Short Stature Related Distress

The Influence of Growth Hormone (GH) Therapy on Short Stature Related Distress a Prospective Randomized Controlled Trial

The research aims to provide data on gender-related psychological impact of GH (Growth Hormone)treatment. Implicit measurable psychological metrics of the therapy outcome on males are to be evaluated in a Prospective Randomized Controlled Trial. As a secondary axis of the study, the investigators aim to provide descriptive documentation of the unique distress signal manifestation implicitly shown by Idiopathic Short Stature (ISS) boys and suggest methods of early detection and measurement of such manifestations that can be used to cement guidelines for the treatment in GH.

Study Overview

• Status: Completed
• Conditions: Idiopathic Short Stature; Self Image
• Intervention / Treatment: Drug: GH treatment (Genotropin); Drug: 1 year treatment with placebo followed by optional 3 years of GH treatment

Detailed Description

Study Design:

A prospective randomized, double blind, placebo controlled trial design, comparing four research groups, with 4 repeated psychological measures (before treatment, after 3 months,after 1 year, and at the end of treatment, after 4 years).

The study will be divided into two segments:

Segment 1:double blind, placebo controlled, will include the first 60 ISS (Idiopathic Short Stature)recruited patients. Patients in this segment will be randomized to one of the growth hormone treatment groups in a ratio of 2:1. 40 patients will be treated 4 years with GH and 20 patients will be treated with placebo at the first year of the study and will have the option to start GH treatment after one year, for a duration of 3 years.

Segment 2:after the enrollment to segment 1 will end, enrollment to segment 2 will begin.Participants in segment 2 (patients with ISS and short stature) will serve as control groups and therefore will not be treated with growth hormone. Patients in Segment 2 will be evaluated only once.

The 4 repeated psychological evaluations (before treatment,after 3 months, after 1 year, and at the end of treatment, after 4 years)will include the following:

1. Implicit Association Test
2. Masculine Self Image
3. Pictures Projective Test
4. The Rosenberg Self Esteem Scale-a self report measure of explicit self- esteem
5. Quality of Life Questionnaire
6. Silhouette Apperception Technique (SAT)- a self report measure assessing children's and adolescent's perception of their own body size compared with that of their age-related peers
7. Child Behavior Checklist

• Study Type: Interventional
• Enrollment (Actual): 60
• Phase: Phase 4

Contacts and Locations

Study Locations

• Israel
  • Petach-Tikva, Israel, 49202
    • Schneider Children's Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 8 years to 17 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: Male

Description

Inclusion Criteria:

1 Segment 1 :Boys at the age range of 8-13 years 2. Segment 2: Boys at the age range of 12-17 years 3. Tanner stage 1 (segment 1 only) 4. more than 2 SD's (standard deviation) below the average height

Exclusion Criteria:

1. Mental retardation,
2. Psychiatric illness,
3. Lack of informed consent\assent.
4. Health status that required chronic treatment with steroids

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: GH treatment; 4 years of GH treatment	Drug: GH treatment (Genotropin); 4 years treatment with GH
Placebo Comparator: Placebo; 1 year treatment with placebo followed by optional 3 years of GH treatment	Drug: 1 year treatment with placebo followed by optional 3 years of GH treatment; 1 year treatment with placebo followed by optional 3 years of GH treatment
No Intervention: Non treatment group

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Psychological Distress score	short stature related distress score as measured by the Height Perception Pictures Test	After 4 years of treatment
Psychological Distress score	short stature related distress score as measured by the Rosenberg Self-Esteem Scale	After 4 years of treatment
Psychological Distress score	short stature related distress score as measured by the Quality of Life questionnaire	After 4 years of treatment
Psychological Distress score	short stature related distress score as measured by Silhouette Apperception Technique questionnaire	After 4 years of treatment

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Perceived self image score	Perceived self image score as measured through Single Category Implicit Association Test	After 4 years of treatment
Perceived self image score	Perceived self image score as measured through the Masculine Self Image questionnaire	After 4 years of treatment

Collaborators and Investigators

• Sponsor: Rabin Medical Center
• Collaborators: Pfizer

Publications and helpful links

General Publications

• Shemesh-Iron M, Lazar L, Lebenthal Y, Nagelberg N, Tenenbaum A, Ezra R, Leffler N, Yackobovitch-Gavan M, Schoenberg-Taz M, Phillip M. Growth hormone therapy and short stature-related distress: A randomized placebo-controlled trial. Clin Endocrinol (Oxf). 2019 May;90(5):690-701. doi: 10.1111/cen.13944. Epub 2019 Mar 7.

Study record dates

Study Major Dates

• Study Start (Actual): October 1, 2011
• Primary Completion (Actual): November 1, 2017
• Study Completion (Actual): November 1, 2017

Study Registration Dates

• First Submitted: November 16, 2010
• First Submitted That Met QC Criteria: November 22, 2010
• First Posted (Estimate): November 23, 2010

Study Record Updates

• Last Update Posted (Actual): December 20, 2017
• Last Update Submitted That Met QC Criteria: December 18, 2017
• Last Verified: December 1, 2017

More Information

Terms related to this study

• Keywords: Psychological distress; GH therapy; Idiopathic Short stature; Self Image
• Additional Relevant MeSH Terms: Endocrine System Diseases; Genetic Diseases, Inborn; Musculoskeletal Diseases; Bone Diseases; Bone Diseases, Developmental; Dwarfism
• Other Study ID Numbers: rmc004981ctil