ANZCTR - Registration

Please note that the copy function is not enabled for this field.
If you wish to modify existing outcomes, please copy and paste the current outcome text into the Update field.

The ANZCTR website will be unavailable from 1pm until 3pm (AEDT) on Wednesday the 30th of October for website maintenance. Please be sure to log out of the system in order to avoid any loss of data.

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers

Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06131983

Registration number

NCT06131983

Ethics application status

Date submitted

8/11/2023

Date registered

15/11/2023

Titles & IDs

Public title

Study of ARO-DUX4 in Adult Patients With Facioscapulohumeral Muscular Dystrophy Type 1

Scientific title

A Phase1/2a Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ARO-DUX4 in Adult Patients With Facioscapulohumeral Muscular Dystrophy Type 1

Secondary ID [1]

ARODUX4-1001

Universal Trial Number (UTN)

Trial acronym

Linked study record

Health condition

Health condition(s) or problem(s) studied:

Facio-Scapulo-Humeral Dystrophy

Condition category

Condition code

Musculoskeletal

Other muscular and skeletal disorders

Neurological

Other neurological disorders

Human Genetics and Inherited Disorders

Other human genetics and inherited disorders

Intervention/exposure

Study type

Interventional

Description of intervention(s) / exposure

Treatment: Drugs - ARO-DUX4 for Injection
Treatment: Drugs - Placebo

Experimental: ARO-DUX4 - ARO-DUX4 for Injection

Placebo comparator: Placebo - (0.9%NaCl)

Treatment: Drugs: ARO-DUX4 for Injection
single or multiple doses of ARO-DUX4 by intravenous (IV) infusion

Treatment: Drugs: Placebo
calculated volume to match active treatment by IV infusion

Intervention code [1]

Treatment: Drugs

Comparator / control treatment

Control group

Outcomes

Primary outcome [1]

Number of Participants With Treatment-Emergent Adverse Events (TEAEs) Over Time Through End of Study (EOS)

Timepoint [1]

Single dose phase: Up to Day 90; Multiple dose phase: Up to Day 360

Secondary outcome [1]

Pharmacokinetics (PK) of ARO-DUX4: Maximum Observed Plasma Concentration (Cmax)

Timepoint [1]

Single dose phase: through 48 hours post-dose, Multiple dose phase: through 24 hours post first and second dose

Secondary outcome [2]

PK of ARO-DUX4: Area Under the Plasma Concentration Versus Time Curve from Zero to 24 Hours (AUC0-24)

Timepoint [2]

Single dose phase: through 48 hours post-dose, Multiple dose phase: through 24 hours post first and second dose

Secondary outcome [3]

PK of ARO-DUX4: Area Under the Plasma Concentration Versus Time Curve from Zero to the Last Quantifiable Plasma Concentration (AUClast)

Timepoint [3]

Single dose phase: through 48 hours post-dose, Multiple dose phase: through 24 hours post first and second dose

Secondary outcome [4]

PK of ARO-DUX4: Area Under the Plasma Concentration Versus Time from Zero to Infinity (AUCinf)

Timepoint [4]

Single dose phase: through 48 hours post-dose, Multiple dose phase: through 24 hours post first and second dose

Secondary outcome [5]

PK of ARO-DUX4: Terminal Elimination Half-Life (t1/2)

Timepoint [5]

Single dose phase: through 48 hours post-dose, Multiple dose phase: through 24 hours post first and second dose

Secondary outcome [6]

PK of ARO-DUX4: Systemic Clearance (CL)

Timepoint [6]

Single dose phase: through 48 hours post-dose, Multiple dose phase: through 24 hours post first and second dose

Secondary outcome [7]

PK of ARO-DUX4: Volume of Distribution (Vss)

Timepoint [7]

Single dose phase: through 48 hours post-dose, Multiple dose phase: through 24 hours post first and second dose

Secondary outcome [8]

