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Effectiveness of rhGH in Children with GHD in GeNeSIS (NCT01088412) - revised

Effectiveness of rhGH in Children with GHD in GeNeSIS (NCT01088412) - revised

Michael O. Thorner

Retired from University of Virginia after serving on faculty for 40 years as a clinical investigator, Division Chief Endocrinology and Metabolism, Chairman of the Department of Medicine

None. Ammonett Pharma will pay Dr. Chen as a consultant to perform biostatistical analysis

Founder and Chief Scientific Office of Ammonett Pharma which is developing ibutamoren for treatment of children with short stature and growth hormone deficiency

15 February 2018

Growth hormone deficiency (GHD) in children is a well-recognized medical condition leading to physical disability and psychological distress associated with extreme short stature starting during childhood and persisting throughout adult life. The condition is rare and is, therefore defined as an "orphan" disease by FDA, EMA and other regulatory authorities.
The only currently approved therapy for GHD is the daily administration of recombinant human growth hormone (rhGH) which must be given by daily subcutaneous injection. The Sponsors of this proposal, Ammonett Pharma, have developed an oral medication, ibutamoren (IB), which can lead to the increased release of endogenous GH in the majority of children diagnosed with GHD and which has the potential to stimulate childhood growth over the long-term, abolishing the physical and psychological symptoms associates with extreme short stature. Because of the existence of internal homeostatic feedback systems which limit the production of GH that can be released upon administration of a GH-releasing agent such as IB, any safety issues associated with exposure to excessive levels of GH are eliminated with the use of IB.
The Sponsor's plan is to test the hypothesis that a sub-population of children with GHD, responsive to IB, can be defined and to demonstrate that, in stimulating growth over the first year of treatment, IB is safe and statistically non-inferior to treatment with injections of rhGH. Such treatment, by its simplicity and increased patient acceptability as compared with daily injections, could greatly increase adherence to a regimen of growth promoting therapy, easing the burden of the disease on patients and families.
As part of the planning of a comparative trial of two doses of IB to standard doses of injected rhGH, the sponsors wish to evaluate data illustrating the effect of starting rhGH in a large population of children who participated in the GeNeSIS register of GHD children treated with Humatrope (Eli Lilly) By evaluating the initial growth response to rhGH in children who fulfill the clinical criteria which the Sponsor believes will predict responsiveness to IB, the Sponsor will be able to define a true control group, optimizing the design of the planned comparative clinical trial. Such an approach will strengthen the evidence required by providers, patient families and others to evaluate the effectives of IB in comparison with that of currently standard therapy.

[{ "PostingID": 5077, "Title": "LILLY-B9R-EW-GDFC", "Description": "The Genetics and Neuroendocrinology of Short Stature International Study (GeNeSIS)

Medicine: Somatropin , Condition: Dwarfism, Growth Hormone Deficiency;
Turner Syndrome;
Infant, Small for Gestational Age;
SHOX Protein, Human, Phase: Not applicable, Clinical Study ID: B9R-EW-GDFC , Sponsor: Lilly." }]

Statistical Analysis Plan

The publication citation will be added after the research is published.