Pasireotide in Prevention of GI Toxicity

What Was Tested

The trial (phase 2) is built around Pasireotide.

It had 1 arm (groups): Pasireotide + Preparatory Regimen (experimental).

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.

The registry classes the interventions as drug.

More on the Plan

The study design will be a non-randomized phase II.

The researchers select matched controls from existing patients who did not take the drug to minimize the time it takes to complete the trial.

The most common regimens combine TBI with cyclophosphamide (TBI/Cy) or busulfan with cyclophosphamide (Bu/Cy) (Appendix E).

However, any regimen meeting the above definition of myeloablative preparatory regimen may be used.

Who Took Part

Enrollment: 37 actual participants took part.

It was open to people of any sex aged from 18 years with no upper age limit.

Healthy volunteers were not accepted; participants needed the condition being studied.

Examples of who could join: 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.

The full record lists 8 inclusion and 30 exclusion criteria; among the exclusions: FCBP must have a current negative serum pregnancy test prior to transplant per institutional practice; active cns disease (related to primary malignancy) at the time of enrollment; patients with existing grade 2 toxicities, except as approved by the investigator.

How the Study Was Run

Design: single-group and open-label.

Everyone knew when Pasireotide was being given (open label), which is simpler to run but leaves more room for expectations to affect what is reported.

Everyone received the same intervention, so there was no separate comparison group inside the trial.

It started in 2015-01-21 with completion listed for 2019-02-28.

It ran at 2 sites in United States (2).

Its stated primary purpose is treatment.

What Was Measured

Researchers chose this as the main measure: 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).

It also tracked 6 secondary outcomes, for example Percentage of Acute GVHD, Maximum Severity of Acute GVHD Compared to Historical Controls, Incidence of Chronic GVHD Compared to Historical Controls, Maximum Severity of Chronic GVHD Compared to Historical Controls, Overall Survival Compared to Historical Controls and Disease Free Survival Compared to Historical Controls.

What This Means in Plain Language

Put simply, this is a phase 2 trial of Pasireotide in 37 people.

Its size is small, so on its own it can only give an early look.

The trial is marked completed; results are posted on the registry, and this page does not restate them.

More context: the Pasireotide compound page, all human trials.