Clinical trial NCT01255358: Intra-Erythrocyte Dexamethasone Sodium Phosphate in Ataxia Teleangiectasia Patients
Official title: Evaluations of Effects of Intra-Erythrocyte Dexamethasone Sodium Phosphate on Neurological Symptoms in Ataxia Teleangiectasia Patients
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Nervous System Disorder
Genetic Syndrome
Keywords: Ataxia Teleangiectasia
AT
ERY-DEX
Dexamethasone
Dexamethasone sodium phosphate
Lead sponsor: Quince Therapeutics S.p.A.
Sponsor class: INDUSTRY
First posted: 2010-12-07
Enrollment: 22
Sex: ALL
Minimum age (years): 3
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The study has the aim to evaluate the improvement in CNS symptoms measured by International Co-operative Ataxia Rating Scale (ICARS) in patients with ataxia teleangectasia (AT), during a period of treatment with ERY-DEX (dexamethasone sodium phosphate ex vivo encapsulated into human autologous erythrocytes).

Detailed description: This is a multi-centre, single arm, open label, 6 months, phase II study to evaluate the effect of ERY-DEX in improving Central Nervous System (CNS) symptoms in patients with Ataxia Teleangectasia (AT). The study consists of a screening period (max duration of 30 days) and a treatment period (duration 6 months).

Eligibility criteria: Inclusion Criteria:

* neurological signs of AT
* patients in autonomous gait or helped by a support
* proven molecular diagnosis of AT
* Males and females aged \> 3 years
* Body weight \>15 kg
* Plasma levels of Lymphocytes CD4+/mm3 \> 500 (for patients aged 3-6 years) or \> 200 (older than 6 years)
* written IC to participate.

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
* Non compliance with the study request
* 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 were pregnant, breast-feeding or were not using adequate contraceptive methods

Primary outcomes:
measure: Mean Change From Baseline in Neurological Symptoms Assessed by Using International Cooperative Ataxia Rating Scale (ICARS) Score - ITT Population
timeFrame: At visits 2 (day 30), 4 (day 90) and 7 (final visit, day 180)
measure: Mean Change From Baseline in Neurological Symptoms Assessed by Using International Cooperative Ataxia Rating Scale (ICARS) Score - PP Population
timeFrame: At visits 2 (day 30), 4 (day 90) and 7 (final visit, day 180)
Secondary outcomes:
measure: Change From V4 to V7 in Investigator Global Assessment (IGA) - ITT Population
timeFrame: At visit 7 (final visit, day 180)
measure: Change From V4 to V7 in Investigator Global Assessment (IGA) - PP Population
timeFrame: At visit 7 (final visit, day 180)
measure: Mean Change From V4 to V7 in Ocular Motility - ITT Population
timeFrame: At visit 7 (final visit, day 180)
measure: Mean Change From V4 to V7 in Ocular Motility - PP Population
timeFrame: At visit 7 (final visit, day 180)
measure: Change From Baseline for Vineland Adaptive Behaviour Scale (VABS) Total Score - ITT Population
timeFrame: At visits 4 (90 days) and 7 (180 days, final visit)
measure: Change From Baseline for Vineland Adaptive Behaviour Scale (VABS) Total Score - PP Population
timeFrame: At visits 4 (90 days) and 7 (180 days, final visit)
measure: Count of Participants on ERY-DEX Showing Treatment Emergent Adverse Events (TEAEs) Including Serious Adverse Events (SAEs)
timeFrame: Throughout the study, until the end of 6 months of treatment (day 180)
Other outcomes:
Locations:
city: Brescia
country: Italy
facility: Spedali Civili
city: Rome
country: Italy
facility: University La Sapienza
Study officials:
affiliation: A.O. Sant'Andrea Rome Italy
name: Luciana Chessa, MD
role: PRINCIPAL_INVESTIGATOR
References: