Effectiveness of Somatostatin Analogues in Patients with hereditary hemorrhagic telangiectasia and symptomatic gastrointestinal bleeding, the SAIPAN-trial: a multicenter, randomized, open-label, parallelgroup, superiority trial.

Phase 3

Recruiting

• Conditions: Hereditary hemorrhagic teleangiectasia (HHT); Rendu-Osler-Weber syndrome; 10018031

• Registration Number: NL-OMON46190
• Lead Sponsor: Radboud Universitair Medisch Centrum

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 15 April 2024

Recruitment & Eligibility

• Status: Recruiting
• Sex: Not specified
• Target Recruitment: 18

Inclusion Criteria

- Patients older than 18 years with written informed consent.
- Diagnosis of HHT: either confirmed by genetic testing or the Curacao criteria (definitediagnosis).
- Presence of IDA in combination with the presence endoscopic proven GI AVM manifestations / telangiectasias confirmed within the last 12 months (upper and/or lower endoscopy and/or capsule endoscopy).
- Endoscopic refractory: at least 1 endoscopic APC / laser /other endoscopic treatment modality performed in the past 5 years.
- Substantial transfusion dependency: at least 4 blood units and / or intravenous iron in the 6 months prior to study inclusion with a:
o At least one serum ferritin below < 30 ug/l within the last 6 months requiring iron
infusion above or equal to 1 g and/or
o Hemoglobin below 5.6 mmol/l (9.0 g/dl) or are in need of transfusions
due to anemia related symptoms within the last 6 months requiring blood transfusion above.

Exclusion Criteria

- liver cirrhosis child-pugh C.
- symptomatic cholecystolithiasis (possible side-effect octreotide).
- pregnancy or nursing women or women having a pregnancy wish during the study period.

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>'Successful response', defined as a decrease of >=50% in the amount of units<br /><br>intravenous iron and/or blood transfusions given.</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>The percentual or mean/median difference between the half year prior to<br /><br>inclusion and the treatment period of a half year between the treatment and<br /><br>observational arm in:<br /><br>- blood and intravenous iron requirements<br /><br>- PROM*s: quality of life (SF-36, EQ-5D), level of fatigue (MFI-20), epistaxis<br /><br>severity (ESS tool), and patient satisfaction<br /><br>- hemoglobin and ferritin levels<br /><br>- number of endoscopic treatments<br /><br>- cost-effectiveness<br /><br>- Safety</p><br>