Trial record 12 of 40 for:    Open Studies | "Dwarfism"

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

This study is currently recruiting participants. (see Contacts and Locations)
Verified April 2014 by Novo Nordisk A/S
Sponsor:
Information provided by (Responsible Party):
Novo Nordisk A/S
ClinicalTrials.gov Identifier:
NCT01927861
First received: August 20, 2013
Last updated: April 29, 2014
Last verified: April 2014
  Purpose

This trial is conducted in Asia. The aim of this 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
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, 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 ] [ Designated as safety issue: No ]
  • Change in height SDS (Standard Deviation Score) [ Time Frame: Week 0, week 208 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Incidence of treatment emergent adverse events (AEs) [ Time Frame: Weeks 0, Week 104 ] [ Designated as safety issue: No ]
  • Incidence of treatment emergent adverse events (AEs) [ Time Frame: Weeks 0, Week 208 ] [ Designated as safety issue: No ]
  • Change in IGF-I (insulin like growth factor I) [ Time Frame: Week 0, week 104 ] [ Designated as safety issue: No ]
  • Change in IGF-I (insulin like growth factor I) [ Time Frame: Week 0, week 208 ] [ Designated as safety issue: No ]
  • Change in HbA1c (Glycosylated haemoglobin) [ Time Frame: Week 0, week 104 ] [ Designated as safety issue: No ]
  • Change in HbA1c (Glycosylated haemoglobin) [ Time Frame: Week 0, week 208 ] [ Designated as safety issue: No ]

Estimated Enrollment: 48
Study Start Date: August 2013
Estimated Study Completion Date: January 2019
Estimated Primary Completion Date: January 2017 (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 11 Years
Genders Eligible for Study:   Both
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

Contacts
Contact: Novo Nordisk clinicaltrials@novonordisk.com

Locations
Japan
Recruiting
Saitama-city, Saitama, Japan, 336-8522
Sponsors and Collaborators
Novo Nordisk A/S
Investigators
Study Director: Global Clinical Registry (GCR, 1452) Novo Nordisk A/S
  More Information

Additional Information:
No publications provided

Responsible Party: Novo Nordisk A/S
ClinicalTrials.gov Identifier: NCT01927861     History of Changes
Other Study ID Numbers: GHLIQUID-4020, U1111-1131-5892
Study First Received: August 20, 2013
Last Updated: April 29, 2014
Health Authority: Japan: Ministry of Health, Labor and Welfare

Additional relevant MeSH terms:
Dwarfism
Genetic Diseases, Inborn
Noonan Syndrome
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

ClinicalTrials.gov processed this record on July 29, 2014