Hereditary Haemorrhagic TelangiectasiaPhase 2NCT03981562
Vitamin D and Hereditary Haemorrhagic Telangiectasia
A Phase 2 study testing Vit D in people with Hereditary Haemorrhagic Telangiectasia, run by St. Paul's Hospital, Canada at 1 site in Canada.
Trial summary
The study aims to assess whether supplementing vitamin D in patients diagnosed with Hereditary Haemorrhagic Telangiectasia (HHT) will decrease the frequency and severity of nosebleeds these patients experience. Read more about this study
- Who is it for?
- Ages 19 years and older
- Conditions the study is for
- Hereditary Haemorrhagic Telangiectasia
- What is being tested?
- Vit D
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 60 participants
- Where
- 1 siteCanada
- Study dates
- 16 Jul 2018to 1 Sep 2020
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 19 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 19 years or older
- Definite diagnosis of HHT using the Curacao criteria;
- HHT patients already on Vitamin D supplementation (these patients will still be included since the study is examining mega-doses specifically)
Who cannot join
- Patients with sinonasal tumours;
- Patients with bleeding disorders;
- Patients with serum levels of 250 or more ng/ml of vitamin D before or during the study supplementation (considered to be toxic levels)
- Patients who are unable to speak English;
- Patients who live outside B.C.
Trial locations and contacts
1 site in 1 country.
Canada1 site, 1 recruiting
- Recruiting
E.N.T Clinic, St. Paul's Hospital
Vancouver, British Columbia, V6Z 1Y6
India Dhillon, BSc, 604-806-9926, idhillon3@providencehealth.bc.ca
Amin R Javer, MD FRCSCFARS
About this study
Official title: Vitamin D Supplementation and Reduction of Severity and Frequency of Epistaxis in Hereditary Haemorrhagic Telangiectasia
The study aims to assess whether supplementing vitamin D in patients diagnosed with Hereditary Haemorrhagic Telangiectasia (HHT) will decrease the frequency and severity of nosebleeds these patients experience. It is hypothesized that the larger the dose of daily vitamin D given to the patients, the less frequent and less severe the nosebleeds will be.
Read the detailed description
- Purpose: The focus of the study is to determine if supplementing vitamin D in patients diagnosed with HHT will reduce the frequency and severity of epistaxis episodes.
- Hypothesis: For patients diagnosed with HHT, daily vitamin D supplementation will reduce the frequency and severity of epistaxis. Additionally, there will be a greater reduction in epistaxis frequency and severity the larger the dose of daily vitamin D supplemented
- Justification: Hereditary haemorrhagic telangiectasia (HHT) is a rare autosomal dominant inherited disorder of the fibrovascular tissue that causes malformation of capillaries (called "telangiectases") and/or larger blood vessels (called "arteriovenous malformations") throughout the body. The formation of these lesions ultimately result in patients experiencing increased tendency for bleeding. These lesions may present in varying locations systemically, however recurrent and severe epistaxis is the most common presentation of HHT. Various medical and surgical treatments exist for management of HHT patients which are aimed at decreasing the frequency and severity of epistaxis episodes. These therapies include: humidification, nasal lubrication, nasal hot saline irrigation, intranasal packing, oestrogen ointment, tranexamic acid, bevacizumab systemic/topically applied, use of propranolol to lower blood pressure, electrocautery and YAG laser. Although all these various options are available, there currently is no consensus toward an optimal solution. Treatment can become especially difficult due to progressive anaemia or when anticoagulant or anti-thrombotic therapy becomes necessary. Therefore, there is a need for establishment of a safe and effective therapy. It has been suggested that vitamin D plays a role in cardiovascular health. Vitamin D plays a key role in establishing the integrity of blood vessels as it has been shown to provide protection of the vascular wall as well has having minor anticoagulation effects. As well, observational retrospective studies have found an association between vitamin D levels and epistaxis bleeding time and severity in HHT patients, with higher serum vitamin D levels being associated with decrease epistaxis bleeding time and severity. Despite evidence of positive effect of vitamin D on HHT, currently no prospective study has been done.
