Clinical Trials List
Protocol NumberITL-2001-CL-311
NCT Number(ClinicalTrials.gov Identfier)NCT06672237
Active
2025-01-03 - 2028-12-31
Phase III
Recruiting3
ICD-10E85.0
Non-neuropathic heredofamilial amyloidosis
ICD-10E85.1
Neuropathic heredofamilial amyloidosis
ICD-10E85.2
Heredofamilial amyloidosis, unspecified
ICD-10E85.3
Secondary systemic amyloidosis
ICD-10E85.4
Organ-limited amyloidosis
ICD-10E85.8
Other amyloidosis
ICD-10E85.9
Amyloidosis, unspecified
ICD-9277.3
Amyloidosis
MAGNITUDE-2: A phase 3, multinational, randomized, double-blind, placebo-controlled trial evaluating the efficacy and safety of NTLA-2001 in participants with transthyretin amyloid disease (ATTRv-PN), a hereditary polyneuropathy.
-
Trial Applicant
MEDPACE TAIWAN LIMITED
-
Sponsor
Medpace Taiwan Limited.
-
Trial scale
Multi-Regional Multi-Center
-
Update
2026/08/27
Investigators and Locations
Co-Principal Investigator
- 江樸田 無
- Ming-Jen Lee 無
- YEN-HUNG LIN 無
- 林昭文 無
- JYH-MING JIMMY JUANG 無
- SHIN-JOE YEH 無
- LI-KAI TSAI 無
- 薛頌儒 無
- HSUEH-WEN HSUEH 無
- SUNG-TSANG HSIEH 無
- Jen Jen and I Su 無
- 范淞斌 無
The Actual Total Number of Participants Enrolled
0 Recruiting
Co-Principal Investigator
- 季康揚 無
- Kon-Ping Lin 無
- Yi-Chu Liao 無
- 蕭丞宗 無
- Wen-Chung Yu
The Actual Total Number of Participants Enrolled
0 Recruiting
Principal Investigator
Long-Sun Ro
The Actual Total Number of Participants Enrolled
0 Recruiting
Condition/Disease
Transthyretinopathy with hereditary polyneuropathy (ATTRv-PN)
Objectives
This is a phase 3, multinational, multicenter, randomized, double-blind, placebo-controlled trial involving approximately 60 eligible participants with ATTRv-PN who have not received TTR silencers (siRNA and ASO) and will be randomly assigned to receive either NTLA-2001 or placebo.
Test Drug
NTLA-2001
Active Ingredient
A clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 gene editing system delivered by lipid nanoparticles (LNPs) for intravenous (IV) administration.
Dosage Form
Intravenous infusion
Dosage
NTLA-2001 (55mg)
Endpoints
• Changes in mNIS+7 score from baseline to month 18
• Changes in serum TTR from baseline to day 29
• Changes in serum TTR from baseline to day 29
Inclution Criteria
1. Males or females aged 18 to 85 years at the time of signing the participant consent form.
2. Diagnosed with ATTRv-PN, including:
a. Data showing a TTR mutation.
b. Neuropathic impairment score (NIS) of 10 to 130 (inclusive).
c. Polyneuropathy disability score (PND) ≤ 3b.
and
d. Abnormalities in the following nerve conduction study (NCS) attributes (total score ≥ 2 from > 1 nerve): fibular motor nerve conduction velocity (MNCV), tibial MNCV, ulnar MNCV, sural nerve distal latency (SNDL), and ulnar SNDL.
3. Karnofsky performance status (KPS) ≥ 60. 4. Male participants must agree to adhere to the following guidelines for at least 4 months after receiving the experimental intervention (or longer if sperm donation is prohibited under national guidelines):
a. Avoid sperm donation.
and
b. When engaging in sexual intercourse with a currently non-pregnant, fertile woman, agree to use male condoms along with the female partner using a highly effective contraceptive method with an annual failure rate of <1%.
