A Study to Evaluate Growth in Participants Treated With Somatropin (Nutropin) Using NuSpin Device

A Phase IV, Open Label, Multicenter, Case-controlled Study of Growth in Patients Using the Nutropin AQ® Nuspin®

This is a Phase IV, open label, case-controlled, observational study to evaluate the age-adjusted cumulative height velocity in pre-pubertal isolated growth hormone deficient (IGHD) and idiopathic short stature (ISS) participants receiving daily doses of somatropin (recombinant human growth hormone [rhGH]; Nutropin) using NuSpin device compared to historical controls from the national cooperative growth study (NCGS).

Study Overview

• Status: Terminated
• Conditions: Growth Hormone Deficiency
• Intervention / Treatment: Device: NuSpin; Drug: Somatropin

• Study Type: Observational
• Enrollment (Actual): 18

Contacts and Locations

Study Locations

• United States
  • Alabama
    • Birmingham, Alabama, United States, 35233
  • Arizona
    • Scottsdale, Arizona, United States, 85258
  • California
    • Sacramento, California, United States, 95819
    • San Diego, California, United States, 92123
  • Colorado
    • Greenwood Village, Colorado, United States, 80111
  • Florida
    • Palm Bay, Florida, United States, 32905
    • Pembrook Pines, Florida, United States, 33028
  • Georgia
    • Atlanta, Georgia, United States, 30342
  • Illinois
    • Wheaton, Illinois, United States, 60187
  • Minnesota
    • Minneapolis, Minnesota, United States, 55455
  • New York
    • Mount Kisco, New York, United States, 10549
    • New Rochelle, New York, United States, 10801
  • Ohio
    • Columbus, Ohio, United States, 43235
  • Tennessee
    • Knoxville, Tennessee, United States, 37916

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years to 14 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Somatropin-naive pre-pubertal participants with growth hormone deficiency or ISS

Description

Inclusion Criteria:

• Bone age less than or equal to (</=) 8 years (females) or </= 10 years (males) as determined by x-ray of the left hand and wrist obtained within six months before enrollment
• Prepubertal males and females by physical exam
• Naive to rhGH therapy
• Diagnosis of IGHD or ISS by standard pharmacologic testing and no other discernable etiology for short stature
• Height standard deviation score (Ht SDS) </= -1.5 (</= 5th percentile) for IGHD participants; Ht SDS </= -2.25 (</= 1.2 percentile) for ISS participants

Exclusion Criteria:

• Short stature etiologies other than IGHD or ISS
• Participants receiving chronic corticosteroid therapy (greater than [>] 3 months) for other medical conditions
• Participants with active malignancy or any other condition that the investigator believes would pose a significant hazard to the participant if rhGH were initiated
• Females with turner syndrome
• Any previous rhGH treatment
• Participation in another simultaneous medical investigation or trial
• Pediatric participants with closed epiphyses
• Participants prescribed rhGH doses outside the variance of NCGS control participant dosing

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Cohort 1: IGHD participants; Isolated growth hormone deficient (IGHD) participants who initiated somatropin (Deoxyribonucleic acid [DNA] origin) (recombinant human growth hormone [rhGH]) using the NuSpin device, will be observed for at least 2 years. The choice of initiation of NuSpin treatment and the treatment regimen will be as per treating physician's discretion, the study protocol does not enforce or specify any treatment regimen.	Device: NuSpin; Device for administration of doses of somatropin.; Drug: Somatropin; Somatropin using either NuSpin or Nutropin AQ Pen, as per standard dosing.; Other Names: Nutropin AQ (Somatropin [DNA Origin] injection)
Cohort 2: ISS participants; Idiopathic short stature (ISS) participants who initiated somatropin (DNA origin) (rhGH) using the NuSpin device, will be observed for at least 2 years. The choice of initiation of NuSpin treatment and the treatment regimen will be as per treating physician's discretion, the study protocol does not enforce or specify any treatment regimen.	Device: NuSpin; Device for administration of doses of somatropin.; Drug: Somatropin; Somatropin using either NuSpin or Nutropin AQ Pen, as per standard dosing.; Other Names: Nutropin AQ (Somatropin [DNA Origin] injection)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Annualized Height Velocity in Response to rhGH Treatment Using the NuSpin Device After Two Years of Treatment	The annualized height velocity (cm per year [cm/yr]) over 2 years was calculated as: [(height in cm at t24 minus (-) height in cm at t0) divided by (date at t24 - date at t0)] multiplied by 365.25, where, t0 is the baseline measurement visit and t24 is the 2-year measurement visit.	Baseline up to Month 24 (Year 2)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Annualized Height Velocity in Response to rhGH Treatment Using the NuSpin Device for First Year of Treatment	The annualized height velocity (cm/yr) after 1 year was calculated as: [(height in cm at t12 - height in cm at t0) divided by (date at t12 - date at t0)] multiplied by 365.25, where, t0 is the baseline measurement visit and t12 is the 1-year measurement visit.	Baseline up to Month 12 (Year 1)
Annualized Height Velocity in Response to rhGH Treatment Using the NuSpin Device During the Second Year of Therapy	The annualized height velocity (cm/yr) for second year was calculated as: [(height in cm at t24 - height in cm at t12) divided by (date at t24 - date at t12)] multiplied by 365.25, where, t12 is the 1-year measurement visit and t24 is the 2-year measurement visit.	Month 12 to Month 24 (Year 1 to Year 2)

Collaborators and Investigators

• Sponsor: Genentech, Inc.
• Investigators: Study Director: D. Aaron Davis, M.D., Genentech, Inc.

Study record dates

Study Major Dates

• Study Start: November 1, 2010
• Primary Completion (Actual): August 1, 2011
• Study Completion (Actual): August 1, 2011

Study Registration Dates

• First Submitted: November 17, 2010
• First Submitted That Met QC Criteria: November 18, 2010
• First Posted (Estimate): November 19, 2010

Study Record Updates

• Last Update Posted (Estimate): December 1, 2016
• Last Update Submitted That Met QC Criteria: October 7, 2016
• Last Verified: October 1, 2016

More Information

Terms related to this study

• Other Study ID Numbers: L4917g; ML01311 (Other Identifier: Hoffmann-La Roche)