- Objectives: Primary Objective To determine if vitamin D supplementation with 1000 IU or 4000 IU will reduce the frequency and severity of epistaxis in HHT patients. Secondary Objective To determine the adequate dosage of vitamin D supplementation required to reduce frequency and severity of epistaxis in HHT patients.
- Research Method
The proposed study is a prospective double-blinded, placebo controlled, randomized control trial.
All the St. Paul's Sinus Centre patients willing to participate in the study will be invited for screening. Currently, the clinic has ~60 patients with HHT; therefore the investigators hope to enrol ~20 patients in each of the three study arms.
Consenting patient will be randomized to ensure equal number of experimental and control patients are in each arm. A closed envelop system will be used to randomize participants within each arm. Patients diagnosed with HHT who are not taking vitamin D supplementation at the time of recruitment will be randomized into one of the following three groups:
- 1000 IU/day vitamin D,
- 4000 IU/day vitamin D, or
- Placebo control Patients who are already taking vitamin D at the time of recruitment will be asked to stop taking the current dose of vitamin D and will be randomized into either the 1000 IU or 4000 IU arm of the study. Patients previously taking Vitamin D will not participate as placebo controls.
Upon enrolment in the study, demographic data will be obtained, including age, gender, and ethnicity. Patient's will also have baseline blood work drawn, standard of care, (full hematological profile, ferritin, aPTT, INR, serum vitamin D, IgE) and be asked to fill out a questionnaire to provide a baseline Epistaxis Severity Score (ESS). This questionnaire is routinely used to assess quality of life in HHT patients and includes six independent predictors of self-described epistaxis severity. Baseline nasal endoscopic scoring will also be performed by endoscopic imaging of the nasal cavity.
The patients will be instructed on dosing of vitamin D supplementation. Patients will continue daily supplementation for three months until the first follow up visit. At the first follow-up visit patients will again have blood work drawn, be asked to fill out the ESS questionnaire, and receive nasal endoscopic scoring.
The last follow-up visit will be at six months and a repeat of the same procedures/tests will be carried out.
- Statistical Analysis:
The primary outcome of this study will be the Epistaxis Severity Score (ESS), which is obtained from patient questionnaires. The secondary outcome will be the Nasal Endoscopic Score. The difference in the ESS and endoscopic score before and after supplementation within and between groups will be analyzed using paired and unpaired students t-tests based on the variance results.
Descriptive statistics (mean, median, SD) will be used to describe demographic and hematological data collected.
- Conditions
- Hereditary Haemorrhagic Telangiectasia
- Being tested
- Vit D, Placebo Oral Tablet
- Keywords
- Epistaxis
- Medical subject headings
- Telangiectasia, Hereditary Hemorrhagic, Epistaxis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Vit DDrug
Patients will take an oral vitamin D supplement once a day for 6 months.
Placebo Oral TabletDrug
Patients will take a placebo oral tablet once a day for 6 months.
| Study group | Role | What they receive |
|---|---|---|
| 1000 IU Vitamin D | Experimental | Not described Receives: Drug: Vit D |
| 4000 IU Vitamin D | Experimental | Not described Receives: Drug: Vit D |
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo Oral Tablet |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Epistaxis Severity Score
A questionnaire that will be given to the patients at each visit which is a major predictor of quality of life in HHT patients.
The score includes six independent predictors of self-described epistaxis severity. The responses will then be weighted by respective coefficients and these added together to give a raw ESS, which will then be divided by the range of the raw score (2.71) and multiplied by 10 to give normalized ESS within the range of 0 to 10 (no epistaxis to severe epistaxis).
Measured: Baseline, 3 months, and 6 months
Secondary outcomes (1)
Change in Modified Lund-Kennedy Score
The score is an objective measure based on Endoscopic sinonasal mucosal inflammation. The score is determined from a range of 0-12 with higher numbers indicating worse inflammation. Endoscopy will determine the density and location of telangiectasias, vascular morphology or patterns, relative percentage of arteriovenous malformations (AVMs), degree of crusting, septal perforation and site.
