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Trial · Interventional

TerminatedPhase 3Results postedRegistry data

Neuromuscular Changes In Small For Gestational Age Children During Somatropin Therapy

Short name SGA-POWER

Overview

Status
Terminated
Phase
Phase 3
Enrollment
23 participants (actual)
Sponsor
Pfizer · Industry
Design
Randomized · Open label (no masking)
Countries
1 country

Germany

Start
Jul 2008
Primary completion
Mar 2011
Completion
Mar 2011
First posted
28 Feb 2008 (estimated)
Last update posted
29 Feb 2012 (estimated)
Conditions
Growth Hormone Therapy · Infant, Small for Gestational Age

Timeline

1 trial · 1 stopped early · 1 with posted results

  • Completed
  • Ongoing
  • Not yet recruiting
  • Stopped
  • Estimated dates (est.)
  • Results posted
20082009201020112012
  1. Ph 3

    SGA-POWER

    NCT00625872

    Jul 2008 to Mar 2011

    Terminated

Registry summary

Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.

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.

Interventions

  • SomatropinDrug
  • SomatropinDrug

Arms2

  • Active comparator

    Treatment Group

    Somatropin for 12 months

  • Other

    Control Group

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

Primary outcomes6

Outcomes the sponsor listed as primary.

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

    Time frame · Baseline and Month 6

    Registry description

    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.

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

    Time frame · Baseline and Month 6

    Registry description

    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.

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

    Time frame · Baseline and Month 6

    Registry description

    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.

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

    Time frame · Baseline and Month 6

    Registry description

    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.

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

    Time frame · Baseline and Month 6

    Registry description

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

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

    Time frame · Baseline and Month 6

    Registry description

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

Secondary outcomes(20)
  1. Change From Baseline in Intellectual Performance of Children Using Kaufmann-Assessment Battery for Children (K-ABC) Test Global Scales at Month 6

    Time frame · Baseline and Month 6

    Registry description

    K-ABC was assessed in children between 2.5-12.5 years. Comprised of 16 subtests; 10 mental processing (intelligence) and 6 achievement subtests. Achievement subtests: expressive vocabulary, faces\&places, arithmetic, riddles, reading/decoding, reading/comprehension. Sixteen subtests were weighted accordingly to form 5 global scales: sequential processing, simultaneous processing, achievement, non-verbal and mental processing composite. Scores were rated as upper extreme \[greater than (\>) 131\], above average (116-130), average (85-115), below average (70-84), lower extreme \[less than (\<) 69\].

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

    Time frame · Baseline, Month 12 and Month 18

    Registry description

    K-ABC was assessed in children between 2.5-12.5 years. Comprised of 16 subtests; 10 mental processing (intelligence) and 6 achievement subtests. Achievement subtests: expressive vocabulary, faces\&places, arithmetic, riddles, reading/decoding, reading/comprehension. Sixteen subtests were weighted accordingly to form 5 global scales: sequential processing, simultaneous processing, achievement, non-verbal and mental processing composite. Scores were rated as upper extreme \[greater than (\>) 131\], above average (116-130), average (85-115), below average (70-84), lower extreme \[less than (\<) 69\].

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

    Time frame · Baseline and Month 6

    Registry description

    The KITAP is a computer aided standardized neuro-cognitive development test which allows examination of a wide range of attention and executive functions such as shift of attention (Distractibility); simple reaction time (Alertness); "Sustained Attention", change of reaction (Flexibility); "Divided Attention", controlled reaction disposition (Go/No go) and "Vigilance". It has been designed appropriately for children between the age of 6 to 10 years to allow optimal motivation during testing and to increase validity of results.

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

    Time frame · Baseline, Month 12 and Month 18

    Registry description

    The KITAP is a computer aided standardized neuro-cognitive development test which allows examination of a wide range of attention and executive functions such as shift of attention (Distractibility); simple reaction time (Alertness); "Sustained Attention", change of reaction (Flexibility); "Divided Attention", controlled reaction disposition (Go/No go) and "Vigilance". It has been designed appropriately for children between the age of 6 to 10 years to allow optimal motivation during testing and to increase validity of results.

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

    Time frame · Baseline and Month 6

    Registry description

    NVLT was assessed for visual memorization that was difficult to verbalize. Test recorded instability index, T-scores\[sum of differences of correct {C} - incorrect {IC} "Yes" answers(1);sum of C "Yes" answers(2);sum of IC "Yes" answers(3);sum of differences of C-IC "Yes" answers with high associative items{ 87%-95%}(4);sum of differences of C-IC "Yes" answers with low associative items{ 54%-64%}(5); difference between difference values for high and low associative items(6)\].Scores were rated as below average(\<40), average(40-60), above average(\>60) and working time ranging between 9-12 minutes.

