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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT02770807




Registration number
NCT02770807
Ethics application status
Date submitted
2/05/2016
Date registered
12/05/2016
Date last updated
29/01/2020

Titles & IDs
Public title
EDS in Ataxia Telangiectasia Patients
Scientific title
Multi-center, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Effects of Intra-Erythrocyte Dexamethasone Sodium Phosphate on Neurological Symptoms in Patients With Ataxia Telangiectasia
Secondary ID [1] 0 0
IEDAT-02-2015
Universal Trial Number (UTN)
Trial acronym
ATTeST
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Nervous System Disease 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
Treatment: Drugs - EDS-EP dose range of ~5-10 mg DSP/infusion
Treatment: Drugs - EDS-EP dose range of ~14-22 mg DSP/infusion
Treatment: Drugs - Placebo

Experimental: EDS-EP dose range of ~5-10 mg DSP/infusion - Drug: EDS-EP dose range of ~5-10 mg DSP/infusion EDS-EP dose range of ~5-10 mg DSP/infusion: A DSP loading quantity of 50.0 mg will be added to the EDS process, by using 2.0 mL of the 25 mg/mL DSP solution to deliver an EDS dose range of ~5-10 mg. DSP is diluted with 11 mL sterile water for injection in the same syringe, for a total of 13.0 mL.
Other Names:
EryDex System end product

Experimental: EDS-EP dose range of ~14-22 mg DSP/infusion - Drug: DSP/infusion EDS-EP dose range of ~14-22 mg DSP/infusion DSP/infusion EDS-EP dose range of ~14-22 mg DSP/infusion: A DSP loading quantity of 125 mg will be added to the EDS process, by using 5.0 mL of the 25 mg/mL DSP solution to deliver an EDS dose range of 14-22 mg. DSP is diluted with 11 mL sterile water for injection in the same syringe, for a total of 16 mL.
Other Names:
EryDex System end product

Placebo Comparator: Placebo EDS infusion - Patients will be treated with autologous erythrocytes prepared with the EDS process using a placebo solution (5 mL of 0.372% NaCl solution) instead of experimental drug (DSP). Placebo is diluted with 11 mL sterile water for injection in the same syringe, for a total of 16 mL.


Treatment: Drugs: EDS-EP dose range of ~5-10 mg DSP/infusion
infusion

Treatment: Drugs: EDS-EP dose range of ~14-22 mg DSP/infusion
infusion

Treatment: Drugs: Placebo
infusion

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Modified International Cooperative Ataxia Rating Scale (mICARS) - Change from baseline analyzed using a Mixed Model Repeated Measures (MMRM) approach
Timepoint [1] 0 0
3 months, 6 months, 9 months, 12 months
Secondary outcome [1] 0 0
Clinical Global Impression of Change (CGI-C) key secondary outcome measure - Change from baseline analyzed using ANCOVA
Timepoint [1] 0 0
3 months, 6 months, 9 months, 12 months
Secondary outcome [2] 0 0
Clinical Global Impression of Severity (CGI-S) of neurological symptoms of AT - Change from baseline
Timepoint [2] 0 0
3 months, 6 months, 9 months, 12 months
Secondary outcome [3] 0 0
Vineland Adaptive Behavior Scales (VABS) - Change from baseline
Timepoint [3] 0 0
3 months, 6 months, 9 months, 12 months

Eligibility
Key inclusion criteria
Main

- Patient meets clinical criteria for diagnosis of AT. The neurological signs of AT
(incoordination of the head and eyes in lateral gaze deflection, gait ataxia
associated with an inappropriately narrow base) must be documented.

- Patient is in autonomous gait or is helped by periodic use of a support.

- Patient will be investigated for the proven genetic diagnosis of AT (prior
documentation or by central laboratory test report).

- Patient is at least 6 years of age, of either sex

- 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.

Main
Minimum age
6 Years
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
General

- 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.

- A disability that may prevent the patient from completing all study requirements.

- Current participation in another clinical study. Medical History and Current Status

- 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).

- Loss/removal of 250 mL or more of blood within the past 4 weeks prior to screening.

- Current neoplastic disease or previous neoplastic disease not in remission for at
least 2 years.

- History of 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 life-threatening morbidity, need
for hospitalization, or mortality.

- Any clinically significant abnormality on standard laboratory examinations
(hematology, biochemistry, urinalysis) at screening that remains abnormal on repeat
testing. Eligibility of patients with abnormal laboratory test values will be
determined by the Investigator in consultation with the Medical Monitor.

- Confirmed hemoglobinopathies, e.g. hemoglobin C disease, sickle cell anemia, or
thalassemia.

- Moderate or severe renal and/or hepatic impairment. Prior/Concomitant Medication

- Any previous oral or parenteral steroid use within 4 weeks before Baseline. Treatment
with inhaled or intranasal steroids for asthma or allergies, as well as use of topical
steroids will be permitted

- Chronic condition or prior allergic reaction representing a contraindication to the
use of dexamethasone or other steroid drugs.

- Has participated in any other trial with an investigational drug and received a dose
within 30 days or 10 half-lives (whichever is greater) from the start of the 30-day
Screening Period.

- Has participated in a previous trial with EDS.

- Requires any concomitant medication prohibited by the protocol.

- Has taken a drug or treatment known to cause major organ system toxicity during the
past year.

- Use of any drug that is a strong inducer/inhibitor of CYP3A4 within 4 weeks before
baseline.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
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
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s

The people analysing the results/data
Intervention assignment
Parallel
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
Royal Children's Hospital - Melbourne
Recruitment postcode(s) [1] 0 0
- Melbourne
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
Hessen
Country [7] 0 0
India
State/province [7] 0 0
Karnataka
Country [8] 0 0
India
State/province [8] 0 0
Kerala
Country [9] 0 0
India
State/province [9] 0 0
Maharashtra
Country [10] 0 0
India
State/province [10] 0 0
Mumbai
Country [11] 0 0
India
State/province [11] 0 0
Tamil Nadu
Country [12] 0 0
India
State/province [12] 0 0
Telangana
Country [13] 0 0
India
State/province [13] 0 0
New Delhi
Country [14] 0 0
Israel
State/province [14] 0 0
Tel HaShomer
Country [15] 0 0
Italy
State/province [15] 0 0
Brescia
Country [16] 0 0
Italy
State/province [16] 0 0
Rome
Country [17] 0 0
Norway
State/province [17] 0 0
Oslo
Country [18] 0 0
Poland
State/province [18] 0 0
Warsaw
Country [19] 0 0
Spain
State/province [19] 0 0
Madrid
Country [20] 0 0
Tunisia
State/province [20] 0 0
Manouba
Country [21] 0 0
United Kingdom
State/province [21] 0 0
Nottinghamshire

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

Ethics approval
Ethics application status

Summary
Brief summary
This is an international, multi-center, one-year, randomized, prospective, double-blind,
placebo-controlled, phase III study, designed to assess the effect of two non-overlapping
dose ranges of EDS EP, administered by IV infusion once per month, on neurological symptoms
of patients with Ataxia Telangiectasia.
Trial website
https://clinicaltrials.gov/show/NCT02770807
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, M.Sc.
Address 0 0
Country 0 0
Phone 0 0
+39 0236504470
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/NCT02770807