Measured: Baseline, 3 months, and 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Jun 2019
- Study start16 Jul 2018
- Primary completion1 Jul 2020 (estimated)
- Study completion1 Sep 2020 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (11)Papers the sponsor has linked to this study.
- Other trials for Hereditary Haemorrhagic TelangiectasiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Patients included in the study will be randomized. Block randomization will be utilized to ensure an equal number of experimental and control patients are in each arm. A closed envelope system will be used to randomize participants within each arm.
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- St. Paul's Hospital, Canada
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Amin Javer, St. Paul's Hospital, Canada
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03981562
- Sponsor's protocol ID
- H18-01337
- Status last verified
- Jun 2019
- First submitted
- 24 May 2019
- Last updated
- 11 Jun 2019
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: All IPD that underlie results in a publication will be available to other researchers.
Publications
Govani FS, Shovlin CL. Hereditary haemorrhagic telangiectasia: a clinical and scientific review. Eur J Hum Genet. 2009 Jul;17(7):860-71. doi: 10.1038/ejhg.2009.35. Epub 2009 Apr 1.
PubMed 19337313Shovlin CL, Guttmacher AE, Buscarini E, Faughnan ME, Hyland RH, Westermann CJ, Kjeldsen AD, Plauchu H. Diagnostic criteria for hereditary hemorrhagic telangiectasia (Rendu-Osler-Weber syndrome). Am J Med Genet. 2000 Mar 6;91(1):66-7. doi: 10.1002/(sici)1096-8628(20000306)91:13.0.co;2-p.
PubMed 10751092Hoag JB, Terry P, Mitchell S, Reh D, Merlo CA. An epistaxis severity score for hereditary hemorrhagic telangiectasia. Laryngoscope. 2010 Apr;120(4):838-43. doi: 10.1002/lary.20818.
PubMed 20087969Chamali B, Finnamore H, Manning R, Laffan MA, Hickson M, Whelan K, Shovlin CL. Dietary supplement use and nosebleeds in hereditary haemorrhagic telangiectasia - an observational study. Intractable Rare Dis Res. 2016 May;5(2):109-13. doi: 10.5582/irdr.2016.01019.
PubMed 27195194Plauchu H, de Chadarevian JP, Bideau A, Robert JM. Age-related clinical profile of hereditary hemorrhagic telangiectasia in an epidemiologically recruited population. Am J Med Genet. 1989 Mar;32(3):291-7. doi: 10.1002/ajmg.1320320302.
PubMed 2729347Al Mheid I, Patel R, Murrow J, Morris A, Rahman A, Fike L, Kavtaradze N, Uphoff I, Hooper C, Tangpricha V, Alexander RW, Brigham K, Quyyumi AA. Vitamin D status is associated with arterial stiffness and vascular dysfunction in healthy humans. J Am Coll Cardiol. 2011 Jul 5;58(2):186-92. doi: 10.1016/j.jacc.2011.02.051.
PubMed 21718915Min B. Effects of vitamin d on blood pressure and endothelial function. Korean J Physiol Pharmacol. 2013 Oct;17(5):385-92. doi: 10.4196/kjpp.2013.17.5.385. Epub 2013 Oct 17.
PubMed 24227938Weber LM, McDonald J, Whitehead K. Vitamin D levels are associated with epistaxis severity and bleeding duration in hereditary hemorrhagic telangiectasia. Biomark Med. 2018 Apr;12(4):365-371. doi: 10.2217/bmm-2017-0229. Epub 2018 Mar 14.
PubMed 29537299Geisthoff UW, Nguyen HL, Roth A, Seyfert U. How to manage patients with hereditary haemorrhagic telangiectasia. Br J Haematol. 2015 Nov;171(4):443-52. doi: 10.1111/bjh.13606. Epub 2015 Jul 23.
PubMed 26205234McDonald J, Bayrak-Toydemir P, Pyeritz RE. Hereditary hemorrhagic telangiectasia: an overview of diagnosis, management, and pathogenesis. Genet Med. 2011 Jul;13(7):607-16. doi: 10.1097/GIM.0b013e3182136d32.
