Intra-Erythrocyte Dexamethasone Sodium Phosphate in Ataxia Telangiectasia Patients

Inclusion Criteria:

1. Patient met clinical criteria for diagnosis of A-T. The neurological signs of A-T (incoordination of the head and eyes in lateral gaze deflection, gait ataxia associated with an inappropriately narrow base) must have been documented. Such signs of A-T illustrated the body systems in which changes were confirmed, but the listed changes were examples and other changes in those systems may have been observed and documented to confirm the diagnosis of A-T.

2. Patient was in autonomous gait or was helped by periodic use of a support (i.e., score for Item 1 of the full ICARS - Walking Capacities between 0 and 4, included).

3. Patient was investigated for the proven genetic diagnosis of A-T (prior documentation or by central laboratory test report).

4. Patient was at least 6 years of age.

5. Body weight was >15 kg.

6. The patient and 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 must have provided assent to participate in the study.

Exclusion criteria

General

1. Females that were:

2. Pregnant or breast-feeding (for European Union [EU] countries only).

3. Of childbearing potential, pregnant, or breast-feeding (for US and Rest of World countries) not using adequate birth control, as determined by their Healthcare Provider.

4. A disability that may have prevented the patient from completing all study requirements.

5. Current participation in another clinical study.

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 increased to <200/mm3 (for patients >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.

10. 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.

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 was 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 EryDex.

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. Used of any drug that is a strong inducer/inhibitor of cytochrome P450 3A4 (CYP3A4) within 4 weeks before baseline.