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Trial details imported from ClinicalTrials.gov

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




Registration number
NCT03563053
Ethics application status
Date submitted
23/05/2018
Date registered
20/06/2018

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
Other interventions - EryDex System

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


Other interventions: 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
Other interventions
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)
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)
Timepoint [2] 0 0
1 year
Primary outcome [3] 0 0
Long term safety/tolerability of EDS-EP: 12-lead ECG (Electrocardiography)
Timepoint [3] 0 0
1 year
Primary outcome [4] 0 0
Long term safety/tolerability of EDS-EP: physical and neurological exam
Timepoint [4] 0 0
1 year
Primary outcome [5] 0 0
Long term safety/tolerability of EDS-EP: Columbia Suicide Severity Rating Scale (CSSRS)
Timepoint [5] 0 0
1 year
Primary outcome [6] 0 0
Long term safety/tolerability of EDS-EP: Bone Mineral Density
Timepoint [6] 0 0
1 year
Primary outcome [7] 0 0
Long term safety/tolerability of EDS-EP: Tanner staging
Timepoint [7] 0 0
1 year
Primary outcome [8] 0 0
Long term safety/tolerability of EDS-EP: sterility test
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)
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
Sex
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
NA
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
12/06/2018
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/12/2021
Actual
Sample size
Target
50
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
Italy
State/province [12] 0 0
Roma
Country [13] 0 0
Poland
State/province [13] 0 0
Warsaw
Country [14] 0 0
Spain
State/province [14] 0 0
Madrid
Country [15] 0 0
Tunisia
State/province [15] 0 0
Tunis

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Quince Therapeutics S.p.A.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Guenter R Janhofer, MD
Address 0 0
EryDel S.p.A.
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
[email protected]
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Undecided
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT03563053