TerminatedPhase 1

A Study of PRX004 in Subjects With Amyloid Transthyretin (ATTR) Amyloidosis

Stopped: Because of the impact of COVID-19 pandemic

United States, Portugal21 participantsStarted 2018-04-05

Plain-language summary

A Phase 1, open-label study of intravenous (IV) PRX004 as a single agent in subjects with hereditary amyloid transthyretin (hATTR) amyloidosis. The study will consist of 3 phases and will enroll up to a total of 36 subjects. A 3+3 dose escalation component to determine the safety, tolerability, PK, PD, and MTD. An expansion component in anticipated PRX004 RP2D cohorts selected from the Dose Escalation Phase. An extended dosing component for eligible subjects from the Dose Escalation or Expansion phases.

Who can participate

Age range18 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Age ≥18 years
✓. Ability to understand and willingness to sign an informed consent form prior to initiation of any study procedures
✓. Diagnosis of amyloidosis determined by polarizing light microscopy of green birefringent material in Congo Red-stained tissue specimens; and confirmed diagnosis of ATTR amyloidosis by immunohistochemistry, mass spectrometry, documentation of an ATTR mutation by gene sequencing, or 99m technetium-3,3-diphosphono-1,2-propanodicarboxylic acid (99mTc-DPD) scans and/or technetium pyrophosphate (PYP) SPECT cardiac imaging. If scintigraphy is used for diagnosis then the grade must be 2 or greater, indicative of transthyretin amyloidosis-cardiomyopathy (ATTR-CM) (Gillmore, 2016)
✓. Known TTR mutation
✓. \[Inclusion Criterion 5 removed in Amendment 2\]
✓. Patients receiving concomitant tafamidis or diflunisal may enroll in the study, providing the dose has been stable for the last 6 months
✓. Karnofsky Performance Status (KPS) ≥60%
✓. Adequate organ function, including all of the following:

Exclusion criteria

What they're measuring

Maximum Tolerated Dose of PRX004

Timeframe: 28 days

Number of subjects with treatment-emergent adverse events and clinically significant changes in ECGs, echocardiograms, cardiac telemetry, vital signs, and laboratory evaluations

Timeframe: 3 months

Trial details

NCT IDNCT03336580

SponsorProthena Biosciences Limited

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2020-07-23

View on ClinicalTrials.gov More Amyloidogenic Transthyretin (ATTR) Amyloidosis trials