Treatment for Growth Failure in Patients With X-Linked Severe Combined Immunodeficiency: Phase 2 Study of Insulin-Like Growth Factor-1

What Was Tested

The trial (phase 1, phase 2) is built around IGF-1 LR3 for insulin resistance, prediabetes & type 2 diabetes.

Those who have XSCID lack white blood cells that protect their bodies from invasion by all types of germs.

IGF-1 is the main hormone responsible for the body's growth and metabolism.

It had 1 arm (groups): Treatment (experimental).

Treatment.

The registry classes the interventions as drug.

More on the Plan

This condition is a fatal inherited immunodeficiency caused by defects in the common cytokine receptor gamma chain (gc), a subunit shared by many cytokine receptors.

The common gamma subunit signals through the Janus kinase/signal transducers and activators of transcription (JAK/STAT) pathway, a complex intracellular signaling pathway used by many cytokines and growth factors, including growth hormone (GH).

This is supported by a report of GH hyporesponsiveness in an XSCID child that was successfully ameliorated following immune reconstitution using bone marrow transplantation (BMT).

Haplo-identical BMTs for XSCID children often achieve only partial immune reconstitution, and many BMT recipients experience ongoing problems with growth failure, achieving heights well below 2 standard deviations for their chronological age.

Who Took Part

6 actual participants took part.

It was open to people of any sex aged from 2 years up to 20 years.

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

Examples of who could join: Have a diagnosis of XSCID. Be between 2 years to 20 years old and have not completed puberty. Consent to permit blood and/or tissue samples for storage. Have a primary care physician at home. Willingness to remain hospitalized for several days.

The full record lists 9 inclusion and 6 exclusion criteria; among the exclusions: have fusion of epiphyseal plates; have any active or suspected neoplasia; have any condition that, in the investigator's opinion, places the patient at undue risk by participating in the study.

How the Study Was Run

Design: single-group and open-label.

Being open-label, the IGF-1 LR3 study did not mask who received what.

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

It started in 2007-06 with completion listed for 2012-12.

It ran at 1 site in United States.

Its stated primary purpose is treatment.

What Was Measured

The main thing measured (primary outcome): Safety of Study Drug (time frame: 1 year). Change in Growth Rate on Study Drug (time frame: During intervention, up to 2 years).

What This Means in Plain Language

Put simply, this is a phase 1, phase 2 trial of IGF-1 LR3 in 6 people with insulin resistance, prediabetes & type 2 diabetes.

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, Insulin resistance, prediabetes & type 2 diabetes research.