  6. Change From Baseline in Intellectual Performance of Children Using Non-verbal Learning Test (NVLT) at Months 12 and 18

    Time frame · Baseline, Month 12 and Month 18

    Registry description

    NVLT was assessed for visual memorization that was difficult to verbalize. Test recorded instability index, T-scores\[sum of differences of correct {C} - incorrect {IC} "Yes" answers(1);sum of C "Yes" answers(2);sum of IC "Yes" answers(3);sum of differences of C-IC "Yes" answers with high associative items{ 87%-95%}(4);sum of differences of C-IC "Yes" answers with low associative items{ 54%-64%}(5); difference between difference values for high and low associative items(6)\].Scores were rated as below average(\<40), average(40-60), above average(\>60) and working time ranging between 9-12 minutes.

  7. Change From Baseline in Intellectual Performance of Children Using Child Behavior Checklist 4-18 Years (CBCL 4-18) at Months 6, 12 and 18

    Time frame · Baseline, Month 6, Month 12 and Month 18

    Registry description

    CBCL was standardized for children ages 4 to 18 years and measured child internalizing and externalizing behaviors and total problems. The 4-18 years' checklist contains 140 questions and responses were recorded on a Likert scale: 0 = Not True, 1 = Somewhat or Sometimes True, 2 = Very True or Often True. The range of possible values was 0-280 (0=good to 280=worst).

  8. Change From Baseline in Peak Jump Power Standard Deviation Score (PJP-SDS; One-leg-jump) at Months 6, 12 and 18

    Time frame · Baseline, Month 6 , Month 12 and Month 18

    Registry description

    PJP was defined as the peak of the calculated power (force multiplied by velocity). It was measured by Leonardo Jumping Platform during one 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.

  9. Change From Baseline in Peak Jump Force Standard Deviation Score (PJF-SDS; One-leg-jump) at Months 6, 12 and 18

    Time frame · Baseline, Month 6, Month 12 and Month 18

    Registry description

    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 one leg jump. The SDS indicates how similar the participant was to the reference population.

  10. Change From Baseline in Maximum Jump Velocity (Vmax; One-leg-jump) at Months 6, 12 and 18

    Time frame · Baseline, Month 6, Month 12 and Month 18

    Registry description

    Vmax was measured by Leonardo Jumping Platform during one leg jump.

  11. Change From Baseline in Five-chair Rising Test- Peak Jump Power (PJP) at Month 6

    Time frame · Baseline and Month 6

    Registry description

    The Chair rising test is a performance test (total power output) to measure neuromuscular function of complex movement in standing up. Test allows diagnostics of movement deficits using Leonardo jump plate. Five stand up test: five repetitions of rising from a chair on jump plate as quickly as possible with arms crossed over the chest (time to perform the tasks, maximal PJP, maximal velocity and maximal PJF). PJP is defined as the peak of the calculated power (force multiplied by velocity).

  12. Change From Baseline in Five-chair Rising Test-Peak Jump Power (PJP) at Months 12 and 18

    Time frame · Baseline, Month 12 and Month 18

    Registry description

    The Chair rising test is a performance test (total power output) to measure neuromuscular function of complex movement in standing up. Test allows diagnostics of movement deficits using Leonardo jump plate. Five stand up test: five repetitions of rising from a chair on jump plate as quickly as possible with arms crossed over the chest (time to perform the tasks, maximal PJP, maximal velocity and maximal PJF). PJP is defined as the peak of the calculated power (force multiplied by velocity).

  13. Change From Baseline in Five-chair Rising Test-Peak Jump Force (PJF) at Month 6

    Time frame · Baseline and Month 6

    Registry description

    The Chair rising test is a performance test (total power output) to measure neuromuscular function of complex movement in standing up. Test allows diagnostics of movement deficits using Leonardo jump plate. Five stand up test: five repetitions of rising from a chair on jump plate as quickly as possible with arms crossed over the chest (time to perform the tasks, maximal PJP, maximal velocity and maximal PJF). PJF is the maximum force of the ascending part of the jump which the participant performed as a counter-movement jump with freely moving arms as high as possible with the head and chest.

  14. Change From Baseline in Five-chair Rising Test-Peak Jump Force (PJF) at Months 12 and 18

    Time frame · Baseline, Month 12 and Month 18

    Registry description

    The Chair rising test is a performance test (total power output) to measure neuromuscular function of complex movement in standing up. Test allows diagnostics of movement deficits using Leonardo jump plate. Five stand up test: five repetitions of rising from a chair on jump plate as quickly as possible with arms crossed over the chest (time to perform the tasks, maximal PJP, maximal velocity and maximal PJF). PJF is the maximum force of the ascending part of the jump which the participant performed as a counter-movement jump with freely moving arms as high as possible with the head and chest.

  15. Change From Baseline in Five-chair Rising Test-Maximum Jump Velocity (Vmax) at Month 6

    Time frame · Baseline and Month 6

    Registry description

    The Chair rising test is a performance test (total power output) to measure neuromuscular function of complex movement in standing up. Test allows diagnostics of movement deficits using Leonardo jump plate. Five stand up test: five repetitions of rising from a chair on jump plate as quickly as possible with arms crossed over the chest (time to perform the tasks, maximal PJP, maximal velocity and maximal PJF). Vmax is defined as the maximum jump velocity.

