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Idiopathic Short StaturePhase 3NCT01746862

A Phase 3 Study to Evaluate the Safety and Efficacy of Saizen® in Children With Idiopathic Short Stature (ISS)

A Phase 3 study testing Saizen® in people with Idiopathic Short Stature, run by Merck KGaA, Darmstadt, Germany at 1 site in South Korea.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

This is an open-label, multi-center, randomized, two-arm parallel, no-treatment group controlled (only for the first 6 months), Phase 3 study in children with ISS. The subjects will be treated with 0.067 milligram/kilogram/day (mg/kg/day) of Saizen®, weight base dose, for 12 months (12 months of treatment in the test… Read more about this study

Who is it for?
Ages 5 years and older
Conditions the study is for
Idiopathic Short Stature
What is being tested?
Saizen®
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
90 participants
Where
1 siteSouth Korea
Study dates
Dec 2012to Mar 2015
Phase
Phase 3
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 5 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Age greater than or equal to 5 years
  • Pre-pubertal; testicular volume less than 4 milliliter (in males) and breast Stage 1 (in females)
  • The official records of height (for example records measured in hospitals or schools) during previous 6 months or more preceding inclusion in the study (self-measurement of the height at home will not be considered as a valid record)
  • Height less than or equal to 3rd percentile compared to same sex, same age
  • Peak serum growth hormone (GH) greater than 10 microgram per liter (mcg/L) in GH stimulation test (results of peak serum GH greater than 10 mcg/L in GH stimulation test within 1 year can be used instead)
  • Naive to GH therapy
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  • Normal birth weight (that is greater than or equal to 3rd percentile when compared to same sex)
  • Normal thyroid function
  • Normal karyotype in girls
  • Written informed consent from parent/guardian
  • Written informed consent from the subject who speaks, understand, read, and write Korean
  • Bone age less than 10 years in boys and less than 9 years in girls, whose difference between the bone and chronological age is no more than 3 years

Who cannot join

  • Puberty development (Tanner stage greater than or equal to 2)
  • Skeletal dysplasia or abnormal body proportions
  • Chronic systemic illness
  • Dysmorphic syndrome
  • Growth Hormone Deficiency
  • Small for Gestational Age (SGA)
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  • Current medication for Attention deficit hyperactivity disorder (ADHD) or hyperactivity disorder
  • Current medication with drugs that may influence secretion or action of growth hormone (such as estrogen, androgen, anabolic steroid, corticosteroid, thyroxine, aromatase inhibitors)
  • Diabetes mellitus
  • Kidney transplantation
  • Acute critical illness, including complications following open heart surgery, abdominal surgery or multiple accidental trauma
  • Acute respiratory failure
  • Malignancy or previous therapy for malignancy
  • Known hypersensitivity to somatotropin or any of its excipients including cresol or glycerol
  • Closed epiphyses, progression or recurrence of an underlying intracranial tumor, chronic renal disease
  • Endocrinologic or metabolic disorders such as Prader-Willi syndrome; Russel-Silver syndrome; Seckel syndrome; Down syndrome; Cushing syndrome; Noonan syndrome; short stature caused by other chromosomal abnormalities
  • The disorders that explain short stature such as psychiatric disorders, nutritional disorders, and chronic debilitating diseases
  • Participation in another clinical trial within the past 3 months
  • Status of legal incapacity or limited legal capacity of the parents or legal guardian

Trial locations and contacts

1 site in 1 country.

South Korea1 site

  • Please contact Merck KGaA Communication Center for Recruiting Sites

    Located in

Other trials in South Korea

About this study

Official title: A Randomized, Open-label, Two-arm Parallel Group, No Treatment Group-controlled, Multicenter Phase III Study to Evaluate the Safety and Efficacy of Saizen® 0.067 mg/kg/Day Subcutaneous Injection in Children With Idiopathic Short Stature

This is an open-label, multi-center, randomized, two-arm parallel, no-treatment group controlled (only for the first 6 months), Phase 3 study in children with ISS. The subjects will be treated with 0.067 milligram/kilogram/day (mg/kg/day) of Saizen®, weight base dose, for 12 months (12 months of treatment in the test group, and 6 months of no treatment and then 6 months of treatment in the control group).

Being tested
Saizen®
Keywords
Saizen®, Recombinant human growth hormone (r-hGH), Idiopathic short stature, Height velocity, Bone age

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Saizen®Drug

    Also called Somatropin, r-hGH

    Subjects in the Saizen test group will receive Saizen (recombinant-human growth hormone [r-hGH]) subcutaneously 6 days per week at a weight based dose of 0.067 milligram per kilogram of body weight per day (mg/kg/day) for 12 months.

