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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT02724228




Registration number
NCT02724228
Ethics application status
Date submitted
12/02/2016
Date registered
31/03/2016
Date last updated
8/04/2019

Titles & IDs
Public title
A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)
Scientific title
A Phase 2, Open-Label, Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children With Achondroplasia
Secondary ID [1] 0 0
2015-004004-30
Secondary ID [2] 0 0
111-205
Universal Trial Number (UTN)
Trial acronym
ACH
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 - Subcutaneous Injection - 111-205 is an open-label, extension study. Subjects receive the same stable dose of BMN 111 received upon completion of the 111-202 study. BMN 111 will be administered in one of the following daily dosing regimens: 15 µg/kg or 30 µg/kg daily.


Treatment: Drugs: BMN 111
BMN 111 will be administered subcutaneously daily.

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] - Number of study participants with treatment-emergent adverse events.
Number of study participants with treatment-emergent serious adverse events
Timepoint [1] 0 0
Through study completion, every 3 months
Secondary outcome [1] 0 0
Growth Velocity [Efficacy] - Annualized growth velocity (cm/yr)
Timepoint [1] 0 0
Up to 5 years or near final adult height
Secondary outcome [2] 0 0
Growth Parameters (Efficacy) - Height standard score (Z-score)
Timepoint [2] 0 0
Up to 5 years or near final adult height
Secondary outcome [3] 0 0
Body Proportions (Efficacy) - Upper-to-lower body segment ratio
Timepoint [3] 0 0
Up to 5 years or near final adult height

Eligibility
Key inclusion criteria
- Have completed 24 months of BMN 111 treatment in Study 111-202.

- Parent(s) or guardian(s) are willing and able to provide written, signed informed
consent. Subjects under the age of majority are willing and able to provide written
assent (if required). Subjects who reach the age of majority in their country will be
asked to provide their own written consent upon reaching the legal age of majority.

- If sexually active, willing to use a highly effective method of contraception while
participating in the study.

- Females >= 10 years old or who have started menses must have a negative pregnancy test
at baseline and be willing to have additional pregnancy tests during the study

- Willing and able to perform all study procedures as physically possible

- Parents/caregivers willing to administer daily injections to the subjects and complete
the required training.
Minimum age
7 Years
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Requires any investigational agent prior to completion of study period.

- 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.

- Concurrent disease or condition that, in the view of the Investigator, would interfere
with study participation or safety evaluations for any reason.

- Permanently discontinued BMN 111 during the 111-202 study.

- Subject is pregnant at Baseline visit or planning to become pregnant (self or partner)
at any time during the study.

- Current chronic therapy with restricted medications.

Study design
Purpose of the study
Treatment
Allocation to intervention
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 2
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
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Murdoch Children's Research Institute - Parkville
Recruitment postcode(s) [1] 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
Illinois
Country [3] 0 0
United States of America
State/province [3] 0 0
Maryland
Country [4] 0 0
United States of America
State/province [4] 0 0
Tennessee
Country [5] 0 0
United States of America
State/province [5] 0 0
Texas
Country [6] 0 0
France
State/province [6] 0 0
Paris
Country [7] 0 0
United Kingdom
State/province [7] 0 0
London

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

Ethics approval
Ethics application status

Summary
Brief summary
This is a Phase 2, Open-Label, Extension Study to Evaluate the Long-Term Safety,
Tolerability, and Efficacy of BMN 111 in Children with Achondroplasia. The primary objective
is to evaluate the long-term safety and tolerability of daily SC injections of BMN 111 in
children with ACH who have completed two years of treatment in the 111-202 study.
Trial website
https://clinicaltrials.gov/show/NCT02724228
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

Summary results
Other publications