A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH) (ACH)

This study is enrolling participants by invitation only.

Sponsor:

Information provided by (Responsible Party):

BioMarin Pharmaceutical

ClinicalTrials.gov Identifier:

NCT02724228

First received: February 12, 2016

Last updated: June 8, 2017

Last verified: June 2017

History of Changes

Purpose

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.

Condition	Intervention	Phase
Achondroplasia	Drug: BMN 111	Phase 2


Study Type:	Interventional
Study Design:	Intervention Model: Single Group Assignment Masking: No masking Primary Purpose: Treatment
Official Title:	A Phase 2, Open-Label, Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children With Achondroplasia

Resource links provided by NLM:

Genetics Home Reference related topics: achondroplasia

Genetic and Rare Diseases Information Center resources: Achondroplasia Mucopolysaccharidosis Type IV

U.S. FDA Resources

Further study details as provided by BioMarin Pharmaceutical:

Primary Outcome Measures:

Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] [ Time Frame: 260 Weeks ]
- Number of study participants with treatment-emergent adverse events.
- Number of study participants with treatment-emergent serious adverse events

Secondary Outcome Measures:

Growth Velocity [Efficacy] [ Time Frame: 260 Weeks ]
Annualized growth velocity (cm/yr)
Growth Parameters (Efficacy) [ Time Frame: 260 weeks ]
Height standard score (Z-score)
Body Proportions (Efficacy) [ Time Frame: 260 weeks ]
Upper-to-lower body segment ratio


Estimated Enrollment:	30
Study Start Date:	January 2016
Estimated Study Completion Date:	December 2022
Estimated Primary Completion Date:	October 2022 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
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, 30 µg/kg, up to 60 µg/kg daily.	Drug: BMN 111 BMN 111 will be administered daily for up to 260 weeks. Other Names: Modified recombinant human C-type natriuretic peptide Vosoritide

Eligibility


Ages Eligible for Study:	7 Years to 16 Years (Child)
Sexes Eligible for Study:	All
Accepts Healthy Volunteers:	No

Criteria

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.
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.

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.
Current chronic therapy with restricted medications.

Contacts and Locations

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02724228

Locations


United States, California
Children's Hospital & Research Center Oakland
Oakland, California, United States, 94609
Harbor - UCLA Medical Center
Torrance, California, United States, 90509
United States, Illinois
Ann and Robert H. Lurie Childrens Hospital of Chicago
Chicago, Illinois, United States, 60611
United States, Maryland
Johns Hopkins McKusick - Institute of Genetic Medicine
Baltimore, Maryland, United States, 21287
United States, Tennessee
Vanderbilt University
Nashville, Tennessee, United States, 37232-2578
United States, Texas
Baylor College of Medicine
Houston, Texas, United States, 77030
Australia, Victoria
Murdoch Children's Research Institute
Parkville, Victoria, Australia, 3052
France
Institut Necker
Paris, France, 75015
United Kingdom
Guys & St. Thomas NHS Foundation Trust Evelina Hospital
London, United Kingdom, SE1 9RT

Sponsors and Collaborators

BioMarin Pharmaceutical

Investigators


Study Director:	Medical Director, MD	BioMarin Pharmaceutical

More Information


Responsible Party:	BioMarin Pharmaceutical
ClinicalTrials.gov Identifier:	NCT02724228 History of Changes
Other Study ID Numbers:	111-205 2015-004004-30 ( EudraCT Number )
Study First Received:	February 12, 2016
Last Updated:	June 8, 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD:	No

Keywords provided by BioMarin Pharmaceutical:


Achondroplasia Dwarfism Bone Diseases, Developmental Bone Diseases

Additional relevant MeSH terms:


Achondroplasia Dwarfism Bone Diseases, Developmental Bone Diseases Musculoskeletal Diseases	Osteochondrodysplasias Genetic Diseases, Inborn Natriuretic Peptide, C-Type Natriuretic Agents Physiological Effects of Drugs

ClinicalTrials.gov processed this record on July 11, 2017

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