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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

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Novo Nordisk A/S
ClinicalTrials.gov Identifier:
NCT01927861
First received: August 20, 2013
Last updated: May 5, 2017
Last verified: May 2017
  Purpose
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 Intervention Phase
Genetic Disorder
Noonan Syndrome
Drug: somatropin
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: 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

Resource links provided by NLM:


Further study details as provided by Novo Nordisk A/S:

Primary Outcome Measures:
  • Change in height SDS (Standard Deviation Score) [ Time Frame: Week 0, week 104 ]
  • Change in height SDS (Standard Deviation Score) [ Time Frame: Week 0, week 208 ]

Secondary Outcome Measures:
  • Incidence of treatment emergent adverse events (AEs) [ Time Frame: Weeks 0, Week 104 ]
  • Incidence of treatment emergent adverse events (AEs) [ Time Frame: Weeks 0, Week 208 ]
  • Change in IGF-I (insulin like growth factor I) [ Time Frame: Week 0, week 104 ]
  • Change in IGF-I (insulin like growth factor I) [ Time Frame: Week 0, week 208 ]
  • Change in HbA1c (Glycosylated haemoglobin) [ Time Frame: Week 0, week 104 ]
  • Change in HbA1c (Glycosylated haemoglobin) [ Time Frame: Week 0, week 208 ]

Enrollment: 51
Actual Study Start Date: August 19, 2013
Estimated Study Completion Date: July 5, 2018
Estimated Primary Completion Date: June 26, 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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.

  Eligibility

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)
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01927861

Locations
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, 232-8555
Novo Nordisk Investigational Site
Kawasaki-shi, Kanagawa, Japan, 216-8511
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
Study Director: Global Clinical Registry (GCR, 1452) Novo Nordisk A/S
  More Information

Additional 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 )
Study First Received: August 20, 2013
Last Updated: May 5, 2017

Additional relevant MeSH terms:
Noonan Syndrome
Syndrome
Dwarfism
Disease
Pathologic Processes
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Genetic Diseases, Inborn
Endocrine System Diseases
Craniofacial Abnormalities
Musculoskeletal Abnormalities
Heart Defects, Congenital
Cardiovascular Abnormalities
Cardiovascular Diseases
Heart Diseases
Congenital Abnormalities
Connective Tissue Diseases

ClinicalTrials.gov processed this record on May 25, 2017