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Investigating the Long-term Efficacy and Safety of Two Doses of NN-220 (Somatropin) in Short Stature Due to Noonan Syndrome

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01927861
Recruitment Status : Completed
First Posted : August 23, 2013
Results First Posted : October 14, 2019
Last Update Posted : October 14, 2019
Sponsor:
Information provided by (Responsible Party):
Novo Nordisk A/S

Brief Summary:
This trial is conducted in Asia. The aim of the trial is to investigate the long-term efficacy and safety of two doses of NN-220 (somatropin) in short stature due to Noonan syndrome.

Condition or disease Intervention/treatment Phase
Genetic Disorder Noonan Syndrome Drug: somatropin Phase 3

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 51 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: A 52-week, Multi-centre, Randomised, Double-blind, Parallel-group, no Treatment Controlled (Open-label) Trial Investigating the Efficacy and Safety of Two Doses of NN-220 in Short Stature With Noonan Syndrome
Actual Study Start Date : August 19, 2013
Actual Primary Completion Date : July 12, 2018
Actual Study Completion Date : July 12, 2018


Arm Intervention/treatment
Experimental: 0.033 mg/kg/day Drug: somatropin
Administered subcutaneously (s.c., under the skin) in a daily regimen for at least 104 weeks. Subject will be offered to continue treatment for another 104 weeks.

Experimental: 0.066 mg/kg/day Drug: somatropin
Administered subcutaneously (s.c., under the skin) in a daily regimen for at least 104 weeks. Subject will be offered to continue treatment for another 104 weeks.




Primary Outcome Measures :
  1. Change in Height SDS (Japanese National Reference Data) [ Time Frame: Baseline, week 104 ]
    Height SDS was calculated using the formula: SDS = (height - mean)/SD, where height was the height variable measured, mean and SD of height by sex and age for the reference population. The scores were centered around zero. Positive SDS indicated greater height and negative SDS indicated lesser height than the mean of the reference population. The change from baseline (week 0) in the height SDS after 104 weeks of treatment was analysed using an analysis of covariance (ANCOVA) model with treatment as a fixed effect and baseline height SDS as a covariate. Positive value in change from baseline indicated that SDS was better than baseline SDS. Missing values were imputed using the last observation carried forward (LOCF) method.


Secondary Outcome Measures :
  1. Height Velocity SDS [ Time Frame: Baseline to week 52 ]
    Height velocity is change in height per year. The height velocity was calculated as the difference between current height (week 52) and height at baseline (week 0) divided by time between those measurement time points and multiplied by 365 days. Height velocity SDS was calculated using the formula: SDS = (height velocity - mean)/SD, where height velocity was the height velocity variable measured, mean and SD of height velocity by sex and age for the reference population. The scores were centered around zero. Positive SDS indicated greater height velocity and negative SDS indicated lesser height velocity than the mean of the reference population.

  2. Height Velocity SDS [ Time Frame: Week 52 to week 104 ]
    Height velocity is change in height per year. The height velocity was calculated as the difference between current height (week 104) and height at week 52 divided by time between those measurement time points and multiplied by 365 days. Height velocity SDS was calculated using the formula: SDS = (height velocity - mean)/SD, where height velocity was the height velocity variable measured, mean and SD of height velocity by sex and age for the reference population. The scores were centered around zero. Positive SDS indicated greater height velocity and negative SDS indicated lesser height velocity than the mean of the reference population.

  3. Height Velocity [ Time Frame: Baseline to week 52 ]
    Height velocity is change in height per year. The height velocity was calculated as the difference between current height (week 52) and height at baseline (week 0) divided by time between those measurement time points and multiplied by 365 days.

  4. Height Velocity [ Time Frame: Week 52 to week 104 ]
    Height velocity is change in height per year. The height velocity was calculated as the difference between current height (week 104) and height at week 52 divided by time between those measurement time points and multiplied by 365 days.

  5. Incidence of Treatment Emergent Adverse Events [ Time Frame: During 104 weeks of treatment ]
    A treatment emergent adverse event (TEAE; for the pivotal phase) was defined as an event that had onset date on or after the date of visit 2 (week 0; start of treatment) and no later than the date of visit 12 (104 weeks; end of pivotal phase). For withdrawal participants (if any), an adverse event with onset date no later than 7 days after the last day of NN-220 treatment was included.

