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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03197766




Registration number
NCT03197766
Ethics application status
Date submitted
23/05/2017
Date registered
23/06/2017

Titles & IDs
Public title
A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
Scientific title
A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
Secondary ID [1] 0 0
2015-003836-11
Secondary ID [2] 0 0
111-301
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Achondroplasia 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - BMN 111
Treatment: Drugs - Placebo

Experimental: Active BMN 111 - Daily subcutaneous injection of 15 micrograms per kilogram BMN111

Placebo comparator: Placebo - Daily subcutaneous injection of placebo


Treatment: Drugs: BMN 111
Subcutaneous injection of 15 µg/kg of BMN 111 daily

Treatment: Drugs: Placebo
Subcutaneous injection of 15 µg/kg of placebo daily
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change From Baseline in Annualized Growth Velocity (AGV) at Week 52
Timepoint [1] 0 0
At Baseline and Week 52
Secondary outcome [1] 0 0
Change From Baseline in Height Z-score at Week 52
Timepoint [1] 0 0
At baseline and Week 52
Secondary outcome [2] 0 0
Change From Baseline in Upper to Lower Segment Body Ratio at Week 52
Timepoint [2] 0 0
At baseline and Week 52
Secondary outcome [3] 0 0
Summary of Subjects Experiencing Adverse Events (AEs) During Treatment
Timepoint [3] 0 0
Up to Week 56

Eligibility
Key inclusion criteria
Inclusion Criteria

* Parent(s) or guardian(s) consent
* 5 to < 18 years old
* ACH, documented and confirmed by genetic testing
* At least a 6-month period of pretreatment growth assessment in Study 111-901 before study entry
* If sexually active, willing to use a highly effective method of contraception
* Ambulatory and able to stand without assistance
Minimum age
5 Years
Maximum age
18 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Exclusion criteria:

* Hypochondroplasia or short stature condition other than ACH
* Have any of the following:

* Hypothyroidism or hyperthyroidism
* Insulin-requiring diabetes mellitus
* Autoimmune inflammatory disease
* Inflammatory bowel disease
* Autonomic neuropathy
* History of any of the following:

* Renal insufficiency defined as serum creatinine > 2 mg/dL
* Chronic anemia
* Baseline systolic blood pressure (BP) < 70 millimeters of mercury (mm Hg) or recurrent symptomatic hypotension (defined as episodes of low BP generally accompanied by symptoms ie, dizziness, fainting) or recurrent symptomatic orthostatic hypotension
* Cardiac or vascular disease

* Have a clinically significant finding or arrhythmia on screening electrocardiogram (ECG) that indicates abnormal cardiac function or conduction or Fridericias corrected QTc-F > 450 msec
* Have an unstable condition likely to require surgical intervention during the study (including progressive cervical medullary compression or severe untreated sleep apnea)
* Decreased growth velocity (< 1.5 cm/yr) over a period of 6 months or evidence of growth plate closure (proximal tibia, distal femur)
* Treated with growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroids in the previous 6 months or treatment greater than 6 months at any time
* Greater than 1 month treatment with oral corticosteroids (low-dose ongoing inhaled steroid for asthma, or intranasal steroids, are acceptable) in the previous 12 months
* Planned or expected to have limb-lengthening surgery during the study period. Subjects with previous limb- lengthening surgery may enroll if surgery occurred at least 18 months prior to the study and healing is complete without sequelae.
* Planned or expected bone-related surgery (ie. surgery involving disruption of bone cortex, excluding tooth extraction), during the study period. Subjects with previous bone-related surgery may enroll if surgery occurred at least 6 months prior to the study and healing is complete without sequelae.
* Had a fracture of the long bones or spine within 6 months prior to screening
* History of severe untreated sleep apnea
* New initiation of sleep apnea treatment (e.g. CPAP or sleep apnea-mitigating surgery) in the previous 2 months prior to screening
* History of hip surgery or hip dysplasia atypical for achondroplastic subjects
* History of clinically significant hip injury in the 30 days prior to screening
* History of slipped capital femoral epiphysis or avascular necrosis of the femoral head
* Abnormal findings on baseline clinical hip exam or imaging assessments that are determined to be clinically significant
* Concurrent disease or condition that would interfere with study participation or safety evaluations, for any reason
* Condition or circumstance that places the subject at high risk for poor treatment compliance or for not completing the study

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s

The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
12/12/2016
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
30/10/2019
Sample size
Target
Accrual to date
Final
121
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
The Children's Hospital at Westmead - Westmead
Recruitment hospital [2] 0 0
Murdoch Children's Research Institute - Parkville
Recruitment postcode(s) [1] 0 0
2145 - Westmead
Recruitment postcode(s) [2] 0 0
3052 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Delaware
Country [3] 0 0
United States of America
State/province [3] 0 0
Georgia
Country [4] 0 0
United States of America
State/province [4] 0 0
Illinois
Country [5] 0 0
United States of America
State/province [5] 0 0
Maryland
Country [6] 0 0
United States of America
State/province [6] 0 0
Missouri
Country [7] 0 0
United States of America
State/province [7] 0 0
Ohio
Country [8] 0 0
United States of America
State/province [8] 0 0
Texas
Country [9] 0 0
United States of America
State/province [9] 0 0
Washington
Country [10] 0 0
United States of America
State/province [10] 0 0
Wisconsin
Country [11] 0 0
Germany
State/province [11] 0 0
Magdeburg
Country [12] 0 0
Germany
State/province [12] 0 0
Münster
Country [13] 0 0
Japan
State/province [13] 0 0
Osaka
Country [14] 0 0
Japan
State/province [14] 0 0
Saitama
Country [15] 0 0
Japan
State/province [15] 0 0
Tokushima
Country [16] 0 0
Spain
State/province [16] 0 0
Barcelona
Country [17] 0 0
Spain
State/province [17] 0 0
Málaga
Country [18] 0 0
Turkey
State/province [18] 0 0
Istanbul
Country [19] 0 0
United Kingdom
State/province [19] 0 0
London
Country [20] 0 0
United Kingdom
State/province [20] 0 0
Sheffield

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
BioMarin Pharmaceutical
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Medical Director, MD
Address 0 0
BioMarin Pharmaceutical
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Undecided
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT03197766