PubMed 21546842Reh DD, Yin LX, Laaeq K, Merlo CA. A new endoscopic staging system for hereditary hemorrhagic telangiectasia. Int Forum Allergy Rhinol. 2014 Aug;4(8):635-9. doi: 10.1002/alr.21339. Epub 2014 Apr 29.
PubMed 24782401
Study at a glance
From the registry record, last updated by the sponsor on 11 Jun 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Vitamin D Supplementation and Reduction of Severity and Frequency of Epistaxis in Hereditary Haemorrhagic Telangiectasia
- ClinicalTrials.gov ID
- NCT03981562
- Acronym / sponsor ID
- H18-01337
- Conditions
- Hereditary Haemorrhagic Telangiectasia
- Keywords
- Epistaxis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- St. Paul's Hospital, CanadaUniversity, hospital or other
- Study start
- 16 Jul 2018 (actual)
- Primary completion
- 1 Jul 2020 (estimated)
- Study completion
- 1 Sep 2020 (estimated)
- First posted
- 11 Jun 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The study aims to assess whether supplementing vitamin D in patients diagnosed with Hereditary Haemorrhagic Telangiectasia (HHT) will decrease the frequency and severity of nosebleeds these patients experience. It is hypothesized that the larger the dose of daily vitamin D given to the patients, the less frequent and less severe the nosebleeds will be.
Detailed description
- Purpose: The focus of the study is to determine if supplementing vitamin D in patients diagnosed with HHT will reduce the frequency and severity of epistaxis episodes.
- Hypothesis: For patients diagnosed with HHT, daily vitamin D supplementation will reduce the frequency and severity of epistaxis. Additionally, there will be a greater reduction in epistaxis frequency and severity the larger the dose of daily vitamin D supplemented
- Justification: Hereditary haemorrhagic telangiectasia (HHT) is a rare autosomal dominant inherited disorder of the fibrovascular tissue that causes malformation of capillaries (called "telangiectases") and/or larger blood vessels (called "arteriovenous malformations") throughout the body. The formation of these lesions ultimately result in patients experiencing increased tendency for bleeding. These lesions may present in varying locations systemically, however recurrent and severe epistaxis is the most common presentation of HHT. Various medical and surgical treatments exist for management of HHT patients which are aimed at decreasing the frequency and severity of epistaxis episodes. These therapies include: humidification, nasal lubrication, nasal hot saline irrigation, intranasal packing, oestrogen ointment, tranexamic acid, bevacizumab systemic/topically applied, use of propranolol to lower blood pressure, electrocautery and YAG laser. Although all these various options are available, there currently is no consensus toward an optimal solution. Treatment can become especially difficult due to progressive anaemia or when anticoagulant or anti-thrombotic therapy becomes necessary. Therefore, there is a need for establishment of a safe and effective therapy. It has been suggested that vitamin D plays a role in cardiovascular health. Vitamin D plays a key role in establishing the integrity of blood vessels as it has been shown to provide protection of the vascular wall as well has having minor anticoagulation effects. As well, observational retrospective studies have found an association between vitamin D levels and epistaxis bleeding time and severity in HHT patients, with higher serum vitamin D levels being associated with decrease epistaxis bleeding time and severity. Despite evidence of positive effect of vitamin D on HHT, currently no prospective study has been done.
- Objectives: Primary Objective To determine if vitamin D supplementation with 1000 IU or 4000 IU will reduce the frequency and severity of epistaxis in HHT patients. Secondary Objective To determine the adequate dosage of vitamin D supplementation required to reduce frequency and severity of epistaxis in HHT patients.
- Research Method
The proposed study is a prospective double-blinded, placebo controlled, randomized control trial.
All the St. Paul's Sinus Centre patients willing to participate in the study will be invited for screening. Currently, the clinic has ~60 patients with HHT; therefore the investigators hope to enrol ~20 patients in each of the three study arms.
