MOL-001

18 evidence topics · 2 sources

Objective

Safety and clinical pharmacology

“safety, PK and PD” (opens the source at this quote in a new tab)

Location and study date

Study location and enrollment period

Attribute	Quoted specification
Clinical research facility	“Celerion”
Location	“Belfast”
Enrollment period	“May through September 2015”

Study design

Phase 1 randomized pharmacology study

“randomised, placebo-controlled, blinded, phase 1 clinical trial” (opens the source at this quote in a new tab)

Eligibility criteria

Healthy nonsmoking adult population

“life-long non-smokers” (opens the source at this quote in a new tab)

Age limits

Eligibility measure	Reported range
Age, years	“18 to 55”

Treatment

Single ascending-dose cohorts

Cohort	Molgramostim dose, µg	Participants
First active-dose cohort	“150”	“4”
Second active-dose cohort	“300”	“4”
Third active-dose cohort	“600”	“4”
Pooled placebo cohort	“placebo”	“6”

Multiple ascending-dose cohorts

Cohort	Daily molgramostim dose, µg	Participants
First active-dose cohort	“300”	“9”
Second active-dose cohort	“600”	“9”
Pooled placebo cohort	“placebo”	“6”

Repeated-dose duration

Treatment component	Reported duration
Consecutive daily doses in the multiple ascending-dose study	“six”

Study outcomes

Rationale for using adverse events as the primary endpoint

Not applicable.

Statistical analysis description

Number of participants (Planned and analyzed)

Total enrollment

“42 subjects were enrolled” (opens the source at this quote in a new tab)

Enrollment by study component

Study component	Participants
Single ascending dose	“18”
Multiple ascending dose	“24”

Description of analysis sets

Pharmacokinetic analysis method

“Non-compartmental PK” (opens the source at this quote in a new tab)

Multiplicity

“All reported p-values were two-sided without adjustment for multiple testing.” (opens the source at this quote in a new tab)

Formal analysis-set definitions

No evidence found.

Results

Participant disposition

Study completion

Disposition measure	Participants
Enrolled and completed the study	“42”

Baseline characteristics

Overall demographic characteristics

Characteristic	Reported value
Age, years, mean and standard deviation	“33.7±11.7”
Age range, years	“(18, 53)”
Male, number and percentage	“40 (95)”
Female, number and percentage	“2 (5)”
White, number and percentage	“40 (95)”
Black, number and percentage	“2 (5)”

Efficacy results

Pharmacokinetic results in healthy volunteers

“healthy people” (opens the source at this quote in a new tab)
“peaked at 2 hours for all three doses and had an elimination half-life of 1.7±0.0 to 5.9±0.9 hours” (opens the source at this quote in a new tab)

Disease-specific efficacy

Not applicable.

Health-related quality of life and patient-reported outcomes

Not applicable.

Safety results

Adverse events by dosing component

Study component	Molgramostim: participants with adverse events	Placebo: participants with adverse events
Single ascending dose	“7 (58%)”	“4 (67%)”
Multiple ascending dose	“15 (83%)”	“6 (100%)”

Serious adverse events

“No serious AEs occurred” (opens the source at this quote in a new tab)

Study limitations

Summary

This study enrolled 42 healthy participants, including two women, and evaluated single doses or six consecutive daily doses. It does not establish efficacy in autoimmune pulmonary alveolar proteinosis or the safety of prolonged daily treatment. The sample size and short exposure limit assessment of uncommon or delayed adverse events. Statistical comparisons were not adjusted for multiple testing.