Primary Objective- Compare the safety and efficacy of subcutaneous somavaratan and daily rhGH during 12 months of treatment.Secondary Objective-Evaluate and compare changes in pharmacodynamic responses (IGF-I, IGF binding protein-3 (IGFBP-3), growth…
ID
Source
Brief title
Condition
- Hypothalamus and pituitary gland disorders
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
Primary efficacy endpoint
- Annual height velocity (cm/yr) after 12 months of treatment with either
somavaratan, or daily rhGH
Secondary outcome
Secondary efficacy endpoints
-IGF-I and IGFBP-3 responses to study drug administration
-changes in height standard deviation score, body weight, body mass index, bone
age
Background summary
Growth Hormone Deficiency (GHD) in children results from a variety of genetic,
neoplastic, inflammatory, post-traumatic and iatrogenic causes. Subjects with
untreated childhood onset GHD will have significant growth failure with
attainment of adult heights significantly less then five feet in many
instances. In addition, there is abnormal body composition with decreased bone
mineralizatoin, decreased lean body mass and increased fat mass. Treatment with
exogenous rhGH initiates a period of accelerated or "catch-up" growth that when
begun at an early age allows attainment of normal adult height and body
composition.
Somavaratan is a recombinant human growth hormone analog, designed to maintain
active drug levels for longer period of time than current daily therapies.
Daily therapy is currently the only approved treatment for adults and children
with GHD. Long-acting GH offers the possibilities of many fewer injections,
enhanced compliance and attainment of improved treatment outcomes.
Study objective
Primary Objective
- Compare the safety and efficacy of subcutaneous somavaratan and daily rhGH
during 12 months of treatment.
Secondary Objective
-Evaluate and compare changes in pharmacodynamic responses (IGF-I, IGF binding
protein-3 (IGFBP-3), growth hormone binding protein (GHBP) and acid labile
subunit (ALS)), bone age, weight, body mass index, height standard deviation
scores, and anti-drug antibody (ADA) and neutralizing antibody (NAb) responses.
Study design
This is a randomized, multi-center, open label study comparing study drug with
commercial available treatment in a parallell group design.
Intervention
Subjects will receive commercial available recombinant human growth hormone
daily ór somavaratan every 15 days during 1 year.
Study burden and risks
Burden and risk:
Pain, bruising or infection due to additional bloodsampling compared to
standard of care, additional radiation exposure to less then 1 mSv due to an
additional X-ray of the hand, requirement to keep a dosing record at home. An
ECG twice; ECG patches could cause a transient rash.
Benefit:
Increase in growth that is similar to that of standard of care. If assigned to
the somavaratan treatment group less frequent treatment injections (twice per
month) as compared to standard of care (daily)
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4200 Bohannon Drive Suite 250
94025 Menlo Park
US
Listed location countries
Age
Inclusion criteria
1. Chronological Age >= 3.0 years and <= 10.0 (girls) and <= 11.0 (boys).;2. Pre-pubertal status: Absent breast development in girls, testicular volume <;4.0 mL in boys.;3. Diagnosis of GHD as documented two or more GH stimulation test results <= 10.0 ng/mL.;4. Height SDS <= -2.0 at screening.;5. Weight for Stature >= 10th percentile.;6. IGF-I SD score <= -1.0 at screening.;7. Delayed bone age (>= 6 months as determined by the central reader). Left hand X-Ray must be obtained within 90 days of screening visit or during screening.;8. Normal thyroid function test results at screening visit (or a minimum of four weeks of thyroxine replacement therapy prior to study drug administration).;9. Available adrenal function test results at screening visit (or in the preceding 6 months) in all subjects without a minimum of four weeks glucocorticoid replacement therapy prior to study drug administration. ;10. Pathology relating to cause of GH deficiency must be stable for at least 6 months prior to screening.;11. Legally authorized representatives must be willing and able to give informed;consent.
Exclusion criteria
1. Prior treatment with any growth promoting agent (e.g., GH, IGF-I, GH releasing hormone (GHRH), gonadotrophins, sex steroids). Up to 10 day exposures to a growth promoting agent for diagnostic purposes are permitted if administered 30 or more days prior to screening. ;2. Documented history of, or current, significant disease.;3. Chromosomal aneuploidy, significant gene mutations (other than those that cause GHD) or confirmed diagnosis of a named syndrome.;4. Birth weight and/or birth length less than 5th percentile for gestational age using gestational age growth charts.;5. A diagnosis of Attention Deficit Hyperactivity Disorder (ADHD), use of ADHD;medications or a likelihood of starting ADHD medications during study participation.;6. Daily use of anti-inflammatory doses of glucocorticoid.;7. Prior history of leukemia, lymphoma, sarcoma or cancer.;8. Treatment with an investigational drug in the 30 days prior to screening.;9. Known allergy to constituents of the study drug formulation.;10. Ocular findings suggestive of increased intracranial pressure and/or retinopathy at screening.;11. Significant spinal abnormalities including scoliosis, kyphosis and spina bifida;variants.;12. Significant abnormality in screening laboratory studies.;13. Current social conditions which would prevent completion of study activities;(e.g., planned family move to a distant location).;14. History of pancreatitis or undiagnosed chronic abdominal pain.;15. History of spinal or total body irradiation.;16. Subjects with other pituitary hormone deficiency who are not treated properly.;17. Unwillingness to provide consent for participation in all trial activities.;18. Unwillingness to accept dose assignments.
Design
Recruitment
Medical products/devices used
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
Register | ID |
---|---|
EudraCT | EUCTR2014-004525-41-NL |
ClinicalTrials.gov | NCT02339090 |
CCMO | NL52440.091.15 |