  6. Change in IGF-I (Insulin-like Growth Factor-I) [ Time Frame: Baseline, week 104 ]
    Change from baseline (within 4 weeks prior to week 0) in IGF-I was evaluated after 104 weeks of treatment. Missing values were imputed using the LOCF method.

  7. Change in HbA1c (Glycosylated Haemoglobin) [ Time Frame: Baseline, week 104 ]
    Change from baseline (within 4 weeks prior to week 0) in HbA1c was evaluated after 104 weeks of treatment.

  8. Change in Clinical Laboratory Tests (Haematology: Erythrocytes) [ Time Frame: Baseline, week 104 ]
    Change from baseline (within 4 weeks prior to week 0) in haematological parameter - erythrocytes. Missing values were imputed using the LOCF method.

  9. Change in Clinical Laboratory Tests (Haematology: Leukocytes and Thrombocytes) [ Time Frame: Baseline, week 104 ]
    Change from baseline (within 4 weeks prior to week 0) in haematological parameter - leukocytes and thrombocytes. Missing values were imputed using the LOCF method.

  10. Change in Clinical Laboratory Tests (Haematology: Haemoglobin) [ Time Frame: Baseline, week 104 ]
    Change from baseline (within 4 weeks prior to week 0) in haematological parameter - haemoglobin. Missing values were imputed using the LOCF method.

  11. Change in Clinical Laboratory Tests (Haematology: Haematocrit) [ Time Frame: Baseline, week 104 ]
    Change from baseline (within 4 weeks prior to week 0) in haematological parameter - haematocrit. Missing values were imputed using the LOCF method.

  12. Change in Clinical Laboratory Tests (Haematology: Neutrophils) [ Time Frame: Baseline, week 104 ]
    Change from baseline (within 4 weeks prior to week 0) in haematological parameter - neutrophils. Missing values were imputed using the LOCF method.

  13. Change in Clinical Laboratory Tests (Haematology: Lymphocytes) [ Time Frame: Baseline, week 104 ]
    Change from baseline (within 4 weeks prior to week 0) in haematological parameter - lymphocytes. Missing values were imputed using the LOCF method.

  14. Change in Clinical Laboratory Tests (Haematology: Monocytes) [ Time Frame: Baseline, week 104 ]
    Change from baseline (within 4 weeks prior to week 0) in haematological parameter - monocytes. Missing values were imputed using the LOCF method.

  15. Change in Clinical Laboratory Tests (Haematology: Eosinophils) [ Time Frame: Baseline, week 104 ]
    Change from baseline (within 4 weeks prior to week 0) in haematological parameter - eosinophils. Missing values were imputed using the LOCF method.

  16. Change in Clinical Laboratory Tests (Haematology: Basophils) [ Time Frame: Baseline, week 104 ]
    Change from baseline (within 4 weeks prior to week 0) in haematological parameter - basophils. Missing values were imputed using the LOCF method.

  17. Change in Clinical Laboratory Tests (Lipids: Total Cholesterol, LDL Cholesterol and HDL Cholesterol) [ Time Frame: Baseline, week 104 ]
    Change from baseline (within 4 weeks prior to week 0) in lipids: total cholesterol, LDL (low-density lipoprotein) cholesterol and HDL (high-density lipoprotein) cholesterol. Missing values were imputed using the LOCF method.

  18. Change in Clinical Laboratory Tests (Biochemistry: AST, ALT, r-GTP and Alkaline Phosphatase) [ Time Frame: Baseline, week 104 ]
    Change from baseline (within 4 weeks prior to week 0) in biochemical parameters - aspartate aminotransferase (AST), alanine aminotransferase (ALT), gamma-glutamyl transpeptidase (r-GTP) and alkaline phosphatase. Missing values were imputed using the LOCF method.

  19. Change in Clinical Laboratory Tests (Biochemistry: Total Protein) [ Time Frame: Baseline, week 104 ]
    Change from baseline (within 4 weeks prior to week 0) in biochemical parameter - total protein. Missing values were imputed using the LOCF method.

