Brief Summary

The study aims at identifying the predictive markers after one month of Saizen therapy in Growth Hormone Deficiency (GHD) and Turner Syndrome children.

Trial Health

On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment

318

participants targeted

Target at P75+ for phase_4

Timeline

Completed

Started May 2005

Typical duration for phase_4

Geographic Reach

12 countries

12 active sites

Status

completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

2.3 years study duration

Study Start

First participant enrolled

May 31, 2005

Completed

6 months until next milestone

First Submitted

Initial submission to the registry

November 18, 2005

Completed

3 days until next milestone

First Posted

Study publicly available on registry

November 21, 2005

Completed

1.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 30, 2007

Completed

Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

September 30, 2007

Completed

10.5 years until next milestone

Results Posted

Study results publicly available

March 20, 2018

Completed

Last Updated

June 26, 2018

Status Verified

March 1, 2018

Enrollment Period

2.3 years

First QC Date

November 18, 2005

Results QC Date

August 24, 2017

Last Update Submit

March 23, 2018

Conditions

Growth Hormone Deficiency Turner Syndrome

Outcome Measures

Primary Outcomes (1)

Change From Baseline in Insulin Like Growth Factor-1 Standard Deviation Score (IGF-1 SDS) at Month 1
IGF-1 SDS was calculated using the Elmlinger reference method. Change in within subject IGF-1 levels (standard deviation scores) at Month 1 from Baseline was assessed. Descriptive statistics were determined for the Baseline and Month 1 assessments, and also for the level of change between these two assessments. If either the Baseline or Month 1 IGF-1 level was missing, then the within-subject change in IGF-1 was assumed to be missing.
Baseline, Month 1

Secondary Outcomes (5)

Change From Baseline in Insulin-like Growth Factor Binding Protein - 3 (IGFBP-3) Level at Month 1
Baseline, Month 1
Change From Baseline in Fasting Glucose Levels at Month 1
Baseline, Month 1
Change From Baseline in Fasting Insulin Levels at Month 1
Baseline, Month 1
Change From Baseline in Homeostasis Model Assessment of Insulin Resistance (HOMA-IR) at Month 1
Baseline, Month 1
Change From Baseline in Bone Alkaline Phosphatase Levels at Month 1
Baseline, Month 1

Study Arms (2)

Turner Syndrome (TS)

EXPERIMENTAL

Drug: Saizen

Growth Hormone Deficiency (GHD)

EXPERIMENTAL

Drug: Saizen

Interventions

SaizenDRUG

Subjects with TS will receive SAIZEN® as subcutaneous injection at a dose of 0.050 milligram per kilogram (mg/kg) of body weight per day (within the recommended dosage 0.045-0.050 mg/kg body weight) for a period of 1 month

Turner Syndrome (TS)

Eligibility Criteria

Age2 Years - 16 Years

Sexall

Healthy VolunteersNo

Age GroupsChild (0-17)

You may qualify if:

One of the following diagnoses and candidacy for SAIZEN® therapy:
A) GHD: documented pre-established diagnosis of GHD with a growth hormone (GH) peak response of \<10 microgram per liter (mcg/L) with 2 GH stimulation tests, without priming with oestradiol.
B) Turner syndrome: documented pre-established diagnosis by karyotype.
Prepubertal status according to Tanner Pre-established history of normal thyroid function or adequate substitution for at least 3 months.
Weight for stature within the population specific normal range (\>5th and \<95th percentiles) for gender Willingness and ability to comply with the protocol for the duration of the study.
Parent's or guardian's written informed consent, given before any study related procedure that is not part of the subject's normal medical care, with the understanding that the subject or parent/guardian may withdraw consent at any time without prejudice to future medical care. If the child is old enough to read and write, a separate assent form will be given.

You may not qualify if:

Acquired GHD due to central nervous system tumour, trauma, infection, infiltration (documented by imaging), and history of irradiation or cranial surgery
Previous treatment with GH, growth hormone-releasing hormone (GHRH), anabolic steroids or any treatment affecting growth.
Previous treatment with corticosteroids, except in case of topical or inhaled corticosteroid administration for atopic disease. Corticosteroids for hormonal substitution are also allowed if the condition and the treatment regimen have been stable for at least 3 months.
Severe associated pathology affecting growth such as malnutrition, malabsorption, or bone dysplasia.
Chronic severe kidney disease.
Chronic severe liver disease.
Chronic infectious disease.
Acute or severe illness during the previous 6 months.
Significant concomitant illness that would interfere with participation or assessment in this study.
Active malignancy (except non-melanomatous skin malignancies that have undergone surgical excision and/or biopsy, diagnosis and treatment to resolution)
History or active Idiopathic intra-cranial hypertension (benign intracranial hypertension or pseudo-tumor cerebri).
Diabetes Mellitus type I \& II.
Any autoimmune disease.
Previous screening failure in this study.
Use of an investigational drug or participation in another clinical study within the last three months.

Contact the study team to confirm eligibility.