Octreotide in Patients With GI Bleeding Due to Rendu-Osler-Weber

Anemia Clinical Trial

— ROW  

Official title:

An Uncontrolled, Pilot-study Assessing the Efficacy of Octreotide Long-acting Release to Decrease Transfusion Requirements and Endoscopy Frequency in Patients With Rendu-Osler-Weber and Gastrointestinal Bleeding

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT02874326
• Other study ID #: NLROW.1012.15
• Status: Active, not recruiting
• Phase: Phase 2
• First received: August 17, 2016
• Last updated: April 18, 2018
• Start date: October 2016
• Est. completion date: October 2018

Study information

• Verified date: April 2018
• Source: Radboud University
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The purpose of this study is to determine whether long-acting octreotide is safe and effective in the treatment of patients with Rendu-Osler-Weber (e.g. HHT).

The study hypothesis is that octreotide is safe and will reduce transfusion requirements and endoscopy frequency in ROW patients with refractory anaemia due to bleeding gastrointestinal telangiectasias.

Description:

Rationale: Rendu-Osler-Weber (ROW) is an autosomal dominant hereditary disease which affects 1 / 5-8000 individuals. It is characterized by arteriovenous malformations (AVMs) and telangiectasias in multiple organs, including the gastrointestinal tract. Patients can be transfusion dependent due to severe gastrointestinal bleeding from those telangiectasias. Endoscopy is not as effective due to the recurrent character of the telangiectasias. Based on literature in patients with non-ROW AVMs and telangiectasias, octreotide might be beneficial for these patients to decrease their transfusion needs.

Objective: To assess the efficacy of octreotide in decreasing the need for transfusions and endoscopic intervention in patients ROW with refractory anaemia due to gastrointestinal bleeding telangiectasias.

Study design: Multicenter, open-label uncontrolled pilot study.

Study population: Patients with ROW and symptomatic gastrointestinal bleeding telangiectasias, who are transfusion and/or endoscopy dependent:

1. Transfusion dependent: at least 2 blood and/or iron infusions in the 6 months before inclusion.

2. Endoscopy dependent: at least one endoscopic intervention with argon plasma coagulation (APC) after the initial/first endoscopic treatment after diagnosis in the half year before inclusion or unsuitable for endoscopy.

Intervention: The intervention is 20 mg Sandostatin long-acting release (LAR) once every four weeks for 26 weeks on top of standard of care.

Main study parameters/endpoints: Primary outcome is response to treatment defined as:

• complete: no endoscopic intervention or transfusion requirements

• partial: a reduction in endoscopic intervention or transfusion requirements

• non-response: an equal or increase in endoscopy frequency or transfusions Important secondary outcomes are the percent change in the number of rebleeds from baseline to endpoint and the number of epistaxis episodes.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 15
• Est. completion date: October 2018
• Est. primary completion date: July 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• Patients with Rendu-Osler-Weber

• Symptomatic gastrointestinal bleeds out of telangiectasias

• Transfusion and / or endoscopy dependent:

Transfusion: at least 2 blood and/or iron infusions in the 6 months before inclusion.

Endoscopy: at least one endoscopy with APC after the initial endoscopic treatment after diagnosis in the half year before inclusion or unsuitable for endoscopic therapy.

Exclusion Criteria:

• liver cirrhosis Child-Pugh C or acute liver failure

• previous unsuccessful treatment with somatostatin analogues (SST) for the same indication (refractory anaemia due to telangiectasias) or current effective treatment with a somatostatin analogue

• severe diseases with life expectancy < 1 year

• patients with left ventricular assist devices (LVAD's)

• Symptomatic cholecystolithiasis (without cholecystectomy)

• pregnancy or nursing women or women who have a pregnancy wish in the study period or who use anticonception inadequate

• current chemotherapy

• patients with a known hypersensitivity to SST analogues or any component of the octreotide LAR formulations

• no understanding of Dutch or English

Related Conditions & MeSH terms

• Anemia
• Gastrointestinal Hemorrhage
• Hemorrhage
• Hereditary Hemorrhagic Telangiectasia
• Telangiectasia, Hereditary Hemorrhagic
• Telangiectasis

Intervention

Drug:
• Octreotide LAR

Locations

Country	Name	City	State
Netherlands	St Antonius Hospital	Nieuwegein	Utrecht
Netherlands	Radboudumc	Nijmegen	Gelderland

Sponsors (2)

Lead Sponsor	Collaborator
Radboud University	St. Antonius Hospital

Country where clinical trial is conducted

Netherlands, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	The percentage of patients who are full responder, partial responder and non-responder at the end of the treatment period	Full responder: no endoscopy and no blood/iron transfusions during treatment period.; Partial responder: a decrease in number of blood/iron transfusions and/or endoscopy during the treatment period compared with the 6 months prior to inclusion.; Non-responder: no decrease in number of blood/iron transfusions and endoscopy during the treatment period compared with the 6 months prior to inclusion.	Comparing the 6 months before inclusion and the study period (26 weeks)
Secondary	The percentual decrease in blood and iron requirements		Comparing the 6 months prior to inclusion and the treatment period of 6 months.
Secondary	The percentual decrease in the number of endoscopic interventions		Comparing the 6 months prior to inclusion and the treatment period of 6 months.
Secondary	The mean/median decrease on the epistaxis severity score (ESS)	Comparing baseline and the end of treatment visit (week 26)	Comparing the 6 months prior to inclusion and the treatment period of 6 months.
Secondary	Change in quality of life using the Short Form (SF)-36 questionnaire		Comparing baseline and end of treatment visit
Secondary	The number, type and severity of adverse events		Study period