  20. Change in Clinical Laboratory Tests (Biochemistry: Blood Urea Nitrogen, Sodium, Potassium, Chloride, Total Calcium and Phosphorus) [ Time Frame: Baseline, week 104 ]
    Change from baseline (within 4 weeks prior to week 0) in biochemical parameters - blood urea nitrogen, sodium, potassium, chloride, total calcium and phosphorus. Missing values were imputed using the LOCF method.

  21. Change in Clinical Laboratory Tests (Biochemistry: Creatinine) [ Time Frame: Baseline, Week 104 ]
    Change from baseline (within 4 weeks prior to week 0) in biochemical parameter - creatinine. Missing values were imputed using the LOCF method.

  22. Change in Glucose Tolerance (AUC of Glucose) Based on the OGTT [ Time Frame: Baseline, week 104 ]
    AUC (area under the curve) of glucose was calculated by the trapezoidal method. Change from baseline (within 4 weeks prior to week 0) in glucose tolerance (AUC of glucose: 30, 60, 90 and 120 min after oral glucose load) at week 104 was evaluated based on the oral glucose tolerance test (OGTT). Change from baseline results are presented as 'ratio to baseline'.

  23. Change in Glucose Tolerance (AUC of Insulin) Based on the OGTT [ Time Frame: Baseline, week 104 ]
    AUC of insulin was calculated by the trapezoidal method. Change from baseline (within 4 weeks prior to week 0) in glucose tolerance (AUC of insulin: 30, 60, 90 and 120 min after oral glucose load) at week 104 was evaluated based on the OGTT. Change from baseline results are presented as 'ratio to baseline'.

  24. Change in Bone Age [ Time Frame: Baseline, week 104 ]
    X-ray picture of carpal bones of left hand was taken for bone age determination. Centralised evaluation of bone age was done by the radius, ulna and short bones (RUS) score method of Tanner-Whitehouse II (TW2). Change from baseline (week 0) in bone age.

  25. Change in Bone Age/Chronological Age [ Time Frame: Baseline, week 104 ]
    X-ray picture of carpal bones of left hand was taken for bone age determination. Centralised evaluation of bone age was done by the RUS score method of Tanner-Whitehouse II (TW2). Change from baseline (week 0) in bone age/chronological age.

  26. Yearly Change in Bone Age/Change in Chronological Age [ Time Frame: Baseline, week 52 ]
    X-ray picture of carpal bones of left hand was taken for bone age determination. Centralised evaluation of bone age was done by the RUS score method of Tanner-Whitehouse II (TW2). Yearly change from baseline (week 0) in bone age/change in chronological age was presented.

  27. Yearly Change in Bone Age/Change in Chronological Age [ Time Frame: Week 52, week 104 ]
    X-ray picture of carpal bones of left hand was taken for bone age determination. Centralised evaluation of bone age was done by the RUS score method of Tanner-Whitehouse II (TW2). Yearly change from week 52 in bone age/change in chronological age was presented.

  28. Change in Vital Signs (Diastolic Blood Pressure and Systolic Blood Pressure) [ Time Frame: Baseline, week 104 ]
    Systolic and diastolic blood pressure were measured after a 5-minute rest in sitting position. Change from baseline (week 0) in systolic blood pressure and diastolic blood pressure.

  29. Change in Vital Signs (Pulse) [ Time Frame: Baseline, week 104 ]
    Pulse was measured after a 5-minute rest in sitting position. Change from baseline (week 0) in pulse.

  30. Change in Urinalysis (Protein, Glucose and Occult Blood) [ Time Frame: Baseline, week 104 ]
    The urinalysis was the measurements of protein, glucose, and occult blood at baseline (within 4 weeks prior to week 0) and week 104 and categorised as negative, trace, 1+, 2+ and 3+. Missing values were imputed using the LOCF method. Number of participants in each category at baseline and week 104 are presented.

  31. Change in Blood Coagulation Test (Prothrombin Time and APTT) [ Time Frame: Baseline, week 104 ]
    Change from baseline (within 4 weeks prior to week 0) in blood coagulation test parameters: Prothrombin time and APTT (activated partial thromboplastin time). Missing values were imputed using the LOCF method.

  32. Change in ECG [ Time Frame: Baseline, week 104 ]
    The ECG was recorded after a 3-minute rest in supine position at baseline (within 4 weeks prior to week 0) and week 104 and categorised as normal, abnormal NCS (not clinically significant) or abnormal CS (clinically significant). Number of participants in each ECG category at baseline and week 104 are presented. Missing values were imputed using the LOCF method.

