Phase 3 Randomized, Placebo-Controlled Study to Assess Safety, Tolerability and Efficacy of Garetosmab in Patients with Fibrodysplasia Ossificans Progressiva

Conditions

Fibrodysplasia Ossificans Progressiva

Brief summary

Number of new HO lesions, Incidence and severity of treatment-emergent adverse events of special interest (AESIs)

Detailed description

Number of clinician-assessed flare-ups., Occurrence of new HO lesions, Total volume of new HO lesions, Occurrence of patient-reported flare-ups, Number of new HO lesions, Occurrence of clinician-assessed flare ups, Number of patient reported flare-ups, Change in joint function assessment by physician using cumulative analog joint involvement scale (CAJIS), Change in pulmonary function as assessed by spirometry, Change in disease severity as assessed by the Patient Global Impression of Severity (PGIS), Change in disease severity as assessed by the Patient’s Global Impression of Change (PGIC)., Change in disease severity as assessed by the Clinician’s Global Impression of Change (CGIC)., Concentration of total activin A in serum over time., Concentrations of garetosmab in serum over time., Incidence of anti-drug antibodies (ADA) to garetosmab over time., Titer of ADA to garetosmab over time.

Related papers

No related papers found yet.

Interventions

DRUGGaretosmab

DRUGPlacebo matching to garetosmab

Eligibility

Sex/Gender

All

Age

18 Years to No maximum

Design outcomes

Primary

Measure	Time frame
Number of new HO lesions, Incidence and severity of treatment-emergent adverse events of special interest (AESIs)	—

Secondary

Measure	Time frame
Number of clinician-assessed flare-ups., Occurrence of new HO lesions, Total volume of new HO lesions, Occurrence of patient-reported flare-ups, Number of new HO lesions, Occurrence of clinician-assessed flare ups, Number of patient reported flare-ups, Change in joint function assessment by physician using cumulative analog joint involvement scale (CAJIS), Change in pulmonary function as assessed by spirometry, Change in disease severity as assessed by the Patient Global Impression of Severity (PGIS), Change in disease severity as assessed by the Patient’s Global Impression of Change (PGIC)., Change in disease severity as assessed by the Clinician’s Global Impression of Change (CGIC)., Concentration of total activin A in serum over time., Concentrations of garetosmab in serum over time., Incidence of anti-drug antibodies (ADA) to garetosmab over time., Titer of ADA to garetosmab over time.	—

Measure

Time frame

Number of clinician-assessed flare-ups., Occurrence of new HO lesions, Total volume of new HO lesions, Occurrence of patient-reported flare-ups, Number of new HO lesions, Occurrence of clinician-assessed flare ups, Number of patient reported flare-ups, Change in joint function assessment by physician using cumulative analog joint involvement scale (CAJIS), Change in pulmonary function as assessed by spirometry, Change in disease severity as assessed by the Patient Global Impression of Severity (PGIS), Change in disease severity as assessed by the Patient’s Global Impression of Change (PGIC)., Change in disease severity as assessed by the Clinician’s Global Impression of Change (CGIC)., Concentration of total activin A in serum over time., Concentrations of garetosmab in serum over time., Incidence of anti-drug antibodies (ADA) to garetosmab over time., Titer of ADA to garetosmab over time.

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Countries

Finland, France, Italy, Netherlands, Poland, Spain

Outcome results

None listed