Efficacy of Thalidomide in the Treatment of Hereditary Hemorrhagic Telangiectasia

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT01485224
• Date of registration: 28/11/2011
• Prospective Registration: No
• Primary sponsor: IRCCS Policlinico S. Matteo
• Public title: Efficacy of Thalidomide in the Treatment of Hereditary Hemorrhagic Telangiectasia THALI-HHT
• Scientific title: Efficacy of Thalidomide in the Treatment of Severe Recurrent Epistaxis in Hereditary Hemorrhagic Telangiectasia (HHT)
• Date of first enrolment: November 2011
• Target sample size: 31
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01485224
• Study type: Interventional
• Study design: Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Italy

Contacts

• Name: Carlo Balduini, M.D.
• Affiliation: Fondazione IRCCS Policlinico S. Matteo, Pavia, Italy

Key inclusion & exclusion criteria

Inclusion Criteria:

- 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 Criteria:

- 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



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Epistaxis

Hereditary Hemorrhagic Telangiectasia

Intervention(s)

Drug: Thalidomide

Primary Outcome(s)

Percentage of patients showing a decrease in the frequency, intensity and duration of epistaxis and in the blood transfusion requirement. [Time Frame: up to 24 weeks]

Secondary Outcome(s)

Time to relapse after the end of treatment [Time Frame: up to 24 weeks after the end of treatment]

Time to response [Time Frame: up to 24 weeks]

Minimum dose of the drug that reduces bleeding [Time Frame: up to 24 weeks]

Number of adverse events [Time Frame: up to 1 year]

Correlations between biological parameters, response to treatment and side effects profile [Time Frame: up to 24 weeks]

Size and number of telangiectasias evaluated by endoscopy of nasal mucosa (recording images of the size and localization of telangiectasias) [Time Frame: up to 24 weeks]

Secondary ID(s)

EudratCT 2011-004096-36

Source(s) of Monetary Support

Please refer to primary and secondary sponsors