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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06153966




Registration number
NCT06153966
Ethics application status
Date submitted
21/11/2023
Date registered
1/12/2023

Titles & IDs
Public title
PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717
Scientific title
A Phase 1/2a Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION717 in Patients With Prion Disease
Secondary ID [1] 0 0
2023-503355-98
Secondary ID [2] 0 0
ION717-CS2
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Prion Disease 0 0
Condition category
Condition code
Infection 0 0 0 0
Studies of infection and infectious agents
Neurological 0 0 0 0
Transmissible spongiform encephalopathies
Neurological 0 0 0 0
Other neurological disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - ION717
Treatment: Drugs - Placebo

Experimental: ION717 + Placebo, Regimen 1 - Participants will receive multiple doses of study drug (ION717 and placebo) during the 30-week double-blind treatment period; the order of doses is blinded. Participants will then receive multiple doses of ION717 during the 70-week open-label extension period.

Experimental: ION717 + Placebo, Regimen 2 - Participants will receive multiple doses of study drug (ION717 and placebo) during the 30-week double-blind treatment period; the order of doses is blinded. Participants will then receive multiple doses of ION717 during the 70-week open-label extension period.


Treatment: Drugs: ION717
ION717 will be administered by IT injection.

Treatment: Drugs: Placebo
Placebo-matching ION717 will be administered by IT injection.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Incidence of treatment-emergent adverse events.
Timepoint [1] 0 0
Baseline up to Week 33
Secondary outcome [1] 0 0
Maximum Observed Plasma Concentration (Cmax) of ION717
Timepoint [1] 0 0
on Day 1 and Week 9
Secondary outcome [2] 0 0
Area Under the Plasma Concentration-time Curve (AUC) of ION717
Timepoint [2] 0 0
on Day 1 and Week 9
Secondary outcome [3] 0 0
Half-life (t1/2?z) of ION717 in Plasma
Timepoint [3] 0 0
on Day 1 and Week 9
Secondary outcome [4] 0 0
Cerebrospinal fluid (CSF) Concentration of ION717
Timepoint [4] 0 0
Pre-dose and at multiple points post-dose up to Week 33
Secondary outcome [5] 0 0
Amount of ION717 Excreted in Urine
Timepoint [5] 0 0
Post-dose on Day 1
Secondary outcome [6] 0 0
Percent Change from Baseline in Prion Protein (PrP) Concentration in CSF
Timepoint [6] 0 0
Pre-dose and at multiple points post-dose up to Week 33

Eligibility
Key inclusion criteria
Key Inclusion Criteria

* A confirmed diagnosis of probable or definite prion disease.
* Early-stage prion disease at the time of Screening.
* Willing to meet all study requirements, including travel to Study Center, procedures, measurements and visits.
* Patients must have a caregiver who is = 18 years old and who is able and willing to facilitate the patient's involvement, to the best of their ability, for the duration of the trial; caregivers must also be able and willing to provide information about themselves and the patient for the duration of the trial.
* Aged = 18 at the time of informed consent.

Key
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Exclusion Criteria

* Clinically significant abnormalities in medical history, laboratory tests or physical examination that would render a patient unsuitable for inclusion.
* Any contraindication or unwillingness to undergo an MRI.
* Obstructive hydrocephalus, presence of a functional ventriculoperitoneal shunt for the drainage of cerebrospinal fluid (CSF) or an implanted central nervous system (CNS) catheter.
* Known brain or spinal disease that would interfere with the LP process, CSF circulation or safety assessment.
* Have any other condition, which, in the opinion of the Investigator would make the patient unsuitable for inclusion or could interfere with the patient participating in or completing the study.

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 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
4/01/2024
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/06/2027
Actual
Sample size
Target
56
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Royal Melbourne Hospital - Parkville
Recruitment postcode(s) [1] 0 0
3050 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Colorado
Country [3] 0 0
United States of America
State/province [3] 0 0
Massachusetts
Country [4] 0 0
United States of America
State/province [4] 0 0
New York
Country [5] 0 0
United States of America
State/province [5] 0 0
Ohio
Country [6] 0 0
Canada
State/province [6] 0 0
Alberta
Country [7] 0 0
Canada
State/province [7] 0 0
Quebec
Country [8] 0 0
France
State/province [8] 0 0
Paris
Country [9] 0 0
Germany
State/province [9] 0 0
Göttingen
Country [10] 0 0
Israel
State/province [10] 0 0
Tel Aviv
Country [11] 0 0
Italy
State/province [11] 0 0
Milan
Country [12] 0 0
Japan
State/province [12] 0 0
Isesaki-shi
Country [13] 0 0
Japan
State/province [13] 0 0
Kodaira-shi
Country [14] 0 0
Japan
State/province [14] 0 0
Shimonoseki-shi
Country [15] 0 0
Spain
State/province [15] 0 0
Barcelona

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Ionis Pharmaceuticals, Inc.
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
Ionis Pharmaceuticals, Inc.
Address 0 0
Country 0 0
Phone 0 0
(844) 221-3587
Fax 0 0
Email 0 0
PrionDisease@clinicaltrialmedia.com
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
Ionis may share anonymized individual participant data, aggregated clinical data, and other types of data that support the results in this study. Data requests from qualified researchers will be considered once all three of the following criteria are met: (1) 12 months from marketing approval of the study drug in both the United States and European Union; (2) 18 months from conclusion of the study; and (3) 6 months from publication of study article. Access would be via a secure environment and is contingent upon approval of a research proposal and entry into an appropriate data use agreement. Requests to access data can be submitted via the website https://vivli.org/ourmember/ionis/.
When will data be available (start and end dates)?
Available to whom?
Available for what types of analyses?
How or where can data be obtained?
IPD available at link: https://vivli.org/ourmember/ionis/


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/NCT06153966