Trial · NCT01111019
Efficacy and Safety Evaluation of Recombinant Human Growth Hormone (r-hGH), Saizen®, on a Population of Children With Hypochondroplasia, Treated at Least 3 Years or Until Near Final Height, When Applicable, in Comparison With a Historic Cohort of Non-treated Children
NCT01111019, Phase 2, Completed, Merck KGaA, Darmstadt, Germany; results first posted 2019-02-15
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Efficacy and Safety Evaluation of Recombinant Human Growth Hormone (r-hGH), Saizen®, on a Population of Children With Hypochondroplasia, Treated at Least 3 Years or Until Near Final Height, When Applicable, in Comparison With a Historic Cohort of Non-treated Children. Trials, record NCT01111019. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01111019
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
Efficacy and Safety Evaluation of Recombinant Human Growth Hormone (r-hGH), Saizen®, on a Population of Children With Hypochondroplasia, Treated at Least 3 Years or Until Near Final Height, When Applicable, in Comparison With a Historic Cohort of Non-treated Children
- NCT number
- NCT01111019
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 19 participants, actual
- Lead sponsor
- Merck KGaA, Darmstadt, Germany
- Conditions
- Hypochondroplasia
- Interventions
- Recombinant human growth hormone (r-hGH)
- First posted
- 2010-04-27
- Last updated
- 2019-02-15
- Results first posted
- 2019-02-15
Source ClinicalTrials.gov
Registered outcomes16
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline in Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects at Year 3 | Baseline (Month 0), Year 3 | 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. |
| Primary | Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects at Year 4 | Year 4 | 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. |
| Secondary | Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects | Year 1, 2, 3, 4, 5, 6, 7, 8 (both male and female); 8.5 (only for female), 9 (only for male) and 9.5 (only for male) | 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). Data was reported by gender. Male subjects were analyzed till 9.5 years and female subjects were analyzed till 8.5 years. 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. |
| Secondary | Change From Baseline in Height of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years | Baseline (Month 0), Year 1, 2, 3, 4, 5, 6, 7, 8 (both male and female); 8.5 (only for female), 9 (only for male) and 9.5 (only for male) | Body proportion was measured in terms of height. Data was reported by gender. Male subjects were analyzed till 9.5 years and female subjects were analyzed till 8.5 years. |
| Secondary | Change From Baseline in Upper Segment (Superior) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years | Baseline (Month 0), Year 1, 2, 3, 4, 5, 6, 7, 8 (both male and female); 8.5 (only for female), 9 (only for male) and 9.5 (only for male) | Body proportion was measured in terms of upper segment for standing and sitting position. Data was reported by gender. Male subjects were analyzed till 9.5 years and female subjects were analyzed till 8.5 years. |
| Secondary | Change From Baseline in Weight of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years | Baseline (Month 0), Year 1, 2, 3, 4, 5, 6, 7, 8 (both male and female); 8.5 (only for female), 9 (only for male) and 9.5 (only for male) | The body weight was measured on a certified and calibrated hospital scale. Data was reported by gender. Male subjects were analyzed till 9.5 years and female subjects were analyzed till 8.5 years. |
| Secondary | Change From Baseline in Body Mass Index (BMI) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years | Baseline (Month 0), Year 1, 2, 3, 4, 5, 6, 7, 8 (both male and female); 8.5 (only for female), 9 (only for male) and 9.5 (only for male) | Body proportion measured as BMI. It was calculated according to the formula BMI = Weight (kilogram)/Height square (meter square). Data was reported by gender. Male subjects were analyzed till 9.5 years and female subjects were analyzed till 8.5 years. |
| Secondary | Change From Baseline in Bone Mineral Density (BMD) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years | Baseline (Month 0), Year 1, 2, 3, 4, 5, 6, 7, 8 (both male and female); 9 (only for male) | Body composition measured as BMD. It was examined at the lumbar spine (L1-L4) and determined annually by dual-energy X-ray absorptiometry. Data was reported by gender. Male subjects were analyzed till 9 years and female subjects were analyzed till 8 years. |
