Long-Term Low-Intervention SafEty and Clinical Outcomes Clinical Study of LivmArli in Patients with Alagille Syndrome in the European Union (LEAP-EU)

Conditions

Alagille syndrome

Brief summary

Safety: Adverse events (AE); The number and proportion of participants who experience an AE; Change in liver function tests from Baseline; Change in FSV levels and INR (as an estimate of vitamin K) from Baseline; Frequency and timing of long-term clinical outcomes (SBD, LT, LT waitlist status, clinically evident portal hypertension, complications of liver cirrhosis, liver carcinoma, disease progression, liver decompensation, liver-related mortality, and all-cause mortality), Efficacy: Change in pruritus severity from Baseline as measured by Clinician Scratch Scale (CSS) score; Change in serum bile acid (sBA) levels from Baseline; Frequency in the use of concomitant medications for the treatment of underlying liver disease, treatment of pruritus, or treatment of cholestasis, Tolerability: Tolerability of starting dose and dose escalation will be assessed by summarizing AEs and/or any other safety information (as available) on starting dose and during dose escalation phase. The cases of dose reduction, treatment interruption, and treatment discontinuation due to AEs/poor tolerability will be also summarized over the whole treatment period.

Related papers

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Interventions

DRUGLivmarli 9.5 mg/mL oral solution

Eligibility

Sex/Gender

All

Age

0 Years to No maximum

Design outcomes

Primary

Measure	Time frame
Safety: Adverse events (AE); The number and proportion of participants who experience an AE; Change in liver function tests from Baseline; Change in FSV levels and INR (as an estimate of vitamin K) from Baseline; Frequency and timing of long-term clinical outcomes (SBD, LT, LT waitlist status, clinically evident portal hypertension, complications of liver cirrhosis, liver carcinoma, disease progression, liver decompensation, liver-related mortality, and all-cause mortality), Efficacy: Change in pruritus severity from Baseline as measured by Clinician Scratch Scale (CSS) score; Change in serum bile acid (sBA) levels from Baseline; Frequency in the use of concomitant medications for the treatment of underlying liver disease, treatment of pruritus, or treatment of cholestasis, Tolerability: Tolerability of starting dose and dose escalation will be assessed by summarizing AEs and/or any other safety information (as available) on starting dose and during dose escalation phase. The cases of	—

Measure

Time frame

Safety: Adverse events (AE); The number and proportion of participants who experience an AE; Change in liver function tests from Baseline; Change in FSV levels and INR (as an estimate of vitamin K) from Baseline; Frequency and timing of long-term clinical outcomes (SBD, LT, LT waitlist status, clinically evident portal hypertension, complications of liver cirrhosis, liver carcinoma, disease progression, liver decompensation, liver-related mortality, and all-cause mortality), Efficacy: Change in pruritus severity from Baseline as measured by Clinician Scratch Scale (CSS) score; Change in serum bile acid (sBA) levels from Baseline; Frequency in the use of concomitant medications for the treatment of underlying liver disease, treatment of pruritus, or treatment of cholestasis, Tolerability: Tolerability of starting dose and dose escalation will be assessed by summarizing AEs and/or any other safety information (as available) on starting dose and during dose escalation phase. The cases of

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Countries

Belgium, France, Germany, Italy, Netherlands, Spain

Outcome results

None listed