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Octreotide in Patients With GI Bleeding Due to Rendu-Osler-Weber (ROW)

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ClinicalTrials.gov Identifier: NCT02874326
Recruitment Status : Unknown
Verified April 2018 by Radboud University Medical Center.
Recruitment status was:  Active, not recruiting
First Posted : August 22, 2016
Last Update Posted : April 20, 2018
Sponsor:
Collaborator:
St. Antonius Hospital
Information provided by (Responsible Party):
Radboud University Medical Center

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.


Condition or disease Intervention/treatment Phase
Hereditary Hemorrhagic Telangiectasia Gastrointestinal Hemorrhage Anemia Drug: Octreotide LAR Phase 2

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:

  1. Transfusion dependent: at least 2 blood and/or iron infusions in the 6 months before inclusion.
  2. 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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 15 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : October 2016
Estimated Primary Completion Date : July 2018
Estimated Study Completion Date : October 2018


Arm Intervention/treatment
Experimental: Active comparator: Octreotide LAR
Sandostatin LAR Sandostatin LAR 20 mg will be administered once every 4 weeks as a intramuscular injection
Drug: Octreotide LAR
Other Names:
  • Sandostatin
  • Dutch registration number (RVG) 18236
  • Anatomical Therapeutic Chemical (ATC) H01CB02




Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. The percentual decrease in blood and iron requirements [ Time Frame: Comparing the 6 months prior to inclusion and the treatment period of 6 months. ]
  2. 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. ]
  3. 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. ]
    Comparing baseline and the end of treatment visit (week 26)

  4. Change in quality of life using the Short Form (SF)-36 questionnaire [ Time Frame: Comparing baseline and end of treatment visit ]
  5. The number, type and severity of adverse events [ Time Frame: Study period ]


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02874326


Locations
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Netherlands
Radboudumc
Nijmegen, Gelderland, Netherlands, 6525 GA
St Antonius Hospital
Nieuwegein, Utrecht, Netherlands, 3430 EM
Sponsors and Collaborators
Radboud University Medical Center
St. Antonius Hospital
Investigators
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Principal Investigator: Joost Drenth, MD PhD Radboud University Medical Center
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Responsible Party: Radboud University Medical Center
ClinicalTrials.gov Identifier: NCT02874326    
Other Study ID Numbers: NLROW.1012.15
First Posted: August 22, 2016    Key Record Dates
Last Update Posted: April 20, 2018
Last Verified: April 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Keywords provided by Radboud University Medical Center:
Sandostatin LAR
Somatostatin
Octreotide
Additional relevant MeSH terms:
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Gastrointestinal Hemorrhage
Telangiectasis
Telangiectasia, Hereditary Hemorrhagic
Hemorrhage
Pathologic Processes
Vascular Diseases
Cardiovascular Diseases
Hemostatic Disorders
Hemorrhagic Disorders
Hematologic Diseases
Vascular Malformations
Cardiovascular Abnormalities
Congenital Abnormalities
Gastrointestinal Diseases
Digestive System Diseases
Octreotide
Gastrointestinal Agents
Antineoplastic Agents, Hormonal
Antineoplastic Agents