Predictive Markers in Chinese Growth Hormone Deficiency (GHD) Children Treated With Saizen®

NCT01187550 | Completed Phase 4 Results

• 1 other identifier: 27709
• Study Type: interventional
• Target: 214
• Locations: 0 countries
• Sites: N/A

Brief Summary

This is an open-label, prospective, multicentric, non-comparative, non-randomized Phase IV interventional study in which subjects pre-diagnosed with Growth Hormone Deficiency (GHD) were treated for 4 weeks with Saizen to compare the response between GHD children born appropriate for gestational age (AGA) and those born small for gestation age (SGA) after 4 weeks of Saizen therapy.

Conditions

Dwarfism, Pituitary

Keywords

Dwarfism, pituitary; Growth hormone

Outcome Measures

Primary Outcomes (1)

• Change From Baseline in Serum Insulin Like Growth Factor-1 Standard Deviation Score (IGF-1 SDS) Levels at Week 4

  Insulin Like Growth Factor-1 Standard Deviation Score (IGF-1 SDS) was calculated as logarithm (log) 10 actual value of IGF-1 - log 10 (mean reference value of IGF-1) divided by log10 reference standard deviation of IGF-1.

  Baseline and Week 4

Secondary Outcomes (5)

• Change From Baseline in Insulin Like Growth Factor Binding Protein-3 (IGFBP-3) Levels at Week 4

  Baseline and Week 4

• Change From Baseline in Fasting Glucose at Week 4

  Baseline and Week 4

• Change From Baseline in Fasting Insulin at Week 4

  Baseline and Week 4

• Change From Baseline in Homeostasis Model Assessment of Insulin Resistance (HOMA-IR) Test at Week 4

  Baseline and Week 4

• Change From Baseline in Lipid Profile at Week 4

  Baseline and Week 4

Interventions

Recombinant human growth hormone (r-hGH) DRUG

Recombinant human growth hormone (r-hGH) administered at dose of 0.033 milligram/kilogram (mg/kg) body weight (0.1 International Unit \[IU\]/kg body weight) per day by subcutaneous injection.

Also known as: Saizen

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Male and female subjects with documented pre-established diagnosis of GHD with a GH peak response of \<10 microgram/liter (mcg/L) with 2 GH stimulation tests, without priming with estradiol
• Subjects with SGA defined as birth weight and/or length at least 2 standard deviations (SDs) below the mean for gestational age
• Subjects with prepubertal status according to Tanner
• Subjects with pre-established history of normal thyroid function or adequate substitution for at least 3 months
• Subjects with weight for stature within the population specific normal range (\>5th and \<95th percentiles) for gender
• Subjects with willingness and ability to comply with the protocol for the duration of the study
• Subjects whose parents or guardians written informed consent given before any study-related procedure that was not part of the subjects normal medical care, with the understanding that the subject or parent/guardian might withdraw consent at any time without prejudice to future medical care. If the child was old enough to read and write, a separate assent form was given

You may not qualify if:

• Subjects who acquired GHD due to central nervous system tumor, trauma, infection, infiltration (documented by imaging), and history of irradiation or cranial surgery
• Subjects with previous treatment with GH, growth hormone releasing hormone (GHRH), anabolic steroids or any treatment affecting growth
• Subjects who had previous treatment with corticosteroids, except in case of topical or inhaled corticosteroid administration for atopic disease. Corticosteroids for hormonal substitution were also allowed if the condition and the treatment regimen had been stable for at least 3 months
• Subjects with severe associated pathology affecting growth such as malnutrition, malabsorption, or bone dysplasia
• Subjects with chronic severe kidney disease
• Subjects with chronic severe liver disease
• Subjects with chronic infectious disease
• Subjects with acute or severe illness during the previous 6 months
• Subjects with significant concomitant illness that would interfere with participation or assessment in this study
• Subjects who had active malignancy (except non-melanomatous skin malignancies that had undergone surgical excision and/or biopsy, diagnosis and treatment to resolution)
• Subjects with history or active idiopathic intra-cranial hypertension (benign intracranial hypertension or pseudo-tumor cerebri)
• Subjects with diabetes mellitus type I \& II
• Subjects with any autoimmune disease
• Subjects who had previous screening failure in this study
• Subjects who had used an investigational drug or participated in another clinical study within the last 3 months

Sponsors & Collaborators

• Merck KGaA, Darmstadt, Germany: lead
• Merck Serono Co., Ltd., China: collaborator

MeSH Terms

Conditions

Dwarfism, Pituitary

Interventions

Growth Hormone; Human Growth Hormone

Condition Hierarchy (Ancestors)

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; Endocrine System Diseases

Intervention Hierarchy (Ancestors)

Pituitary Hormones, Anterior; Pituitary Hormones; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Peptides; Amino Acids, Peptides, and Proteins

Results Point of Contact

• Title: Merck KGaA Communication Center
• Organization: Merck Serono, a division of Merck KGaA

service@merckgroup.com

+49-6151-72-5200

Study Officials

• Medical Responsible

  Merck Serono Co., Ltd., China

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 22, 2010
• First Posted: August 24, 2010
• Study Start: March 1, 2007
• Primary Completion: September 1, 2008
• Study Completion: April 1, 2009
• Last Updated: February 13, 2014
• Results First Posted: August 7, 2012

Record last verified: 2014-01