| Secondary | Change From Baseline in Percent Body Fat Mass of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years | Baseline (Month 0), Year 1, 2, 3, 4, 5, 6, 7, 8 (both male and female); 9 (only for male) | Body composition measured as percentage of body fat mass. It was measured by Dual X-ray absorptiometry. Data was reported by gender. Male subjects were analyzed till 9 years and female subjects were analyzed till 8 years. |
| Secondary | Change From Baseline in Lean Body Mass (LBM) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years | Baseline (Month 0), Year 1, 2, 3, 4, 5, 6, 7, 8 (both male and female); 9 (only for male) | Body composition measured as LBM. It was measured by Dual X-ray absorptiometry. Data was reported by gender. Male subjects were analyzed till 9 years and female subjects were analyzed till 8 years. |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 registered outcomesOutcomes16
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change From Baseline in Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects at Year 3 | Baseline (Month 0), Year 3 | Intention to Treat (ITT) population included all subjects who received at least one dose of treatment with at least an efficacy assessment available during the treatment period. | Standard Deviation Score (SDS) | MEAN |
| PRIMARY | Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects at Year 4 | Year 4 | Intention to Treat (ITT) population included all subjects who received at least one dose of treatment with at least an efficacy assessment available during the treatment period. | Standard Deviation Score (SDS) | MEAN |
| SECONDARY | Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects | Year 1, 2, 3, 4, 5, 6, 7, 8 (both male and female); 8.5 (only for female), 9 (only for male) and 9.5 (only for male) | Intention to Treat (ITT) population included all subjects who received at least one dose of treatment with at least an efficacy assessment available during the treatment period. Here Number analyzed signifies those subjects who were evaluable for this outcome measure for specified category. | Standard Deviation Score (SDS) | MEDIAN |
| SECONDARY | Change From Baseline in Height of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years | Baseline (Month 0), Year 1, 2, 3, 4, 5, 6, 7, 8 (both male and female); 8.5 (only for female), 9 (only for male) and 9.5 (only for male) | Intention to Treat (ITT) population included all subjects who received at least one dose of treatment with at least an efficacy assessment available during the treatment period. Here Number Analyzed signifies those subjects who were evaluable for the specified category. | Centimeter (cm) | MEAN |
| SECONDARY | Change From Baseline in Upper Segment (Superior) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years | Baseline (Month 0), Year 1, 2, 3, 4, 5, 6, 7, 8 (both male and female); 8.5 (only for female), 9 (only for male) and 9.5 (only for male) | ITT population. Here Number analyzed signifies those subjects who were evaluable for this outcome measure for specified category. Number Analyzed was 0 at certain time points because there was no data collected at the specified time points. | Centimeter (cm) | MEAN |
| SECONDARY | Change From Baseline in Weight of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years | Baseline (Month 0), Year 1, 2, 3, 4, 5, 6, 7, 8 (both male and female); 8.5 (only for female), 9 (only for male) and 9.5 (only for male) | ITT population. Here Number analyzed signifies those subjects who were evaluable for this outcome measure for specified category. | Kilogram (kg) | MEAN |
| SECONDARY | Change From Baseline in Body Mass Index (BMI) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years | Baseline (Month 0), Year 1, 2, 3, 4, 5, 6, 7, 8 (both male and female); 8.5 (only for female), 9 (only for male) and 9.5 (only for male) | Intention to Treat (ITT) population included all subjects who received at least one dose of treatment with at least an efficacy assessment available during the treatment period. Here Number analyzed signifies those subjects who were evaluable for this outcome measure for specified category. | Kilogram per meter square (kg/m^2) | MEAN |
| SECONDARY | Change From Baseline in Bone Mineral Density (BMD) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years | Baseline (Month 0), Year 1, 2, 3, 4, 5, 6, 7, 8 (both male and female); 9 (only for male) | ITT population. Here Number analyzed signifies those subjects who were evaluable for this outcome measure for specified category. Number Analyzed was 0 at certain time points because there was no data collected at the specified time points. | Gram per square centimeter (g/cm^2) | MEAN |
