Additional identifiers
EudraCT number
2008-001185-91
ClinicalTrials.gov number
Protocol/serial number
EUDRA-CT: 2008-001185-91
Study information
Scientific title
Central randomised, double-blind, crossover, multicentre clinical trial of betamethasone and placebo in children with ataxia teleangectasia
Acronym
BETA-AT clinical trial
Study hypothesis
1. Betamethasone determines a larger decrease of neurological symptoms than placebo
2. Betamethasone determines a larger increase of health-related quality of life than placebo
2006 case study results in http://www.ncbi.nlm.nih.gov/pubmed/17030666.
As of 29/09/2009 this record was updated to include amended anticipated start and end dates; the initial dates at the time of registration were as follows:
Initial anticipated start date: 15/07/2008
Initial anticipated end date: 30/04/2009
Ethics approval
The local ethics committee (Azienda Ospedaliera Universitaria Senese Comitato Etico Locale - Sperimentazione Clinica Medicinali) gave approval on the 4th June 2008.
Study design
Phase II, central randomised, double-blind, crossover, multicentre clinical trial
Primary study design
Interventional
Secondary study design
Randomised controlled trial
Trial setting
Not specified
Trial type
Treatment
Patient information sheet
Not available in web format, please use the contact details below to request a patient information sheet
Condition
Ataxia teleangectasia (AT)
Intervention
Central randomised, double-blind, crossover study of betamethasone versus placebo, at the dose of 0.05 mg/kg every 12 hours. The substances (betamethasone or placebo) will be administered orally for 30 days (each branch). Between the day 11 and 20, they will slightly tapered for 10 days; then again administered at a full dosage for 10 days. Each branch of the trial will be followed by a washout period of 30 days. Between the day 1 and 10 of each wash out period, the substances will be slightly tapered.
Added 01/10/2009:
Plasma levels of betamethasone were tested 1 day before entrance into the study and at the 31st day of each arm of the trial.
Intervention type
Drug
Phase
Phase II
Drug names
Betamethasone
Primary outcome measure
Check of the neurological symptoms (Ataxia International Cooperative Ataxia Rating Scale) before and after the drug or placebo. Test schedule: 1 day before the entrance and at the 31st day within each branch of the trial.
Secondary outcome measures
Check of the general health status and quality of life before and after the drug or placebo. Test schedule: 1 day before the entrance and at the 31st day within each branch of the trial.
Overall trial start date
11/11/2008
Overall trial end date
01/07/2009
Reason abandoned (if study stopped)
Eligibility
Participant inclusion criteria
1. Proven molecular diagnosis of A-T (alpha-fetoprotein [AFP] level more than twice the upper limit of normal and demonstration of ATM protein deficiency by Western blot)
2. Evident neurological signs of ataxia (uncoordination of head and eyes in lateral gaze deflection, gait ataxia associated with an inappropriately narrow base)
3. Aged greater than or equal to 3 years, either sex
4. Plasma CD4+ lymphocytes/mm^3 greater than or equal to 500 (3 - 6 years) or greater than or equal to 200 (greater than 6 years)
5. Written informed consent to participate from the parents and verbal consent to participate from the patient, if able to understand the main concepts and aims of the study
Participant type
Patient
Age group
Child
Gender
Both
Target number of participants
25
Participant exclusion criteria
1. Confinement to a wheelchair (i.e. inability to walk)
2. Current or previous neoplastic disease
3. History of severe impairment of the immunological system (i.e. history of serious infectious disease)
4. Presence of other chronic conditions (i.e. diabetes, mental delay, osteoporosis, etc) representing a contraindication to the use of a steroid drug
5. Noncompliance with the aims and methods of the study
Recruitment start date
11/11/2008
Recruitment end date
01/07/2009
Locations
Countries of recruitment
Italy
Trial participating centre
Department of Paediatrics
Siena
I-53100
Italy
Sponsor information
Organisation
Fondazione Monte Paschi di Siena (Italy)
Sponsor details
Via Banchi di Sotto
34
Siena
I-53100
Italy
Sponsor type
Research organisation
Website
Funders
Funder type
Research organisation
Funder name
Fondazione Monte Paschi di Siena (Italy)
Alternative name(s)
Funding Body Type
Funding Body Subtype
Location
Results and Publications
Publication and dissemination plan
Not provided at time of registration
Intention to publish date
Participant level data
Not provided at time of registration
Basic results (scientific)
Publication list