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Treatment of growth hormone deficient children is currently limited to daily injections of growth hormone. Missed injections reduce therapeutic effectiveness, resulting in suboptimal growth.1,2

Growth hormone (GH) deficiency is the consequence of inadequate secretion of growth hormone from the pituitary gland. This results in low GH in the body, insufficient production of downstream signaling molecules required for growth, and the subsequent lack of growth. In children, this rare disorder is called pediatric growth hormone deficiency (PGHD).

Secretion of GH is under strict hormonal feedback control. Growth hormone-releasing hormone (GHRH) and ghrelin are stimulators of GH production, while somatostatin activity inhibits GH production.

GH is an important hormone regulating growth in childhood and adolescence.


PGHD can be congenital (children are born with the condition), acquired (brain tumor, head injuries or other causes), iatrogenic (induced by treatment for cancer), or idiopathic (of unknown cause). Consequences of PGHD can range from severe to moderate.




In children, PGHD results in and manifests as slower-than-normal growth.  Children are usually diagnosed by approximately seven years of age due to short stature and stunted growth velocity, although some children are not diagnosed until delay in pubertal growth spurt. About 1 in 3500 children are diagnosed with PGHD.3

PGHD is generally diagnosed by clinical observations and multiple tests, including radiographic assessment of bone age and tests of other hormones.

GH release stimulation tests are often needed to confirm PGHD diagnosis.


Current treatment options are limited to daily injectable formulations of recombinant human growth hormone (rhGH). Treatment should be started as soon as PGHD diagnosis is confirmed. Long-term (seven years or longer) treatment is not uncommon to achieve “catch-up” growth and reach full height potential. Unfortunately, daily injections can be painful, potentially leading to missed doses and sub-optimal growth.1,2

Treatment response is assessed by measurement of height and growth velocity. Treatment is usually continued until final height, completion of skeletal bone maturation (epiphyseal closure), or both have been recorded.

The main therapeutic goal of growth hormone treatment in children with PGHD is to enable these children to achieve normal height, with accompanying improvement of the psychosocial problems related to short stature.



Lumos Pharma is developing LUM-201, a once daily tablet that, if successful will provide an alternative to daily injections of growth hormone.

LUM-201, also known as ibutamoren, is an orally-administered small molecule that promotes the secretion of GH from the pituitary gland.

LUM-201 has been observed to increase the amplitude of endogenous pulsatile GH secretion, which mimics the natural pattern of GH secretion.

LUM-201 has been studied in more than 1200 patients (more than 150 children) and was generally well-tolerated.*

 Important Information – *At the doses tested previously in the Merck Trials, LUM-201 was generally well-tolerated in children with the most commonly reported adverse events being digestive systems events, including appetite increase. Mild elevations in liver enzymes without accompanying changes in bilirubin were also reported.

Pediatric Growth Hormone Deficiency has a number of different causes; not all patients will benefit from treatment with LUM-201. Our Phase 2 clinical trial tests the use of predictive enrichment markers (PEMs) to test for pituitary function.  The use of PEMs may enable clinicians to determine which patients are more likely to benefit from LUM-201.

PEM-Positive Patients

  • Have a functional but reduced hypothalamic pituitary growth hormone axis
  • More likely to respond to LUM-201
  • May represent 50-60% of PGHD patients

PEM-Negative Patients

  • Have a non-functional hypothalamic pituitary growth hormone axis
  • Not likely to respond to LUM-201
  • May represent 40-50% of PGHD patients

Lumos Pharma intends to develop LUM-201 as an orally-administered product to treat a subset of children with pediatric growth hormone deficiency (PGHD). Recombinant human GH is approved for use in many other rare indications beyond PGHD including Turner syndrome, Children Born Small for Gestational Age, Prader-Willi Syndrome, Idiopathic Short Stature (ISS), and several others. Lumos Pharma plans to develop LUM-201 for the treatment of several of these additional indications.

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Development of a Predictive Enrichment Marker for Oral GH Secretagogue LUM-201 in Children with Growth Hormone Deficiency

  • George M Bright, MD
  • Minh-Ha T Do, PhD
  • John C McKew, PhD
  • Werner F Blum, MD
  • Michael O Thorner, MB, BS, DSc

Journal of the Endocrine Society – bvab030

Published:  25 February 2021

Corroboration of Height Velocity Prediction Markers for rhGH with an Oral GH Secretagogue Treatment in Children with GHD

  • Werner F Blum, MD
  • George M Bright, MD
  • Minh-Ha T Do, PhD
  • John C McKew, PhD
  • Haiying Chen, MD, PhD
  • Michael O Thorner, MB, BS, DSc

Journal of the Endocrine Society – bvab029

Published:  25 February 2021

Optimizing Patient Management and Adherence for children receiving Growth Hormone – Carlo L. Acerini, Katarzyna Wac, Peter Bang, and Dagmar Lehwalder – Frontiers in Endocrinology

Short Stature: Comparison of WHO and National Growth Standards/References for Height – Henrik Thybo Christesen, Birgitte Tønnes Pedersen, Effie Pournara, Isabelle Oliver Petit, Pétur Benedikt Júlíusson – PLOSone

The challenge of growth hormone deficiency diagnosis and treatment during the transition from puberty into adulthood – Elena Inzaghi and Stefano Cianfarani – Frontiers In Endocrinology

Non-Compliance with Growth Hormone Treatment in Children Is Common and Impairs Linear Growth – Wayne S. Cutfield, Jose ́ G. B. Derraik, Alistair J. Gunn, Kyle Reid, Theresa Delany, Elizabeth Robinson, Paul L. Hofman – PLOS One

Growth Hormone Research Society Perspective on Biomarkers of GH Action in Children and Adults – Gudmundur Johannsson, Martin Bidlingmaier, Beverly M K Biller, Margaret Boguszewski, Felipe F Casanueva, Philippe Chanson, Peter E Clayton, Catherine S Choong, David Clemmons, Mehul Dattani, Jan Frystyk, Ken Ho, Andrew R Hoffman, Reiko Horikawa, Anders Juul, John J Kopchick, Xiaoping Luo, Sebastian Neggers, Irene Netchine, Daniel S Olsson, Sally Radovick, Ron Rosenfeld, Richard J Ross, Katharina Schilbach, Paulo Solberg, Christian Strasburger, Peter Trainer, Kevin C J Yuen, Kerstin Wickstrom, Jens O L Jorgensen, and the Growth Hormone Research Society


1. Rosenfeld, R. et al, Compliance and Persistence in Pediatric and Adult Patients Receiving Growth Hormone Therapy, Endocrine Practice, 2008, 14(2):143-154

2. Cutfield, W.S. et al, Non-Compliance with Growth Hormone Treatment in Children Is Common and Impairs Linear Growth, PLOS ONE, 2011, 6(1): e16223

3. Lindsay Journal of Pediatrics, 1994, 125(1):29-35