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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04601051




Registration number
NCT04601051
Ethics application status
Date submitted
19/10/2020
Date registered
23/10/2020
Date last updated
8/09/2023

Titles & IDs
Public title
Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2001 in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN) and Patients With Transthyretin Amyloidosis-Related Cardiomyopathy (ATTR-CM)
Scientific title
Phase 1 Two-Part (Open-label, Single Ascending Dose (Part 1) and Open-label, Single Dose Expansion (Part 2)) Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2001 in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN) and Patients With Transthyretin Amyloidosis-Related Cardiomyopathy (ATTR-CM)
Secondary ID [1] 0 0
2020-002034-32
Secondary ID [2] 0 0
ITL-2001-CL-001
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Condition category
Condition code

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Comparator / control treatment
Control group

Outcomes

Eligibility
Key inclusion criteria
Polyneuropathy

* Male and/or female participants 18 to 80 years of age inclusive, at the time of signing the informed consent
* Diagnosis of polyneuropathy (PN) due to transthyretin (TTR) amyloidosis (ATTR)
* Must have a body weight of at least 45 kilograms (kg) at Screening visit
* Lack of access to approved treatments for ATTR and/or progression of hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN) despite use of approved treatment for ATTRv-PN

Polyneuropathy
Minimum age
18 Years
Maximum age
90 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Amyloidosis attributable to non-TTR protein, e.g., amyloid light-chain (AL) amyloidosis
* Known leptomeningeal transthyretin amyloidosis
* Use of any of the following TTR-directed therapy for ATTR within certain timeframe:

1. Patisiran
2. Inotersen
3. Vutrisiran
4. Tafamidis
5. Diflunisal
6. Doxycycline and/or tauroursodeoxycholic acid
7. Any other investigational agent for the treatment of ATTRv-PN:
* Other protocol defined Inclusion/Exclusion criteria may apply

Cardiomyopathy Inclusion Criteria (UK only):

* Male and/or female participants 18 to 90 years of age inclusive, at the time of signing the informed consent
* Diagnosis of transthyretin (ATTR) amyloidosis with cardiomyopathy, classified as hereditary ATTR amyloidosis with cardiomyopathy (ATTRv-CM) or wild type cardiomyopathy (ATTRwt-CM).
* Must have a body weight of at least 45 kilograms (kg) at Screening visit
* New York Heart Association (NYHA) Class I-III heart failure
* At least 1 prior hospitalization for heart failure and/or clinical evidence of heart failure.
* Able to complete =150 meters on the 6-minute walk test (6-MWT) during the Screening period.

Cardiomyopathy Exclusion Criteria (UK only):

* Amyloidosis attributable to non-TTR protein, e.g., amyloid light-chain (AL) amyloidosis
* Known leptomeningeal transthyretin amyloidosis
* Use of any of the following TTR-directed therapy for ATTR within certain timeframes:

1. Patisiran
2. Inotersen
3. Vutrisiran
4. Tafamidis
5. Diflunisal
6. Doxycycline and/or tauroursodeoxycholic acid
7. Investigational TTR stabilizer (e.g., AG-10)
* Participants with heart failure that in the opinion of the investigator is caused by ischemic heart disease, hypertension, or uncorrected valvular disease and not primarily due to transthyretin amyloid cardiomyopathy.
* Participants with a history of sustained ventricular tachycardia or aborted ventricular fibrillation or with a history of atrioventricular (AV) nodal or sinoatrial (SA) nodal dysfunction for which a pacemaker is indicated but will not be placed. Pacemaker or defibrillator placement, initiation of or change in anti-arrhythmic medication within 28 days prior to study drug administration.
* Other protocol defined Inclusion/Exclusion criteria may apply

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised 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
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Other
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
5/11/2020
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/08/2026
Actual
Sample size
Target
Accrual to date
Final
72
Recruitment in Australia
Recruitment state(s)

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Intellia Therapeutics
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
This study will be conducted to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of NTLA-2001 in participants with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN) and participants with hereditary transthyretin amyloidosis with cardiomyopathy (ATTRv-CM) or wild type cardiomyopathy (ATTRwt-CM)
Trial website
https://clinicaltrials.gov/study/NCT04601051
Trial related presentations / publications
Public notes
This record is viewable in the ANZCTR as it had previously listed Australia and/or New Zealand as a recruitment site, however these sites have since been removed

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
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT04601051