A Trial to Evaluate the Correlation Between Spontaneous Catch-up Growth, Clinical Response to Saizen (Recombinant Human Growth Hormone, r-hGH) and Gene Expression Profiling in Children Small for Gestational Age (SGA)

NCT01067352 | Terminated Phase 3 Results

• 1 other identifier: IMP23681
• Study Type: interventional
• Target: 25
• Locations: 1 country
• Sites: 1

Brief Summary

This open, multicentric, randomized, controlled study is planned to evaluate the correlation between gene expression, spontaneous catch-up growth and therapeutic response to Saizen in SGA children.

Conditions

Infant, Small for Gestational Age

Keywords

Infant, small for gestational age; Growth hormone; Saizen

Outcome Measures

Primary Outcomes (1)

• Correlation Between Gene Expression Profiling and Catch-up Growth in Small for Gestational Age (SGA) Children

  Gene expression profiling:analysis of ribonucleic acid (RNA) extracted from body tissue or fluids using Clontech Atlas Human Array to study level of activation of genes in tissue analyzed. Analysis was performed to identify possible correlation between catch-up growth (either spontaneous or drug-induced after Week 48) and therapeutic response to rhGH. Spontaneous catch up growth:shown by SGA participants having length more than third percentile at Week 96 without any treatment;drug induced growth was by SGA participants having length more than third percentile at Week 96 with drug treatment.

  Baseline and Week 48

Secondary Outcomes (2)

• Percentage of Untreated Participants Who Showed a Spontaneous Catch-up Growth

  Baseline through Week 96

• Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and AEs Leading to Study Drug Discontinuation

  Baseline through Week 96

Study Arms (3)

Group A (A1)

EXPERIMENTAL

Participants were allocated to Group A if were still third percentile for height (according to the Tanner reference table) at the age of 4-6 years. Group A would be then randomized to receive Saizen at the daily dose of 0.035 milligram (mg)/kilogram (kg) (Group A1) or no treatment (Group A2) for two years.

Drug: Recombinant human growth hormone (r-hGH)

Group A (A2)

NO INTERVENTION

Participants were allocated to Group A if were still third percentile for height (according to the Tanner reference table) at the age of 4-6 years. Group A would be then randomized to receive no treatment (Group A2) for two years.

Group B

NO INTERVENTION

Participants were allocated to Group B being third percentile (thus showing a spontaneous catch-up growth).

Interventions

Recombinant human growth hormone (r-hGH) DRUG

Recombinant human GH were administered subcutaneously (s.c) at the daily dose of 0.067 mg/kg of body weight to Group A1.

Also known as: Saizen

Group A (A1)

Eligibility Criteria

• Age: 4 Years - 6 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• SGA at birth (defined as a length at birth equal or below the tenth percentile according to the Italian reference table of Bertini and Fabris)
• Age of 24 Months
• Caucasic
• Born at term (i.e. after the 37th completed week of gestation)
• Height equal or below (Group A) or up (Group B) the third percentile at the age of 24 months according to the Tanner reference table
• Sufficient GH secretion (more than 10 nanogram (ng)/milliliter (ml)) at least to one of the tests commonly used at that age (glucagon, Levo-dopa, arginine, clonidine, Growth Hormone Releasing Hormone (GHRH), GH integrated secretion)
• Normal level of Thyroid-stimulating hormone (THS), Free Triiodothyronine (FT3), Free Thyroxine (FT4), Insulin-like growth factor 1(IGF-1), insulin and haemoglobin A1c (HbA1c)
• Normal level of Immunoglobulin A (IgA)
• Children parents willing to comply with the protocol for the whole duration of the study
• A written Informed Consent before the baseline visit must be obtained from the parent(s) / legal guardian(s)

You may not qualify if:

• Congenital malformations (including Silver-Russel syndrome)
• Known abnormal karyotype, especially in girls
• Twins
• Severe psychomotor retardation
• Previous or ongoing treatment with anabolic steroids or r-hGH
• Treatments interfering with the immune system (including bacterial lysate)
• Severe chronic illnesses
• Autoimmune diseases

Sponsors & Collaborators

• Merck KGaA, Darmstadt, Germany: lead
• Merck Serono S.P.A., Italy: collaborator

Study Sites (1)

Merck Serono S.p.A.

Roma, Italy

Location

MeSH Terms

Interventions

Growth Hormone; Human Growth Hormone

Intervention Hierarchy (Ancestors)

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

Limitations and Caveats

The primary and secondary efficacy objectives were not met because of poor participant enrollment and no quality RNA samples obtained to evaluate (RNA degradation in nearly all of the blood samples).

Results Point of Contact

• Title: Medical Responsible
• Organization: Merck Serono S.p.A., Italy, an affiliate of Merck KGaA, Darmstadt, Germany

service@merckgroup.com

+49-6151-72-5200

Study Officials

• Medical Responsible

  Merck Serono S.P.A., Italy

  STUDY DIRECTOR

Publication Agreements

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

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 10, 2010
• First Posted: February 11, 2010
• Study Start: February 1, 2004
• Primary Completion: July 1, 2009
• Study Completion: July 1, 2009
• Last Updated: December 27, 2013
• Results First Posted: December 14, 2011

Record last verified: 2013-12

Locations

IT (1)