To assess the efficacy of octreotide LAR in decreasing the need for transfusions and endoscopic intervention in patients ROW with refractory anaemia due to gastrointestinal bleeding telangiectasias.
ID
Source
Brief title
Condition
- Gastrointestinal haemorrhages NEC
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
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
Secondary outcome
Important secondary outcomes are the percent change in the number of rebleeds
from baseline to endpoint and the number of epistaxis episodes.
Background summary
Rendu-Osler-Weber (ROW) is an autosomal dominant hereditary disease which
affects 1 / 5-8000 individuals. It is characterized by ateriovenous
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 LAR
might be beneficial for these patients to decrease their transfusion needs.
Study objective
To assess the efficacy of octreotide LAR 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.
Intervention
The intervention is 20 mg octreotide LAR once every four weeks for 26 weeks on
top of standard of care.
Study burden and risks
The burden consist of extra visits and blood samples (3 times), and two times a
questionnaire. There is a risk for the known side-effects of octreotide LAR.
The potential benefit for participating patients is that Sandostatin may reduce
the need for transfusions and endoscopic intervention.
Geert Grooteplein-Zuid 10
Nijmegen 6500 HB
NL
Geert Grooteplein-Zuid 10
Nijmegen 6500 HB
NL
Listed location countries
Age
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 endoscopy.
Exclusion criteria
- liver cirrhosis Child-Pugh C or acute liver failure
- Symptomatic cholecystolithiasis (without cholecystectomie)
- previous unsuccessful treatment with somatostatin analogues for the same indication (refractory anaemia due to telangiectasias) or current effective treatment with a somatostatin analogue
- current successful treatment with thalidomide
- severe diseases with life expectancy < 1 year
- patients with left ventricular assist devices (LVAD*s)
- pregnancy or nursing women or women who have a pregnancy wish in the studyperiod or who use anticonception inadequate
- current chemotherapy
- patients with a known hypersensitivity to SST analogues or any component of the pasireotide LAR formulations
- systemic cancer currently undergoing chemotherapy or radiation therapy
- no understanding of Dutch or English
Design
Recruitment
Medical products/devices used
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| EudraCT | EUCTR2016-001340-19-NL |
| CCMO | NL57309.100.16 |