Status and phase
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Treatments
Study type
Funder types
Identifiers
About
Objectives:
The objective of study was to evaluate the safety and the efficacy of EryDex (Dexamethasone sodium phosphate encapsulated in autologous erythrocytes, using the EryDex System - EDS) at two dose levels (low dose and high dose DSP/infusion), compared to placebo, on Neurological Symptoms in Patients With Ataxia Telangiectasia.
Initial Double-Blind Treatment Period (0 to 6 Months)
Primary Efficacy Objective:
• Evaluate the effect of EryDex at two dose levels (low dose and high dose DSP/infusion), compared to placebo, on central nervous system (CNS) symptoms measured by the change in the Modified International Cooperative Ataxia Rating Scale (mICARS) from baseline to Month 6 (Visit 9) in patients with ataxia telangiectasia (A-T).
Secondary Efficacy Objectives:
Safety Objectives:
• Evaluate the safety and tolerability of two non-overlapping doses of EryDex, compared to placebo, in patients with A-T over the 12-month double-blind study duration.
Extension Treatment Period (6-12 Months):
Primary Objective:
• Evaluate the efficacy of EryDex at two dose levels (low dose and high dose DSP/infusion) compared to placebo, in treating CNS symptoms in A-T patients during longer-term treatment (up to 12 months), as measured by the mICARS.
Secondary Objectives:
Full description
This was an international, multi-center, one-year, randomized, prospective, double-blind, placebo-controlled, phase III study.
This study was divided into three periods: Screening (Days -30 to -1), 6-month Initial Treatment Period (Months 1-6; Visits 1-9), and 6-month Extension Treatment Period (Months 7-12; Visits 10-15).
A total of 175 patients, of the 180 planned, met all selection criteria at baseline, and were randomized in a 1:1:1 fashion to one of the two EDS-EP dose levels or placebo.
These patients were randomly assigned to receive one of the two doses of EDS-EP or placebo, as follows:
The initial 6-month treatment period was considered complete when the endpoint assessment (at Visit 9/Month 6 or at early discontinuation) was performed for all patients. All patients who completed the assessments over the initial 6 months of the trial were eligible to continue in an additional 6-month, double-blind, placebo- controlled extension, designed to collect information on the longer-term safety and efficacy of the trial treatments.
Following completion of the 6-month Initial Treatment Period, patients that met all entry criteria were re-randomized and treated as follows:
Patients originally randomized to one of the two dose levels of EryDex (low dose or high dose; Groups 1 or 2) continued in the same treatment arm.
Patients originally randomized to the Placebo group (Group 3) were re-allocated as defined at the initial randomization in equal proportions (1:1) and received either the EryDex low dose or high dose as follows:
Enrollment
Sex
Ages
Volunteers
Inclusion and exclusion criteria
Inclusion Criteria:
Exclusion criteria
General
Females that were:
A disability that may have prevented the patient from completing all study requirements.
Current participation in another clinical study.
Medical History and Current Status
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 increased 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.
Any other severe, unstable, or serious disease or condition that in the Investigator's opinion 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 was 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 EryDex.
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.
Used of any drug that is a strong inducer/inhibitor of cytochrome P450 3A4 (CYP3A4) within 4 weeks before baseline.
Primary purpose
Allocation
Interventional model
Masking
176 participants in 3 patient groups, including a placebo group
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Data sourced from clinicaltrials.gov
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