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Trial · Interventional

CompletedPhase 2Results postedRegistry data

Pasireotide in Prevention of GI Toxicity

Overview

Status
Completed
Phase
Phase 2
Enrollment
37 participants (actual)
Sponsor
Anthony Sung, MD · Other
Design
Allocation not applicable · Open label (no masking)
Countries
1 country

United States

Start
21 Jan 2015
Primary completion
28 Feb 2019
Completion
15 Oct 2019
First posted
13 Aug 2014 (estimated)
Last update posted
17 Nov 2021
Conditions
Hematological Malignancies

Timeline

1 trial · 1 completed · 1 with posted results

  • Completed
  • Ongoing
  • Not yet recruiting
  • Stopped
  • Estimated dates (est.)
  • Results posted
201520162017201820192020
  1. Ph 2

    Pasireotide in Prevention of GI Toxicity

    NCT02215070

    21 Jan 2015 to 15 Oct 2019

Registry summary

Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.

The purpose of this study is to evaluate if the drug, Pasireotide, is safe and effective in reducing the gastrointestinal side effects of the drugs received to prepare for allogeneic stem cell transplant. The study will also evaluate if Pasireotide is effective in reducing acute and chronic Graft-versus-Host-Disease (GvHD) after transplant.

Interventions

  • PasireotideDrug

Arms1

  • Experimental

    Pasireotide + Preparatory Regimen

    Eligible subjects will receive pasireotide daily for 5 days before stem cell transplant, the day of the stem cell transplant, and daily for 8 days following the stem cell transplant. Preparatory regimen will be given 4 days before stem cell transplant.

Primary outcomes1

Outcomes the sponsor listed as primary.

  1. Percentage of GI Toxicity From the Preparatory Regimen and the GVHD Prophylaxis in Stem Cell Transplantation (SCT) Patients Who Are Treated With Pasireotide

    Time frame · 30 Days

    Registry description

    Number of participants who experience grades III-IV GI toxicity

Secondary outcomes(6)
  1. Percentage of Acute GVHD

    Time frame · 100 days

    Registry description

    Number of participants who experience acute GVHD

  2. Maximum Severity of Acute GVHD Compared to Historical Controls

    Time frame · 100 days

    Registry description

    Assess maximum severity of acute GVHD scored as stage 1 (least severe) through stage 4 (most severe) using BMT CTN, 2013 standards

  3. Incidence of Chronic GVHD Compared to Historical Controls

    Time frame · 1 year

    Registry description

    Measure the number of participants who experience chronic GVHD

  4. Maximum Severity of Chronic GVHD Compared to Historical Controls

    Time frame · 1 year

    Registry description

    Assess maximum severity of chronic GVHD scored as none, mild, moderate or severe using 2014 NIH Consensus Criteria

  5. Overall Survival Compared to Historical Controls

    Time frame · 1 year

    Registry description

    Rate of overall survival of participants at one year post transplant

  6. Disease Free Survival Compared to Historical Controls

    Time frame · 1 year

    Registry description

    Rate of disease free survival of participants at one year post transplant

Posted results

Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.

Primary outcome results

Primary outcomeCount of participants· 30 Days

Percentage of GI Toxicity From the Preparatory Regimen and the GVHD Prophylaxis in Stem Cell Transplantation (SCT) Patients Who Are Treated With Pasireotide

GroupValue (Participants)
Pasireotide + Preparatory Regimen21
Secondary and other outcome results(7)
Secondary outcomeCount of participants· 100 days

Percentage of Acute GVHD

GroupValue (Participants)
Pasireotide + Preparatory Regimen15
Secondary outcomeMedian· 100 days

Maximum Severity of Acute GVHD Compared to Historical Controls

GroupValue (units on a scale)
Pasireotide + Preparatory Regimen2
Secondary outcomeCount of participants· 1 year

Incidence of Chronic GVHD Compared to Historical Controls

GroupValue (Participants)
Pasireotide + Preparatory Regimen16
Historical Controls22
Secondary outcomeCount of participants· 1 year

Maximum Severity of Chronic GVHD Compared to Historical Controls

GroupValue (Participants)
Pasireotide + Preparatory Regimen8
Historical Controls11
Secondary outcomeNumber· 1 year

Overall Survival Compared to Historical Controls

GroupValue (percentage of participants)
Pasireotide + Preparatory Regimen63
Historical Controls82
Secondary outcomeNumber· 1 year

Disease Free Survival Compared to Historical Controls

GroupValue (percentage of participants)
Pasireotide + Preparatory Regimen40
Historical Controls78
Other pre specified outcome· 100 days

Citrulline and Fecal Calprotectin Levels Will be Measured

GroupValue

Adverse events, as posted

Adverse events as posted: participants affected out of participants at risk, by group
GroupDeathsSerious eventsOther events
Pasireotide + Preparatory RegimenEligible subjects will receive pasireotide daily for 5 days before stem cell transplant, the day of the stem cell transplant, and daily for 8 days following the stem cell transplant. Preparatory regimen will be given 4 days before stem cell transplant.1 / 268 / 2626 / 26
Historical ControlsContemporaneous controls were matched using the following parameters: Transplant diagnosis (acute leukemias, lymphomas, myelodysplastic syndrome \[MDS\]/myeloproliferative neoplasm \[MPN\]/other), donor (related, unrelated), graft (bone marrow, peripheral blood progenitor cell \[PBPC\], and cord), GVHD prophylaxis (tacrolimus/methotrexate), age, hematopoietic cell transplantation-specific comorbidity index (HCT-CI), and conditioning regimen (TBI-based, chemotherapy only).0 / 00 / 00 / 0

Each cell reads participants affected / participants at risk, as the registry posted them.

Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works

Studies0

Studies that cite this trial.

No study cites this trial.

Eligibility

Who could join, as posted.

Sex
All
Age
from 18 Years
Healthy volunteers
Not accepted
Inclusion and exclusion criteria, as posted
Inclusion Criteria: * 18 years of age or older at the time of study enrollment. * Histologically confirmed diagnosis for which an allogeneic transplant is utilized. * Plan to receive an allogeneic transplant from a 4-6/6 single or dual umbilical cord blood graft, or a 7-8/8 HLA-matched sibling or unrelated donor (High resolution HLA-A, B, C, DRB1). * Meet standard criteria as defined by the institution for a myeloablative allogeneic stem cell transplantation, with myeloablative defined as using conditioning regimens containing: * TBI ≥ 1200 cGy, or * Busulfan ≥ 12.8mg/kg * Patient must have given written informed consent according to FDA guidelines. * Willingness and ability to comply with scheduled visits, treatment plans, laboratory tests and other study procedures. Exclusion Criteria: * Female patients who are pregnant or lactating, or are of childbearing potential (FCBP, defined as all women physiologically capable of becoming pregnant) and not practicing an effective method of contraception/birth control * FCBP must have a current negative serum pregnancy test prior to transplant per institutional practice. * Use of an investigational drug within 1 month prior to dosing. Concurrent enrollment on other clinical research studies that contain an interventional therapy is not permitted while subjects are receiving pasireotide or within 5 half-lives of finishing pasireotide. However, subjects may concurrently enroll in non-interventional studies (e.g. biobanking, mobile health tracking). * Active CNS disease (related to primary malignancy) at the time of enrollment. * Patients with existing grade 2 toxicities, except as approved by the investigator. * Any of the following diseases or conditions: Cardiac: * History of unexplained syncope or family history of idiopathic sudden death. * Sustained or clinically significant cardiac arrhythmias. * Risk factors for Torsades de Pointes such as: * Uncontrolled hypokalemia * Uncontrolled hypomagnesemia or hypermagnesemia * Cardiac failure (New York Heart Association Class II or higher) * Clinically significant/symptomatic bradycardia (HR \< 50), or high-grade AV block. * Known diagnosis of QT prolongation (QTc ≥ 470) or family history of long QT syndrome * Concomitant disease(s) that could prolong QT such as autonomic neuropathy (caused by diabetes, or Parkinson's disease), HIV, cirrhosis, uncontrolled hypothyroidism or cardiac failure. * Concomitant medications known to prolong the QT interval during the same time as pasireotide is to be administered (unless approved by PI and QTc \< 470; standard transplant medications that are known to prolong the QT (e.g. azoles, ondansetron, etc.) are permitted but caution is advised and patients should be closely monitored). Endocrine: * Uncontrolled diabetes at the time of cytoreduction. All patients with diabetes must be optimized on their diabetes regimen prior to initiating pasireotide. • If a patient is diabetic: uncontrolled diabetes as defined by HbA1c \> 8 per cent despite adequate therapy * Patients who are not biochemically euthyroid. Patients with known history of hypothyroidism are eligible if they are on adequate and stable replacement thyroid hormone therapy for at least 3 months. * Known diagnosis of hypocortisolism * Known diagnosis of pituitary hormone deficiency. * Known hypersensitivity to somatostatin analogs or any component of the pasireotide LAR or s.c. formulations. Infectious: * Uncontrolled (not being treated) infections at the time of cytoreduction. * A positive HIV test result (ELISA and Western blot) or history of known HIV. An HIV test will not be required; however, previous medical history will be reviewed. Gastrointestinal: * Moderately impaired hepatic function (Child-Pugh B) or severe hepatic impairment (Child-Pugh C) * Known gallbladder or bile duct disease, symptomatic cholelithiasis, acute or chronic pancreatitis. * Known malabsorption syndrome, short bowel or chologenic diarrhea not controlled by specific therapeutic means. Hematologic: * Abnormal coagulation (PT or aPTT \> 30% above normal limits). * Continuous anticoagulation therapy. Patients who were on anticoagulant therapy must complete a washout period of at least 10 days and have confirmed normal coagulation parameters before study inclusion. Miscellaneous: * Major surgery/surgical therapy for any cause within 1 month prior to pasireotide administration. Patients should have recovered and have a good clinical condition before entering the study. * Any co-morbid condition which, in the view of the Principal Investigator, renders the patient at high risk from treatment complications. Patients with a history of non-compliance to medical regimens or who are considered potentially unreliable or will not be able to complete the entire study.

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