HealthyPhase 1NCT04935294
Study of Danicopan in Participants With Normal Kidney Function and Participants With Kidney Dysfunction
A Phase 1 study testing Danicopan in people with Healthy and Renal Dysfunction, run by Alexion Pharmaceuticals, Inc. at 4 sites in United States.
Trial summary
This was an open-label, nonrandomized, multi-center, single-dose, parallel group study to evaluate the effect of severe renal impairment (RI) on the safety, tolerability, pharmacokinetics, and pharmacodynamics of danicopan (ACH-0144471) compared to demographically-matched healthy participants with normal renal… Read more about this study
- Who is it for?
- Ages 18 to 75 yearsHealthy volunteers can take part
- Conditions the study is for
- Healthy, Renal Dysfunction
- What is being tested?
- Danicopan
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 16 participants
- Where
- 4 sitesUnited States
- Study dates
- 24 Jan 2018to 29 May 2018
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Body mass index (BMI) in the range of 18.0 to 40.0 kilograms (kg)/meter squared, inclusive, with a minimum body weight of 50 kg at screening.
- Stable creatinine clearance.
- Participants with RI must have met the following additional criteria to be enrolled in this study
- A stable medication regimen was required. Concomitant medications must have been approved by the Sponsor and Investigator (or designee) or pre-specified in the protocol.
- Abnormal laboratory values must not have been clinically relevant at screening or check-in.
- Participants were in good general health at screening and check-in, allowing for the concurrent illnesses associated with chronic kidney disease.
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- Stable severe RI.
- Healthy matched control participants with normal renal function must have met the following additional criteria to be enrolled in this study
- In good health, determined by no clinically significant findings from medical history, physical examination, 12-lead electrocardiogram, and vital sign measurements at screening or check-in, as determined by the Investigator (or designee).
- Participants had normal renal function.
- Participants must have been demographically matched to a renally impaired participant by age (± 10 years), sex, and BMI (± 20%) at screening.
Who cannot join
- History of any medical or psychiatric condition or disease (aside from RI for RI participants) that might have limited the participant's ability to complete or participate in this clinical study, confound the results of the study, or pose an additional risk to the participant by their participation in the study.
- History of procedures that could alter absorption or excretion of orally administered drugs.
- Body temperature ≥ 38°Celsius on Day - 1 or Day 1 predose; history of febrile illness, or other evidence of infection, within 14 days prior to study drug administration.
- History or presence of drug or alcohol abuse within previous year; current tobacco/nicotine user; positive for alcohol and/or drug screen at screening.
- Participation in any other investigational study drug trial in which receipt of an investigational study drug occurred within 5 half-lives (if known) or 30 days prior to check-in, whichever was longer.
- For matched control participants, use of any prescription medications/products within 14 days prior to check-in and use of any over-the-counter, nonprescription preparations within 7 days prior to check-in, unless deemed acceptable by the Investigator (or designee).
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- Donation of whole blood from 3 months prior to study drug administration, or of plasma from 30 days prior to study drug administration; receipt of blood products within 6 months prior to check-in.
- Participant required dialysis within the last 3 months prior to check-in.
- Participant did not produce sufficient urine output to permit urine sampling at screening and/or check-in; history of urinary incontinence prior to check-in.
- History of kidney transplant or actively on a transplant waiting list prior to check-in.
- Any acute or chronic non-renal condition prior to check-in that would have limited the participant's ability to complete or participate in this clinical study.
- Any major surgery within 4 weeks of study drug administration.
Trial locations and contacts
4 sites in 1 country.
United States4 sites
Clinical Trial Site
Miami, Florida, 33014
Clinical Trial Site
Minneapolis, Minnesota, 55404
Clinical Trial Site
Orlando, Florida, 32809
Clinical Trial Site
Saint Paul, Minnesota, 55114
About this study
Official title: A Phase I, Open-Label, Parallel Group Study To Evaluate ACH-0144471 Safety, Tolerability, And Pharmacokinetics In Subjects With Normal Renal Function And Subjects With Renal Dysfunction
This was an open-label, nonrandomized, multi-center, single-dose, parallel group study to evaluate the effect of severe renal impairment (RI) on the safety, tolerability, pharmacokinetics, and pharmacodynamics of danicopan (ACH-0144471) compared to demographically-matched healthy participants with normal renal function.
- Conditions
- Healthy, Renal Dysfunction
- Being tested
- Danicopan
- Keywords
- Danicopan, ALXN2040, ACH-0144471, Renal Impairment, Pharmacokinetics, Pharmacodynamics, Factor D Inhibitor, Safety
- Medical subject headings
- Renal Insufficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DanicopanDrug
Also called ALXN2040, ACH-0144471 (formerly), ACH-4471, ACH4471
Oral tablet.
| Study group | Role | What they receive |
|---|---|---|
| Group 1: Matched Control Group Of Healthy Participants | Experimental | Participants received a single 200-milligram (mg) treatment on Day 1. Receives: Drug: Danicopan |
| Group 2: Severe RI And Not On Dialysis | Experimental | Participants received a single 200-mg treatment on Day 1. Receives: Drug: Danicopan |
What is being measured
The primary outcome is the main question the study is designed to answer.