  33. Change in Height SDS (Japanese National Reference Data) [ Time Frame: Baseline, week 208 ]
    Height SDS was calculated using the formula: SDS = (height - mean)/SD, where height was the height variable measured, mean and SD of height by sex and age for the reference population. The scores were centered around zero. Positive SDS indicated greater height and negative SDS indicated lesser height than the mean of the reference population. The change from baseline (week 0) in the height SDS after 208 weeks of treatment was analysed. Positive value in change from baseline indicated that SDS was better than baseline SDS. Missing values were imputed using the LOCF method.

  34. Change in Height SDS (Noonan Syndrome Reference Data in Japanese) [ Time Frame: Baseline, week 208 ]
    Height SDS was calculated using the formula: Z=[(value/M)^L-1]/(S*L); where L, M and S are skewness (L), median (M) and coefficient of variation (S) of Japanese Noonan syndrome' height provided for each sex and age. For each participant, a standard deviation score Z (SDS) was calculated based on age and sex referring to the values L, M and S. The scores were centered around zero. Positive SDS indicated greater height and negative SDS indicated lesser height than the mean of the reference population. The change from baseline (week 0) in the height SDS after 208 weeks of treatment was analysed. Positive value in change from baseline indicated that SDS was better than baseline SDS. Missing values were imputed using the LOCF method.

  35. Height Velocity [ Time Frame: Week 104 to week 156 ]
    Height velocity is change in height per year. The height velocity was calculated as the difference between current height (week 156) and height at week 104 divided by time between those measurement time points and multiplied by 365 days. Missing values were imputed using the LOCF method.

  36. Height Velocity [ Time Frame: Week 156 to week 208 ]
    Height velocity is change in height per year. The height velocity was calculated as the difference between current height (week 208) and height at week 156 divided by time between those measurement time points and multiplied by 365 days. Missing values were imputed using the LOCF method.

  37. Height Velocity SDS [ Time Frame: Week 104 to week 156 ]
    Height velocity is change in height per year. The height velocity was calculated as the difference between current height (week 156) and height at week 104 divided by time between those measurement time points and multiplied by 365 days. Height velocity SDS was calculated using the formula: SDS = (height velocity - mean)/SD, where height velocity was the height velocity variable measured, mean and SD of height velocity by sex and age for the reference population. The scores were centered around zero. Positive SDS indicated greater height velocity and negative SDS indicated lesser height velocity than the mean of the reference population.

  38. Height Velocity SDS [ Time Frame: Week 156 to week 208 ]
    Height velocity is change in height per year. The height velocity was calculated as the difference between current height (week 208) and height at week 156 divided by time between those measurement time points and multiplied by 365 days. Height velocity SDS was calculated using the formula: SDS = (height velocity - mean)/SD, where height velocity was the height velocity variable measured, mean and SD of height velocity by sex and age for the reference population. The scores were centered around zero. Positive SDS indicated greater height velocity and negative SDS indicated lesser height velocity than the mean of the reference population.

  39. Incidence of Treatment Emergent AEs [ Time Frame: Week 0 to week 234 (208 weeks treatment period + 26 weeks extended treatment period) + 7 days (follow-up period) ]
    A treatment emergent AE (TEAE) was defined as an event that had onset date on or after the date of visit 2 (week 0; start of treatment) and no later than 7 days after the last day of NN-220 treatment.

  40. Change in IGF-I [ Time Frame: Baseline, week 208 ]
    Change from baseline (within 4 weeks prior to week 0) in IGF-I was evaluated after 208 weeks of treatment. Missing values were imputed using the LOCF method.

  41. Change in HbA1c [ Time Frame: Baseline, week 208 ]
    Change from baseline (within 4 weeks prior to week 0) in HbA1c was evaluated after 208 weeks of treatment.

  42. Change in Clinical Laboratory Tests (Haematology: Erythrocytes) [ Time Frame: Baseline, week 208 ]
    Change from baseline (within 4 weeks prior to week 0) in haematological parameter - erythrocytes. Missing values were imputed using the LOCF method.