  16. Change From Baseline in Five-chair Rising Test-Maximum Jump Velocity (Vmax) at Months 12 and 18

    Time frame · Baseline, Month 12 and Month 18

    Registry description

    The Chair rising test is a performance test (total power output) to measure neuromuscular function of complex movement in standing up. Test allows diagnostics of movement deficits using Leonardo jump plate. Five stand up test: five repetitions of rising from a chair on jump plate as quickly as possible with arms crossed over the chest (time to perform the tasks, maximal PJP, maximal velocity and maximal PJF). Vmax is defined as the maximum jump velocity.

  17. Change From Baseline in Five-chair Rising Test (Time to Perform the Tasks) at Month 6

    Time frame · Baseline and Month 6

    Registry description

    Chair rising test is performance test (total power output) to measure neuromuscular function of complex movement in standing up. Test allows diagnostics of movement deficits using Leonardo jump plate. Five stand up test: 5 repetitions of rising from a chair on jump plate as quickly as possible with arms crossed over chest (time to perform tasks, maximal PJP, maximal velocity and maximal PJF). Time to perform task includes: Average (avg) rise time which is avg time to perform 1 rise, avg time per test is the avg time to perform 1 test (rise and sitting down) and total time to perform 5 tests.

  18. Change From Baseline in Five-chair Rising Test (Time to Perform the Tasks) at Months 12 and 18

    Time frame · Baseline, Month 12 and Month 18

    Registry description

    Chair rising test is performance test (total power output) to measure neuromuscular function of complex movement in standing up. Test allows diagnostics of movement deficits using Leonardo jump plate. Five stand up test: 5 repetitions of rising from a chair on jump plate as quickly as possible with arms crossed over chest (time to perform tasks, maximal PJP, maximal velocity and maximal PJF). Time to perform task includes: Average (avg) rise time which is avg time to perform 1 rise, avg time per test is the avg time to perform 1 test (rise and sitting down) and total time to perform 5 tests.

  19. Change From Baseline in One-chair Rising Test-Peak Jump Power (PJP) at Months 6, 12 and 18

    Time frame · Baseline, Month 6 , Month 12 and Month 18

    Registry description

    The Chair rising test is a performance test (total power output) to measure neuromuscular function of complex movement to stand up. Test allows diagnostics of movement deficits using Leonardo jump plate. One stand up test: rising from a chair on the jump plate as quickly as possible with arms crossed over the chest (analysis of time, PJP, PJF and time of fastest rising). PJP is defined as the peak of the calculated power (force multiplied by velocity).

  20. Change From Baseline in One-chair Rising Test-Peak Jump Force (PJF) at Months 6, 12 and 18

    Time frame · Baseline, Month 6 , Month 12 and Month 18

    Registry description

    The Chair rising test is a performance test (total power output) to measure neuromuscular function of complex movement to stand up. Test allows diagnostics of movement deficits using Leonardo jump plate. One stand up test: rising from a chair on the jump plate as quickly as possible with arms crossed over the chest (analysis of time, PJP, PJF and time of fastest rising). PJF is the maximum force of the ascending part of the jump which the participant performed as a counter-movement jump with freely moving arms as high as possible with the head and chest.

Posted results

Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.

Primary outcome results

Primary outcomeLeast squares mean· Baseline and Month 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

GroupValue (Watt/kilogram (W/kg))Spread
Somatropin-0.151.01
Control Arm0.280.51
Primary outcomeLeast squares mean· 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

GroupValue (W/kg)Spread
Somatropin-1.020.43
Control Arm0.590.27
Primary outcomeLeast squares mean· 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

GroupValue (Newtons)Spread
Somatropin-0.700.62
Control Arm-0.550.29
Secondary and other outcome results(5)
Secondary outcomeMean· Baseline and Month 6

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

GroupValue (Units on a scale)Spread
Somatropin25.78.35
Control Arm28.58.33
Secondary outcomeMean· Baseline, Month 12 and Month 18

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

GroupValue (Units on a scale)
Somatropinnot posted
Control Armnot posted
Secondary outcomeMean· Baseline and Month 6

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

GroupValue (Seconds)Spread
Somatropin554.3201.08
Control Arm514.5127.41
Secondary outcomeMean· 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 Months 12 and 18

GroupValue (Seconds)
Somatropinnot posted
Control Armnot posted
Secondary outcomeMean· Baseline and Month 6

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

GroupValue (Units on a scale)Spread
Somatropin0.30.15
Control Arm0.20.21

Adverse events, as posted

Adverse events as posted: participants affected out of participants at risk, by group
GroupDeathsSerious eventsOther events
SomatropinSomatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals.–0 / 1210 / 12
Control ArmNo treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.–0 / 117 / 11

Each cell reads participants affected / participants at risk, as the registry posted them.

Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works

Studies0

Studies that cite this trial.

No study cites this trial.

Eligibility

Who could join, as posted.

Sex
All
Age
from 6 Years up to 10 Years
Healthy volunteers
Not accepted
Inclusion and exclusion criteria, as posted
Inclusion Criteria: * 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). * Growth velocity SDS \< 0 during the last year before inclusion (Brandt/Reinken 1988). Exclusion Criteria: * 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.

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