Use of BETAmethasone in Ataxia Teleangectasia
| ISRCTN | ISRCTN08774933 |
|---|---|
| DOI | https://doi.org/10.1186/ISRCTN08774933 |
| EudraCT/CTIS number | 2008-001185-91 |
| Secondary identifying numbers | EUDRA-CT: 2008-001185-91 |
- Submission date
- 28/10/2008
- Registration date
- 20/11/2008
- Last edited
- 30/12/2020
- Recruitment status
- No longer recruiting
- Overall study status
- Completed
- Condition category
- Nervous System Diseases
Prospectively registered
Protocol
Statistical analysis plan
Results
Individual participant data
Plain English summary of protocol
Not provided at time of registration
Contact information
Dr Raffaella Zannolli
Scientific
Scientific
Department of Paediatrics
Policlinico le Scotte
Viale Bracci 8
Siena
I-53100
Italy
Study information
| Study design | Phase II, central randomised, double-blind, crossover, multicentre clinical trial |
|---|---|
| Primary study design | Interventional |
| Secondary study design | Randomised controlled trial |
| Study setting(s) | Not specified |
| Study type | Treatment |
| Participant information sheet | Not available in web format, please use the contact details below to request a patient information sheet |
| Scientific title | Central randomised, double-blind, crossover, multicentre clinical trial of betamethasone and placebo in children with ataxia teleangectasia |
| Study acronym | BETA-AT clinical trial |
| Study objectives | 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(s) | The local ethics committee (Azienda Ospedaliera Universitaria Senese Comitato Etico Locale - Sperimentazione Clinica Medicinali) gave approval on the 4th June 2008. |
| Health condition(s) or problem(s) studied | Ataxia telangiectasia (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 |
| Pharmaceutical study type(s) | |
| Phase | Phase II |
| Drug / device / biological / vaccine name(s) | 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 study start date | 11/11/2008 |
| Completion date | 01/07/2009 |
Eligibility
| Participant type(s) | Patient |
|---|---|
| Age group | Child |
| Lower age limit | 3 Years |
| Sex | Both |
| Target number of participants | 25 |
| Total final enrolment | 13 |
| Key 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 |
| Key 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 |
| Date of first enrolment | 11/11/2008 |
| Date of final enrolment | 01/07/2009 |
Locations
Countries of recruitment
- Italy
Study participating centre
Department of Paediatrics
Siena
I-53100
Italy
I-53100
Italy
Sponsor information
Fondazione Monte Paschi di Siena (Italy)
Research organisation
Research organisation
Via Banchi di Sotto, 34
Siena
I-53100
Italy
| Website | http://www.fondazionemps.it |
|---|---|
"ROR" |
https://ror.org/022pga911 |
Funders
Funder type
Research organisation
Fondazione Monte Paschi di Siena (Italy)
No information available
Results and Publications
| Intention to publish date | |
|---|---|
| Individual participant data (IPD) Intention to share | No |
| IPD sharing plan summary | Not provided at time of registration |
| Publication and dissemination plan | Not provided at time of registration |
| IPD sharing plan |
Study outputs
| Output type | Details | Date created | Date added | Peer reviewed? | Patient-facing? |
|---|---|---|---|---|---|
| Results article | results | 01/09/2012 | 30/12/2020 | Yes | No |
Editorial Notes
30/12/2020: The following changes have been made:
1. Publication reference added.
2. The final enrolment number has been added from the reference.
