Health Condition 1: null- Ataxia Telangiectasia
Conditions
Interventions
Intervention1: EDS-EP dose range of approx 5-10 mg and 14-22 mg: Group 1: EDS-EP dose range of approx 5-10 mg DSP/infusion Group 2: EDS-EP dose range of approx 14-22 mg DSP/infusion
Frequency: Once
Sponsors
EryDel SpA
CliniRx Tangent Research India Pvt Ltd
Eligibility
Inclusion criteria
Inclusion criteria: 1. Patient meets clinical criteria for diagnosis of AT. The neurological signs of AT (in coordination of the head and eyes in lateral gaze deflection, gait ataxia associated with an inappropriately narrow base) must be documented. 2. Patient is in autonomous gait or is helped by periodic use of a support. 3. Patient will be investigated for the proven genetic diagnosis of AT (prior documentation or by central laboratory test report). 4. Patient is at least 6 years of age, of either sex. 5. Body weight > 15 kg. 6. 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.
Exclusion criteria
Exclusion criteria: General 1. Females that are of childbearing potential, pregnant, or are breast-feeding. 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 another clinical study. Medical History and Current Status 4. CD4+ lymphocytes count 6 years). 5. Loss/removal of 250 mL or more of blood within the past 4 weeks prior to screening. 6. Current neoplastic disease or previous neoplastic disease not in remission for at least 2 years. 7. History of severe impairment of the immunological system. 8. Severe or unstable pulmonary disease. 9. Uncontrolled diabetes. Patients with diabetes that has been stabilized (i.e. no hypoglycemic or hyperglycemic episodes in the past 3 months) will be eligible. 10. Any other severe, unstable, or serious disease or condition that in the Investigatorââ?¬•s opinion would put the patient at risk for imminent lifethreatening morbidity, need for hospitalization, or mortality. 11. 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. 12. Confirmed hemoglobinopathies, e.g. hemoglobin C disease, sickle cell anemia, or thalassemia. 13. Moderate or severe renal and/or hepatic impairment. Prior/Concomitant Medication. 14. 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. 15. Chronic condition or prior allergic reaction representing a contraindication to the use of dexamethasone or other steroid drugs. 16. 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. 17. Has participated in a previous trial with EDS. 18. Requires any concomitant medication prohibited by the protocol. 19. Has taken a drug or treatment known to cause major organ system toxicity during the past year. 20. Use of any drug that is a strong inducer/inhibitor of CYP3A4 within 4 weeks before baseline.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| To evaluate the effect of two dose ranges (approx 5-10 and approx 14-22 mg DSP/infusion) compared to placebo, on CNS symptoms measured by the ââ?¬Ë?Modifiedââ?¬â?¢ ICARS in patients with AT. Timepoint: 6 Months and 12 Months | — |
Secondary
| Measure | Time frame |
|---|---|
| To evaluate the effect of EDS-EP, compared to placebo, in this population on the following efficacy measures: 1. CGI-S of neurological symptoms of AT 2. Adaptive behavior measured by the VABS scaleTimepoint: 6 months;To evaluate the safety and tolerability of EDS-EP compared to placebo in AT patients, based on the occurrence of Treatment-Emergent Adverse Events (TEAEs), including Serious AEs and discontinuations due to AEs, and changes in vital signs, laboratory parameters, ECGs and physical/neurological examination findings.Timepoint: 6 months | — |
Countries
Australia, Belgium, Costa Rica, Germany, India, Israel, Italy, Norway, Poland, Spain, Tunisia, Turkey, United Kingdom, United States of America
Contacts
Public ContactShiv Issar
CliniRx Tangent Research India Pvt Ltd
Outcome results
None listed