospital and Medical Research Centre Department of Pediatric Neurology
  • All India Institute of Medical Sciences Child Neurology Division Department of Pediatrics
  • Dipartimento Neuroscienze umane e salute mentale, Policlinico Umberto I Università La SapienzaRecruiting
  • Department of Clinical Immunology - The Children's Memorial Health Institute
  • Servicio de Neurolgia Pediatrica, Hospital Materno-Infantil La Paz
  • Razi Hospital, Clinical Investigation Center-NeuroscienceRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

active drug

Arm Description

~14-22 mg dexamethasone sodium phosphate (DSP)

Outcomes

Primary Outcome Measures

Long term safety/tolerability of EDS-EP: # of pts with treatment related Adverse Events (CTCAE4.0)
AEs will be listed and summarized by body system and preferred term. Incidence of AEs(%),intensity and relatedness to study drug will be reported. SAEs and events newly occurring or worsening after administration of study medication will be summarized. AEs that result in death/discontinuation of the study medication will be listed separately
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)
Values will be listed and summarized as normal or abnormal, according to local normal ranges, depending on the lab assessment (not possible to provide unique range and unit of measure due to changes from lab to lab). Abnormal and clinically notable values will be identified and listed for each parameter
Long term safety/tolerability of EDS-EP: 12-lead ECG (Electrocardiography)
Results will be listed and summarized as normal or abnormal, according guidelines (PR interval: < 100 msec or > 210 msec; QRS interval: < 50 msec or > 120 msec; QTc interval: > 450 msec; Heart rate: < 50 bpm (sinus bradycardia) or > 120 bpm (sinus tachycardia); Morphology: presence of T-wave inversion, abnormal R-waves, pathological Q-waves, significant ST elevation or depression). Abnormal and clinically notable values will be identified and listed
Long term safety/tolerability of EDS-EP: physical and neurological exam
Results will be listed and summarized as normal or abnormal, according to local assessment. Examination includes general appearance,skin,neck(including thyroid),eyes/ears,nose,mouth,throat,lungs,heart, abdomen,back,lymphnodes,extremities,nervous system. Abnormal and clinically notable values will be identified and listed for each parameter
Long term safety/tolerability of EDS-EP: Columbia Suicide Severity Rating Scale (CSSRS)
Outcome will be listed and summarized on the basis of the answers: answer of "yes" to any of the 6 questions may indicate a need for referral to a trained mental health professional and an answer of "yes" to questions 4, 5 or 6 indicate high-risk
Long term safety/tolerability of EDS-EP: Bone Mineral Density
Z-scores will be listed and summarized as normal or abnormal, following guidelines provided in the 2013 International Society for Clinical Densitometry Official Pediatric Position. Abnormal and clinically notable values will be identified and listed for each parameter
Long term safety/tolerability of EDS-EP: Tanner staging
The results (stages from I to V, according to the age, from 0 to 18) will be listed and summarized. Abnormal and clinically notable values will be identified and listed for each parameter
Long term safety/tolerability of EDS-EP: sterility test
Outcome can be Positive or negative. Results will be listed and summarized

Secondary Outcome Measures

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)
Descriptive analyses will be performed to evaluate the long-term effects of EDS-EP on health-related Quality of Life (QoL), assessed by EQ-5D-5L scale. EQ-5D-5L descriptive system scoring: from 5, min/worst, to 25, best/max); EQ-VAS (EQ Visual Analogue scale) scoring: from 0, min/worst, to 100, best/max

Full Information

First Posted
May 23, 2018
Last Updated
August 6, 2021
Sponsor
Erydel
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1. Study Identification

Unique Protocol Identification Number
NCT03563053
Brief Title
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
Acronym
OLE-IEDAT
Official Title
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
Study Type
Interventional

2. Study Status

Record Verification Date
August 2021
Overall Recruitment Status
Unknown status
Study Start Date
June 12, 2018 (Actual)
Primary Completion Date
December 2021 (Anticipated)
Study Completion Date
December 2021 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Erydel

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Ataxia Telangiectasia

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Model Description
Open-label, Long-term, Extension Treatment
Masking
None (Open Label)
Allocation
N/A
Enrollment
50 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
active drug
Arm Type
Experimental
Arm Description
~14-22 mg dexamethasone sodium phosphate (DSP)
Intervention Type
Combination Product
Intervention Name(s)
EryDex System
Intervention 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
Primary Outcome Measure Information:
Title
Long term safety/tolerability of EDS-EP: # of pts with treatment related Adverse Events (CTCAE4.0)
Description
AEs will be listed and summarized by body system and preferred term. Incidence of AEs(%),intensity and relatedness to study drug will be reported. SAEs and events newly occurring or worsening after administration of study medication will be summarized. AEs that result in death/discontinuation of the study medication will be listed separately
Time Frame
1 year
Title
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)
Description
Values will be listed and summarized as normal or abnormal, according to local normal ranges, depending on the lab assessment (not possible to provide unique range and unit of measure due to changes from lab to lab). Abnormal and clinically notable values will be identified and listed for each parameter
Time Frame
1 year
Title
Long term safety/tolerability of EDS-EP: 12-lead ECG (Electrocardiography)
Description
Results will be listed and summarized as normal or abnormal, according guidelines (PR interval: < 100 msec or > 210 msec; QRS interval: < 50 msec or > 120 msec; QTc interval: > 450 msec; Heart rate: < 50 bpm (sinus bradycardia) or > 120 bpm (sinus tachycardia); Morphology: presence of T-wave inversion, abnormal R-waves, pathological Q-waves, significant ST elevation or depression). Abnormal and clinically notable values will be identified and listed
Time Frame
1 year
Title
Long term safety/tolerability of EDS-EP: physical and neurological exam
Description
Results will be listed and summarized as normal or abnormal, according to local assessment. Examination includes general appearance,skin,neck(including thyroid),eyes/ears,nose,mouth,throat,lungs,heart, abdomen,back,lymphnodes,extremities,nervous system. Abnormal and clinically notable values will be identified and listed for each parameter
Time Frame
1 year
Title
Long term safety/tolerability of EDS-EP: Columbia Suicide Severity Rating Scale (CSSRS)
Description
Outcome will be listed and summarized on the basis of the answers: answer of "yes" to any of the 6 questions may indicate a need for referral to a trained mental health professional and an answer of "yes" to questions 4, 5 or 6 indicate high-risk
Time Frame
1 year
Title
Long term safety/tolerability of EDS-EP: Bone Mineral Density
Description
Z-scores will be listed and summarized as normal or abnormal, following guidelines provided in the 2013 International Society for Clinical Densitometry Official Pediatric Position. Abnormal and clinically notable values will be identified and listed for each parameter
Time Frame
1 year
Title
Long term safety/tolerability of EDS-EP: Tanner staging
Description
The results (stages from I to V, according to the age, from 0 to 18) will be listed and summarized. Abnormal and clinically notable values will be identified and listed for each parameter
Time Frame
1 year
Title
Long term safety/tolerability of EDS-EP: sterility test
Description
Outcome can be Positive or negative. Results will be listed and summarized
Time Frame
1 year
Secondary Outcome Measure Information:
Title
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)
Description
Descriptive analyses will be performed to evaluate the long-term effects of EDS-EP on health-related Quality of Life (QoL), assessed by EQ-5D-5L scale. EQ-5D-5L descriptive system scoring: from 5, min/worst, to 25, best/max); EQ-VAS (EQ Visual Analogue scale) scoring: from 0, min/worst, to 100, best/max
Time Frame
1 year

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Years
Accepts Healthy Volunteers
No
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 Females that are pregnant, or are breast-feeding (for EU countries only); 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. Severe or unstable pulmonary disease. 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 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. Requires any other concomitant medication prohibited by the protocol. Use of any drug that is a strong inducer/inhibitor of CYP3A4.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Irene Maccabruni, BS
Phone
+393458415028
Email
irene.maccabruni@erydel.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Guenter R Janhofer, MD
Organizational Affiliation
EryDel S.p.A.
Official's Role
Study Director
Facility Information:
Facility Name
Ataxia Center and HD Center of Excellence - UCLA
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
Country
United States
Individual Site Status
Completed
Facility Name
Division of Pediatric Allergy and Immunology - Johns Hopkins Hospital
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21287-3923
Country
United States
Individual Site Status
Completed
Facility Name
Cincinnati Children's Hospital Medical Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Individual Site Status
Completed
Facility Name
Department of Pediatrics Division of Child and Adolescent Neurology Mitochondrial Clinic - University of Texas Medical School
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Individual Site Status
Completed
Facility Name
Department of Neurology Royal Children's Hospital
City
Parkville
State/Province
Victoria
ZIP/Postal Code
3052
Country
Australia
Individual Site Status
Completed
Facility Name
Laboratory of Pediatric Immunology UZ Leuven
City
Leuven
ZIP/Postal Code
3000
Country
Belgium
Individual Site Status
Completed
Facility Name
Klinik für Kinder- und Jugendmedizin, Allergologie, pneumologie und Mukoviszidose, Universitätsklinikum Frankfurt
City
Frankfurt
ZIP/Postal Code
60590
Country
Germany
Individual Site Status
Completed
Facility Name
National Institute of Mental Health and Neurosciences (NIMHANS) Department of Neurology
City
Bangalore
ZIP/Postal Code
560 029
Country
India
Individual Site Status
Completed
Facility Name
Vijaya Health Centre Department of Neurology
City
Chennai
ZIP/Postal Code
600 026
Country
India
Individual Site Status
Completed
Facility Name
Nizam's Institute of Medical Sciences Department of Neurology
City
Hyderabad
ZIP/Postal Code
500 082
Country
India
Individual Site Status
Completed
Facility Name
Jaslok Hospital and Research Center Department of Pediatric Neurology
City
Mumbai
ZIP/Postal Code
400 026
Country
India
Individual Site Status
Completed
Facility Name
P.D. Hinduja National Hospital and Medical Research Centre Department of Pediatric Neurology
City
Mumbai
ZIP/Postal Code
400016
Country
India
Individual Site Status
Completed
Facility Name
All India Institute of Medical Sciences Child Neurology Division Department of Pediatrics
City
New Delhi
ZIP/Postal Code
110 029
Country
India
Individual Site Status
Completed
Facility Name
Dipartimento Neuroscienze umane e salute mentale, Policlinico Umberto I Università La Sapienza
City
Roma
Country
Italy
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Vincenzo Leuzzi, MD
Email
vincenzo.leuzzi@uniroma1.it
Facility Name
Department of Clinical Immunology - The Children's Memorial Health Institute
City
Warsaw
ZIP/Postal Code
04-730
Country
Poland
Individual Site Status
Completed
Facility Name
Servicio de Neurolgia Pediatrica, Hospital Materno-Infantil La Paz
City
Madrid
ZIP/Postal Code
28046
Country
Spain
Individual Site Status
Completed
Facility Name
Razi Hospital, Clinical Investigation Center-Neuroscience
City
Tunis
Country
Tunisia
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Riadh Gouider, MD
Email
riadh.gouider@gnet.tn

12. IPD Sharing Statement

Plan to Share IPD
Undecided

Learn more about this trial

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

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