EDS in Ataxia Telangiectasia Patients (ATTeST)

This is an interventional treatment trial for Nervous System Disease focused on measuring Ataxia Teleangiectasia, AT, EryDex system, Dexamethasone, Dexamethasone sodium phosphate Read More

Main Inclusion Criteria:

• 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 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.