Open-label single-arm study

12 evidence topics · 2 sources

Objective

ALP-response assessment

“ALP” (opens the source at this quote in a new tab)

Location and study date

Study population: Mexico

“Mexican” (opens the source at this quote in a new tab)

Study design

Open-label design

“open-label” (opens the source at this quote in a new tab)

Eligibility criteria

Prior treatment: ongoing UDCA

“ongoing treatment with UDCA” (opens the source at this quote in a new tab)

Treatment

Open-label, single-arm study: regimen

Field	Quoted finding
Regimen	“4 mg for 16 weeks”

Study outcomes

Rationale for using ALP reduction as the primary endpoint

Prognostic association of ALP and bilirubin

“Combining levels of alkaline phosphatase and bilirubin increased the ability to predict patient survival times.” (opens the source at this quote in a new tab)

Statistical analysis description

Number of participants (Planned and analyzed)

Open-label, single-arm study: enrollment

Field	Quoted finding
Enrolled patients	“7”

Description of analysis sets

Primary analysis population

“modified intent-to-treat” (opens the source at this quote in a new tab)

Results

Participant disposition

Open-label, single-arm study: termination

Field	Quoted finding
Reason for study termination	“lack of enrollment”

Baseline characteristics

Open-label, single-arm study: baseline characteristics

Field	Quoted finding
Sex	“all patients were female”
Mean age, years	“51.1 ± 10.0”
Mean baseline ALP, U/L	“230 ± 103”

Efficacy results

Open-label, single-arm study: ALP response

Field	Quoted finding
Mean ALP reduction at Week 16	“48 ± 23%”

Health-related quality of life and patient-reported outcomes

No evidence found.

Safety results

No evidence found.

Study limitations

Summary

Only seven participants were enrolled, all female, and the study ended because of lack of enrollment. Its open-label, single-arm design cannot separate treatment effects from changes that would have occurred without saroglitazar. The 16-week biochemical assessment does not establish a transplantation or survival benefit.