Hereditary Hemorrhagic TelangiectasiaPhase 2ROWNCT02874326
Octreotide in Patients With GI Bleeding Due to Rendu-Osler-Weber
A Phase 2 study testing Octreotide LAR in people with Hereditary Hemorrhagic Telangiectasia and Gastrointestinal Hemorrhage, run by Radboud University Medical Center at 2 sites in Netherlands.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hereditary Hemorrhagic Telangiectasia, Gastrointestinal Hemorrhage, Anemia
- What is being tested?
- Octreotide LAR
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 2 sitesNetherlands
- Study dates
- Oct 2016to Oct 2018
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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
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- current chemotherapy
- patients with a known hypersensitivity to SST analogues or any component of the octreotide LAR formulations
- no understanding of Dutch or English
Trial locations and contacts
2 sites in 1 country.
Netherlands2 sites
St Antonius Hospital
Nieuwegein, Utrecht, 3430 EM
Radboudumc
Nijmegen, Gelderland, 6525 GA
About this study
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
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.
Read the 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.
- Being tested
- Octreotide LAR
- Keywords
- Sandostatin LAR, Somatostatin, Octreotide
- Medical subject headings
- Telangiectasia, Hereditary Hemorrhagic, Gastrointestinal Hemorrhage, Anemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Octreotide LARDrug
Also called Sandostatin, Dutch registration number (RVG) 18236, Anatomical Therapeutic Chemical (ATC) H01CB02
| Study group | Role | What they receive |
|---|---|---|
| Active comparator: Octreotide LAR | Experimental | Sandostatin LAR Sandostatin LAR 20 mg will be administered once every 4 weeks as a intramuscular injection Receives: Drug: Octreotide LAR |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Comparing the 6 months before inclusion and the study period (26 weeks)
Secondary outcomes (5)
The percentual decrease in blood and iron requirements
Measured: Comparing the 6 months prior to inclusion and the treatment period of 6 months.
The percentual decrease in the number of endoscopic interventions
Measured: Comparing the 6 months prior to inclusion and the treatment period of 6 months.
The mean/median decrease on the epistaxis severity score (ESS)
Comparing baseline and the end of treatment visit (week 26)
Measured: Comparing the 6 months prior to inclusion and the treatment period of 6 months.
Change in quality of life using the Short Form (SF)-36 questionnaire
Measured: Comparing baseline and end of treatment visit
The number, type and severity of adverse events
Measured: Study period
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Aug 2016 (estimated)
- Study startOct 2016
- Primary completionJul 2018 (estimated)
- Study completionOct 2018 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hereditary Hemorrhagic TelangiectasiaStudies of the same condition, open and under way.
- More trials run by Radboud University Medical CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Radboud University Medical Center
- Sponsor type
- University, hospital or other
- Collaborators
- St. Antonius Hospital
- Responsible party
- Sponsor
- Investigators
- Joost Drenth, MD PhD, Principal investigator, Radboud University Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02874326
- Sponsor's protocol ID
- NLROW.1012.15
- Acronym
- ROW
- Status last verified
- Apr 2018
- First submitted
- 17 Aug 2016
- Last updated
- 20 Apr 2018
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Apr 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT02874326
- Acronym / sponsor ID
- ROW / NLROW.1012.15
- Keywords
- Sandostatin LAR, Somatostatin, Octreotide
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 15 participants (estimated)
- Lead sponsor
- Radboud University Medical CenterUniversity, hospital or other
- Collaborators
- St. Antonius Hospital
- Study start
- Oct 2016 (actual)
- Primary completion
- Jul 2018 (estimated)
- Study completion
- Oct 2018 (estimated)
- First posted
- 22 Aug 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Active comparator: Octreotide LAR | Experimental | Drug: Octreotide LARSandostatin LAR Sandostatin LAR 20 mg will be administered once every 4 weeks as a intramuscular injection |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (9 listed)
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Octreotide LARAlso: Sandostatin, Dutch registration number (RVG) 18236, Anatomical Therapeutic Chemical (ATC) H01CB02 | Drug | Active comparator: Octreotide LAR |
Primary outcome
The percentage of patients who are full responder, partial responder and non-responder at the end of the treatment period
Time frame: Comparing the 6 months before inclusion and the study period (26 weeks)
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.
5 secondary outcomes
The percentual decrease in blood and iron requirements
Time frame: Comparing the 6 months prior to inclusion and the treatment period of 6 months.
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.
The mean/median decrease on the epistaxis severity score (ESS)
Time frame: Comparing the 6 months prior to inclusion and the treatment period of 6 months.
Change in quality of life using the Short Form (SF)-36 questionnaire
Time frame: Comparing baseline and end of treatment visit
The number, type and severity of adverse events
Time frame: Study period
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Apr 2018.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Joost Drenth, MD PhD principal investigator
Radboud University Medical Center
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Octreotide LAR in people with Hereditary Hemorrhagic Telangiectasia and Gastrointestinal Hemorrhage, run by Radboud University Medical Center at 2 sites in Netherlands.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 20 Apr 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Netherlands. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The percentage of patients who are full responder, partial responder and non-responder at the end of the treatment period (time frame: Comparing the 6 months before inclusion and the study period (26 weeks)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Apr 2018.