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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT03563053




Registration number
NCT03563053
Ethics application status
Date submitted
23/05/2018
Date registered
20/06/2018
Date last updated
29/01/2020

Titles & IDs
Public 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
Scientific 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
Secondary ID [1] 0 0
IEDAT-03-2018
Universal Trial Number (UTN)
Trial acronym
OLE-IEDAT
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Ataxia Telangiectasia 0 0
Condition category
Condition code
Neurological 0 0 0 0
Other neurological disorders
Neurological 0 0 0 0
Neurodegenerative diseases
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Cardiovascular 0 0 0 0
Diseases of the vasculature and circulation including the lymphatic system

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Combination Product - EryDex System

Experimental: active drug - ~14-22 mg dexamethasone sodium phosphate (DSP)


Combination Product: EryDex System
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

Intervention code [1] 0 0
Combination Product
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
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
Timepoint [1] 0 0
1 year
Primary outcome [2] 0 0
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
Timepoint [2] 0 0
1 year
Primary outcome [3] 0 0
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
Timepoint [3] 0 0
1 year
Primary outcome [4] 0 0
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
Timepoint [4] 0 0
1 year
Primary outcome [5] 0 0
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
Timepoint [5] 0 0
1 year
Primary outcome [6] 0 0
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
Timepoint [6] 0 0
1 year
Primary outcome [7] 0 0
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
Timepoint [7] 0 0
1 year
Primary outcome [8] 0 0
Long term safety/tolerability of EDS-EP: sterility test - Outcome can be Positive or negative. Results will be listed and summarized
Timepoint [8] 0 0
1 year
Secondary outcome [1] 0 0
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
Timepoint [1] 0 0
1 year

Eligibility
Key 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.
Minimum age
6 Years
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
N/A
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Department of Neurology Royal Children's Hospital - Parkville
Recruitment postcode(s) [1] 0 0
3052 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Maryland
Country [3] 0 0
United States of America
State/province [3] 0 0
Ohio
Country [4] 0 0
United States of America
State/province [4] 0 0
Texas
Country [5] 0 0
Belgium
State/province [5] 0 0
Leuven
Country [6] 0 0
Germany
State/province [6] 0 0
Frankfurt
Country [7] 0 0
India
State/province [7] 0 0
Bangalore
Country [8] 0 0
India
State/province [8] 0 0
Chennai
Country [9] 0 0
India
State/province [9] 0 0
Hyderabad
Country [10] 0 0
India
State/province [10] 0 0
Mumbai
Country [11] 0 0
India
State/province [11] 0 0
New Delhi
Country [12] 0 0
Poland
State/province [12] 0 0
Warsaw
Country [13] 0 0
Spain
State/province [13] 0 0
Madrid

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Erydel
Address
Country

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/show/NCT03563053
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Irene Maccabruni, BS
Address 0 0
Country 0 0
Phone 0 0
+393458415028
Fax 0 0
Email 0 0
irene.maccabruni@erydel.com
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT03563053