Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12619001020178
Ethics application status
Approved
Date submitted
26/06/2019
Date registered
16/07/2019
Date last updated
16/07/2019
Date data sharing statement initially provided
16/07/2019
Type of registration
Prospectively registered

Titles & IDs
Public title
A pilot study assessing the effectiveness of oral Propranolol in preventing epistasis in patients with Hereditary Haemorrhagic Telangiectasia.
Scientific title
A pilot study assessing the effectiveness of oral Propranolol in preventing epistasis in patients with Hereditary Haemorrhagic Telangiectasia.
Secondary ID [1] 298606 0
none
Universal Trial Number (UTN)
U1111-1236-0176
Trial acronym
ProHHT
Linked study record
None

Health condition
Health condition(s) or problem(s) studied:
Hereditary Haemorrhagic Telangiectasia 313460 0
Condition category
Condition code
Human Genetics and Inherited Disorders 311891 311891 0 0
Other human genetics and inherited disorders
Cardiovascular 311954 311954 0 0
Diseases of the vasculature and circulation including the lymphatic system

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
The medication Propranolol oral tablets will be prescribed for the patients at a dose of 40mg once daily for 6 months. Adherence will be monitored using a 'drug dispensation and accountability log". This will record exactly what is dispensed to the patient and on return of the propranolol bottle it will be recorded how many tablets were actually taken by the patient to measure compliance.
Intervention code [1] 314866 0
Treatment: Drugs
Comparator / control treatment
No control group
Control group
Uncontrolled

Outcomes
Primary outcome [1] 320558 0
Change in Epistasis. Measured using Epistaxis scoring sheet.
Timepoint [1] 320558 0
6 months post baseline
Secondary outcome [1] 371999 0
Change in Blood transfusions administered to patient. Recorded in CRF but also captured on Epistaxis scoring sheet.
Timepoint [1] 371999 0
6 Months post baseline
Secondary outcome [2] 372000 0
Change in Quality of Life. This will be measured by using the SF36 questionnaire before and after treatment.
Timepoint [2] 372000 0
Baseline and 6 months post baseline.

Eligibility
Key inclusion criteria
Participants with a diagnosis of HHT according to curacao criteria score of >4
Participants with an Epistaxis severity score of >4 despite conventional treatment
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Absolute contraindication to Propranolol e.g. Bradycardia
Pregnancy or breast feeding
Planned surgical intervention for epistaxis control
Deemed by cardiologist as unsuitable for taking Propranolol medication

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
allocation is not concealed
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Not applicable
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
pilot study to demonstrate effectiveness of Propranolol for HHT patients.
Phase
Not Applicable
Type of endpoint/s
Efficacy
Statistical methods / analysis
This is a pilot study. It is not powered, but if the intervention appears to benefit HHT patients a larger powered study will be developed.

Recruitment
Recruitment status
Not yet recruiting
Date of first participant enrolment
Anticipated
6/08/2019
Actual
Date of last participant enrolment
Anticipated
5/02/2020
Actual
Date of last data collection
Anticipated
7/10/2020
Actual
Sample size
Target
15
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
QLD
Recruitment hospital [1] 14129 0
Royal Brisbane & Womens Hospital - Herston
Recruitment postcode(s) [1] 26896 0
4029 - Royal Brisbane Hospital
Recruitment postcode(s) [2] 26939 0
4029 - Herston

Funding & Sponsors
Funding source category [1] 303147 0
Hospital
Name [1] 303147 0
ENT CLinical Trials Fund, RBWH
Country [1] 303147 0
Australia
Primary sponsor type
Hospital
Name
Royal brisbane and Women's Hospital
Address
Butterfield Street, Herston , QLD 4029
Country
Australia
Secondary sponsor category [1] 303144 0
None
Name [1] 303144 0
Address [1] 303144 0
Country [1] 303144 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 303696 0
Royal Brisbane and Women's Hospital HREC
Ethics committee address [1] 303696 0
Lower Ground Ned Hanlon Building, RBWH, Butterfield St., Herston, QLD, 4029
Ethics committee country [1] 303696 0
Australia
Date submitted for ethics approval [1] 303696 0
22/03/2019
Approval date [1] 303696 0
17/05/2019
Ethics approval number [1] 303696 0
HREC/QRBW/2019/48585

Summary
Brief summary

This study will review the intervention of administration of oral propranolol tablets to patients with HHT (Hereditary Haemorrhagic Telangiectasia) . Patients will be monitored to see if Propranolol helps reduce epistaxis (nosebleeds) . The most common symptom of HHT is having recurrent, spontaneous nosebleeds. Propranolol has been used for a similar purpose in another group of patients with good effect. If it should work it would be a very simple and cost effective way to reduce patient symptoms and approve quality of life for these patients..
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 94526 0
Dr James Earnshaw
Address 94526 0
ENT Clinic, Royal Brisbane and Women's Hospital, Herston, QLD 4029.
Country 94526 0
Australia
Phone 94526 0
+61 736360946
Fax 94526 0
Email 94526 0
[email protected]
Contact person for public queries
Name 94527 0
Ms Kathryn Girling
Address 94527 0
ENT Clinic Research Office, Level 8 Ned Hanlon building, RBWH, Butterfield St., Herston, QLD 4029
Country 94527 0
Australia
Phone 94527 0
+61 736460946
Fax 94527 0
Email 94527 0
[email protected]
Contact person for scientific queries
Name 94528 0
Dr James Earnshaw
Address 94528 0
ENT Clinic Research Office, Level 8 Ned Hanlon building, RBWH, Butterfield St., Herston, QLD 4029
Country 94528 0
Australia
Phone 94528 0
+61 736460946
Fax 94528 0
Email 94528 0
[email protected]

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment
Study in Pilot stage only


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

Documents added manually
No documents have been uploaded by study researchers.

Documents added automatically
No additional documents have been identified.