Proof-of-concept Trial of Apraglutide in Acute Graft Versus Host Disease (aGVHD)

NCT ID: NCT05415410

Last Updated: 2025-10-20

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE2

Total Enrollment

31 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-05-25

Study Completion Date

2024-12-17

Brief Summary

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The aim of this trial is to assess safety and efficacy of apraglutide in subjects with steroid refractory gastrointestinal aGVHD.

Detailed Description

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This is an international, multicenter, randomized proof-of-concept trial to evaluate safety, tolerability, efficacy, durability of response, and clinical outcomes of apraglutide administration to subjects with steroid-refractory (SR) aGVHD of the lower GI tract being treated with systemic steroids (SS) and ruxolitinib (RUX).

Conditions

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GVHD

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

SINGLE

Participants

Study Groups

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Apraglutide Low Dose

Low-dose, weight-based apraglutide subcutaneous (SC) injections (1.4 to 3.5 mg) once weekly for up to 13 weeks (with optional treatment up to an additional 13 weeks), for participants with body weight of more than 50.0 kg.

Group Type EXPERIMENTAL

Apraglutide

Intervention Type DRUG

Apraglutide is a new, synthetic glucagon-like peptide 2 (GLP-2) receptor agonist which acts as a gut targeted regenerative approach that is intestinotrophic with a mode of action that improves absorption and enhances gut barrier function.

Apraglutide High Dose

High-dose, weight-based apraglutide SC injections (3.5 to 7.6 mg) once weekly for up to 13 weeks (with optional treatment up to an additional 13 weeks), for participants with body weight of more than 50.0 kg.

Group Type EXPERIMENTAL

Apraglutide

Intervention Type DRUG

Apraglutide is a new, synthetic glucagon-like peptide 2 (GLP-2) receptor agonist which acts as a gut targeted regenerative approach that is intestinotrophic with a mode of action that improves absorption and enhances gut barrier function.

Apraglutide Standard Dose

Standard-dose apraglutide SC injections (1.4 mg) once weekly for up to 13 weeks (with optional treatment up to an additional 13 weeks), for participants with body weight between 40.0 kg to 49.9 kg.

Group Type EXPERIMENTAL

Apraglutide

Intervention Type DRUG

Apraglutide is a new, synthetic glucagon-like peptide 2 (GLP-2) receptor agonist which acts as a gut targeted regenerative approach that is intestinotrophic with a mode of action that improves absorption and enhances gut barrier function.

Interventions

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Apraglutide

Apraglutide is a new, synthetic glucagon-like peptide 2 (GLP-2) receptor agonist which acts as a gut targeted regenerative approach that is intestinotrophic with a mode of action that improves absorption and enhances gut barrier function.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Able to give informed consent and agree to follow the details of participation as outlined in the protocol
* Male or female subjects aged 12 years or above at the time of consent and who weigh a minimum of 40 kg. Only subjects aged 18 years and above will be included in Germany.
* Clinically confirmed steroid refractory lower GI-aGVHD (MAGIC stage 1-4) prior to randomization
* Have undergone alloSCT from any donor source, any conditioning regimen
* Treated with SS plus RUX (RUX starts concomitantly to apraglutide or a maximum of 72 hours before apraglutide initiation)
* Women of childbearing potential (WOCBP): highly effective method of contraception and refrain from donating eggs during the trial and for 4 weeks after the End of Trial (EOT) visit
* Male subjects with partner WOCBP: contraception and abstention from sperm donation during the trial and for 2 weeks after the EOT visit

Exclusion Criteria

* Treatment with any systemic GVHD therapy other than SS and RUX including methotrexate and mycophenolate mofetil at the time of randomization / Day 0
* Concomitant treatment with Janus kinase inhibitor other than RUX at the time of randomization
* Failed alloSCT due to relapse of underlying malignant disease
* Presence of SR GI-aGVHD occurring after donor lymphocyte infusion for pre-emptive treatment of malignancy recurrence
* Any use of enteral glutamine or GLP analogs or known ADA, within 6 months prior to randomization / Day 0
* Significant organ system failures (respiratory renal hepatic and cardiac)
* Presence of relapsed primary malignancy or treatment for relapse after alloHSCT
* Presence or history of GI tumors (including the hepatobiliary system and pancreas) within the last five years before randomization
* Presence of colonic polyps not removed
* Active clinically uncontrolled infection or active tuberculosis
* Known chronic GVHD
* Known active GI inflammation not related to GI-aGVHD
* Major abdominal surgery in the last 6-months prior to randomization or history of clinically significant intestinal adhesions
* Abnormal liver function tests
Minimum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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VectivBio AG

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Tomasz Masior

Role: STUDY_DIRECTOR

VectivBio AG

Locations

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Stanford Cancer Center

Stanford, California, United States

Site Status

University of Iowa

Iowa City, Iowa, United States

Site Status

Massachusetts General Hospital

Boston, Massachusetts, United States

Site Status

The Ohio State University

Columbus, Ohio, United States

Site Status

South Austin Medical Center

Austin, Texas, United States

Site Status

Universitätsklinikum Köln (AoeR)

Cologne, , Germany

Site Status

Universitaetsklinikum Duesseldorf

Düsseldorf, , Germany

Site Status

Universitätsklinikum Freiburg

Freiburg im Breisgau, , Germany

Site Status

Martin Luther Universität Halle-Wittenberg

Halle, , Germany

Site Status

Universitätsklinikum Hamburg-Eppendorf

Hamburg, , Germany

Site Status

Universitätsmedizin der Johannes Gutenberg - Universität Mainz

Mainz, , Germany

Site Status

Instituto Portugues de Oncologia do Porto Francisco Gentil

Porto, , Portugal

Site Status

Hospital Universitario Virgen del Rocio

Seville, , Spain

Site Status

Countries

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United States Germany Portugal Spain

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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TA799-101

Identifier Type: -

Identifier Source: org_study_id

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