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

NCT ID: NCT02874326

Last Updated: 2018-04-20

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: PHASE2
• Total Enrollment: 15 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2016-10-31
• Study Completion Date: 2018-10-31

Brief 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.

Detailed 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.

Conditions

Hereditary Hemorrhagic Telangiectasia; Gastrointestinal Hemorrhage; Anemia

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Active comparator: Octreotide LAR

Sandostatin LAR Sandostatin LAR 20 mg will be administered once every 4 weeks as a intramuscular injection

Group Type: EXPERIMENTAL

Octreotide LAR

Intervention Type: DRUG

Interventions

Octreotide LAR

Intervention Type: DRUG

Other Intervention Names

Sandostatin; Dutch registration number (RVG) 18236; Anatomical Therapeutic Chemical (ATC) H01CB02

Eligibility Criteria

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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

St. Antonius Hospital

OTHER

Sponsor Role: collaborator

Radboud University Medical Center

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Joost Drenth, MD PhD

Role: PRINCIPAL_INVESTIGATOR

Radboud University Medical Center

Locations

Radboudumc

Nijmegen, Gelderland, Netherlands

St Antonius Hospital

Nieuwegein, Utrecht, Netherlands

Countries

Netherlands

Other Identifiers

NLROW.1012.15

Identifier Type: -

Identifier Source: org_study_id