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Trial record 3 of 3 for:    ntla-2001
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Long-Term Follow-Up (LTFU) of Subjects Dosed With NTLA-2001

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ClinicalTrials.gov Identifier: NCT05697861
Recruitment Status : Recruiting
First Posted : January 26, 2023
Last Update Posted : March 26, 2024
Sponsor:
Information provided by (Responsible Party):
Intellia Therapeutics

Study Description
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Brief Summary:
This is a follow-up study of subjects who received NTLA-2001 in a previous clinical trial as an observational evaluation of the long-term effects of the investigational therapy.

Condition or disease
Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy Wild-Type Transthyretin Cardiac Amyloidosis

Study Design
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Study Type : Observational
Estimated Enrollment : 72 participants
Observational Model: Case-Only
Time Perspective: Prospective
Official Title: Long-Term Follow-Up of Subjects Treated With NTLA-2001
Actual Study Start Date : July 10, 2023
Estimated Primary Completion Date : March 2038
Estimated Study Completion Date : March 2038

Resource links provided by the National Library of Medicine


Groups and Cohorts
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Outcome Measures
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Primary Outcome Measures :
  1. Incidence of Treatment-related SAEs and Protocol-specified AESIs [ Time Frame: up to 15 years ]
    Long-term safety assessment of any treatment-related SAEs and protocol-specified AESIs


Secondary Outcome Measures :
  1. PD biomarkers for ATTR including serum TTR and serum prealbumin [ Time Frame: up to 15 years ]
    Long-term assessment of serum TTR and serum prealbumin


Biospecimen Retention:   Samples Without DNA
serum

Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Participating sites in an Intellia-sponsored clinical study of NTLA-2001.
Criteria

Inclusion Criteria:

  1. A subject has completed or discontinued from an Intellia-sponsored clinical study in which a complete or partial dose of NTLA-2001 was received.
  2. A subject has provided informed consent for the LTFU study.

Exclusion Criteria:

None

Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05697861


Contacts
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Contact: Trial Manager at Intellia 1-833-888-0387 clinicalscience@intelliatx.com

Locations
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New Zealand
Clinical Trial Site Recruiting
Auckland, New Zealand
Sweden
Clinical Trial Site Recruiting
Umea, Sweden
United Kingdom
Clinical Trial Site Recruiting
London, United Kingdom
Sponsors and Collaborators
Intellia Therapeutics
More Information
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Responsible Party: Intellia Therapeutics
ClinicalTrials.gov Identifier: NCT05697861    
Other Study ID Numbers: ITL-2001-CL-999
2022-003405-30 ( EudraCT Number )
First Posted: January 26, 2023    Key Record Dates
Last Update Posted: March 26, 2024
Last Verified: March 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Additional relevant MeSH terms:
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Polyneuropathies
Amyloid Neuropathies
Amyloid Neuropathies, Familial
Cardiomyopathies
Amyloidosis
Heart Diseases
Cardiovascular Diseases
Proteostasis Deficiencies
Metabolic Diseases
 Peripheral Nervous System Diseases
Neuromuscular Diseases
Nervous System Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Amyloidosis, Familial
Metabolism, Inborn Errors