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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03424018




Registration number
NCT03424018
Ethics application status
Date submitted
28/12/2017
Date registered
6/02/2018

Titles & IDs
Public title
An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
Scientific title
A Phase 3, Open-Label Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children With Achondroplasia
Secondary ID [1] 0 0
2017-002404-28
Secondary ID [2] 0 0
111-302
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

Experimental: BMN 111 -


Treatment: Drugs: BMN 111
Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change from baselines in mean annualized growth velocity
Timepoint [1] 0 0
Through study completion, an average of 1 year
Secondary outcome [1] 0 0
Changes in health-related quality of life as measured by the Quality of Life in Short-Statured Youth questionnaire
Timepoint [1] 0 0
Through study completion, every 6-12 months
Secondary outcome [2] 0 0
Potential changes in daily activity performance as measured by Activities of Daily Living questionnaire
Timepoint [2] 0 0
Through study completion, every 12 months
Secondary outcome [3] 0 0
Characterize maximum concentration (Cmax) of BMN 111 in plasma
Timepoint [3] 0 0
Through study completion, every 12 months
Secondary outcome [4] 0 0
Characterize the area under the plasma concentration time-curve from time 0 to infinity (AUC0-8)
Timepoint [4] 0 0
Through study completion, every 12 months
Secondary outcome [5] 0 0
Characterize the area under the plasma concentration time-curve from time 0 to the last measurable concentration (AUC0-t)
Timepoint [5] 0 0
Through study completion, every 12 months
Secondary outcome [6] 0 0
Characterize the elimination half-life of BMN 111 (t1/2)
Timepoint [6] 0 0
Through study completion, every 12 months
Secondary outcome [7] 0 0
Characterize the apparent clearance of drug
Timepoint [7] 0 0
Through study completion, every 12 months
Secondary outcome [8] 0 0
Characterize the apparent volume of distribution based upon the terminal phase (Vz/F)
Timepoint [8] 0 0
Through study completion, every 12 months
Secondary outcome [9] 0 0
Characterize the amount of time BMN 111 is present at maximum concentration (Tmax)
Timepoint [9] 0 0
Through study completion, every 12 months
Secondary outcome [10] 0 0
BMN 111 Activity Biomarkers
Timepoint [10] 0 0
Through study completion, every 12 months
Secondary outcome [11] 0 0
Evaluate change from baseline in body proportion ratios of the extremities
Timepoint [11] 0 0
Through study completion, every 6 months
Secondary outcome [12] 0 0
Effect of BMN 111 on bone morphology and quality
Timepoint [12] 0 0
Through study completion, every 12 months for DXA or 2 years for X-ray
Secondary outcome [13] 0 0
Final Adult Height
Timepoint [13] 0 0
Up to at least 16 years of age for females and 18 years of age for males

Eligibility
Key inclusion criteria
* Must have completed Study 111-301
* Female >= 10 years old or who have begun menses must have a negative pregnancy test at the Baseline Visit and be willing to have additional pregnancy tests during the study
* If sexually active, willing to use a highly effective method of contraception while participating in the study
* Are willing and able to perform all study procedures
* Parent(s) or guardian(s) are willing and able to provide written, signed informed consent after the nature of the study has been explained and prior to performance of any research-related procedure. Also, subjects under the age of majority are willing and able to provide written assent (if required by local regulations or the IRB/IEC) after the nature of the study has been explained and prior to performance of any research-related procedure. Subjects who reach the age of majority in their country while the study is ongoing will be asked to provide their own written consent again upon reaching the legal age of majority.
Minimum age
6 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Permanently discontinued BMN 111 or placebo prior to completion of the 111-301 study
* Have a clinically significant finding or arrhythmia on Baseline ECG that indicates abnormal cardiac function
* Evidence of decreased growth velocity (<1.5 cm/year) as assessed over a period of at least 6 months or of growth plate closure (proximal tibia, distal femur) through bilateral lower extremity X-rays.
* Require any investigational agent prior to completion of study period
* Current therapy with medications known to alter renal function
* Pregnant or breastfeeding or plan to become pregnant during study
* Concurrent disease or condition that, in the view of the investigator, would interfere with study participation or safety evaluations, for any reason.
* Have a condition or circumstance that, in the view of the investigator, 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
NA
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
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
12/12/2017
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/06/2031
Actual
Sample size
Target
Accrual to date
Final
119
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
Address 0 0
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)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT03424018