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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
Date last updated
8/10/2024

Titles & IDs
Public title
Extension Treatment Using EryDex System in Patients With AT Who Participated in the ATTeST-IEDAT-02-2015 Study
Scientific title
Open-label, Long-term, Extension Treatment Using Intra-Erythrocyte Dexamethasone Sodium Phosphate (EryDex System) in Patients With Ataxia Telangiectasia Who Participated in the ATTeST-IEDAT-02-2015 Study
Secondary ID [1] 0 0
2018-000338-36
Secondary ID [2] 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
Genetic Syndrome 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) for 12 months. The duration of EryDex treatment could be extended further and continue until patients eventually withdrew consent, or the Investigator decided to discontinue treatment based on a risk / benefit assessment.


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

EryDex treatment consisted of a dose range correspondent to the ATTeST High Dose (obtained by loading 125 mg of DSP to the EryDex process and that, in the ATTeST, resulted in a mean of 17.4 ± 5.4 mg (mean ± standard deviation) of DSP infused to patients) administered via ex vivo encapsulation into EDS that were infused into the patient with A-T.

The dose of EryDex treatment was selected to allow collection of long-term safety data on the dose of erythrocyte encapsulated DSP that was considered more effective among the two different doses that were employed in the ATTeST study, based on the analysis of the ATTeST blinded, Phase 3 study data.
Intervention code [1] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Treatment-Emergent Adverse Event (TEAE), Treatment-emergent Serious Adverse Events (TESAE), and Adverse Events of Special Interest (AESI) Throughout the Study
Timepoint [1] 0 0
From Baseline (Visit 1 - Day 0) to Follow-up (~60 days after last infusion, i.e. up to 50.5 months)
Secondary outcome [1] 0 0
Change From Baseline in Quality of Life Using EQ-5D-5L Scale to Month 36
Timepoint [1] 0 0
From Baseline (Visit 1- Day 0) to Month 36
Secondary outcome [2] 0 0
Number of Patients With Improving, Stable or Worsening Score Using a Clinical Global Impression of Change (CGI-C) From Baseline (Visit 1- Day 0) to Month 36
Timepoint [2] 0 0
From Baseline (Visit 1- Day 0) to Month 36
Secondary outcome [3] 0 0
Number of Patients With None to Severe (0 to 4) Scores in Clinical Global Impression of Severity (CGI-S)-Structured of Neurological Symptoms of AT From Baseline (Visit 1 - Day 0) to Month 36
Timepoint [3] 0 0
From Baseline (Visit 1- Day 0) to Month 36
Secondary outcome [4] 0 0
Change From Baseline of the Modified International Cooperative Ataxia Rating Scale (mICARS) Until Month 36
Timepoint [4] 0 0
From Baseline (Visit 1- Day 0) to Month 36

Eligibility
Key inclusion criteria
1. Patient completed the double-blind period in the ATTeST study and completed the final (Visit 15 / Month 12) Efficacy Assessments of ATTeST or discontinued the study during the COVID-19 pandemic.
2. Patient tolerated the study medication, without any evidence of steroid adverse events, or treatment-related severe events / 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, provided written informed consent to participate. If consent was provided solely by the caregiver in accordance with local regulations, the patient also was asked to provide their assent to participate in the study.
5. Patient did not present safety contraindication for continuation of treatment, as determined by the Principal Investigator (PI) according to the procedures described below.

Moreover, patients who were discontinued from the ATTeST study during the COVID-19 pandemic were eligible to receive the EryDex treatment in the IEDAT-03-2018 study, in the absence of safety contraindications to continuation of the treatment, and after signing the informed consent.

There were no de novo enrolled patients.
Minimum age
6 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Patients who met one or more of the following criteria were not considered to be eligible to participate in the study:

General

1. Females that were:

1. Pregnant, or were breast-feeding (for EU countries only)
2. Of childbearing potential, pregnant, or were breast-feeding (for US and Rest of World countries).

Females of childbearing potential using adequate birth control, as determined by their Health Care Provider, were eligible.
2. A disability that may prevent the patient from completing all study requirements.
3. Current participation in another clinical study with another investigational drug.

Medical History and Current Status
4. Cluster differential 4 positive (CD4+) lymphocytes count < 400 / mm3 (for patients 6 years of age) or < 150 / mm3 (for patients > 6 years). In presence of oral infections, like oral candidiasis, documented at the screening or recurrent as per medical history documentation, the limit increases to < 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 had been stabilized (i.e., no hypoglycemic or hyperglycemic episodes in the past 3 months) were 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 life-threatening morbidity, need for hospitalization, or mortality.
10. Eligibility of patients with abnormal laboratory test values were determined by the Investigator.
11. Confirmed haemoglobinopathies, e.g., haemoglobin C disease, sickle cell anaemia, 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 EryDex treatment administered in the ATTeST 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 were permitted.
15. Requires any other concomitant medication prohibited by the protocol.
16. Use of any drug that is a strong inducer / inhibitor of Cytochrome P450 3A4 (CYP3A4).

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
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
Stopped early
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
Actual
2/09/2022
Sample size
Target
Accrual to date
Final
104
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
Tamil Nadu
Country [8] 0 0
India
State/province [8] 0 0
Bangalore
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
Italy
State/province [11] 0 0
Roma
Country [12] 0 0
Norway
State/province [12] 0 0
Oslo
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
Country [16] 0 0
United Kingdom
State/province [16] 0 0
Nottinghamshire

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

Ethics approval
Ethics application status

Summary
Brief summary
Primary Objective

To monitor and evaluate the long-term safety and tolerability of EDS-EP in AT patients.

Secondary Objective

To evaluate the long-term effect of EDS-EP on health-related Quality of Life (QoL; EQ-5D-5L scale).

Exploratory Objective:

To evaluate the long-term effect of EDS-EP in treating central nervous system (CNS) symptoms, as measured by the "Modified" International Cooperative Ataxia Rating Scale (mICARS), and Clinical Global Impression of severity and change (CGI-S/C).
Trial website
https://clinicaltrials.gov/study/NCT03563053
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Guenter R Janhofer, MD, PhD
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



Summary Results

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