| SECONDARY | Change From Baseline in Percent Body Fat Mass of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years | Baseline (Month 0), Year 1, 2, 3, 4, 5, 6, 7, 8 (both male and female); 9 (only for male) | ITT population. Here Number analyzed signifies those subjects who were evaluable for this outcome measure for specified category. Number Analyzed was 0 at certain time points because there was no data collected at the specified time points. | Percentage (%) of body fat mass | MEAN |
| SECONDARY | Change From Baseline in Lean Body Mass (LBM) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years | Baseline (Month 0), Year 1, 2, 3, 4, 5, 6, 7, 8 (both male and female); 9 (only for male) | ITT population. Here Number analyzed signifies those subjects who were evaluable for this outcome measure for specified category. Number Analyzed was 0 at certain time points because there was no data collected at the specified time points. | Gram (g) | MEAN |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 outcomesResults by group215
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change From Baseline in Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects at Year 3 | r-hGH (Saizen®) | 1.39 | Standard Deviation 0.9 | Standard Deviation Score (SDS) | |
| Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects at Year 4 | r-hGH (Saizen®) | 25.6 | Standard Deviation 3.5 | Standard Deviation Score (SDS) | |
| Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects | r-hGH (Saizen®) | Male: Year 1 | -1.82 | Full Range -3.4 to -1.4 | Standard Deviation Score (SDS) |
| Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects | r-hGH (Saizen®) | Male: Year 2 | -1.51 | Full Range -3.4 to -1.1 | Standard Deviation Score (SDS) |
| Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects | r-hGH (Saizen®) | Male: Year 3 | -1.30 | Full Range -3.7 to -1.0 | Standard Deviation Score (SDS) |
| Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects | r-hGH (Saizen®) | Male: Year 4 | -1.49 | Full Range -3.7 to -1.0 | Standard Deviation Score (SDS) |
| Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects | r-hGH (Saizen®) | Male: Year 5 | -1.51 | Full Range -3.5 to -1.0 | Standard Deviation Score (SDS) |
| Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects | r-hGH (Saizen®) | Male: Year 6 | -2.88 | Full Range -3.8 to -2.0 | Standard Deviation Score (SDS) |
| Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects | r-hGH (Saizen®) | Male: Year 7 | -3.9 | Full Range -3.9 to -3.9 | Standard Deviation Score (SDS) |
| Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects | r-hGH (Saizen®) | Male: Year 8 | -3.90 | Full Range -3.9 to -3.9 | Standard Deviation Score (SDS) |
1 to 10 of 215Source ClinicalTrials.gov
See all 215 results by groupParticipant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | r-hGH (Saizen®) | 19 | |
| Overall Study | COMPLETED | r-hGH (Saizen®) | 16 | |
| Overall Study | NOT COMPLETED | r-hGH (Saizen®) | 3 |
Source ClinicalTrials.gov
Adverse events69
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Neutropenia | Blood and lymphatic system disorders | r-hGH (Saizen®) | 1 | 19 | |
| other | Thrombopenia | Blood and lymphatic system disorders | r-hGH (Saizen®) | 1 | 19 | |
| other | Leucopenia | Blood and lymphatic system disorders | r-hGH (Saizen®) | 1 | 19 | |
| other | Lymphopenia | Blood and lymphatic system disorders | r-hGH (Saizen®) | 1 | 19 | |
| other | Angina pectoris | Cardiac disorders | r-hGH (Saizen®) | 1 | 19 | |
| other | Precocious puberty | Endocrine disorders | r-hGH (Saizen®) | 1 | 19 | |
| other | Hypothyroidism | Endocrine disorders | r-hGH (Saizen®) | 1 | 19 | |
| other | Dental cyst | Gastrointestinal disorders | r-hGH (Saizen®) | 1 | 19 | |
| other | Constipation | Gastrointestinal disorders | r-hGH (Saizen®) | 1 | 19 | |
| other | Abdominal Pain | Gastrointestinal disorders | r-hGH (Saizen®) | 5 | 19 |
1 to 10 of 69Source ClinicalTrials.gov
See all 69 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | r-hGH (Saizen®) | 0 | 19 |
| Total, other adverse events | r-hGH (Saizen®) | 18 | 19 |
| Total, serious adverse events | r-hGH (Saizen®) | 1 | 19 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 25323764 | DERIVED |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
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