  43. Change in Clinical Laboratory Tests (Haematology: Leukocytes and Thrombocytes) [ Time Frame: Baseline, Week 208 ]
    Change from baseline (within 4 weeks prior to week 0) in haematological parameter - leukocytes and thrombocytes. Missing values were imputed using the LOCF method.

  44. Change in Clinical Laboratory Tests (Haematology: Haemoglobin) [ Time Frame: Baseline, week 208 ]
    Change from baseline (within 4 weeks prior to week 0) in haematological parameter - haemoglobin. Missing values were imputed using the LOCF method.

  45. Change in Clinical Laboratory Tests (Haematology: Haematocrit) [ Time Frame: Baseline, week 208 ]
    Change from baseline (within 4 weeks prior to week 0) in haematological parameter - haematocrit. Missing values were imputed using the LOCF method.

  46. Change in Clinical Laboratory Tests (Haematology: Neutrophils) [ Time Frame: Baseline, week 208 ]
    Change from baseline (within 4 weeks prior to week 0) in haematological parameter - neutrophils. Missing values were imputed using the LOCF method.

  47. Change in Clinical Laboratory Tests (Haematology: Lymphocytes) [ Time Frame: Baseline, week 208 ]
    Change from baseline (within 4 weeks prior to week 0) in haematological parameter - lymphocytes. Missing values were imputed using the LOCF method.

  48. Change in Clinical Laboratory Tests (Haematology: Monocytes) [ Time Frame: Baseline, week 208 ]
    Change from baseline (within 4 weeks prior to week 0) in haematological parameter - monocytes. Missing values were imputed using the LOCF method.

  49. Change in Clinical Laboratory Tests (Haematology: Eosinophils) [ Time Frame: Baseline, week 208 ]
    Change from baseline (within 4 weeks prior to week 0) in haematological parameter - eosinophils. Missing values were imputed using the LOCF method.

  50. Change in Clinical Laboratory Tests (Haematology: Basophils) [ Time Frame: Baseline, week 208 ]
    Change from baseline (within 4 weeks prior to week 0) in haematological parameter - basophils. Missing values were imputed using the LOCF method.

  51. Change in Clinical Laboratory Tests (Lipids: Total Cholesterol, LDL Cholesterol and HDL Cholesterol) [ Time Frame: Baseline, week 208 ]
    Change from baseline (within 4 weeks prior to week 0) in lipids: total cholesterol, LDL cholesterol and HDL cholesterol. Missing values were imputed using the LOCF method.

  52. Change in Clinical Laboratory Tests (Biochemistry: AST, ALT, r-GTP and Alkaline Phosphatase) [ Time Frame: Baseline, week 208 ]
    Change from baseline (within 4 weeks prior to week 0) in biochemical parameters - AST, ALT, r-GTP and alkaline phosphatase. Missing values were imputed using the LOCF method.

  53. Change in Clinical Laboratory Tests (Biochemistry: Total Protein) [ Time Frame: Baseline, week 208 ]
    Change from baseline (within 4 weeks prior to week 0) in biochemical parameter - total protein. Missing values were imputed using the LOCF method.

  54. Change in Clinical Laboratory Tests (Biochemistry: Blood Urea Nitrogen, Sodium, Potassium, Chloride, Total Calcium and Phosphorus) [ Time Frame: Baseline, week 208 ]
    Change from baseline (within 4 weeks prior to week 0) in biochemical parameters - blood urea nitrogen, sodium, potassium, chloride, total calcium and phosphorus. Missing values were imputed using the LOCF method.

  55. Change in Clinical Laboratory Tests (Biochemistry: Creatinine) [ Time Frame: Baseline, week 208 ]
    Change from baseline (within 4 weeks prior to week 0) in biochemical parameters - creatinine. Missing values were imputed using the LOCF method.

  56. Change in Glucose Tolerance (AUC of Glucose) Based on the OGTT [ Time Frame: Baseline, week 208 ]
    AUC of glucose was calculated by the trapezoidal method. Change from baseline (within 4 weeks prior to week 0) in glucose tolerance (AUC of glucose: 30, 60, 90 and 120 min after oral glucose load) at week 208 was evaluated based on the OGTT. Change from baseline results are presented as 'ratio to baseline'.

