To determine if SSA are effective in decreasing transfusion requirements and improving quality of life while being cost-effective.
ID
Source
Brief title
Condition
- Gastrointestinal vascular conditions
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
'Successful response', defined as a decrease of >=50% in the amount of units
intravenous iron and/or blood transfusions given.
Secondary outcome
The percentual or mean/median difference between the half year prior to
inclusion and the treatment period of a half year between the treatment and
observational arm in:
- blood and intravenous iron requirements
- PROM*s: quality of life (SF-36, EQ-5D), level of fatigue (MFI-20), epistaxis
severity (ESS tool), and patient satisfaction
- hemoglobin and ferritin levels
- number of endoscopic treatments
- cost-effectiveness
- Safety
Background summary
Rendu-Osler-Weber (ROW or HHT) 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 might
be beneficial for these patients to decrease their transfusion needs.
Study objective
To determine if SSA are effective in decreasing transfusion requirements and
improving quality of life while being cost-effective.
Study design
International, multicenter, open label RCT with a parallell observational
control group. (Phase III)
Intervention
Self-administerend subcutaneous injection with 0.1 mg short-acting Octreotide
twice daily for 26 weeks.
Study burden and risks
Self-administered subcutaneous Octreotide-injection. The risks are the known
side effects of subcutaneous Octreotide treatment.
Furthermore, the participants has to visit the hospital 4 times for check-ups
with bloodsamples.
Geert Grooteplein Zuid 10
Nijmegen 6500 HB
NL
Geert Grooteplein Zuid 10
Nijmegen 6500 HB
NL
Listed location countries
Age
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.
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 | EUCTR2018-004179-11-NL |
| CCMO | NL68092.091.18 |
| Other | NTR nummer volgt |