Insulin-like Growth Factor 1 Receptor (IGF-1R) Antibody AMG479 (Ganitumab) in Combination With the Src Family Kinase (SFK) Inhibitor Dasatinib in People With Embryonal and Alveolar Rhabdomyosarcoma

What Was Tested

This registered phase 1, phase 2 trial tested IGF-1 LR3.

Background: Rhabdomyosarcoma (RMS) is the most common soft tissue sarcoma of childhood.

Two types are embryonal RMS (ERMS) and alveolar RMS (ARMS).

Dasatinib may block over-expression of a certain enzyme.

It had three arms (groups): one (experimental), one (experimental) and one (experimental).

The registry classes the interventions as drug.

More on the Plan

Background: * Rhabdomyosarcoma (RMS) is the most common soft tissue sarcoma of childhood.

The annual incidence in the United States is 4-7 cases per million children under 15 years, which represents 250 new cases per year.

Downstream signaling cascades of IGF-1R further regulate tumor cell proliferation, survival, and metastasis through the mitogen-activated protein kinase (MAPK)/extracellular signal-regulated kinase 1/2 (ERK) and phosphatidylinositol 3-kinases (PI3Ks)/mammalian target of rapamycin (mTOR) pathways.

A number of these have been tested in the clinical setting.

Who Took Part

Enrollment: 14 actual participants took part.

It was open to people of any sex aged from 2 years with no upper age limit.

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

Examples of who could join: Patients must have measurable disease. Patients must be able to undergo appropriate imaging studies to monitor tumor response. Archival tissue of tumors (slides or blocks (blocks preferred)) must be available for analysis. If tissue is not available, patients willing to undergo a pre-treatment biopsy may enroll. There is no maximum number of prior medical therapies. There must be no curative or life prolonging treatments available.

The full record lists 27 inclusion and 13 exclusion criteria; among the exclusions: patients who are receiving any other investigational agents; history of allergic reactions attributed to compounds of similar chemical or biologic composition to dasatinib or ganitumab or other agents used in study; patients who require concurrent treatment with antithrombotic and/or anti-platelet agents (e.g., warfarin, heparin, low molecular weight heparin, aspirin, and/or ibuprofen).

How the Study Was Run

The set-up was non-randomized, sequential and open-label.

Nothing was hidden in this open-label IGF-1 LR3 study: the participants and staff both knew the treatment.

It started in 2017-07-07 with completion listed for 2021-09-15.

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

Its stated primary purpose is treatment.

What Was Measured

The main thing measured (primary outcome): Phase II: Number of Participants Who Experience an Objective Clinical Response (CR or PR) When Treated With Ganitumab Plus Dasatinib. Phase II: Number of Participants That is Without Progression at 4 Months (time frame: At 4 months).

It also tracked 4 secondary outcomes, for example Phase I: Number of Participants With Grade ≥3 Adverse Events Related to Treatment With Ganitumab and Dasatinib and Phase II: Number of Participants With Grade ≥3 Adverse Events Related to Treatment With Ganitumab and Dasatinib.

What This Means in Plain Language

Put simply, this is a phase 1, phase 2, non-randomized trial of IGF-1 LR3 in 14 people.

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

It ended early (terminated); results are posted on the registry, and this page does not restate them.

More context: the IGF-1 LR3 compound page, all human trials.