PK of ARO-DUX4: Recovery of Unchanged Drug in Urine Over 0-24 Hours (Amount Excreted: Ae)

Timepoint [8]

Single dose phase: through 48 hours post-dose, Multiple dose phase: through 24 hours post first and second dose

Secondary outcome [9]

PK of ARO-DUX4: Fraction of Drug Excreted in Urine as Percent of Intravenous (IV) Dose (Fe)

Timepoint [9]

Single dose phase: through 48 hours post-dose, Multiple dose phase: through 24 hours post first and second dose

Secondary outcome [10]

PK of ARO-DUX4: Renal Clearance (CLr)

Timepoint [10]

Single dose phase: through 48 hours post-dose, Multiple dose phase: through 24 hours post first and second dose

Eligibility

Key inclusion criteria

* Genetically confirmed FSHD1 based on Screening evaluation or source verifiable medical record
* Clinical severity score between 3 and 8 (scale, 0 to 10)
* Must have eligible lower extremity muscle for biopsy as determined from MRI by a central reader
* A 12-lead electrocardiogram (ECG) at Screening with no abnormalities that may compromise participant's safety in the study
* Participants of childbearing potential and their partners must use highly effective contraception during the study and for at least 12 weeks following the end of study or last dose of study medication, whichever is later. Males must not donate sperm during the study from Day 1 until at least 12 weeks following the end of study or last dose of study medication, whichever is later.

Minimum age

18 Years

Maximum age

70 Years

Sex

Both males and females

Can healthy volunteers participate?

No

Key exclusion criteria

* Human Immunodeficiency Virus (HIV) infection as shown by presence of anti-HIV antibody (seropositive) at Screening
* Seropositive for hepatitis B (HBV) or hepatitis C (HCV) at Screening
* Uncontrolled hypertension
* Severe cardiovascular disease
* History of thrombolic events
* Platelet count less that the lower limit of normal at Screening
* History or presence of: a hypercoagulable state, nephrotic range proteinuria, antiphospholipid antibody syndrome, myeloproliferative disease, inability to ambulate, use of hormone-based contraceptives.
* Any contraindication to muscle biopsy or MRI

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

Parallel

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

26/02/2024

Date of last participant enrolment

Anticipated

Actual

Date of last data collection

Anticipated

1/06/2025

Actual

Sample size

Target

52

Accrual to date

Final

Recruitment in Australia

Recruitment state(s)

NSW,QLD

Recruitment hospital [1]

Research Site - Liverpool

Recruitment hospital [2]

Research Site - Birtinya

Recruitment postcode(s) [1]

2170 - Liverpool

Recruitment postcode(s) [2]

4575 - Birtinya

Recruitment outside Australia

Country [1]

New Zealand

State/province [1]

Auckland

Funding & Sponsors

Primary sponsor type

Commercial sector/industry

Name

Arrowhead Pharmaceuticals

Address

Country

Ethics approval

Ethics application status

Summary

Brief summary

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of ARO-DUX4 in participants with facioscapulohumeral muscular dystrophy Type 1 (FSHD1). In Part 1 of the study, participants will receive a single dose of ARO-DUX4 or placebo. In Part 2 of the study, participants will receive 2 or 4 doses of ARO-DUX4 or placebo. Participants who complete Part 1 will have the option to re-screen and re-randomize into Part 2. All participants will undergo pre- and post-dose MRI-guided muscle biopsies (a total of 2 biopsies). Participants who complete Part 1 and enroll in Part 2 will be required to undergo an additional screening biopsy. Participants completing Part 1 or Part 2 may have the option to continue to receive drug in an open-label extension study or may be eligible to participate in later-stage clinical studies.

Trial website

https://clinicaltrials.gov/study/NCT06131983

Trial related presentations / publications

Public notes

Contacts

Principal investigator

Name

Address

Country

Phone

Fax

Email

Contact person for public queries

Name

Medical Monitor

Address

Country

Phone

626-304-3400

Fax

Email

[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/NCT06131983