Phase 3
Completed N=51
Investigating the Long-term Efficacy and Safety of Two Doses of NN-220 (Somatropin) in Short Stature Due to Noonan Syndrome
Genetic Disorder · Noonan Syndrome
Source: ClinicalTrials.gov NCT01927861 ↗
Enrolled (actual)
51
Serious AEs
37.3%
Results posted
Oct 2019
Primary outcomePrimary: Change in Height SDS (Japanese National Reference Data) — 0.84; 1.47 Standard deviation score — p=< 0.0001
◆ Published Evidence
Established
24citations · ~4 / year
Long-term efficacy and safety of two doses of Norditropin<sup>®</sup> (somatropin) in Noonan syndrome: a 4-year randomized, double-blind, multicenter trial in Japanese patients.
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.
Linked Publications (3)
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Long-term efficacy and safety of two doses of Norditropin<sup>®</sup> (somatropin) in Noonan syndrome: a 4-year randomized, double-blind, multicenter trial in Japanese patients.
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Efficacy and safety of two doses of Norditropin<sup>®</sup> (somatropin) in short stature due to Noonan syndrome: a 2-year randomized, double-blind, multicenter trial in Japanese patients.
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Long-term effectiveness and safety of daily growth hormone therapy in Japanese children with Noonan syndrome: a post-marketing surveillance study.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change in Height SDS (Japanese National Reference Data) |
0.85; 1.84 | — |
| SECONDARY Height Velocity SDS |
-0.73; 0.92 | — |
| SECONDARY Height Velocity |
5.64; 6.11 | — |
| SECONDARY Incidence of Treatment Emergent Adverse Events |
265; 306 | — |
| SECONDARY Change in IGF-I (Insulin-like Growth Factor-I) |
90.4; 159.1 | — |
| SECONDARY Change in HbA1c (Glycosylated Haemoglobin) |
0.14; 0.13 | — |
| SECONDARY Change in Clinical Laboratory Tests (Haematology: Erythrocytes) |
0.121; 0.095 | — |
| SECONDARY Change in Clinical Laboratory Tests (Haematology: Leukocytes and Thrombocytes) |
-1.50; -1.50; -27.5; -56.6 | — |
| SECONDARY Change in Clinical Laboratory Tests (Haematology: Haemoglobin) |
0.46; 0.46 | — |
| SECONDARY Change in Clinical Laboratory Tests (Haematology: Haematocrit) |
1.24; 1.26 | — |
| SECONDARY Change in Clinical Laboratory Tests (Haematology: Neutrophils) |
-1.23; 4.88 | — |
| SECONDARY Change in Clinical Laboratory Tests (Haematology: Lymphocytes) |
-0.14; -5.70 | — |
| SECONDARY Change in Clinical Laboratory Tests (Haematology: Monocytes) |
0.08; 0.56 | — |
| SECONDARY Change in Clinical Laboratory Tests (Haematology: Eosinophils) |
1.30; 0.42 | — |
| SECONDARY Change in Clinical Laboratory Tests (Haematology: Basophils) |
-0.01; -0.12 | — |
| SECONDARY Change in Clinical Laboratory Tests (Lipids: Total Cholesterol, LDL Cholesterol and HDL Cholesterol) |
0.031; -0.110; -0.005; -0.173; 0.036; 0.056 | — |
| SECONDARY Change in Clinical Laboratory Tests (Biochemistry: AST, ALT, r-GTP and Alkaline Phosphatase) |
-5.4; -7.4; -1.28; -1.81; 1.0; -3.5 | — |
| SECONDARY Change in Clinical Laboratory Tests (Biochemistry: Total Protein) |
0.02; 0.05 | — |
| SECONDARY Change in Clinical Laboratory Tests (Biochemistry: Blood Urea Nitrogen, Sodium, Potassium, Chloride, Total Calcium and Phosphorus) |
-0.10; -0.15; 0.9; 1.4; -0.13; -0.09 | — |
| SECONDARY Change in Clinical Laboratory Tests (Biochemistry: Creatinine) |
5.5; 6.7 | — |
| SECONDARY Change in Glucose Tolerance (AUC of Glucose) Based on the OGTT |
1.03; 1.10 | — |
| SECONDARY Change in Glucose Tolerance (AUC of Insulin) Based on the OGTT |
1.52; 2.38 | — |
| SECONDARY Change in Bone Age |
3.89; 4.68 | — |
| SECONDARY Change in Bone Age/Chronological Age |
0.049; 0.149 | — |
| SECONDARY Yearly Change in Bone Age/Change in Chronological Age |
0.87; 1.05 | — |
| SECONDARY Change in Vital Signs (Diastolic Blood Pressure and Systolic Blood Pressure) |
5.8; 3.5; 6.8; 5.8 | — |
| SECONDARY Change in Vital Signs (Pulse) |
-15.3; -3.8 | — |
| SECONDARY Change in Urinalysis (Protein, Glucose and Occult Blood) |
23; 22; 2; 4; 0; 0 | — |
| SECONDARY Change in Blood Coagulation Test (Prothrombin Time and APTT) |
0.70; 1.20; 0.70; 0.80 | — |
| SECONDARY Change in ECG |
15; 14; 10; 12; 0; 0 | — |
| SECONDARY Change in Height SDS (Japanese National Reference Data) |
0.85; 1.84 | — |
| SECONDARY Change in Height SDS (Noonan Syndrome Reference Data in Japanese) |
0.96; 1.92 | — |
| SECONDARY Incidence of Treatment Emergent AEs |
507; 539 | — |
| SECONDARY Change in IGF-I |
133.5; 217.2 | — |
| SECONDARY Change in HbA1c |
0.19; 0.20 | — |
Eligibility 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)
Data sourced from ClinicalTrials.gov (NCT01927861) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.