  57. Change in Glucose Tolerance (AUC of Insulin) Based on the OGTT [ Time Frame: Baseline, week 208 ]
    AUC of insulin was calculated by the trapezoidal method. Change from baseline (within 4 weeks prior to week 0) in glucose tolerance (AUC of insulin: 30, 60, 90 and 120 min after oral glucose load) at week 208 was evaluated based on the OGTT. Change from baseline results are presented as 'ratio to baseline'.

  58. Change in Bone Age [ Time Frame: Baseline, week 208 ]
    X-ray picture of carpal bones of left hand was taken for bone age determination. Centralised evaluation of bone age was done by the RUS score method of Tanner-Whitehouse II (TW2). Change from baseline (week 0) in bone age.

  59. Change in Bone Age/Chronological Age [ Time Frame: Baseline, week 208 ]
    X-ray picture of carpal bones of left hand was taken for bone age determination. Centralised evaluation of bone age was done by the RUS score method of Tanner-Whitehouse II (TW2). Change from baseline (week 0) in bone age/chronological age.

  60. Yearly Change in Bone Age/Change in Chronological Age [ Time Frame: Week 104, week 156 ]
    X-ray picture of carpal bones of left hand was taken for bone age determination. Centralised evaluation of bone age was done by the RUS score method of Tanner-Whitehouse II (TW2). Yearly change from week 104 in bone age/change in chronological age was presented.

  61. Yearly Change in Bone Age/Change in Chronological Age [ Time Frame: Week 156, week 208 ]
    X-ray picture of carpal bones of left hand was taken for bone age determination. Centralised evaluation of bone age was done by the RUS score method of Tanner-Whitehouse II (TW2). Yearly change from week 156 in bone age/change in chronological age was presented.

  62. Change in Vital Signs (Diastolic Blood Pressure and Systolic Blood Pressure) [ Time Frame: Baseline, week 208 ]
    Systolic and diastolic blood pressure were measured after a 5-minute rest in sitting position. Change from baseline (week 0) in systolic blood pressure and diastolic blood pressure. Missing values were imputed using the LOCF method.

  63. Change in Vital Signs (Pulse) [ Time Frame: Baseline, week 208 ]
    Pulse was measured after a 5-minute rest in sitting position. Change from baseline (week 0) in pulse. Missing values were imputed using the LOCF method.

  64. Change in Urinalysis (Protein, Glucose and Occult Blood) [ Time Frame: Baseline, week 208 ]
    The urinalysis was the measurements of protein, glucose, and occult blood at baseline (within 4 weeks prior to week 0) and week 208 and categorised as negative, trace, 1+, 2+ and 3+. Number of participants in each category at baseline and week 208 are presented. Missing values were imputed using the LOCF method.

  65. Change in Blood Coagulation Test (Prothrombin Time and APTT) [ Time Frame: Baseline, week 208 ]
    Change from baseline (within 4 weeks prior to week 0) in blood coagulation test parameters: prothrombin time and APTT. Missing values were imputed using the LOCF method.

  66. Change in ECG [ Time Frame: Baseline, week 208 ]
    The ECG was recorded after a 3-minute rest in supine position at baseline (within 4 weeks prior to week 0) and week 208 and categorised as normal, abnormal NCS or abnormal CS. Number of participants in each ECG category at baseline and week 208 are presented. Missing values were imputed using the LOCF method.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   3 Years to 10 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Japanese children with Noonan syndrome clinically diagnosed in one of the following ways: 1. Clinically diagnosed by at least two medical experts using van der Burgt score list, 2. Clinically diagnosed by one medical expert using van der Burgt score list and diagnosed by result of genetic testing for Noonan syndrome, 3. Clinically diagnosed by one medical expert using van der Burgt score list and diagnosed by the same medical expert based on the results of centralised evaluation of facial change using van der Burgt score list
  • Height SDS (standard deviation score): -2 SDS or below (according to the Japanese reference data)
  • Age: boys 3 to below 11 years, girls 3 to below 10 years
  • Height records must be available within the period between 40 and 64 weeks prior to Visit 1 (screening)
  • Prepubertal children (definition for girls breast and pubes of Tanner stage is I, and none of menses, and for boys testicular volume below 4 mL, and pubes and penis of Tanner stage is I)

Exclusion Criteria:

