A Phase 2, Open-Label, Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children with Achondroplasia

Conditions

Achondroplasia

Brief summary

Safety will be evaluated by the incidence of AEs, serious adverse events (SAEs), laboratory test results (urinalysis, chemistry, hematology), changes in vital signs, physical examination, and echocardiogram (ECHO), X-rays/QCT results, and clinical hip assessment.

Detailed description

Efficacy will be assessed by change from baseline in height growth velocity (annualized to cm/year), height, final adult height, growth parameters, and in body proportions. These changes will be assessed by anthropometric measurements and measurement ratios. Growth parameters (anthropometric measurements) may include but are not limited to height, standing height, sitting height, weight, head circumference, upper and lower arm and leg length, and arm span., Body proportion measurements may include but are not limited to upper: lower body segment ratio, upper arm: forearm length ratio, upper leg: lower leg length ratio, and arm span: standing height ratio. Final adult height defined as the height achieved at 16 years of age for females, and 18 years of age for males.

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Interventions

DRUGVoxzogo 0.56 mg powder and solvent for solution for injection

DRUGVoxzogo 1.2 mg powder and solvent for solution for injection

Eligibility

Sex/Gender

All

Age

0 Years to 17 Years

Design outcomes

Primary

Measure	Time frame
Safety will be evaluated by the incidence of AEs, serious adverse events (SAEs), laboratory test results (urinalysis, chemistry, hematology), changes in vital signs, physical examination, and echocardiogram (ECHO), X-rays/QCT results, and clinical hip assessment.	—

Secondary

Measure	Time frame
Efficacy will be assessed by change from baseline in height growth velocity (annualized to cm/year), height, final adult height, growth parameters, and in body proportions. These changes will be assessed by anthropometric measurements and measurement ratios. Growth parameters (anthropometric measurements) may include but are not limited to height, standing height, sitting height, weight, head circumference, upper and lower arm and leg length, and arm span., Body proportion measurements may include but are not limited to upper: lower body segment ratio, upper arm: forearm length ratio, upper leg: lower leg length ratio, and arm span: standing height ratio. Final adult height defined as the height achieved at 16 years of age for females, and 18 years of age for males.	—

Measure

Time frame

Efficacy will be assessed by change from baseline in height growth velocity (annualized to cm/year), height, final adult height, growth parameters, and in body proportions. These changes will be assessed by anthropometric measurements and measurement ratios. Growth parameters (anthropometric measurements) may include but are not limited to height, standing height, sitting height, weight, head circumference, upper and lower arm and leg length, and arm span., Body proportion measurements may include but are not limited to upper: lower body segment ratio, upper arm: forearm length ratio, upper leg: lower leg length ratio, and arm span: standing height ratio. Final adult height defined as the height achieved at 16 years of age for females, and 18 years of age for males.

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Countries

France

Outcome results

None listed