Condition
Growth Hormone Deficiency
Growth hormone deficiency occurs when the pituitary gland makes too little growth hormone, which drives growth in children and helps adults maintain muscle, bone and metabolism. The strongest evidence is for macimorelin, an oral test that trials found identified adult cases with accuracy comparable to the standard insulin tolerance test. CJC-1295 raised growth hormone levels in healthy adults, but that evidence is moderate and measured hormone levels rather than patient outcomes.
Sources: PMID 29860473; PMID 16352683
- Updated
- How we grade
- 46 studies cited
- Supplements studied
- 0
- Medicines and peptides
- 2
- Graded outcomes
- 7
Prescription medicines and peptides studied, for context
| Grade | Outcome | Effect | Size | Studies | People | Studies list |
|---|---|---|---|---|---|---|
| GH Deficiency Diagnosis Phase 3: 82-87% sensitivity, 92-96% specificity for AGHD diagnosis. Optimal GH cutoff 2.7-5.1 ng/mL. Accuracy comparable to insulin tolerance test (ITT). First FDA-approved oral GH stimulation test. | Improves (the measure goes up) | Large effect | 13 studies | |||
Studies that measured gh deficiency diagnosis
| ||||||
| Diagnostic Safety No hypoglycemia risk unlike ITT. Well-tolerated single oral dose. Contraindicated only with strong CYP3A4 inhibitors. Can be used in patients where ITT is contraindicated (elderly, cardiovascular disease, seizure history). | Improves (the measure goes up) | Large effect | 13 studies | |||
Studies that measured diagnostic safety
| ||||||
| GH Stimulation Response Robust GH release in healthy subjects. Peak response at 30-90 minutes. Reproducible test with low inter- and intra-individual variability. Blunted response (<5.1 ng/mL) indicates GH deficiency. | Improves (the measure goes up) | Large effect | 12 studies | |||
Studies that measured gh stimulation response
| ||||||
| Grade | Outcome | Effect | Size | Studies | People | Studies list |
|---|---|---|---|---|---|---|
| Growth Hormone Levels 2-10 fold increase in plasma GH for 6+ days | Improves (the measure goes up) | Large effect | 24 studies | 120 people | ||
Studies that measured growth hormone levels | ||||||
| IGF-1 Levels 1.5-3 fold increase sustained for 9-11 days | Changes; see studies | Large effect | 24 studies | 120 people | ||
Studies that measured igf-1 levels | ||||||
| GH Pulse Amplitude 7.5-fold increase in GH pulse amplitude | Improves (the measure goes up) | Large effect | 23 studies | 60 people | ||
Studies that measured gh pulse amplitude
| ||||||
| Body Length/Growth Normalized growth in GH-deficient models | Improves (the measure goes up) | Moderate effect | 15 studies | 40 people | ||
Studies that measured body length/growth
| ||||||
Key findings
- GH Deficiency DiagnosisImproves (the measure goes up)
- Diagnostic SafetyImproves (the measure goes up)
- GH Stimulation ResponseImproves (the measure goes up)
Safety notes in the studies
- No hypoglycemia risk unlike ITT.
Studies cited
46 studies from PubMed
- Analysis of growth hormone releasing hormone and its analogs in urine using nano liquid chromatography coupled with quadrupole/orbitrap mass spectrometry.
- Molecular recognition of two approved drugs Macimorelin and Anamorelin by the growth hormone secretagogue receptor.
- Macimorelin Acetate for the Diagnosis of Childhood-onset Growth Hormone Deficiency
- Pilot clinical trial of macimorelin to assess safety and efficacy in patients with cancer cachexia
- Diagnosis and testing for growth hormone deficiency across the ages: a global view of the accuracy, caveats, and cut-offs for diagnosis.
- Early detection of cannabinoids in biological samples based on their affinity interaction with the growth hormone secretagogue receptor.
- An antibody-free, ultrafiltration-based assay for the detection of growth hormone-releasing hormones in urine at low pg/mL concentrations using nanoLC-HRMS/MS.
- Advances in the detection of growth hormone releasing hormone synthetic analogs.
- Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Macimorelin in Children with Suspected Growth Hormone Deficiency: An Open-Label, Group Comparison, Dose-Escalation Trial
- Current concepts of the diagnosis of adult growth hormone deficiency.
- Growth hormone deficiency in adults with Cushing's disease.
- Translational potential of the ghrelin receptor agonist macimorelin for seizure suppression in pharmacoresistant epilepsy.
- Comparison of magnetic bead surface functionalities for the immunopurification of growth hormone-releasing hormones prior to liquid chromatography-high resolution mass spectrometry.
- Sensitivity and specificity of the macimorelin test for diagnosis of AGHD
- Safety, tolerability, pharmacokinetics, and pharmacodynamics of macimorelin in healthy adults: Results of a single-dose, randomized controlled study
- Thorough QT/QTc Study Evaluating the Effect of Macimorelin on Cardiac Safety Parameters in Healthy Participants
- Growth hormone secretagogues: history, mechanism of action, and clinical development
- An immuno polymerase chain reaction screen for the detection of CJC-1295 and other growth-hormone-releasing hormone analogs in equine plasma.
- An update on the diagnosis of growth hormone deficiency
- Adult growth hormone deficiency: clinical advances and approaches to improve adherence.
- Macimorelin as a Diagnostic Test for Adult GH Deficiency
- An update on the diagnosis of growth hormone deficiency.
- Netnography of Female Use of the Synthetic Growth Hormone CJC-1295: Pulses and Potions.
- Qualitative identification of growth hormone-releasing hormones in human plasma by means of immunoaffinity purification and LC-HRMS/MS.
- Anticonvulsant effect of a ghrelin receptor agonist in 6Hz corneally kindled mice.
- Expanded test method for peptides >2 kDa employing immunoaffinity purification and LC-HRMS/MS.
- The macimorelin-stimulated growth hormone test for adult growth hormone deficiency diagnosis.
- Macimorelin (AEZS-130)-Stimulated Growth Hormone (GH) Test: Validation of a Novel Oral Stimulation Test for the Diagnosis of Adult GH Deficiency
- Protective but not anticonvulsant effects of ghrelin and JMV-1843 in the pilocarpine model of Status epilepticus.
- Immunoaffinity purification of peptide hormones prior to liquid chromatography-mass spectrometry in doping controls.
- Safety and tolerability of subcutaneous CJC-1295 in healthy adults: Phase I clinical trial
- Ghrelin stimulation of growth hormone-releasing hormone neurons is direct in the arcuate nucleus.
- Activation of the GH/IGF-1 axis by CJC-1295, a long-acting GHRH analog, results in serum protein profile changes in normal adult subjects
- Pulsatile vs continuous growth hormone secretion: clinical implications
- Prolonged stimulation of growth hormone (GH) and insulin-like growth factor I secretion by CJC-1295, a long-acting analog of GH-releasing hormone, in healthy adults
- Once-daily administration of CJC-1295, a long-acting growth hormone-releasing hormone (GHRH) analog, normalizes growth in the GHRH knockout mouse
- Growth hormone-releasing hormone and growth hormone secretagogues: clinical applications
- Pulsatile secretion of growth hormone (GH) persists during continuous stimulation by CJC-1295, a long-acting GH-releasing hormone analog.
- Human growth hormone-releasing factor (hGRF)1-29-albumin bioconjugates activate the GRF receptor on the anterior pituitary in rats: identification of CJC-1295 as a long-lasting GRF analog
- Pharmacokinetics, safety, and tolerability of increasing repeat doses of CJC-1295 in healthy volunteers
- New active series of growth hormone secretagogues.
- Growth hormone-releasing peptides, ghrelin, and growth hormone secretagogues
- Growth Hormone Stimulation Tests in Assessing Adult Growth Hormone Deficiency.
- Effects of intranasal growth hormone-releasing hormone on sleep and sleep-related growth hormone release in aged men
- GHRH increases slow-wave sleep and REM sleep in humans
- Induction of a prolonged increase in growth hormone (GH) levels by GH-releasing hormone