Condition category
Nervous System Diseases
Date applied
28/10/2008
Date assigned
20/11/2008
Last edited
01/10/2009
Prospective/Retrospective
Retrospectively registered
Overall trial status
Completed
Recruitment status
No longer recruiting

Plain English Summary

Not provided at time of registration

Trial website

Contact information

Type

Scientific

Primary contact

Dr Raffaella Zannolli

ORCID ID

Contact details

Department of Paediatrics
Policlinico le Scotte
Viale Bracci 8
Siena
I-53100
Italy

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 measures

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

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

http://www.fondazionemps.it

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

Results - basic reporting

Publication summary

Publication citations

Additional files

Editorial Notes