achondroplasia MedDRA version: 20.0 Level: LLT Classification code 10000452 Term: Achondroplasia System Organ Class: 100000004850
Conditions
Sponsors
BioMarin Pharmaceutical Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. 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 18 are willing and able to provide written assent (if required by local regulations or the IRB/EC) after the nature of the study has been explained and prior to performance of any research-related procedure. Subjects who reach the age of 18 years while the study is ongoing will be asked to provide their own written consent. 2. 5 to =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Have hypochondroplasia or short stature condition other than ACH (e.g., trisomy 21, pseudoachondroplasia) 2. Have any of the following: Hypothyroidism or hyperthyroidism; Insulin-requiring diabetes mellitus; Autoimmune inflammatory disease (including celiac disease, lupus (SLE), juvenile dermatomyositis, scleroderma, and others); Inflammatory bowel disease; Autonomic neuropathy 3. Have a history of any of the following: Renal insufficiency defined as serum creatinine > 2 mg/dl; Chronic anemia; Baseline systolic blood pressure (BP) 450 msec 5. Have an unstable condition likely to require surgical intervention during the study (including progressive cervical medullary compression or severe untreated sleep apnea) 6. Evidence of decreased growth velocity (AGV 1 month treatment with oral corticosteroids (low-dose ongoing inhaled steroid for asthma, or intranasal steroids, are acceptable) in the previous 12 months 14. 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 screening and healing is complete without sequelae. 15. Planned or expected bone-related surgery (ie. surgery involving disruption of bone cortex, excluding tooth extraction), during the study period.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Secondary Objective: •Evaluate change from baseline in height Z-score in subjects treated with BMN 111 compared with control subjects in the placebo group at 52 weeks •Evaluate change from baseline in upper:lower segment body ratio in subjects treated with BMN 111 compared with control subjects in the placebo group at 52 weeks •Evaluate safety and tolerability of BMN 111 in children with ACH •Evaluate the pharmacokinetics of BMN 111 •Evaluate change from baseline in bone metabolism biomarkers ;Primary end point(s): The primary efficacy endpoint is the change from baseline in annualized growth velocity (AGV) at Week 52 (12- month).;Timepoint(s) of evaluation of this end point: Anthropometric measurements: Screen, Day 1, Week 13, Week 26, Week 39, Week 52;Main Objective: Evaluate change from baseline in annualized growth velocity at 52 weeks in subjectstreated with BMN 111 compared with control subjects in the placebo group | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): The secondary efficacy endpoints include the change from baseline in height Z-score and the change from baseline in upper:lower body segment ratio. Safety will be evaluated by assessment of AEs, SAEs, laboratory test results (urinalysis, chemistry, hematology), changes in vital signs, physical examination, ECG, X-rays/DXA, clinical hip assessment, and anti-BMN 111 immunogenicity assessments. PK sampling will be carried out over the 12-month study period in subjects randomized to BMN 111 or placebo. ; Timepoint(s) of evaluation of this end point: Anthropometric measurements: Screen, Day 1, Week 13, Week 26, Week 39, Week 52 Clinical labs (urinalysis, chemistry, hematology): Screen, Day 1, Day 10, Week 6, Week 13, Week 26, Week 39, Week 52, Week 54 Vital signs and AEs: Screen, Day 1, Day 2, Day 3, Day 10, Week 6, Week 26, Week 39, Week 52, Week 54 (follow-up) Physical exam: Screen, Day 1, Day 10, Week 6, Week 13, Week 26, Week 39, Week 52, Week 54 (follow-up) ECG: Screen, Day 1, Day 10, Week 13, Week 26, Week 39, Week 52, Week 54 (follow-up) X-Ray/DXA: Screen, Week 26, Week 52 Clinical hip assessment: Screen, Week 26, Week 52 Anti-BMN 111 immunogenicity: Day 1, Week 13, Week 26, Week 39, Week 52, Week 54 (follow-up) PK: Day 1 (full), Week 13 (partial), Week 26 (full), Week 39 (partial), Week 52 (full) | — |
Countries
Australia, Germany, Japan, Spain, Turkey, United Kingdom, United States
Contacts
Public ContactClinical Trials Information
BioMarin Pharmaceutical Inc.
Outcome results
None listed