Clinical trial • Not applicable • Rare Disease

ZICLUMERAN; SINGLE GUIDE RNA TARGETING THE HUMAN TTR GENE for Hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN) | Transthyretin amyloidosis-related cardiomyopathy (ATTR-CM)

Not applicable trial of ZICLUMERAN; SINGLE GUIDE RNA TARGETING THE HUMAN TTR GENE for Hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN)…

Overview

Trial Therapeutic Area
Rare Disease
Trial Disease
Hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN) | Transthyretin amyloidosis-related cardiomyopathy (ATTR-CM)
Trial Stage
Not applicable
Drug Modality
Gene therapy|mRNA|Other RNA
Orphan Drug
Yes

Key dates

Initial CTIS Submission Date
08-04-2024
First CTIS Authorization Date
16-04-2024

Trial design

Not applicable trial across 2 sites in Sweden, France.

Target Sample Size
63

Eligibility

Recruits 63 Vulnerable population selected. The JSON states: "2. A participant has provided informed consent for the LTFU study." Subject information and informed consent forms are listed among submitted documents. No further details on assent or age-specific consent processes are provided in the JSON..

Pregnancy Exclusion
5. Female participants must agree to the following until 7 months after the last administration of study intervention in the parent study: a. Use a protocol-specified highly effective method of contraception, if a WOCBP. This is not required for female participants who are postmenopausal or surgically sterile, as defined in Section 10.3.1. AND b. Not undergo oocyte retrieval for in vitro fertilization for at least 7 months after the last administration of study intervention. The time frame may be extended beyond 7 months if oocyte retrieval is contraindicated based on country-specific guidelines.
Vulnerable Population
Vulnerable population selected. The JSON states: "2. A participant has provided informed consent for the LTFU study." Subject information and informed consent forms are listed among submitted documents. No further details on assent or age-specific consent processes are provided in the JSON.

Inclusion criteria

  • {"criterion_text":"- 1. A participant has completed or discontinued from an Intellia-sponsored clinical study in which a complete or partial dose of NTLA-2001 was received.\n- 2. A participant has provided informed consent for the LTFU study.\n- 3. A participant is willing to attend study visits, complete protocol-required follow-up schedule, and comply with the study requirements.\n- 4. Male participants must agree to the following until 4 months after the last administration of study intervention in the parent study: a. Refrain from donating sperm. The time frame may be extended beyond 4 months if sperm donation is contraindicated based on country-specific guidelines. AND b. Use a male condom, in addition to the female partner using a highly effective contraceptive method with a failure rate of < 1% per year, when having sexual intercourse with a WOCBP (Section 10.3.1).\n- 5. Female participants must agree to the following until 7 months after the last administration of study intervention in the parent study: a. Use a protocol-specified highly effective method of contraception, if a WOCBP. This is not required for female participants who are postmenopausal or surgically sterile, as defined in Section 10.3.1. AND b. Not undergo oocyte retrieval for in vitro fertilization for at least 7 months after the last administration of study intervention. The time frame may be extended beyond 7 months if oocyte retrieval is contraindicated based on country-specific guidelines."}

Exclusion criteria

  • {"criterion_text":"- None."}

Endpoints

Primary endpoints

  • {"endpoint_text":"- For ATTR-CM and ATTRv-PN participants, incidence of: • Treatment-related SAEs • Protocol-specified AESIs","definition_or_measurement_approach":""}

Secondary endpoints

  • {"endpoint_text":"- PD biomarkers for ATTR over time: • Serum TTR • Serum prealbumin","definition_or_measurement_approach":""}

Recruitment

Planned Sample Size
63
Recruitment Window Months
181
Consent Approach
Participants must provide informed consent. The JSON includes subject information and informed consent form documents (e.g. L1_SIS and ICF_Main_Intellia; 2023-507385-11_NIFC_Main) and participant-facing materials in English and French. No details on assent or age-specific consent processes are provided in the JSON.

Geography

Total Number Of Sites
2
Total Number Of Participants
15

Sweden

Earliest CTIS Part Ii Submission Date
15-03-2024
Latest Decision Or Authorization Date
27-08-2025
Processing Time Days
530
Number Of Sites
1
Number Of Participants
9

Sites

Site Name
Region Vaesterbotten
Department Name
Norrlands universitetssjukhus, Daniel Naezéns väg, 907 37 Umeå, Medicincentrum E:41
Contact Person Name
Björn Pilebro
Number Of Participants
9

France

Earliest CTIS Part Ii Submission Date
07-10-2024
Latest Decision Or Authorization Date
28-08-2025
Processing Time Days
325
Number Of Sites
1
Number Of Participants
6

Sites

Site Name
Assistance Publique Hopitaux De Paris
Department Name
Neurology
Contact Person Name
Andoni ECHANIZ-LAGUNA
Contact Person Email
andoni.echaniz-laguna@aphp.fr
Number Of Participants
6

Sponsor

Primary sponsor

Full Name
Intellia Therapeutics Inc.
Organisation Type
Pharmaceutical company
Country Of Registered Address
United States

Contract research organisations

Name
Charles River Laboratories Montreal ULC
Responsibilities
Sample management (contact email MTL-SampleManagement@crl.com); sponsor duties code: 4
Name
Medpace Finland Oy
Responsibilities
Roles indicated by sponsor duties codes: 1,12,13,4,5 (contact email RS-Advisor-Support@medpace.com)

Third parties

  • {"country":"Canada","full_name":"Charles River Laboratories Montreal ULC","duties_or_roles":"[{\"id\":756430,\"code\":\"4\"}]","organisation_type":"Pharmaceutical company"}
  • {"country":"Finland","full_name":"Medpace Finland Oy","duties_or_roles":"[{\"id\":756431,\"code\":\"1\"},{\"id\":756432,\"code\":\"12\"},{\"id\":756433,\"code\":\"13\"},{\"id\":756434,\"code\":\"4\"},{\"id\":756435,\"code\":\"5\"}]","organisation_type":"Pharmaceutical company"}

Investigational products

Investigational Product Name
NTLA-2001
Active Substance
ZICLUMERAN; SINGLE GUIDE RNA TARGETING THE HUMAN TTR GENE
Modality
Gene therapy|mRNA|Other RNA
Routes Of Administration
Intravenous
Route
Intravenous
Authorisation Status
prodAuthStatus: 1
Orphan Designation
Yes
Maximum Dose
1 mg/kg

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