  • Children with known or suspected hypersensitivity against human growth hormone (hGH) or related products (including any components of the trial products)
  • Children with diabetic type diagnosed with the Japanese Diabetes Society Classification
  • Children with history or presence of active malignancy
  • Children who have received GH (growth hormone) treatment
  • Children who have received systemic administration of the following medications within two years prior to Visit 1 (screening): Thyroid hormone (except replacement therapy), antithyroid hormone, androgen, oestrogen, progesterone, anabolic steroid, adrenocortical steroid treatment period for at least 13 weeks), derivative of gonadotropin releasing hormone and somatomedin C (IGF-I)

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01927861


Locations
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Japan
Novo Nordisk Investigational Site
Asahikawa, Hokkaido, Japan, 078-8510
Novo Nordisk Investigational Site
Fukuoka, Japan, 812-8582
Novo Nordisk Investigational Site
Fukuoka, Japan, 830-0011
Novo Nordisk Investigational Site
Iruma-gun, Saitama, Japan, 350 0495
Novo Nordisk Investigational Site
Kanagawa, Japan, 216-8511
Novo Nordisk Investigational Site
Kanagawa, Japan, 232-8555
Novo Nordisk Investigational Site
Kyoto, Japan, 602-8566
Novo Nordisk Investigational Site
Maebashi-shi, Gunma, Japan, 371-8511
Novo Nordisk Investigational Site
Miyazaki, Japan, 889-1692
Novo Nordisk Investigational Site
Nagoya, Aichi, Japan, 467-8602
Novo Nordisk Investigational Site
Niigata-shi, Niigata, Japan, 951 8520
Novo Nordisk Investigational Site
Oita, Japan, 879-5593
Novo Nordisk Investigational Site
Osaka, Japan, 534-0021
Novo Nordisk Investigational Site
Osaka, Japan, 594-1101
Novo Nordisk Investigational Site
Saitama-city, Saitama, Japan, 336-8522
Novo Nordisk Investigational Site
Saitama-shi, Saitama, Japan, 330-8777
Novo Nordisk Investigational Site
Sapporo, Hokkaido, Japan, 065-8611
Novo Nordisk Investigational Site
Sendai-shi, Miyagi, Japan, 980 8574
Novo Nordisk Investigational Site
Shizuoka, Japan, 431-3192
Novo Nordisk Investigational Site
Tochigi, Japan, 329-0498
Novo Nordisk Investigational Site
Tokyo, Japan, 113-8519
Novo Nordisk Investigational Site
Tokyo, Japan, 113-8655
Novo Nordisk Investigational Site
Tokyo, Japan, 157 8535
Novo Nordisk Investigational Site
Tokyo, Japan, 160-8582
Novo Nordisk Investigational Site
Tokyo, Japan, 162-8666
Novo Nordisk Investigational Site
Tokyo, Japan, 183-8561
Novo Nordisk Investigational Site
Zentsuji, Kagawa, Japan, 765-8507
Sponsors and Collaborators
Novo Nordisk A/S
Investigators
Layout table for investigator information
Study Director: Global Clinical Registry (GCR, 1452) Novo Nordisk A/S
  Study Documents (Full-Text)

Documents provided by Novo Nordisk A/S:
Study Protocol  [PDF] November 26, 2018
Statistical Analysis Plan  [PDF] October 28, 2016


Additional Information:
Layout table for additonal information
Responsible Party: Novo Nordisk A/S
ClinicalTrials.gov Identifier: NCT01927861     History of Changes
Other Study ID Numbers: GHLIQUID-4020
U1111-1131-5892 ( Other Identifier: WHO )
JapicCTI-132336 ( Registry Identifier: JAPIC )
First Posted: August 23, 2013    Key Record Dates
Results First Posted: October 14, 2019
Last Update Posted: October 14, 2019
Last Verified: September 2019
Additional relevant MeSH terms:
Layout table for MeSH terms
Dwarfism
Noonan Syndrome
Genetic Diseases, Inborn
Syndrome
Disease
Pathologic Processes
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Endocrine System Diseases
Craniofacial Abnormalities
Musculoskeletal Abnormalities
Heart Defects, Congenital
Cardiovascular Abnormalities
Cardiovascular Diseases
Heart Diseases
Congenital Abnormalities
Connective Tissue Diseases