Octreotide in Patients With GI Bleeding Due to Rendu-Osler-Weber (ROW)
Primary Purpose
Hereditary Hemorrhagic Telangiectasia, Gastrointestinal Hemorrhage, Anemia
Status
Unknown status
Phase
Phase 2
Locations
Netherlands
Study Type
Interventional
Intervention
Octreotide LAR
Sponsored by
About this trial
This is an interventional treatment trial for Hereditary Hemorrhagic Telangiectasia focused on measuring Sandostatin LAR, Somatostatin, Octreotide
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
Sites / Locations
- Radboudumc
- St Antonius Hospital
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Active comparator: Octreotide LAR
Arm Description
Sandostatin LAR Sandostatin LAR 20 mg will be administered once every 4 weeks as a intramuscular injection
Outcomes
Primary Outcome Measures
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.
Secondary Outcome Measures
The percentual decrease in blood and iron requirements
The percentual decrease in the number of endoscopic interventions
The mean/median decrease on the epistaxis severity score (ESS)
Comparing baseline and the end of treatment visit (week 26)
Change in quality of life using the Short Form (SF)-36 questionnaire
The number, type and severity of adverse events
Full Information
NCT ID
NCT02874326
First Posted
August 17, 2016
Last Updated
April 18, 2018
Sponsor
Radboud University Medical Center
Collaborators
St. Antonius Hospital
1. Study Identification
Unique Protocol Identification Number
NCT02874326
Brief Title
Octreotide in Patients With GI Bleeding Due to Rendu-Osler-Weber
Acronym
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
Study Type
Interventional
2. Study Status
Record Verification Date
April 2018
Overall Recruitment Status
Unknown status
Study Start Date
October 2016 (Actual)
Primary Completion Date
July 2018 (Anticipated)
Study Completion Date
October 2018 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Radboud University Medical Center
Collaborators
St. Antonius Hospital
4. Oversight
Data Monitoring Committee
No
5. Study Description
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:
Transfusion dependent: at least 2 blood and/or iron infusions in the 6 months before inclusion.
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hereditary Hemorrhagic Telangiectasia, Gastrointestinal Hemorrhage, Anemia
Keywords
Sandostatin LAR, Somatostatin, Octreotide
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
15 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Active comparator: Octreotide LAR
Arm Type
Experimental
Arm Description
Sandostatin LAR Sandostatin LAR 20 mg will be administered once every 4 weeks as a intramuscular injection
Intervention Type
Drug
Intervention Name(s)
Octreotide LAR
Other Intervention Name(s)
Sandostatin, Dutch registration number (RVG) 18236, Anatomical Therapeutic Chemical (ATC) H01CB02
Primary Outcome Measure Information:
Title
The percentage of patients who are full responder, partial responder and non-responder at the end of the treatment period
Description
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.
Time Frame
Comparing the 6 months before inclusion and the study period (26 weeks)
Secondary Outcome Measure Information:
Title
The percentual decrease in blood and iron requirements
Time Frame
Comparing the 6 months prior to inclusion and the treatment period of 6 months.
Title
The percentual decrease in the number of endoscopic interventions
Time Frame
Comparing the 6 months prior to inclusion and the treatment period of 6 months.
Title
The mean/median decrease on the epistaxis severity score (ESS)
Description
Comparing baseline and the end of treatment visit (week 26)
Time Frame
Comparing the 6 months prior to inclusion and the treatment period of 6 months.
Title
Change in quality of life using the Short Form (SF)-36 questionnaire
Time Frame
Comparing baseline and end of treatment visit
Title
The number, type and severity of adverse events
Time Frame
Study period
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Joost Drenth, MD PhD
Organizational Affiliation
Radboud University Medical Center
Official's Role
Principal Investigator
Facility Information:
Facility Name
Radboudumc
City
Nijmegen
State/Province
Gelderland
ZIP/Postal Code
6525 GA
Country
Netherlands
Facility Name
St Antonius Hospital
City
Nieuwegein
State/Province
Utrecht
ZIP/Postal Code
3430 EM
Country
Netherlands
12. IPD Sharing Statement
Plan to Share IPD
No
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Octreotide in Patients With GI Bleeding Due to Rendu-Osler-Weber
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