A Phase 2, Randomized, Controlled, Multicenter Study of Vosoritide in Children With Idiopathic Short Stature

Conditions

Idiopathic Short Stature (ISS)

Brief summary

Dose-Finding Phase: Change from baseline in AGV at 6 months Long-Term Phase: Change from baseline in height and height Z-score at 4 years

Detailed description

1. Incidence of treatment-emergent adverse events over the course of the study, 2. Change from baseline in height Z-score at 6 months, 3. Change from baseline in height at each visit and at FAH, 4. Change from baseline in height Z-score at each visit and at FAH, 5. PK parameters (eg, Tmax, Cmax, AUC0-t, AUC0-inf, t1/2, CL/F, Vz/F), 6. Change from baseline at prespecified timepoints in: • urine cGMP • serum CXM, 7. Change from baseline in bone age/chronological age at prespecified timepoints, 8. Change from baseline at each visit in the following, as measured by DXA: • total body (less head) BMD Z-score • lumbar spine BMD Z-score • total body (less head) BMC • lumbar spine BMC

Related papers

No related papers found yet.

Interventions

DRUGmodified recombinant human C-type natriuretic peptide (rhCNP)

DRUGVoxzogo 0.56 mg powder and solvent for solution for injection

DRUGLyophilized powder for reconstitution

DRUGVoxzogo 1.2 mg powder and solvent for solution for injection

Eligibility

Sex/Gender

All

Age

0 Years to 64 Years

Design outcomes

Primary

Measure	Time frame
Dose-Finding Phase: Change from baseline in AGV at 6 months Long-Term Phase: Change from baseline in height and height Z-score at 4 years	—

Secondary

Measure	Time frame
1. Incidence of treatment-emergent adverse events over the course of the study, 2. Change from baseline in height Z-score at 6 months, 3. Change from baseline in height at each visit and at FAH, 4. Change from baseline in height Z-score at each visit and at FAH, 5. PK parameters (eg, Tmax, Cmax, AUC0-t, AUC0-inf, t1/2, CL/F, Vz/F), 6. Change from baseline at prespecified timepoints in: • urine cGMP • serum CXM, 7. Change from baseline in bone age/chronological age at prespecified timepoints, 8. Change from baseline at each visit in the following, as measured by DXA: • total body (less head) BMD Z-score • lumbar spine BMD Z-score • total body (less head) BMC • lumbar spine BMC	—

Measure

Time frame

1. Incidence of treatment-emergent adverse events over the course of the study, 2. Change from baseline in height Z-score at 6 months, 3. Change from baseline in height at each visit and at FAH, 4. Change from baseline in height Z-score at each visit and at FAH, 5. PK parameters (eg, Tmax, Cmax, AUC0-t, AUC0-inf, t1/2, CL/F, Vz/F), 6. Change from baseline at prespecified timepoints in: • urine cGMP • serum CXM, 7. Change from baseline in bone age/chronological age at prespecified timepoints, 8. Change from baseline at each visit in the following, as measured by DXA: • total body (less head) BMD Z-score • lumbar spine BMD Z-score • total body (less head) BMC • lumbar spine BMC

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Countries

France, Germany, Italy

Outcome results

None listed