5. Female participants must:
a. Be infertile.
and
b. Agree not to donate eggs (ovaries, ovum cells) for at least 7 months after receiving the experimental intervention.
6. Participants must provide written informed consent before undergoing any assessments specified in the trial protocol.
7. Participants must agree to abstain from alcohol during the screening period and for 64 days after receiving the experimental intervention.
1. Other known causes of sensorimotor or autonomic neuropathy (e.g., autoimmune diseases, monogammaglobulinemia, etc.).
2. Amyloidosis attributable to non-TRT proteins (e.g., amyloid light chain amyloidosis), or known primary amyloidosis or leptomeningeal amyloidosis.
3. Known diabetes mellitus.
4. Poorly controlled hypothyroidism or hyperthyroidism.
5. New York Heart Association (NYHA) grade III or IV heart failure (HF).
6. History of any myocardial infarction, unstable angina, severe aortic stenosis, cerebrovascular accident or transient ischemic attack, symptomatic peripheral artery disease, pulmonary embolism, or deep vein thrombosis within 3 months prior to the scheduled first intervention. Such history must be documented more than 3 months prior to the scheduled first intervention, and data must show optimal clinical management and stable status.
7. Known history of cirrhosis.
8. A family or personal history of severe or uncontrolled thrombotic disease.
9. A known or suspected systemic viral, parasitic, or fungal infection, or antibiotic treatment for a bacterial infection within 14 days prior to the first trial intervention.
10. Active or chronic hepatitis B or C infection, or a positive result for hepatitis B surface antigen (HBsAg) or hepatitis C virus (HCV) antibody (Ab).
11. A history of being positive for human immunodeficiency virus (HIV).
Note: Local HIV testing is only required if mandated by local or national regulations.
12. A history of active malignancy within 3 years prior to screening or during the screening period, except for the following:
a. Basal cell carcinoma of the skin.
b. Squamous cell carcinoma of the skin that has undergone curative excision.
c. Cervical carcinoma in situ that has received curative treatment.
d. Non-metastatic prostate adenocarcinoma that has been stabilized by a medical oncologist with hormone therapy, or that can be managed with observation as the appropriate course of action.
13. Pregnant or breastfeeding.
14. Underwent major surgery within 3 months prior to the scheduled initial administration of the trial intervention, or is scheduled to undergo major surgery at any point during the trial period.
15. Previously received a TTR gene silencing agent (small interfering RNA, siRNA or antisense oligonucleotide, ASO).
16. Currently using tafamidis, diflunisal, acoramidis, doxycycline, or tauro-ursodesoxy cholic acid (TUDCA); if any of these medications have been previously used, a 30-day cleansing period must be completed before the first administration of the trial intervention.
17. Received warfarin or heparin/heparin derivative antithrombotic therapy within 14 days prior to the first administration of the trial intervention, or is expected to require warfarin antithrombotic therapy until day 64.
Note: apixaban, dabigatran, edoxaban, or rivaroxaban are permitted, provided the dose remains stable for 28 days prior to screening, stable during screening, and is expected to remain stable for 90 days after the first administration of the trial intervention.
18. Plan to begin potentially hepatotoxic therapy (e.g., amiodarone) within 64 days of the first administration of the trial intervention.
19. Have received a liver, heart, or other solid organ transplant; a bone marrow transplant; or are expected to receive a transplant within one year prior to screening.
Note: Prior or planned corneal transplants are not excluded.
20. Received any deactivated, subunit, or messenger ribonucleic acid (mRNA)/lip nanoparticle (LNP) vaccine within 14 days prior to the trial intervention, any live vaccine within 28 days prior to the trial intervention, or scheduled to receive any vaccine within 28 days after the trial intervention.
