Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Recombinant human growth hormone · 1 trial · 1 indication
Height was calculated in standardized units expressed as the standard deviation score (SDS), where SDS = (x - m)/sigma, in which x represents the height variable measured by sex and age, and m and sigma are the statistical parameters (mean and standard deviation) for the Sempe reference population. The reference population was the historical cohort consisting of non-treated subjects with Hypochondroplasia (HCH). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) a participant's value was relative to the mean of the reference population. The scores were centered around zero. Negative score indicated smaller height for the respective age/gender.
Height was calculated in standardized units expressed as the standard deviation score (SDS), where SDS = (x - m)/sigma, in which x represents the height variable measured by sex and age, and m and sigma are the statistical parameters (mean and standard deviation) for the Sempe reference population. The reference population was the historical cohort consisting of non-treated subjects with Hypochondroplasia (HCH). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) a participant's value was relative to the mean of the reference population. The scores were centered around zero. Negative score indicated smaller height for the respective age/gender.
| Arm | Type | Description |
|---|---|---|
| r-hGH (Saizen®) | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| Recombinant human growth hormone (r-hGH) | DRUG | Subjects will receive a single subcutaneous injection of recombinant human growth hormone (r-hGH) equivalent to a dose of 0.057 milligram per kilogram per day (mg/kg/day). The dose will be subsequently adjusted during the trial and subjects will be treated for at least 3 years or until near final height is reached. |
Inclusion Criteria: * Male or female children with hypochondroplasia defined by a disproportional short limb height and a X-ray evidence of shortening of the long bones and failure of increase in the interpedicular distance between lumbar vertebrae L1 and L5 * Result of genetic analysis for mutatio...
Recombinant human growth hormone is being studied for use in hypochondroplasia, a rare genetic condition affecting bone growth. It is intended to improve growth in children with this condition, with treatment lasting at least three years or until near final height is reached.
Recombinant human growth hormone is developed by Merck & Company, Inc., a pharmaceutical company traded on the New York Stock Exchange under the ticker symbol MRK.
Recombinant human growth hormone is in Phase 2 clinical development for hypochondroplasia. It is an investigational therapy, meaning it has not yet been approved by regulatory authorities and is still being evaluated in clinical trials.
Recombinant human growth hormone has been studied in one completed Phase 2 clinical trial, identified as NCT01111019. This trial evaluated the efficacy and safety of the drug in children with hypochondroplasia in France, enrolling 19 participants aged 3 years and older.
Yes, recombinant human growth hormone is also known as Saizen. The clinical trial NCT01111019 specifically evaluated the Saizen brand of recombinant human growth hormone in children with hypochondroplasia.