Predictive Value of the Insulin-like Growth Factor-1 (IGF-1) Generation Test for the Growth Response to Growth Hormone Treatment (PRED-IGF)

This study has been withdrawn prior to enrollment.
(Participant enrollment was not feasible)
Sponsor:
Information provided by (Responsible Party):
Ipsen
ClinicalTrials.gov Identifier:
NCT01438801
First received: August 24, 2011
Last updated: December 19, 2013
Last verified: December 2013
  Purpose

The evaluation of a standardized diagnostic test to predict the growth response in a 1 year trial with Growth Hormone (GH) treatment (carried out in the context of regular patient care) in non GH deficient short children with low serum insulin-like growth factor-1 (IGF-1).


Condition Intervention Phase
Idiopathic Short Stature
Drug: Nutropin [Somatropin (rDNA origin) for injection]
Phase 4

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Diagnostic
Official Title: Predictive Value of Baseline and Stimulated Serum IGF-1 and IGFBP-3 During a Dose-escalation IGF-1 Generation Test for the 1 Year Growth Response to Growth Hormone (GH) Therapy in Short Children With Low IGF-1 and a Normal GH Peak in a Provocation Test

Resource links provided by NLM:


Further study details as provided by Ipsen:

Primary Outcome Measures:
  • Peak serum IGF-1 level with GH (1.4 mg/m2/day) change from baseline. [ Time Frame: Week 2 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Delta IGF-1 SDS on 0.7 & 1.4 mg GH/m2/day [ Time Frame: Baseline, week 2 ] [ Designated as safety issue: No ]
  • Delta insulin-like growth factor binding protein-3 (IGFBP-3) standard deviation score (SDS) on 0.7 & 1.4 mg GH/m2/day [ Time Frame: Baseline, week 2 ] [ Designated as safety issue: No ]
  • Delta of the ratio IGF-1:IGFBP-3 SDS on 0.7 mg & 1.4 mg GH/m2/day [ Time Frame: Baseline, week 2 ] [ Designated as safety issue: No ]
  • Delta of the ratio IGF-1:IGFBP-2 (insulin-like growth factor binding protein-2) SDS on 0.7 mg & 1.4 mg GH/m2/day [ Time Frame: Baseline, week 2 ] [ Designated as safety issue: No ]
  • Peak IGFBP-3 SDS on 0.7 mg & 1.4 mg GH/m2/day [ Time Frame: Baseline, week 2 ] [ Designated as safety issue: No ]
  • Peak of the ratio IGF-1:IGFBP-3 SDS on 0.7 mg & 1.4 mg GH/m2/day [ Time Frame: Baseline, week 2 ] [ Designated as safety issue: No ]
  • Peak of the ratio IGF-1:IGFBP-2 SDS on 0.7 mg & 1.4 mg GH/m2/day [ Time Frame: Baseline, week 2 ] [ Designated as safety issue: No ]

Enrollment: 0
Study Start Date: May 2013
Study Completion Date: July 2013
Primary Completion Date: July 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: NutropinAq Drug: Nutropin [Somatropin (rDNA origin) for injection]
Doses in the diagnostic protocol are 0.7 and 1.4 mg/m2/day, injected by subcutaneous injection at bedtime for 2 weeks, divided by washout periods of preferably 4 weeks (accepted range 4-6 weeks). An additional period of 2 weeks on 2.8 mg/m2/day (after a washout period of 4-6 weeks) will be added if the IGF-I response on either dosage is insufficient (serum IGF-I SDS <0).

  Eligibility

Ages Eligible for Study:   2 Years to 10 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Age 2.0 - 9.0 years for females, 2.0 - 10.0 yrs for males.
  • Prepubertal stage (Tanner 1 for breast (B1) in females, or Tanner 1 for genitals (G1) for males).
  • Bone age < 10 'years' (males) or < 9 'years' (females) according to Greulich and Pyle. The bone age will be read by the pediatric endocrinologist responsible for the pre-study screening visit.
  • Height SDS < -2.5 for ethnically adequate references. For children of Dutch or Western European origin the 1997 nation-wide references for Dutch children will be used. For children of Moroccan or Turkish origin, the respective reference charts will be used. For children of other ethnicities, the 1977 North American (NCHS/WHO) reference will be used, as these charts have been accepted by WHO as world-wide standard from the age of 5 years.

Exclusion Criteria:

  • Has a history of hypersensitivity to growth hormone or phenol (conservative added to GH in NutropinAq), or drugs with a similar chemical structure.
  • Has abnormal baseline findings, any other medical condition(s) or laboratory findings that, in the opinion of the Investigator, might jeopardise the subject's safety or decrease the chance of obtaining satisfactory data needed to achieve the objective(s) of the study.
  • Has a birth weight and/or length below -2 SDS for Swedish reference charts. Patients will not be excluded due to an unknown birth weight or length.
  • Has a known cause of short stature, or any significant concomitant disease that is likely to interfere with growth or with the study schedule/objectives, or is a known contraindication to GH treatment.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01438801

Sponsors and Collaborators
Ipsen
Investigators
Study Director: Gino Ciotti, MD Ipsen
  More Information

No publications provided

Responsible Party: Ipsen
ClinicalTrials.gov Identifier: NCT01438801     History of Changes
Other Study ID Numbers: A-95-58035-017
Study First Received: August 24, 2011
Last Updated: December 19, 2013
Health Authority: Netherlands: The Central Committee on Research Involving Human Subjects (CCMO)

Additional relevant MeSH terms:
Dwarfism
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Genetic Diseases, Inborn
Endocrine System Diseases

ClinicalTrials.gov processed this record on April 20, 2014