21. Received any investigational drug within 6 months prior to the first administration of the trial intervention.
22. Participants meet any of the following laboratory criteria:
a. Aspartate aminotransferase (AST), alanine aminotransferase (ALT), or total bilirubin > 1.5 × upper limit of normal (ULN) (see the exceptions for Gilbert's syndrome below).
a. For participants with a history of Gilbert's syndrome, total bilirubin > 3.0 × ULN.
b. Estimated glomerular filtration rate (eGFR) < 30 mL/min/1.73 m², as measured by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) formula.
c. Platelet count < 100,000 cells/mm³.
d. Vitamin A < lower limit of normal (LLN).
e. International normalized ratio (INR) > 2.0 (> 3.5 for participants receiving anticoagulant therapy).
f. Vitamin B12 < LLN.
23. Known hypersensitivity to any LNP component, or prior LNP treatment with any serious/severe/life-threatening treatment-related abnormal laboratory test results or adverse event (AE).
24. Unable or unwilling to take vitamin A supplements during the trial.
25. Unable or unwilling to undergo the required pre-treatment medication regimen.
26. The trial administrator believes the expected survival is less than 24 months.
27. A history of alcohol or drug abuse within the 3 years prior to screening.
28. Any condition, abnormal laboratory test results, or other reason deemed by the trial administrator to potentially adversely affect participant safety, impair the assessment of trial results, or hinder adherence to the trial protocol.
29. Unwilling to comply with the trial procedures outlined in the trial protocol, including follow-up, or unwilling to fully cooperate with the trial administrator's instructions.
2. Diagnosed with ATTRv-PN, including:
a. Data showing a TTR mutation.
b. Neuropathic impairment score (NIS) of 10 to 130 (inclusive).
c. Polyneuropathy disability score (PND) ≤ 3b.
and
d. Abnormalities in the following nerve conduction study (NCS) attributes (total score ≥ 2 from > 1 nerve): fibular motor nerve conduction velocity (MNCV), tibial MNCV, ulnar MNCV, sural nerve distal latency (SNDL), and ulnar SNDL.
3. Karnofsky performance status (KPS) ≥ 60. 4. Male participants must agree to adhere to the following guidelines for at least 4 months after receiving the experimental intervention (or longer if sperm donation is prohibited under national guidelines):
a. Avoid sperm donation.
and
b. When engaging in sexual intercourse with a currently non-pregnant, fertile woman, agree to use male condoms along with the female partner using a highly effective contraceptive method with an annual failure rate of <1%.
5. Female participants must:
a. Be infertile.
and
b. Agree not to donate eggs (ovaries, ovum cells) for at least 7 months after receiving the experimental intervention.
6. Participants must provide written informed consent before undergoing any assessments specified in the trial protocol.
7. Participants must agree to abstain from alcohol during the screening period and for 64 days after receiving the experimental intervention.
1. Other known causes of sensorimotor or autonomic neuropathy (e.g., autoimmune diseases, monogammaglobulinemia, etc.).
2. Amyloidosis attributable to non-TRT proteins (e.g., amyloid light chain amyloidosis), or known primary amyloidosis or leptomeningeal amyloidosis.
3. Known diabetes mellitus.
4. Poorly controlled hypothyroidism or hyperthyroidism.
5. New York Heart Association (NYHA) grade III or IV heart failure (HF).
6. History of any myocardial infarction, unstable angina, severe aortic stenosis, cerebrovascular accident or transient ischemic attack, symptomatic peripheral artery disease, pulmonary embolism, or deep vein thrombosis within 3 months prior to the scheduled first intervention. Such history must be documented more than 3 months prior to the scheduled first intervention, and data must show optimal clinical management and stable status.
7. Known history of cirrhosis.
8. A family or personal history of severe or uncontrolled thrombotic disease.
9. A known or suspected systemic viral, parasitic, or fungal infection, or antibiotic treatment for a bacterial infection within 14 days prior to the first trial intervention.
10. Active or chronic hepatitis B or C infection, or a positive result for hepatitis B surface antigen (HBsAg) or hepatitis C virus (HCV) antibody (Ab).
11. A history of being positive for human immunodeficiency virus (HIV).
Note: Local HIV testing is only required if mandated by local or national regulations.