Consenting patient will be randomized to ensure equal number of experimental and control patients are in each arm. A closed envelop system will be used to randomize participants within each arm. Patients diagnosed with HHT who are not taking vitamin D supplementation at the time of recruitment will be randomized into one of the following three groups:
- 1000 IU/day vitamin D,
- 4000 IU/day vitamin D, or
- Placebo control Patients who are already taking vitamin D at the time of recruitment will be asked to stop taking the current dose of vitamin D and will be randomized into either the 1000 IU or 4000 IU arm of the study. Patients previously taking Vitamin D will not participate as placebo controls.
Upon enrolment in the study, demographic data will be obtained, including age, gender, and ethnicity. Patient's will also have baseline blood work drawn, standard of care, (full hematological profile, ferritin, aPTT, INR, serum vitamin D, IgE) and be asked to fill out a questionnaire to provide a baseline Epistaxis Severity Score (ESS). This questionnaire is routinely used to assess quality of life in HHT patients and includes six independent predictors of self-described epistaxis severity. Baseline nasal endoscopic scoring will also be performed by endoscopic imaging of the nasal cavity.
The patients will be instructed on dosing of vitamin D supplementation. Patients will continue daily supplementation for three months until the first follow up visit. At the first follow-up visit patients will again have blood work drawn, be asked to fill out the ESS questionnaire, and receive nasal endoscopic scoring.
The last follow-up visit will be at six months and a repeat of the same procedures/tests will be carried out.
- Statistical Analysis:
The primary outcome of this study will be the Epistaxis Severity Score (ESS), which is obtained from patient questionnaires. The secondary outcome will be the Nasal Endoscopic Score. The difference in the ESS and endoscopic score before and after supplementation within and between groups will be analyzed using paired and unpaired students t-tests based on the variance results.
Descriptive statistics (mean, median, SD) will be used to describe demographic and hematological data collected.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 3
- Model description
- Patients included in the study will be randomized. Block randomization will be utilized to ensure an equal number of experimental and control patients are in each arm. A closed envelope system will be used to randomize participants within each arm.
| Arm | Type | Receives |
|---|---|---|
| 1000 IU Vitamin D | Experimental | Drug: Vit D |
| 4000 IU Vitamin D | Experimental | Drug: Vit D |
| Placebo | Placebo comparator | Drug: Placebo Oral Tablet |
Who may qualify
- Ages
- 19 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- 19 years or older
- Definite diagnosis of HHT using the Curacao criteria;
- HHT patients already on Vitamin D supplementation (these patients will still be included since the study is examining mega-doses specifically)
Key exclusion criteria (5 listed)
- Patients with sinonasal tumours;
- Patients with bleeding disorders;
- Patients with serum levels of 250 or more ng/ml of vitamin D before or during the study supplementation (considered to be toxic levels)
- Patients who are unable to speak English;
- Patients who live outside B.C.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Vit DPatients will take an oral vitamin D supplement once a day for 6 months. | Drug | 1000 IU Vitamin D, 4000 IU Vitamin D |
| Placebo Oral TabletPatients will take a placebo oral tablet once a day for 6 months. | Drug | Placebo |
Primary outcome
Change in Epistaxis Severity Score
Time frame: Baseline, 3 months, and 6 months
A questionnaire that will be given to the patients at each visit which is a major predictor of quality of life in HHT patients. The score includes six independent predictors of self-described epistaxis severity. The responses will then be weighted by respective coefficients and these added together to give a raw ESS, which will then be divided by the range of the raw score (2.71) and multiplied by 10 to give normalized ESS within the range of 0 to 10 (no epistaxis to severe epistaxis).
1 secondary outcome
Change in Modified Lund-Kennedy Score
Time frame: Baseline, 3 months, and 6 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Jun 2019.
- Background: PubMed 19337313Govani FS, Shovlin CL. Hereditary haemorrhagic telangiectasia: a clinical and scientific review. Eur J Hum Genet. 2009 Jul;17(7):860-71. doi: 10.1038/ejhg.2009.35. Epub 2009 Apr 1.
