Predictive Markers in GHD and TS Children Treated With SAIZEN®

This study has been completed.
Sponsor:
Information provided by:
Merck KGaA
ClinicalTrials.gov Identifier:
NCT00256126
First received: November 18, 2005
Last updated: March 26, 2014
Last verified: March 2014
  Purpose

The study aims at identifying the predictive markers after one month of Saizen therapy in Growth Hormone Deficiency (GHD) and Turner Syndrome children. The study will recruit approximately 360 children in several countries worldwide. The study lasts for about the first one month of daily growth hormone treatment. There will be three clinic visits during the month of the study. There is an initial visit, then a visit before growth hormone treatment starts and finally a visit at the fourth week of treatment. The study requires two additional blood tests to a regular Saizen treatment follow-up. One sample is taken before growth hormone injections start and one additional blood sample is taken at the fourth week of treatment.


Condition Intervention Phase
Growth Hormone Deficiency
Drug: Saizen
Phase 4

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase IV Open-label Study of Predictive Markers in Growth Hormone Deficient and Turner Syndrome Pre-pubertal Children Treated With SAIZEN®

Resource links provided by NLM:


Further study details as provided by Merck KGaA:

Primary Outcome Measures:
  • Changes in serum IGF-1 levels after one month in Growth Hormone Deficiency (GHD) and Turner Syndrome (TS) children [ Time Frame: After one month ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • The changes of IGBP-3 levels [ Time Frame: After one month ] [ Designated as safety issue: No ]
  • The changes of glycemia and insulinemia, insulin resistance (HOMA-IR analysis) [ Time Frame: After one month ] [ Designated as safety issue: No ]
  • The changes of alkaline phosphatase [ Time Frame: After one month ] [ Designated as safety issue: No ]

Enrollment: 318
Study Start Date: May 2005
Study Completion Date: October 2007
Primary Completion Date: October 2007 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 1 Drug: Saizen
blood sampling (10 ml) at baseline and one month (10 ml)

  Eligibility

Ages Eligible for Study:   2 Years to 16 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • One of the following diagnoses and candidacy for SAIZEN® therapy:

A)GHD: documented pre-established diagnosis of GHD with a GH peak response of <10 μg/L with 2 GH stimulation tests, without priming with oestradiol.

B)Turner syndrome: documented pre-established diagnosis by karyotype.

  • Prepubertal status according to Tanner Pre-established history of normal thyroid function or adequate substitution for at least 3 months.
  • Weight for stature within the population specific normal range (>5th and <95th percentiles) for gender Willingness and ability to comply with the protocol for the duration of the study.
  • Parent's or guardian's written informed consent, given before any study related procedure that is not part of the subject's normal medical care, with the understanding that the subject or parent/guardian may withdraw consent at any time without prejudice to future medical care. If the child is old enough to read and write, a separate assent form will be given.

Exclusion Criteria:

  • Acquired GHD due to central nervous system tumour, trauma, infection, infiltration (documented by imaging), and history of irradiation or cranial surgery
  • Previous treatment with GH, GHRH, anabolic steroids or any treatment affecting growth.
  • Previous treatment with corticosteroids, except in case of topical or inhaled corticosteroid administration for atopic disease. Corticosteroids for hormonal substitution are also allowed if the condition and the treatment regimen have been stable for at least 3 months.
  • Severe associated pathology affecting growth such as malnutrition, malabsorption, or bone dysplasia.
  • Chronic severe kidney disease.
  • Chronic severe liver disease.
  • Chronic infectious disease.
  • Acute or severe illness during the previous 6 months.
  • Significant concomitant illness that would interfere with participation or assessment in this study.
  • Active malignancy (except non-melanomatous skin malignancies that have undergone surgical excision and/or biopsy, diagnosis and treatment to resolution)
  • History or active Idiopathic intra-cranial hypertension (benign intracranial hypertension or pseudo-tumor cerebri).
  • Diabetes Mellitus type I & II.
  • Any autoimmune disease.
  • Previous screening failure in this study.
  • Use of an investigational drug or participation in another clinical study within the last three months.
  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: NCT00256126

Locations
Argentina
Local Medical Information Office
Buenos Aires, Argentina
Australia
Local Medical Information Office
Sydney, Australia
Austria
Local Medical Information Office
Vienna, Austria
Canada
Local Medical Information Office
Mississauga, Canada
France
Local Medical InformationOffice
Paris, France
Germany
Local Medical Information Office
Munich, Germany
Italy
Local Medical Information Office
Rome, Italy
Norway
Local Medical Information Office
Oslo, Norway
Russian Federation
Local Medical Information Office
Russia, Russian Federation
Singapore
Local Medical Information Office
Singapore, Singapore
Spain
Local Medical Information Office
Madrid, Spain
Sweden
Local Medical Information Office
Stockholm, Sweden
United Kingdom
Local Medical Information Office
Feltham, United Kingdom
Sponsors and Collaborators
Merck KGaA
Investigators
Study Chair: Clement Olivier Merck Serono International S.A., an affiliate of Merck KGaA, Darmstadt, Germany
  More Information

Additional Information:
Publications:
Responsible Party: Clement Olivier, Merck Serono International SA, an affiliate of Merck KGaA Darmstadt, Germany
ClinicalTrials.gov Identifier: NCT00256126     History of Changes
Other Study ID Numbers: 24531
Study First Received: November 18, 2005
Last Updated: March 26, 2014
Health Authority: United Kingdom: National Health Service

Additional relevant MeSH terms:
Dwarfism, Pituitary
Endocrine System Diseases
Dwarfism
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Bone Diseases, Endocrine
Hypopituitarism
Pituitary Diseases
Hypothalamic Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases

ClinicalTrials.gov processed this record on August 19, 2014