Effectiveness of somatostatin analogues for GI bleeding in patients with hereditary hemorrhagic telangiectasia

• Register: EUCTR
• Last refreshed on: 24 March 2025
• Main ID: EUCTR2018-004179-11-IT
• Date of registration: 17/08/2021
• Prospective Registration: Yes
• Primary sponsor: RADBOUD UNIVERSITY MEDICAL CENTER
• Public title: Effectiveness of somatostatin analogues for GI bleeding in patients with hereditary hemorrhagic telangiectasia
• Scientific title: Effectiveness of Somatostatin Analogues in Patients with hereditary hemorrhagic telangiectasia and symptomatic gastrointestinal bleeding, SAIPAN-trial: a multicentre, randomized, open-label, parallel-group, superiority trial. - SAIPAN study
• Date of first enrolment: 03/09/2021
• Target sample size: 38
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2018-004179-11
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: yes Single blind: no Double blind: no Parallel group: yes Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: yes Other specify the comparator: standard of care Number of treatment arms in the trial: 2
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

Denmark, France, Germany, Italy, Netherlands, United Kingdom

Contacts

• Name: Servizio Informazione sperimentazio
• Affiliation: Radboudumc

• Name: Servizio Informazione sperimentazio
• Affiliation: Radboudumc

Key inclusion & exclusion criteria

Inclusion criteria:
· Patients older than 18 years with written informed consent.
· Diagnosis of HHT: either confirmed by genetic testing or the Curaçao criteria
(definite diagnosis)
· Presence of 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, or 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:
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 Haemoglobin below 5.6 mmol/l (9.0 g/dl) or are in need of transfusions due to anaemia
related symptoms within the last 6 months requiring blood transfusion above.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 20
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 20

Exclusion criteria:
· Liver cirrhosis Child-Pugh C,
· Insuloma,
· Uncontrolled diabetes mellitus as defined by HbA1c >64 mmol/ml, despite adequate
therapy
· Juvenile Polyposis Syndrome
· Symptomatic cholecystolithiasis (possible side-effect octreotide)


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Hereditary hemorrhagic telangiectasia (HHT), also known as Osler–Weber–Rendu disease (in specific patients with gastrointestinal bleeding and transfusion dependency)
MedDRA version: 21.0 Level: PT Classification code 10019883 Term: Hereditary haemorrhagic telangiectasia System Organ Class: 10010331 - Congenital, familial and genetic disorders

Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Intervention(s)

Product Name: octreotide a azione pronta 0,1 mg
Product Code: [OCTREOTIDE ACETATO]
Pharmaceutical Form: Concentrate for solution for injection/infusion
INN or Proposed INN: OCTREOTIDE ACETATO
CAS Number: 83150-76-9
Current Sponsor code: OPR00000697
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 1-

Primary Outcome(s)

Primary end point(s): ‘Successful response’, defined as a decrease of =50% in the amount of units
intravenous iron and/or blood transfusions given between the number of transfusions at
baseline (26 weeks prior to study inclusion) and during the treatment study period (26
weeks).
NB Patients included based on intravenous iron requirements and needing bloodtransfusion
during the study period are treatment failures.

Timepoint(s) of evaluation of this end point: 26 weeks prior to study inclusion and during the treatment study period (26 weeks)

Main Objective: To investigate the effectiveness of somatostatin analogues in decreasing the
transfusion requirements in patients with HHT and GI bleeding who are refractory to
endoscopic therapy.

Secondary Objective: To investigate the effectiveness of octreotide on: decreasing the endoscopic treatment
frequency, increasing the health-related quality of life, decreasing fatigue and
epistaxis symptoms, and to assess the effects of long-term octreotide on adverse
events and cost- effectiveness.

Secondary Outcome(s)

Secondary end point(s): 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
- number of endoscopic treatments
- cost-effectiveness (see economic evaluation)
The percentual or mean/median difference between the value at baseline and after a
half year between the treatment and observational arm in:
- PROMs: quality of life (SF-36), level of fatigue (multidimensional fatigue inventory
(MFI)-20), epistaxis severity (ESS tool), and patient satisfaction
The percentual or mean/median difference after a half year between the treatment and
observational arm in:
- (S)AE.
The percentual or mean/median difference between the value at baseline, after 4 weeks,
12 weeks, and after a half year between the treatment and observational arm in:
- hemoglobin and ferritin levels

Timepoint(s) of evaluation of this end point: 0 weeks, 4 weeks, 12 weeks, 26 weeks, 30 weeks (follow up)

Secondary ID(s)

2018-4898

2018-004179-11-NL

Source(s) of Monetary Support

ZonMw

Ethics review

Status: Approved
Approval date: 21/06/2021
Contact: