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    Clinical Trial Results:
    Therapeutic effect of propranolol in a series of patients with von Hippel-Lindau disease and retinal hemangioblastomas in short, medium and long term treatment.

    Summary
    EudraCT number
    		 2014-003671-30
    Trial protocol
    		 ES  
    Global end of trial date
    01 Sep 2016

    Results information
    Results version number
    		 v1(current)
    This version publication date
    04 Jun 2022
    First version publication date
    04 Jun 2022
    Other versions
    Summary report(s)
    		 Evaluation of the safety and effectiveness of oral propranolol in patients with von Hippel-Lindau disease and retinal hemangioblastomas: phase III clinical trial

    Trial information

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    Trial identification
    Sponsor protocol code
    VHL-HOPE-2014-1
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 -
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Alianza VHL
    Sponsor organisation address
    C/ SANTA MARTA, 24, OLÍAS DEL REY, Spain, 45280
    Public contact
    Susi Martínez Gómez, ALIANZA ESPAÑOLA DE FAMILIAS DE VON HIPPEL-LINDAU, 34 616050514, info@alianzavhl.org
    Scientific contact
    Karina Villar, ALIANZA ESPAÑOLA DE FAMILIAS DE VON HIPPEL-LINDAU, 34 607680759, kvillar@jccm.es
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    01 Sep 2016
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    01 Sep 2016
    Global end of trial reached?
    Yes
    Global end of trial date
    01 Sep 2016
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    TO EVALUATE THE EFFECTIVENESS OF PROPRANOLOL IN CONTROLLING THE GROWTH OF PAPILLARY AND JUXTAPAPILLARY RETINAL HEMANGIOBLASTOMAS (HB)
    Protection of trial subjects
    All patients had the mobile phone number of their ophthalmologist for possible consultations
    Background therapy
    		 -
    Evidence for comparator
    		 No comparator treatment
    Actual start date of recruitment
    15 Sep 2014
    Long term follow-up planned
    Yes
    Long term follow-up rationale
    		 Safety, Efficacy, Scientific research
    Long term follow-up duration
    		 2 Years
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Spain: 7
    Worldwide total number of subjects
    7
    EEA total number of subjects
    7
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    0
    Children (2-11 years)
    0
    Adolescents (12-17 years)
    1
    Adults (18-64 years)
    6
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 The association of patients Alianza VHL informed about and invited their members to participate in the study. Seven VHL patients from different regions of Spain were included. All of them had a positive genetic diagnosis of VHL.

    Pre-assignment
    Screening details
    		 The participants should meet one of the following two criteria: Papillary or juxtapapillary hemangioblastomas, non-eligible for standard treatment (laser photocoagulation or cryotherapy) due to the high risk of iatrogenic visual loss.Peripheral retinal hemangioblastomas for which patients had rejected standard treatments.

    Period 1
    Period 1 title
    Baseline period
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 Treatment arm
    Arm description
    		 The pharmaceutical form and strength used was propranolol 40 mg, film-coated tablets, 1 every 8 hours up to a total dosage of 120 mg/day, achieved in 7–10 days. An experienced cardiologist in handling propranolol established the dosage regimen. The patients did not receive other treatments during the study.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Propranolol hydrochloride
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Film-coated tablet
    Routes of administration
    Oral use
    Dosage and administration details
    propranolol 40 mg, film-coated tablets, 1 every 8 hours

    Number of subjects in period 1
    		Treatment arm
    Started
    7
    Completed
    7
    Period 2
    Period 2 title
    Final evaluation
    Is this the baseline period?
    		 No
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 Treatment arm
    Arm description
    		 The pharmaceutical form and strength used was propranolol 40 mg, film-coated tablets, 1 every 8 hours up to a total dosage of 120 mg/day, achieved in 7–10 days. An experienced cardiologist in handling propranolol established the dosage regimen. The patients did not receive other treatments during the study.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Propranolol hydrochloride
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Film-coated tablet
    Routes of administration
    Oral use
    Dosage and administration details
    propranolol 40 mg, film-coated tablets, 1 every 8 hours

    Number of subjects in period 2
    		Treatment arm
    Started
    7
    Completed
    6
    Not completed
    1
         Physician decision
    1

    Baseline characteristics

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    End points

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    End points reporting groups
    Reporting group title
    Treatment arm
    Reporting group description
    		 The pharmaceutical form and strength used was propranolol 40 mg, film-coated tablets, 1 every 8 hours up to a total dosage of 120 mg/day, achieved in 7–10 days. An experienced cardiologist in handling propranolol established the dosage regimen. The patients did not receive other treatments during the study.
    Reporting group title
    Treatment arm
    Reporting group description
    		 The pharmaceutical form and strength used was propranolol 40 mg, film-coated tablets, 1 every 8 hours up to a total dosage of 120 mg/day, achieved in 7–10 days. An experienced cardiologist in handling propranolol established the dosage regimen. The patients did not receive other treatments during the study.

    Primary: Number and size of all the retinal hemangioblastomas

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    End point title
    		 Number and size of all the retinal hemangioblastomas [1]
    End point description
    End point type
    Primary
    End point timeframe
    The primary endpoint of the study was the number and size of all the retinal hemangioblastomas. The follow-up visits were scheduled at the Ophthalmology Department at baseline and at months 1, 3, 6, 9 and 12 of treatment.
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: the number of patients and tumors is so small that statistical analysis is meaningless. This study has only a descriptive objective.
    End point values
    		 Treatment arm
    Number of subjects analysed
    7
    Units: cm
        number (not applicable)
    7
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    At baseline, and at 1, 3, 6, 9 and 12 months
    Assessment type
    		 Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    17.1
    Reporting groups
    Reporting group title
    Treatment arm
    Reporting group description
    		 -

    Serious adverse events
    		 Treatment arm
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 7 (0.00%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Frequency threshold for reporting non-serious adverse events: 1%
    Non-serious adverse events
    		 Treatment arm
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    1 / 7 (14.29%)
    Vascular disorders
    Hypotension
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? No

    Interruptions (globally)
    Were there any global interruptions to the trial? No

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported

    Online references
    http://www.ncbi.nlm.nih.gov/pubmed/31245608
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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