Study to Assess the Efficacy and Safety of Eutropin in Prader-Willi Syndrome

A Phase III, Multi-center, Randomized, Comparative, Parallel, Open Study to Assess the Efficacy and Safety After Treatment of Eutropin® Inj. Compared to Genotropin® in Infants/Toddlers With Prader-Willi Syndrome

Evaluate the efficacy and safety after treatment of Eutropin® inj. compared to Genotropin® in infants/toddlers with Prader-Willi syndrome

Study Overview

• Status: Completed
• Conditions: Prader-Willi Syndrome
• Intervention / Treatment: Drug: Genotropin; Drug: Eutropin

• Study Type: Interventional
• Enrollment (Actual): 34
• Phase: Phase 3

Contacts and Locations

Study Locations

• Korea, Republic of
  • Seoul, Korea, Republic of
    • Asan Medical Center
  • Seoul, Korea, Republic of
    • Samsung Medical Center
  • Suwon, Korea, Republic of
    • Ajou University Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

• Inclusion Criteria:

  1. Pediatric patients with PWS confirmed by methylation PCR genetic testing
  2. Prepubertal pediatric patients (Tanner's Pubertal stage I) at screening
  3. Pediatric patients who have never been treated with hGH prior to screening, or who had been treated with hGH for less than 6 months if they had a treatment history, and whose last administration was made 6 months prior to screening
  4. Pediatric patients with normal thyroid function at screening (Those with normal function through a hormonal therapy were allowable.)
  5. Pediatric patients whose parents or LARs signed the informed consent form in writing after receiving the explanation about the purpose, method, effects, etc. of the clinical study, and who also signed the informed consent form in writing if they are capable of reading and understanding writing.

• Exclusion Criteria:

  1. Pediatric patients who are accompanied by other causes for growth retardation as follows except for PWS at screening

     : Chronic renal failure (including the case in which renal transplantation has been undergone), Silver-Russell syndrome, Turner's syndrome, Seckel syndrome, Down's syndrome, Noonan syndrome, Cushing's syndrome, congenital infections, psychiatric disorders, chronic debilitating diseases, etc.

  2. Pediatric patients with malignancy or a history of malignancy at screening
  3. Pediatric patients with severe respiratory disturbance, or sleep apnoea or a history of respiratory infections with an unknown cause at screening. However, those whose condition had been confirmed to be eligible to participate in the clinical study on investigator's judgment were allowed to participae in the study.
  4. Pediatric patients with impaired fasting glucose, diabetes, and diabetic retinopathy at screening
  5. Pediatric patients whose epiphyses are closed with a growth rate of ≤1 cm/year at screening
  6. Pediatric patients who are being administered any drug that may have an effect on the secretion and actions of hGH (estrogen, androgen, anabolic steroids, corticosteroids, GnRH analogs, thyroxine, aromatase inhibitors, etc.) or anticonvulsants and cyclosporin at screening, and have been administered any of them for a long period of time within 6 months prior to screening (However, those who have been administered a thyroxine preparation for ≥4 weeks on a stable dose [allowable in case the investigator determines the dose is stable even though it is changeable based upon the weight of the pediatric patient] were allowed to participate in the clinical study.)
  7. Pediatric patients who are being administered any drug (e.g. methylphenidate) for treatment of hyperactivity disorders including attention deficit hyperactivity disorder (ADHD) at screening
  8. Pediatric patients who are hypersensitive to somatropin or any excipient of the investigational product (cresol or glycerol) or who have a relevant history of hypersensitivity
  9. Pediatric patients who have participated in any other clinical studies after enrolled in this study or who had participated in any other clinical studies within 3 months prior to enrollment in this clinical study
  10. Pediatric patients in whom this clinical study is considered to be difficult to be conducted for any other reasons on investigator's judgment

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Eutropin; Eutropin 0.24mg/kg/week	Drug: Eutropin
Active Comparator: Genotropin; Genotropin 0.24mg/kg/week	Drug: Genotropin

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Change from baseline in height SDS (Standard Deviation Score)	baseline and 52 weeks
Change from baseline in Lean body mass (g)	baseline and 52 weeks
Change from baseline in Percent body fat (%)	baseline and 52 weeks

Secondary Outcome Measures

Outcome Measure	Time Frame
Change from baseline in height velocity (cm/year)	baseline, 16, 28 and 52 weeks
Change from baseline in head circumference (cm)	baseline, 16, 28 and 52 weeks
Change from baseline in cognitive development (score) by Bayley Scale	baseline, 28 and 52 weeks
Change from baseline in motor development (score) by Bayley Scale	baseline, 28 and 52 weeks
Change from baseline in weight SDS	baseline 16, 28 and 52 weeks
Change from baseline in BMI (kg/m2) (Body Mass Index)	baseline, 16, 28 and 52 weeks
Change from baseline in Bone age (month)	baseline and 52 weeks
Change from baseline in Bone mineral density (g/cm)	baseline and 52 weeks
Change from baseline in height (cm)	baseline, 16, 28 and 52 weeks
Change from baseline in height SDS	baseline, 16 and 28 weeks
Change from baseline in IGF-1 (ng/mL) and IGF-1 SDS	baseline, 28, and 52 weeks
Change from baseline in IGFBP-3 (ng/mL) and IGFBP-3 SDS	baseline, 28, and 52 weeks

Collaborators and Investigators

• Sponsor: LG Life Sciences

Publications and helpful links

General Publications

• Yang A, Choi JH, Sohn YB, Eom Y, Lee J, Yoo HW, Jin DK. Effects of recombinant human growth hormone treatment on growth, body composition, and safety in infants or toddlers with Prader-Willi syndrome: a randomized, active-controlled trial. Orphanet J Rare Dis. 2019 Sep 11;14(1):216. doi: 10.1186/s13023-019-1195-1.

Study record dates

Study Major Dates

• Study Start: June 1, 2014
• Primary Completion (Actual): December 1, 2017
• Study Completion (Actual): December 1, 2017

Study Registration Dates

• First Submitted: July 25, 2014
• First Submitted That Met QC Criteria: July 28, 2014
• First Posted (Estimate): July 30, 2014

Study Record Updates

• Last Update Posted (Actual): June 27, 2019
• Last Update Submitted That Met QC Criteria: June 24, 2019
• Last Verified: June 1, 2019

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Nervous System Diseases; Neurologic Manifestations; Neurobehavioral Manifestations; Disease; Congenital Abnormalities; Overnutrition; Nutrition Disorders; Genetic Diseases, Inborn; Intellectual Disability; Abnormalities, Multiple; Chromosome Disorders; Obesity; Syndrome; Prader-Willi Syndrome
• Other Study ID Numbers: LG-HGCL007