Patients with Ataxia-Teleangectasia (AT) MedDRA version: 9.1 Level: LLT Classification code 10003594
Conditions
Interventions
Product Name: Dexamethasone sodium phosphate
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: Dexamethasone
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration n
Sponsors
ERYDEL S.P.A.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: • Neurological signs of AT (un-coordination of the head and eyes in lateral gaze deflection, gait ataxia associated with an inappropriately narrow base) • Patients in autonomous gait or helped by a support • Proven molecular diagnosis of AT (at least one ATM mutation) and/or ATM protein deficiency by Western blot • Males and females aged >3 years • Body weight > 15 Kg • Plasma levels of CD4+ lymphocytes/mm3 =500 (for patients aged 3-6 years) or =200 (for patients older than 6 years) • Written informed consent to participate from the patient or from the parents (or from a legal acceptable representative), if able or not to understand the aims of the trial, respectively Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: • Current or previous neoplastic disease • History of severe impairment of the immunological system • Chronic conditions representing a contraindication to the use of steroid drugs • Noncompliance with the study request • Have participated in any other investigational trial within 30 days from Screening Period • Any previous steroid assumption within 30 days before starting ERY-DEX • Have any other significant disease that in the Investigator’s opinion would exclude the patient from the trial • Females of childbearing potential who are pregnant, breast-feeding or are not using adequate contraceptive methods
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: • To evaluate the improvement in central nervous system (CNS) symptoms measured by International Co-operative Ataxia Rating Scale (ICARS) in patients with Ataxia-Teleangectasia (AT), during a period of treatment ERY-DEX (dexamethasone sodium phosphate ex vivo encapsulated into human autologous erythrocytes).;Secondary Objective: • To evaluate the effect of ERY-DEX on Treatment-Emergent Adverse Events (TEAEs), including SAEs and laboratory parameters • To evaluate the effect of ERY-DEX on global health status of the patient by an Investigator Global Assessment • To evaluate the effect of ERY-DEX on ocular motility measured by an ad hoc form • To evaluate the effect of ERY-DEX on adaptive behaviour measured by the Vineland Adaptive Behavior Scale (VABS) • To evaluate the effect of ERY-DEX on special laboratory parameters • To evaluate the pharmacokinetic (PK) profile of dexamethasone sodium phosphate administered through ERY-DEX (in a sub-set of patients);Primary end point(s): The aim of the study is to measure ICARS improvement after six months of treatment with dexamethasone sodium phosphate | — |
Countries
Italy
Outcome results
None listed