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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05083364




Registration number
NCT05083364
Ethics application status
Date submitted
8/10/2021
Date registered
19/10/2021

Titles & IDs
Public title
Study of ARO-C3 in Adult Healthy Volunteers and Patients With Complement Mediated Renal Disease
Scientific title
A Phase 1/2a Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and/or Pharmacodynamics of ARO-C3 in Adult Healthy Volunteers and in Adult Patients With Complement-Mediated Renal Disease
Secondary ID [1] 0 0
AROC3-1001
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
C3 Glomerulopathy 0 0
IgA Nephropathy 0 0
Condition category
Condition code
Renal and Urogenital 0 0 0 0
Kidney disease
Inflammatory and Immune System 0 0 0 0
Autoimmune diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - ARO-C3
Treatment: Drugs - Placebo

Experimental: ARO-C3 (Healthy Volunteers) - 1 or 2 doses of ARO-C3 by subcutaneous (sc) injection

Placebo comparator: Placebo (Healthy Volunteers) - placebo calculated volume to match active treatment by sc injection

Experimental: ARO-C3 (Adult Patients with C3G or IgAN) - 3 doses of ARO-C3 by sc injection


Treatment: Drugs: ARO-C3
ARO-C3 for sc injection

Treatment: Drugs: Placebo
sterile normal saline (0.9% NaCl) for sc injection
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Participants with Adverse Events (AEs) and/or Serious Adverse Events (SAEs) at Day 169
Timepoint [1] 0 0
up to day 169 (End of Study [EOS])
Secondary outcome [1] 0 0
Pharmacokinetics (PK) of ARO-C3: Maximum Observed Plasma Concentration (Cmax)
Timepoint [1] 0 0
up to 48 hours post-dose
Secondary outcome [2] 0 0
PK of ARO-C3: Area under the Plasma Concentration Versus Time Curve from Zero to 24Hours (AUC0-24)
Timepoint [2] 0 0
up to 48 hours post-dose
Secondary outcome [3] 0 0
PK of ARO-C3: Area Under the Plasma Versus Time Concentration Curve from Zero to the Last Quantifiable Plasma Concentration (AUClast)
Timepoint [3] 0 0
up to 48 hours post-dose
Secondary outcome [4] 0 0
PK of ARO-C3: Area Under the Plasma Concentration Versus Time Curve from Zero Extrapolated to Infinity (AUCinf) PK of ARO-C3:
Timepoint [4] 0 0
up to 48 hours post-dose
Secondary outcome [5] 0 0
PK of ARO-C3: Terminal Elimination Half-Life (t1/2)
Timepoint [5] 0 0
up to 48 hours post-dose
Secondary outcome [6] 0 0
PK of ARO-C3: Apparent Total Body Clearance of ARO-C3 from Plasma (CL)
Timepoint [6] 0 0
up to 48 hours post-dose
Secondary outcome [7] 0 0
PK of ARO-C3: Volume of Distribution (Vz/F)
Timepoint [7] 0 0
up to 48 hours post-dose
Secondary outcome [8] 0 0
Pharmacodynamics (PD): Change From Baseline in Complement 3 (C3) up to Day 169
Timepoint [8] 0 0
Baseline, through Day 169 (EOS)
Secondary outcome [9] 0 0
PD: Percent Change From Baseline in C3 up to Day 169
Timepoint [9] 0 0
Baseline, through Day 169 (EOS)

Eligibility
Key inclusion criteria
Inclusion Criteria (All Participants):