Participants With Treatment-emergent Adverse Events
Measured: Day 1 through Day 10 (+/- 2 days)
Secondary outcomes (6)
Area Under The Plasma Concentration-time Curve Extrapolated To Infinity (AUC0-inf) Of Danicopan
Measured: Up to 72 hours postdose
Maximum Observed Plasma Concentration (Cmax) Of Danicopan
Measured: Up to 72 hours postdose
Time To Reach The Maximum Observed Plasma Concentration (Tmax) Of Danicopan
Measured: Up to 72 hours postdose
Alternative Pathway Activity As Measured By Wieslab Assay
Measured: Up to 72 hours postdose
Change From Baseline In Plasma Bb Fragment Of Complement Factor B Concentration
Measured: Baseline, Up to 72 hours postdose
Change From Baseline In Complement Factor D Concentration
Measured: Baseline, Up to 72 hours postdose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Jun 2021
- Study start24 Jan 2018
- Primary completion29 May 2018
- Study completion29 May 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for HealthyStudies of the same condition, open and under way.
- More trials run by Alexion Pharmaceuticals, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Basic science
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 16 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Achillion, a wholly owned subsidiary of Alexion
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04935294
- Sponsor's protocol ID
- ACH471-009
- Status last verified
- Jun 2021
- First submitted
- 21 Jun 2021
- Last updated
- 23 Jun 2021
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Jun 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I, Open-Label, Parallel Group Study To Evaluate ACH-0144471 Safety, Tolerability, And Pharmacokinetics In Subjects With Normal Renal Function And Subjects With Renal Dysfunction
- ClinicalTrials.gov ID
- NCT04935294
- Acronym / sponsor ID
- ACH471-009
- Conditions
- Healthy, Renal Dysfunction
- Keywords
- Danicopan, ALXN2040, ACH-0144471, Renal Impairment, Pharmacokinetics, Pharmacodynamics, Factor D Inhibitor, Safety
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 16 participants (actual)
- Lead sponsor
- Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Achillion, a wholly owned subsidiary of Alexion
- Study start
- 24 Jan 2018 (actual)
- Primary completion
- 29 May 2018 (actual)
- Study completion
- 29 May 2018 (actual)
- First posted
- 23 Jun 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This was an open-label, nonrandomized, multi-center, single-dose, parallel group study to evaluate the effect of severe renal impairment (RI) on the safety, tolerability, pharmacokinetics, and pharmacodynamics of danicopan (ACH-0144471) compared to demographically-matched healthy participants with normal renal function.
Study design
- Primary purpose
- Basic science
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Group 1: Matched Control Group Of Healthy Participants | Experimental | Drug: DanicopanParticipants received a single 200-milligram (mg) treatment on Day 1. |
| Group 2: Severe RI And Not On Dialysis | Experimental | Drug: DanicopanParticipants received a single 200-mg treatment on Day 1. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- Body mass index (BMI) in the range of 18.0 to 40.0 kilograms (kg)/meter squared, inclusive, with a minimum body weight of 50 kg at screening.
- Stable creatinine clearance.
- Participants with RI must have met the following additional criteria to be enrolled in this study
- A stable medication regimen was required. Concomitant medications must have been approved by the Sponsor and Investigator (or designee) or pre-specified in the protocol.
- Abnormal laboratory values must not have been clinically relevant at screening or check-in.
Key exclusion criteria (12 listed)
- History of any medical or psychiatric condition or disease (aside from RI for RI participants) that might have limited the participant's ability to complete or participate in this clinical study, confound the results of the study, or pose an additional risk to the participant by their participation in the study.
- History of procedures that could alter absorption or excretion of orally administered drugs.
- Body temperature ≥ 38°Celsius on Day - 1 or Day 1 predose; history of febrile illness, or other evidence of infection, within 14 days prior to study drug administration.
- History or presence of drug or alcohol abuse within previous year; current tobacco/nicotine user; positive for alcohol and/or drug screen at screening.
- Participation in any other investigational study drug trial in which receipt of an investigational study drug occurred within 5 half-lives (if known) or 30 days prior to check-in, whichever was longer.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| DanicopanAlso: ALXN2040, ACH-0144471 (formerly), ACH-4471, ACH4471Oral tablet. | Drug | Group 1: Matched Control Group Of Healthy Participants, Group 2: Severe RI And Not On Dialysis |
Primary outcome
Participants With Treatment-emergent Adverse Events
Time frame: Day 1 through Day 10 (+/- 2 days)
6 secondary outcomes
Area Under The Plasma Concentration-time Curve Extrapolated To Infinity (AUC0-inf) Of Danicopan
Time frame: Up to 72 hours postdose
Maximum Observed Plasma Concentration (Cmax) Of Danicopan
Time frame: Up to 72 hours postdose
Time To Reach The Maximum Observed Plasma Concentration (Tmax) Of Danicopan
Time frame: Up to 72 hours postdose
Alternative Pathway Activity As Measured By Wieslab Assay
Time frame: Up to 72 hours postdose
Change From Baseline In Plasma Bb Fragment Of Complement Factor B Concentration
Time frame: Baseline, Up to 72 hours postdose
Change From Baseline In Complement Factor D Concentration
Time frame: Baseline, Up to 72 hours postdose
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Jun 2021.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Danicopan in people with Healthy and Renal Dysfunction, run by Alexion Pharmaceuticals, Inc. at 4 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 23 Jun 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Participants With Treatment-emergent Adverse Events (time frame: Day 1 through Day 10 (+/- 2 days)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Jun 2021.