Vitamin D and Hereditary Haemorrhagic Telangiectasia

NCT03981562 | Unknown Phase 2

• 1 other identifier: H18-01337
• Study Type: interventional
• Target: 60
• Locations: 1 country
• Sites: 1

Brief 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. 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.

Conditions

Hereditary Haemorrhagic Telangiectasia

Keywords

Epistaxis

Outcome Measures

Primary Outcomes (1)

• 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).

  Baseline, 3 months, and 6 months

Secondary Outcomes (1)

• Change in Modified Lund-Kennedy Score

  Baseline, 3 months, and 6 months

Study Arms (3)

1000 IU Vitamin D

EXPERIMENTAL

Drug: Vit D

4000 IU Vitamin D

EXPERIMENTAL

Drug: Vit D

Placebo

PLACEBO COMPARATOR

Drug: Placebo Oral Tablet

Interventions

Vit D DRUG

Patients will take an oral vitamin D supplement once a day for 6 months.

1000 IU Vitamin D; 4000 IU Vitamin D

Placebo Oral Tablet DRUG

Patients will take a placebo oral tablet once a day for 6 months.

Placebo

Eligibility Criteria

• Age: 19 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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)

You may not qualify if:

• 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.

Sponsors & Collaborators

• St. Paul's Hospital, Canada: lead

Study Sites (1)

E.N.T Clinic, St. Paul's Hospital

Vancouver, British Columbia, V6Z 1Y6, Canada

RECRUITING

Related Publications (11)

• 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.

  PMID: 19337313 BACKGROUND

• Shovlin 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.

  PMID: 10751092 BACKGROUND

• Hoag 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.

  PMID: 20087969 BACKGROUND

• Chamali 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.

  PMID: 27195194 BACKGROUND

• Plauchu 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.

  PMID: 2729347 BACKGROUND

• Al 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.

  PMID: 21718915 BACKGROUND

• Min 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.

  PMID: 24227938 BACKGROUND

• Weber 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.

  PMID: 29537299 BACKGROUND

• Geisthoff 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.

  PMID: 26205234 BACKGROUND

• McDonald 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.

  PMID: 21546842 BACKGROUND

• Reh 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.

  PMID: 24782401 BACKGROUND

Related Links

• Osler- Weber-Rendu Disease (Hereditary Hemorrhagic Telangiectasia).
• Vitamin D: fact sheet for health professionals.

MeSH Terms

Conditions

Telangiectasia, Hereditary Hemorrhagic; Epistaxis

Condition Hierarchy (Ancestors)

Hemostatic Disorders; Vascular Diseases; Cardiovascular Diseases; Telangiectasis; Hemorrhagic Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Vascular Malformations; Cardiovascular Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Nose Diseases; Respiratory Tract Diseases; Otorhinolaryngologic Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Signs and Symptoms, Respiratory; Signs and Symptoms

Central Study Contacts

Amin Javer, MD FRCSCFARS

CONTACT

6048069926; sinusdoc@me.com

India Dhillon, BSc

CONTACT

6048069926; idhillon3@providencehealth.bc.ca

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Masking Details: The study is double blinded, the investigators and the patients throughout the data collection and data analysis period will not be aware of the vitamin D dosage given.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Director, St. Paul's Sinus Centre

Model Details: 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.

Study Record Dates

• First Submitted: May 24, 2019
• First Posted: June 11, 2019
• Study Start: July 16, 2018
• Primary Completion: July 1, 2020
• Study Completion: September 1, 2020
• Last Updated: June 11, 2019

Record last verified: 2019-06

Data Sharing

• IPD Sharing: Will share

Locations

CA (1)