Neuromuscular Changes In Small For Gestational Age Children During Somatropin Therapy

NCT00625872 | Terminated Phase 3 Results

• 1 other identifier: A6281283
• Study Type: interventional
• Target: 23
• Locations: 1 country
• Sites: 10

Brief Summary

The planned study focuses on the effect of a one year Somatropin treatment (0.035 mg/kg/d or 0.067 mg/kg/d) in short children born SGA on neuromuscular function and cognitive performance.

Conditions

Growth Hormone Therapy; Infant, Small for Gestational Age

Outcome Measures

Primary Outcomes (6)

• Change From Baseline in Peak Jump Power Standard Deviation Score (PJP-SDS; Two-leg-jump) in Full Analysis Set (FAS) Population at Month 6

  Peak jump power (PJP) was defined as the peak of the calculated power (force multiplied by velocity). It was measured by Leonardo Jumping Platform during two-leg jump. The participant performs 3 jumps and the highest peak (PJP) of the 3 recordings was selected for further calculations. The SDS indicates how similar the participant was to the reference population.

  Baseline and Month 6

• Change From Baseline in Peak Jump Power Standard Deviation Score (PJP-SDS; Two-leg-jump) in Per Protocol (PP) Population at Month 6

  PJP was defined as the peak of the calculated power (force multiplied by velocity). It was measured by Leonardo Jumping Platform during two-leg jump. The participant performs 3 jumps and the highest peak (PJP) of the 3 recordings was selected for further calculations. The SDS indicates how similar the participant was to the reference population.

  Baseline and Month 6

• Change From Baseline in Peak Jump Force Standard Deviation Score (PJF-SDS; Two-leg-jump) in Full Analysis Set (FAS) Population at Month 6

  PJF was defined as the maximum of force of the ascending part of the jump which the participant performed as a counter-movement jump with freely moving arms and as high as possible with the head and chest. It was measured by Leonardo Jumping Platform during two-leg jump. The SDS indicates how similar the participant was to the reference population.

  Baseline and Month 6

• Change From Baseline in Peak Jump Force Standard Deviation Score (PJF-SDS; Two-leg-jump) in Per Protocol (PP) Population at Month 6

  PJF was defined as the maximum of force of the ascending part of the jump which the participant performed as a counter-movement jump with freely moving arms and as high as possible with the head and chest. It was measured by Leonardo Jumping Platform during two-leg jump. The SDS indicates how similar the participant was to the reference population.

  Baseline and Month 6

• Change From Baseline in Maximum Jump Velocity (Vmax; Two-leg-jump) in Full Analysis Set (FAS) Population at Month 6

  Vmax was measured by Leonardo Jumping Platform during two-leg jump.

  Baseline and Month 6

• Change From Baseline in Maximum Jump Velocity (Vmax; Two-leg-jump) in Per Protocol (PP) Population at Month 6

  Vmax was measured by Leonardo Jumping Platform during two-leg jump.

  Baseline and Month 6

Secondary Outcomes (44)

• Change From Baseline in Intellectual Performance of Children Using Kaufmann-Assessment Battery for Children (K-ABC) Test Global Scales at Month 6

  Baseline and Month 6

• Change From Baseline in Intellectual Performance of Children Using Kaufmann-Assessment Battery for Children (K-ABC) Test Global Scales at Months 12 and 18

  Baseline, Month 12 and Month 18

• Change From Baseline in Intellectual Performance of Children Using Kinderversion Der Testbatterie Zur Aufmerksamkeitsprüfung für Kinder (KITAP) Test at Month 6

  Baseline and Month 6

• Change From Baseline in Intellectual Performance of Children Using Kinderversion Der Testbatterie Zur Aufmerksamkeitsprüfung für Kinder (KITAP) Test at Months 12 and 18

  Baseline, Month 12 and Month 18

• Change From Baseline in Intellectual Performance of Children Using Non-verbal Learning Test (NVLT) at Month 6

  Baseline and Month 6

Other Outcomes (3)

• Change From Baseline in Bone Density Using Peripheral Quantitative Computed Tomography (pqCT) at 6 or 12 or 18 Months

  Baseline, Month 6, Month 12 and Month 18

• Change From Baseline in Bone Structure Using Peripheral Quantitative Computed Tomography (pqCT) at 6 or 12 or 18 Months

  Baseline, Month 6, Month 12 and Month 18

• Change From Baseline in Bone Stability Using Peripheral Quantitative Computed Tomography (pqCT) at 6 or 12 or 18 Months

  Baseline, Month 6, Month 12 and Month 18

Study Arms (2)

Treatment Group

ACTIVE COMPARATOR

Somatropin for 12 months

Drug: Somatropin

Control Group

OTHER

In the first 6 months no intervention, afterwards Somatropin for 12 months

Drug: Somatropin

Interventions

Somatropin DRUG

Patients will be randomized at baseline in a 1:1 ratio into treatment group or control group. After six months the control group will undergo GH therapy with a higher dose of 0.067 mg/kg/day, too. All patients are treated with growth hormone for 12 months.

Treatment Group

Eligibility Criteria

• Age: 6 Years - 10 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Pre-pubertal boys between 6 and 10 years of age or girls between 6 and 9 years of age.
• Birth length- and/or birth weight-SDS adjusted to gestational age \< -2.0 (Voigt et al. 2002, Voigt et al. 2006 or Lawrence et al. 1989).
• Current height-SDS \< -2.5 (Brandt/Reinken 1992) and parental adjusted height-SDS below -1 (Hermanussen and Cole 2003).

You may not qualify if:

• Severe SGA (birth weight or length \< -4 SD) and clinically relevant dysmorphic features.
• Severe pre-maturity (GA \< 32 weeks of gestation).
• Severe perinatal complications like asphyxia, sepsis, necrotizing enterocolitis (NEC), respiratory distress syndrome, if associated with long-term sequelae (like short bowel syndrome, bronchopulmonary dysplasia (BPD), cerebral palsy etc).
• Inability to perform one- or two leg jumps from a standing position.
• Prior GH treatment.

Sponsors & Collaborators

• Pfizer: lead

Study Sites (10)

Pfizer Investigational Site

Berlin, 13353, Germany

Location

Pfizer Investigational Site

Bonn, 53113, Germany

Location

Pfizer Investigational Site

Cologne, 50937, Germany

Location

Pfizer Investigational Site

Datteln, 45711, Germany

Location

Pfizer Investigational Site

Dresden, 01307, Germany

Location

Pfizer Investigational Site

Erlangen, 91054, Germany

Location

Pfizer Investigational Site

Essen, 45122, Germany

Location

Pfizer Investigational Site

Heidelberg, 69120, Germany

Location

Pfizer Investigational Site

Kiel, 24105, Germany

Location

Pfizer Investigational Site

München, 80337, Germany

Location

Related Links

• To obtain contact information for a study center near you, click here.

MeSH Terms

Interventions

Human Growth Hormone

Intervention Hierarchy (Ancestors)

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

Limitations and Caveats

The study was prematurely discontinued, therefore not all data was powered.

Results Point of Contact

• Title: Pfizer ClinicalTrials.gov Call Center
• Organization: Pfizer, Inc.

ClinicalTrials.gov_Inquiries@pfizer.com

1-800-718-1021

Study Officials

• Pfizer CT.gov Call Center

  Pfizer

  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 19, 2008
• First Posted: February 28, 2008
• Study Start: July 1, 2008
• Primary Completion: March 1, 2011
• Study Completion: March 1, 2011
• Last Updated: February 29, 2012
• Results First Posted: February 29, 2012

Record last verified: 2012-01

Locations

DE (10)