NCT04889690

Brief Summary

This was a multiple ascending dose, randomized, double-blind study assessing the safety, tolerability, pharmacokinetics, and pharmacodynamics of danicopan in healthy participants. Four different doses (75 milligrams \[mg\], 200 mg, 500 mg, 800 mg) and dose-matched placebo were administered under fasted conditions.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
45

participants targeted

Target at P50-P75 for phase_1 healthy

Timeline
Completed

Started May 2016

Typical duration for phase_1 healthy

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

May 21, 2016

Completed
8 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 11, 2017

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

January 11, 2017

Completed
4.3 years until next milestone

First Submitted

Initial submission to the registry

May 12, 2021

Completed
5 days until next milestone

First Posted

Study publicly available on registry

May 17, 2021

Completed
Last Updated

May 17, 2021

Status Verified

May 1, 2021

Enrollment Period

8 months

First QC Date

May 12, 2021

Last Update Submit

May 12, 2021

Conditions

Healthy

Keywords

DanicopanALXN2040ACH-0144471Factor D InhibitorPharmacokineticsPharmacodynamicsAscending Dose

Outcome Measures

Primary Outcomes (1)

  • Incidence Of Serious Adverse Events, Grade 3 Or 4 Adverse Events (AEs), AEs Leading To Discontinuation, And Clinically Significant Laboratory Abnormalities And Electrocardiogram Abnormalities

    Day 1 through Day 42

Secondary Outcomes (5)

  • Maximum Observed Plasma Concentration (Cmax) Of Danicopan

    Up to 16 hours postdose

  • Time To Maximum Observed Plasma Concentration (Tmax) Of Danicopan

    Up to 16 hours postdose

  • Area Under The Plasma Concentration Versus Time Curve Over The Dosing Interval (AUCtau) Of Danicopan

    Up to 16 hours postdose

  • Activity Of Danicopan As Measured By Alternative Pathway (AP) Wieslab Assay

    Up to 16 hours postdose

  • Relationship Between AP Inhibition And Danicopan Plasma Concentrations

    Up to 16 hours postdose

Study Arms (4)

Cohort 1: 200 mg

EXPERIMENTAL

All participants under fasted conditions received 200 mg of danicopan or placebo twice daily (BID) over a 14-day period.

Drug: DanicopanDrug: Placebo

Cohort 2: 500 mg

EXPERIMENTAL

All participants under fasted conditions received 500 mg of danicopan or placebo BID over a 14-day period.

Drug: DanicopanDrug: Placebo

Cohort 3: 800 mg

EXPERIMENTAL

All participants under fasted conditions received 800 mg of danicopan or placebo BID over a 14-day period.

Drug: DanicopanDrug: Placebo

Cohort 4: 75 mg

EXPERIMENTAL

All participants under fasted conditions received 75 mg of danicopan or placebo thrice daily (TID) over a 7-day period.

Drug: DanicopanDrug: Placebo

Interventions

DanicopanDRUG
Also known as: ACH-0144471 (formerly), ALXN2040, ACH-4471, ACH4471, 4471
Cohort 1: 200 mgCohort 2: 500 mgCohort 3: 800 mgCohort 4: 75 mg
PlaceboDRUG
Cohort 1: 200 mgCohort 2: 500 mgCohort 3: 800 mgCohort 4: 75 mg

Eligibility Criteria

Age25 Years - 55 Years
Sexall
Healthy VolunteersYes
Age GroupsAdult (18-64)

You may qualify if:

  • Healthy was defined as having no clinically relevant abnormalities identified by a detailed medical history, physical exam, blood pressure and heart rate measurements, 12-lead electrocardiogram, and clinical laboratory tests.
  • Body mass index of 18 to 30 kilograms (kg)/meter squared with a minimum body weight of 50 kg.

You may not qualify if:

  • History or clinically relevant evidence of significant cardiovascular, pulmonary, hepatic, renal, hematological, gastrointestinal, endocrine, immunologic, dermatologic, neurological, or psychiatric disease.
  • Any condition possibly affecting drug absorption (including gastrectomy and cholecystectomy).
  • Body temperature greater than or equal to 38°Celcius on Day -1 or Day 1, Hour 0; history of febrile illness or other evidence of infection within 14 days prior to first study drug administration.
  • Current tobacco/nicotine user; consumption of any alcohol within 72 hours before first study drug administration or have a history of regular alcohol consumption exceeding 21 drinks/week within 6 months of screening; positive urine drug screen at screening or Day -1.
  • Clinically significant laboratory abnormalities at either Screening or Day -1.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Clinical Trial Site

Auckland, New Zealand

Location

MeSH Terms

Interventions

danicopanrhoA GTP-Binding Protein

Intervention Hierarchy (Ancestors)

rho GTP-Binding ProteinsMonomeric GTP-Binding ProteinsGTP-Binding ProteinsGTP PhosphohydrolasesAcid Anhydride HydrolasesHydrolasesEnzymesEnzymes and CoenzymesCarrier ProteinsProteinsAmino Acids, Peptides, and ProteinsIntracellular Signaling Peptides and Proteins

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
DOUBLE
Who Masked
PARTICIPANT, INVESTIGATOR
Purpose
BASIC SCIENCE
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

May 12, 2021

First Posted

May 17, 2021

Study Start

May 21, 2016

Primary Completion

January 11, 2017

Study Completion

January 11, 2017

Last Updated

May 17, 2021

Record last verified: 2021-05

Data Sharing

IPD Sharing
Will not share

Locations

NZ(1)