12. A history of active malignancy within 3 years prior to screening or during the screening period, except for the following:
a. Basal cell carcinoma of the skin.
b. Squamous cell carcinoma of the skin that has undergone curative excision.
c. Cervical carcinoma in situ that has received curative treatment.
d. Non-metastatic prostate adenocarcinoma that has been stabilized by a medical oncologist with hormone therapy, or that can be managed with observation as the appropriate course of action.
13. Pregnant or breastfeeding.
14. Underwent major surgery within 3 months prior to the scheduled initial administration of the trial intervention, or is scheduled to undergo major surgery at any point during the trial period.
15. Previously received a TTR gene silencing agent (small interfering RNA, siRNA or antisense oligonucleotide, ASO).
16. Currently using tafamidis, diflunisal, acoramidis, doxycycline, or tauro-ursodesoxy cholic acid (TUDCA); if any of these medications have been previously used, a 30-day cleansing period must be completed before the first administration of the trial intervention.
17. Received warfarin or heparin/heparin derivative antithrombotic therapy within 14 days prior to the first administration of the trial intervention, or is expected to require warfarin antithrombotic therapy until day 64.
Note: apixaban, dabigatran, edoxaban, or rivaroxaban are permitted, provided the dose remains stable for 28 days prior to screening, stable during screening, and is expected to remain stable for 90 days after the first administration of the trial intervention.
18. Plan to begin potentially hepatotoxic therapy (e.g., amiodarone) within 64 days of the first administration of the trial intervention.
19. Have received a liver, heart, or other solid organ transplant; a bone marrow transplant; or are expected to receive a transplant within one year prior to screening.
Note: Prior or planned corneal transplants are not excluded.
20. Received any deactivated, subunit, or messenger ribonucleic acid (mRNA)/lip nanoparticle (LNP) vaccine within 14 days prior to the trial intervention, any live vaccine within 28 days prior to the trial intervention, or scheduled to receive any vaccine within 28 days after the trial intervention.
21. Received any investigational drug within 6 months prior to the first administration of the trial intervention.
22. Participants meet any of the following laboratory criteria:
a. Aspartate aminotransferase (AST), alanine aminotransferase (ALT), or total bilirubin > 1.5 × upper limit of normal (ULN) (see the exceptions for Gilbert's syndrome below).
a. For participants with a history of Gilbert's syndrome, total bilirubin > 3.0 × ULN.
b. Estimated glomerular filtration rate (eGFR) < 30 mL/min/1.73 m², as measured by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) formula.
c. Platelet count < 100,000 cells/mm³.
d. Vitamin A < lower limit of normal (LLN).
e. International normalized ratio (INR) > 2.0 (> 3.5 for participants receiving anticoagulant therapy).
f. Vitamin B12 < LLN.
23. Known hypersensitivity to any LNP component, or prior LNP treatment with any serious/severe/life-threatening treatment-related abnormal laboratory test results or adverse event (AE).
24. Unable or unwilling to take vitamin A supplements during the trial.
25. Unable or unwilling to undergo the required pre-treatment medication regimen.
26. The trial administrator believes the expected survival is less than 24 months.
27. A history of alcohol or drug abuse within the 3 years prior to screening.
28. Any condition, abnormal laboratory test results, or other reason deemed by the trial administrator to potentially adversely affect participant safety, impair the assessment of trial results, or hinder adherence to the trial protocol.
29. Unwilling to comply with the trial procedures outlined in the trial protocol, including follow-up, or unwilling to fully cooperate with the trial administrator's instructions.
Exclusion Criteria
Participants are ineligible to participate in this trial if they meet any of the following criteria at the time of screening:
1. Other known causes of sensorimotor or autonomic neuropathy (e.g., autoimmune diseases, monogammaglobulinemia, etc.).
2. Amyloidosis attributable to non-TR proteins (e.g., amyloid light chain amyloidosis), or a known history of primary amyloidosis or leptomeningeal amyloidosis.
