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Saizen

Phase 3

Fibromyalgia | Small molecule | Musculoskeletal |Merck & Company, Inc.|Last Updated: Sep 26, 2017

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindACTIVE_CONTROLLED
Total Trials1
Total Enrollment113

FDA Designations

No designations recorded

Clinical trial landscape

Saizen · 3 trials · 3 indications

Phase 3 3
NCT01746862A Phase 3 Study to Evaluate the Safety and Efficacy of Saizen® in Children With Idiopathic Short Stature (ISS)Idiopathic Short Stature
COMPLETED90 Analytics
NCT00933686Growth Hormone in Neuroendocrine Dysfunction With Severe Fibromyalgia SyndromeFibromyalgia
COMPLETED113 Analytics
NCT00249821Optimization of the Dosage Regimen of Growth Hormone Therapy in Children Born Small for Gestational AgeSmall Gestational Age (SGA)
COMPLETED22 Analytics
PHASE3COMPLETED
A Phase 3 Study to Evaluate the Safety and Efficacy of Saizen® in Children With Idiopathic Short Stature (ISS)
Idiopathic Short StatureUnlock trial analytics
PHASE3COMPLETED
Growth Hormone in Neuroendocrine Dysfunction With Severe Fibromyalgia Syndrome
FibromyalgiaUnlock trial analytics
PHASE3COMPLETED
Optimization of the Dosage Regimen of Growth Hormone Therapy in Children Born Small for Gestational Age
Small Gestational Age (SGA)Unlock trial analytics

Study Endpoints

Primary Endpoints

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

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.

Percentage of Participants With Less Than 11 Tender Points at Month 6
Month 6

The musculoskeletal component in form of pain on palpation in at least 11 of 18 tender point sites is required to fulfill the American College of Rheumatology (ACR) criteria for fibromyalgia syndrome, An important response is considered when the number of tender points falls below 11 since it is the threshold for fibromyalgia diagnosis.

Percentage of Participants With Less Than 11 Tender Points at Month 12
Month 12

The musculoskeletal component in form of pain on palpation in at least 11 of 18 tender point sites is required to fulfill the ACR criteria for fibromyalgia syndrome, An important response is considered when the number of tender points falls below 11 since it is the threshold for fibromyalgia diagnosis.

Height Velocity
Month 12

Height Velocity (HV) is the change in height since the previous year´s measurement and more precisely: HV = {(h-hp)/(d-dp)} \* 365.25 \[centimeter (cm)/year\] where h is current height in cm, hp is previous height in cm, closest to 1 year previous, d is the current date and dp is the date of measurement of previous height, closest to 1 year previous. Additionally, d and dp have to be within 0.6 years and 1.5 years. HV is the mean height velocity over the interval between d and dp but is displayed as HV at d.

Secondary Endpoints

Change From Baseline in Height Velocity at Month 12
Baseline, Month 12
Change From Baseline in Height at Month 6 and 12
Baseline, Month 6, Month 12
Change From Baseline in Height Standard Deviation Score (SDS) at Month 6 and 12
Baseline, Month 6, Month 12
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Study Design & Arms

AllocationRANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Saizen Test GroupEXPERIMENTAL -
Saizen Control GroupACTIVE_COMPARATOR -
Saizen®ACTIVE_COMPARATOR -
Placebo + Saizen®ACTIVE_COMPARATOR -
Saizen® 0.057 mg/kg/dayEXPERIMENTALSubjects who met all inclusion/exclusion criteria were randomly assigned to 0.057 mg/kg/day in this multi-center study. Subjects were stratified at randomization according to the Height-Standard Deviation Score (H-SDS) at this time (less than \[\<\] -2 SDS or greater than \[\>\] -2 SDS)
Saizen® 0.035 mg/kg/dayEXPERIMENTALSubjects who met all inclusion/exclusion criteria were randomly assigned to 0.035 mg/kg/day in this multi-center study. Subjects were stratified at randomization according to the H-SDS at this time (\< -2 SDS or \> -2 SDS)

Interventions

NameTypeDescription
Saizen®DRUGSubjects 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.
Placebo and Saizen®DRUGPlacebo matched to Saizen® will be administered for the first 6 months followed by treatment with Saizen® (Somatropin) 0.006 mg/kg subcutaneously daily for next 6 months. Saizen® dose will be titrated (an increase of 0.2 milligram per day) if the increase in insulin-like growth factor 1 (IGF-1) is less than 50 percent of the baseline value. Dose titrations will be made at Month 7 and 9.
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Eligibility Criteria

Age Range5 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: * 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...

Countries:South KoreaSpain
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Competitive Landscape -Fibromyalgia 5 trials

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Frequently asked questions about Saizen

What is Saizen used for?

Saizen is used for fibromyalgia, idiopathic short stature, and being born small for gestational age (SGA). It is a small molecule being developed by Merck & Company, Inc. (MRK) and is currently in Phase 3 clinical development for these indications.

Who makes Saizen?

Merck & Company, Inc. (MRK) makes Saizen. The drug is a small molecule in Phase 3 clinical development for fibromyalgia, idiopathic short stature, and small gestational age (SGA).

What phase is Saizen in?

Saizen is in Phase 3 clinical development. It is investigational and not yet approved, with completed Phase 3 trials in fibromyalgia, idiopathic short stature, and small gestational age (SGA).

What clinical trials is Saizen in?

Saizen has completed Phase 3 trials including NCT00249821 in children born small for gestational age, NCT00933686 in fibromyalgia, and NCT01746862 in idiopathic short stature. These trials are completed, with enrollment ranging from 22 to 113 participants.

Is Saizen the same as growth hormone?

Saizen is a growth hormone therapy used in conditions like small gestational age and idiopathic short stature. The trials evaluate growth hormone therapy regimens, indicating Saizen is a form of growth hormone treatment.