* Willing to provide written informed consent and to comply with study requirements
* Female participants must be non-pregnant/non-lactating
* Healthy volunteers must be willing to be vaccinated with a meningococcal and pneumococcal vaccine. C3G and IgAN participants must have been vaccinated or willing to undergo vaccination
* All participants must be willing to be vaccinated or have a history of vaccination for Haemophilus influenzae
* Body Mass Index (BMI) between 18.0 and 35.0 kg/m2
* 12-lead electrocardiogram (ECG) at Screening with no abnormalities that may compromise participant's safety at discretion of investigator
* Participants of childbearing potential must use highly effective contraception during the study and for at least 12 weeks following the end of the study or last dose of study drug, whichever is later. Males must not donate sperm during the study and for at least 12 weeks following the end of the study or last dose of study drug, whichever is later.
* No abnormal finding of clinical relevance at the Screening evaluation that, in the opinion of the investigator, could adversely impact participant safety or study results

Inclusion Criteria (C3G and IgAN Participants):

* Diagnosis of C3G or IgAN
* Clinical evidence of ongoing disease based on significant proteinuria
* Estimated glomerular filtration rate =30 mL/Min/1.73 m2 at Screening and currently not on dialysis
* Must be on a maximally recommended or tolerated dose of an angiotensin converting enzyme inhibitor (ACEI) or angiotensin receptor blocker (ARB)
Minimum age
18 Years
Maximum age
70 Years
Sex
Both males and females
Can healthy volunteers participate?
Yes
Key exclusion criteria
Exclusion Criteria (All Participants):

* Seropositive for human immunodeficiency virus (HIV) infection,hepatitis B virus, or hepatitis C virus
* History of recurrent or chronic infections
* Uncontrolled hypertension
* Regular use of alcohol within 30 days prior to Screening
* Use of illicit drugs within 1 year prior to Screening or positive urine drug screen at Screening
* History of meningococcal infection
* History of asplenia or splenectomy
* Known contraindication or history of anaphylactic reaction to any vaccine or vaccine component or prophylactic antibiotics planned for use in the study
* Any medical or surgical condition that, in the opinion of the investigator, would expose the participant to a significant safety risk or compromise the results of the study

Note: Additional Inclusion/Exclusion criteria may apply per protocol

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Other
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
2/02/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/06/2025
Actual
Sample size
Target
60
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
Research Site 1 - Camperdown
Recruitment hospital [2] 0 0
Research Site 3 - Concord
Recruitment hospital [3] 0 0
Research Site 2 - Clayton
Recruitment postcode(s) [1] 0 0
2050 - Camperdown
Recruitment postcode(s) [2] 0 0
2139 - Concord
Recruitment postcode(s) [3] 0 0
3168 - Clayton
Recruitment outside Australia
Country [1] 0 0
Georgia
State/province [1] 0 0
Tbilisi
Country [2] 0 0
Germany
State/province [2] 0 0
Cologne
Country [3] 0 0
Germany
State/province [3] 0 0
Erlangen
Country [4] 0 0
Germany
State/province [4] 0 0
Halle
Country [5] 0 0
Germany
State/province [5] 0 0
Mainz
Country [6] 0 0
Korea, Republic of
State/province [6] 0 0
Busan
Country [7] 0 0
Korea, Republic of
State/province [7] 0 0
Gyeonggi-do
Country [8] 0 0
Korea, Republic of
State/province [8] 0 0
Daegu
Country [9] 0 0
Korea, Republic of
State/province [9] 0 0
Soeul
Country [10] 0 0
New Zealand
State/province [10] 0 0
Auckland
Country [11] 0 0
Thailand
State/province [11] 0 0
Bangkok
Country [12] 0 0
Thailand
State/province [12] 0 0
Chiang Mai
Country [13] 0 0
United Kingdom
State/province [13] 0 0
Cambridge
Country [14] 0 0
United Kingdom
State/province [14] 0 0
Coventry
Country [15] 0 0
United Kingdom
State/province [15] 0 0
Leicester
Country [16] 0 0
United Kingdom
State/province [16] 0 0
Liverpool
Country [17] 0 0
United Kingdom
State/province [17] 0 0
Newcastle
Country [18] 0 0
United Kingdom
State/province [18] 0 0
Oxford

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Arrowhead Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Clinical Operations Lead
Address 0 0
Country 0 0
Phone 0 0
626-304-3400
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT05083364