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Efficacy of Thalidomide in the Treatment of Hereditary Hemorrhagic Telangiectasia

NCT01485224 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 31

Last updated 2017-08-29

No results posted yet for this study

Summary

Hereditary hemorrhagic telangiectasia (HHT) (OMIM 187300 and 600376), also known as Rendu-Osler-Weber syndrome, is an autosomal dominant disease and has a prevalence between 1:5000 and 1:8000 in different populations. Clinically, the occurrence of mucocutaneous and gastrointestinal telangiectasias and of systemic arteriovenous malformations is commonly observed. Recurrent and severe epistaxis, due to the presence of telangiectasias in nasal mucosa, is the most common presentation of HHT, frequently leading to severe anemia requiring intravenous iron and blood transfusions. Although not life threatening, severe epistaxis has a great impact on quality of life in HHT patients and it represents the most important impediment in daily activities, that poses therapeutic challenge. Recently, angiogenesis has been implicated in the pathogenesis of HHT. Circulating concentrations of both TGF-beta and vascular endothelial growth factor (VEGF) are significantly elevated and therefore, anti-angiogenic substances may be effective in the treatment of vascular malformations in this disease. Thalidomide functions as a potent immunosuppressive and antiangiogenic agent. The aim of this study is to assess the clinical effects of thalidomide therapy on the severity of epistaxis in subjects with HHT who are refractory to standard therapies.

Conditions

  • Hereditary Hemorrhagic Telangiectasia
  • Epistaxis

Interventions

DRUG

Thalidomide

Eligible patients will receive thalidomide at a starting dose of 50 mg/day by mouth at bedtime for 4 weeks. In the event of unsatisfactory/no response, thalidomide dosage will be progressively increased by 50 mg/day every 4 weeks until complete or partial response, to a maximum dose of 200 mg/day. Treatment will be continued until one of the following criteria is met: * 8 additional weeks of treatment after the achievement of complete response * 16 additional weeks of treatment after the achievement of partial response * 24 weeks of treatment completed without response * unacceptable toxicity. Then, patients will be followed off of thalidomide for 24 weeks.

Sponsors & Collaborators

  • Fondazione IRCCS Policlinico San Matteo di Pavia

    lead OTHER

Principal Investigators

  • Carlo Balduini, M.D. · Fondazione IRCCS Policlinico S. Matteo, Pavia, Italy

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2011-11-30
Primary Completion
2014-10-31
Completion
2016-10-11

Countries

  • Italy

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01485224 on ClinicalTrials.gov