Status:
COMPLETED
Efficacy of Thalidomide in the Treatment of Hereditary Hemorrhagic Telangiectasia
Lead Sponsor:
Fondazione IRCCS Policlinico San Matteo di Pavia
Conditions:
Hereditary Hemorrhagic Telangiectasia
Epistaxis
Eligibility:
All Genders
18+ years
Phase:
PHASE2
Brief 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 diffe...
Detailed Description
In the management of HHT epistaxis, multiple approaches have been tried, including electrocautery, laser, embolization, arterial ligation, but all approaches are largely palliative with variable resul...
Eligibility Criteria
Inclusion
- Diagnosis of HHT, according to the diagnostic criteria world-wide recognized (Curacao criteria), with severe recurrent epistaxis (grade 2-3 according to the criteria proposed by Pagella et al., i.e. at least one episode of overt bleeding/week requiring at least one blood transfusion during the last three months), and refractory to mini-invasive surgical procedures, i.e. argon plasma coagulation. For these patients, there is no effective treatment option currently available
- Age \> 18 years
- Ability of signing written informed consent
- Women of childbearing potential:
- declared intention not to start a pregnancy throughout the study and for four weeks following the date of the last dose of thalidomide (safe contraception, see Celgene guidelines, "Programma di Prevenzione della Gravidanza")
- negative serum pregnancy test obtained within 48 hours prior to the first dose of Thalidomide
- declared intention to undergo pregnancy tests periodically while on the study medication
- Males with female partner of childbearing potential:
- declared intention not to father throughout the study and for one week following the date of the last dose of thalidomide (safe contraception, see Celgene guidelines, "Programma di Prevenzione della Gravidanza")
- Estimated life expectancy must be greater than 10 months
Exclusion
- Pregnant or lactating women, or potentially fertile (both males and females) who have not agreed to avoid pregnancy during the trial period and for four weeks (females) or one week (males) following the date of the last dose of thalidomide
- Neurological diseases
- Psychiatric illness that would prevent granting of informed consent
- Active cardiovascular disease
- High risk for thromboembolic events (comorbidities, such as diabetes or uncontrolled infections, malignancy, immobility, prior history of thromboembolic events, use of erythropoietic agents or other agents such as hormone replacement therapy, central venous catheter, anti-cardiolipin, or anti-beta2 glycoprotein antibodies)
- Patients with rare hereditary problems of galactose intolerance, the Lapp lactase deficiency or glucose-galactose malabsorption since thalidomide capsules contain lactose
Key Trial Info
Start Date :
November 1 2011
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
October 11 2016
Estimated Enrollment :
31 Patients enrolled
Trial Details
Trial ID
NCT01485224
Start Date
November 1 2011
End Date
October 11 2016
Last Update
August 29 2017
Active Locations (1)
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1
Clinica Medica 3, Fondazione IRCCS Policlinico S. Matteo
Pavia, Italy, 27100