  • Saizen®Drug

    Also called Somatropin, r-hGH

    Subjects in the Saizen control group will receive no treatment for the first 6 months and thereafter will receive Saizen (r-hGH) subcutaneously 6 days per week at a weight based dose of 0.067 mg/kg/day for the next 6 months.

Study groups in NCT01746862
Study groupRoleWhat they receive
Saizen Test GroupExperimentalNot described
Receives: Drug: Saizen®
Saizen Control GroupActive comparatorNot described
Receives: Drug: Saizen®

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Change From Baseline in Height Velocity at Month 6 Using Last Observation Carried Forward (LOCF) Method

    Baseline height is defined as the last available height measurement before randomization. Baseline height velocity = ([Baseline height minus height measurement obtained at least 6 months prior] / 6) * 12. Height velocity at Month 6 = ([Month 6 height minus height measurement obtained at least 6 months prior] / 6) * 12.

    Measured: Baseline, Month 6

Secondary outcomes (7)

  1. Change From Baseline in Height Velocity at Month 12

    Baseline height is defined as the last available height measurement before randomization. Baseline height velocity = ([Baseline height minus height measurement obtained at least 12 months prior] / 12) * 12. Height velocity at Month 12 = ([Month 12 height minus height measurement obtained at least 12 months prior] / 12) * 12.

    Measured: Baseline, Month 12

  2. Change From Baseline in Height at Month 6 and 12

    Measured: Baseline, Month 6, Month 12

  3. Change From Baseline in Height Standard Deviation Score (SDS) at Month 6 and 12

    Height SDS was calculated as: Height SDS = (measured height - population mean) / population standard deviation, where mean and standard deviation were based on the Korean standard growth charts. SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) a subject's value was relative to the mean of the reference population. The scores were centred around zero. Negative score indicated a subject was smaller for their age/gender.

    Measured: Baseline, Month 6, Month 12

  4. Change From Baseline in Serum Concentration of Insulin-like Growth Factor-I (IGF-I) at Month 6 and 12

    Measured: Baseline, Month 6, Month 12

  5. Change From Baseline in Serum Concentration of Insulin Like Growth Factor Binding Protein-3 (IGFBP-3) at Month 6 and 12

    Measured: Baseline, Month 6, Month 12

  6. Percentage of Adherence to Study Treatment

    Percentage of adherence to study treatment (adherence rate) was defined as the actual number of received treatments divided by the scheduled number of treatments multiplied by 100. The adherence rate for 6 months was calculated from Baseline to 6 months for the Saizen Test Group and from Month 6 to Month 12 for the Saizen Control Group.

    Measured: 6 months post-dose (Saizen Test Group and Saizen Control Group); 12 months post-dose (Saizen Test Group)

  7. Number of Subjects With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs

    An adverse event (AE) was defined as any untoward medical occurrence which does not necessarily have a causal relationship with this the study drug. An AE was defined as any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of study drug, whether or not considered related to the study drug. A serious adverse event (SAE) was an AE that resulted in any of the following outcomes: death; life threatening; persistent/significant disability/incapacity; initial or prolonged inpatient hospitalization; congenital anomaly/birth defect or was otherwise considered medically important. For the Saizen Test Group, TEAEs were defined as events that occurred or worsened at or after the first administration of treatment and for the Saizen Control Group, TEAEs were defined as events that occurred or worsened at or after the randomization.

    Measured: Baseline up to Month 13

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted11 Dec 2012 (estimated)
  2. Study startDec 2012
  3. Primary completionAug 2014
  4. Study completionMar 2015

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
90 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01746862
Sponsor's protocol ID
EMR200104-533
Status last verified
Aug 2016
First submitted
30 Nov 2012
Last updated
18 Oct 2016

Publications

  • Chung WY, Yoo HW, Hwang JS, Ko CW, Kim HS, Jin DK, Lee KH, Han HS, Paranchothy P, Suh BK. Effect of Growth Hormone Therapy on Height Velocity in Korean Children with Idiopathic Short Stature: A Phase III Randomised Controlled Trial. Horm Res Paediatr. 2018;90(1):44-53. doi: 10.1159/000491016. Epub 2018 Aug 15.

    PubMed 30110706

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Saizen® in people with Idiopathic Short Stature, run by Merck KGaA, Darmstadt, Germany at 1 site in South Korea.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 12 inclusion and 19 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 18 Oct 2016. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in South Korea. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 18 Oct 2016). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change From Baseline in Height Velocity at Month 6 Using Last Observation Carried Forward (LOCF) Method (time frame: Baseline, Month 6). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01746862, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Oct 2016.

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