- Background: PubMed 10751092Shovlin CL, Guttmacher AE, Buscarini E, Faughnan ME, Hyland RH, Westermann CJ, Kjeldsen AD, Plauchu H. Diagnostic criteria for hereditary hemorrhagic telangiectasia (Rendu-Osler-Weber syndrome). Am J Med Genet. 2000 Mar 6;91(1):66-7. doi: 10.1002/(sici)1096-8628(20000306)91:13.0.co;2-p.
- Background: PubMed 20087969Hoag JB, Terry P, Mitchell S, Reh D, Merlo CA. An epistaxis severity score for hereditary hemorrhagic telangiectasia. Laryngoscope. 2010 Apr;120(4):838-43. doi: 10.1002/lary.20818.
- Background: PubMed 27195194Chamali B, Finnamore H, Manning R, Laffan MA, Hickson M, Whelan K, Shovlin CL. Dietary supplement use and nosebleeds in hereditary haemorrhagic telangiectasia - an observational study. Intractable Rare Dis Res. 2016 May;5(2):109-13. doi: 10.5582/irdr.2016.01019.
- Background: PubMed 2729347Plauchu H, de Chadarevian JP, Bideau A, Robert JM. Age-related clinical profile of hereditary hemorrhagic telangiectasia in an epidemiologically recruited population. Am J Med Genet. 1989 Mar;32(3):291-7. doi: 10.1002/ajmg.1320320302.
- Background: PubMed 21718915Al Mheid I, Patel R, Murrow J, Morris A, Rahman A, Fike L, Kavtaradze N, Uphoff I, Hooper C, Tangpricha V, Alexander RW, Brigham K, Quyyumi AA. Vitamin D status is associated with arterial stiffness and vascular dysfunction in healthy humans. J Am Coll Cardiol. 2011 Jul 5;58(2):186-92. doi: 10.1016/j.jacc.2011.02.051.
- Background: PubMed 24227938Min B. Effects of vitamin d on blood pressure and endothelial function. Korean J Physiol Pharmacol. 2013 Oct;17(5):385-92. doi: 10.4196/kjpp.2013.17.5.385. Epub 2013 Oct 17.
- Background: PubMed 29537299Weber LM, McDonald J, Whitehead K. Vitamin D levels are associated with epistaxis severity and bleeding duration in hereditary hemorrhagic telangiectasia. Biomark Med. 2018 Apr;12(4):365-371. doi: 10.2217/bmm-2017-0229. Epub 2018 Mar 14.
- Background: PubMed 26205234Geisthoff UW, Nguyen HL, Roth A, Seyfert U. How to manage patients with hereditary haemorrhagic telangiectasia. Br J Haematol. 2015 Nov;171(4):443-52. doi: 10.1111/bjh.13606. Epub 2015 Jul 23.
- Background: PubMed 21546842McDonald J, Bayrak-Toydemir P, Pyeritz RE. Hereditary hemorrhagic telangiectasia: an overview of diagnosis, management, and pathogenesis. Genet Med. 2011 Jul;13(7):607-16. doi: 10.1097/GIM.0b013e3182136d32.
- Background: PubMed 24782401Reh DD, Yin LX, Laaeq K, Merlo CA. A new endoscopic staging system for hereditary hemorrhagic telangiectasia. Int Forum Allergy Rhinol. 2014 Aug;4(8):635-9. doi: 10.1002/alr.21339. Epub 2014 Apr 29.
- Osler- Weber-Rendu Disease (Hereditary Hemorrhagic Telangiectasia).Link provided by the sponsor.
- Vitamin D: fact sheet for health professionals.Link provided by the sponsor.
- Individual participant dataSharing planned: Yes. All IPD that underlie results in a publication will be available to other researchers.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Vit D in people with Hereditary Haemorrhagic Telangiectasia, run by St. Paul's Hospital, Canada at 1 site in Canada.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 11 Jun 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Amin Javer, MD FRCSCFARS (6048069926, sinusdoc@me.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Epistaxis Severity Score (time frame: Baseline, 3 months, and 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03981562, which also lists 11 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Jun 2019.