3. Known diabetes mellitus.
4. Poorly controlled hypothyroidism or hyperthyroidism.
5. New York Heart Association (NYHA) grade III or IV heart failure (HF).
6. A history of myocardial infarction, unstable angina, severe aortic stenosis, cerebrovascular accident or transient ischemic attack, symptomatic peripheral artery disease, pulmonary embolism, or deep vein thrombosis within 3 months prior to the scheduled first interventional treatment. Regarding the above medical history, it must be performed more than 3 months prior to the scheduled first trial intervention, and data must show optimal clinical management and stable condition.
7. Known history of cirrhosis.
8. Known family or personal history of severe or uncontrolled thrombotic disease.
9. Known or suspected systemic viral, parasitic, or fungal infection, or antibiotic treatment for bacterial infection within 14 days prior to the first trial intervention.
10. Active or chronic hepatitis B or C infection, or a positive hepatitis B surface antigen (HBsAg) or hepatitis C virus (HCV) antibody (Ab) test result.
11. History of positive human immunodeficiency virus (HIV) status.
Note: Local HIV testing is only required if mandated by local or national regulations.
12. A history of active malignancy within 3 years prior to screening or during the screening period, except for:
a. Basal cell carcinoma of the skin.
b. Squamous cell carcinoma of the skin that has undergone curative resection.
c. Cervical carcinoma in situ that has received curative treatment.
d. Non-metastatic prostate adenocarcinoma that has been stabilized with hormone therapy by an oncologist, or that can be managed with observation alone as appropriate.
e. Ductal carcinoma in situ of the breast that has undergone radical treatment and for which the participant has received at least 6 months of stable anti-hormone therapy.
f. Melanoma in situ that has been completely resected and for which no cancer cells were found at the tissue margins.
13. Pregnancy or breastfeeding.
14. Having undergone major surgery within 3 months prior to the scheduled initial administration of the trial intervention, or scheduled to undergo major surgery at any time during the trial period.
15. Previous treatment with a TTR gene silencing agent (small interfering RNA, siRNA or antisense oligonucleotide, ASO).
16. Currently using tafamidis, diflunisal, acoramidis, doxycycline, or tauro-ursodesoxycholic acid (TUDCA); if any of these medications have been previously used, a 30-day clearance period must be completed before the first administration of the trial intervention.
17. Received warfarin or heparin/heparin derivative antithrombotic therapy within 14 days prior to the first administration of the trial intervention, or is expected to require warfarin antithrombotic therapy until day 64.
Note: The use of apixaban, dabigatran, edoxaban, or rivaroxaban is permitted, provided that the dose remains stable for 28 days prior to screening, stable during screening, and is expected to remain stable for 90 days after the first administration of the trial intervention.
18. Plans to begin potentially hepatotoxic therapy (e.g., amiodarone) within 64 days of administering the trial intervention.
19. Has received a liver, heart, or other solid organ transplant; a bone marrow transplant; or is expected to receive a transplant within one year prior to screening.
Note: Prior or planned corneal transplants are not excluded.
20. Has received any deactivated, subunit, or messenger ribonucleic acid (mRNA)/lipid nanoparticle (LNP) vaccine within 14 days prior to administering the trial intervention; has received any live vaccine within 28 days prior to administering the trial intervention; or is scheduled to receive any vaccine within 28 days of administering the trial intervention.
21. Received any investigational drug within 6 months prior to the first administration of the investigational intervention.
22. Participants meet any of the following laboratory criteria:
a. Aspartate aminotransferase (AST), alanine aminotransferase (ALT), or total bilirubin > 1.5 × upper limit of normal (ULN) (see the exceptions for Gilbert's syndrome below).
a. For participants with a history of Gilbert's syndrome, total bilirubin > 3.0 × ULN.
b. Estimated glomerular filtration rate (eGFR) < 30 mL/min/1.73 m² as measured by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) formula.
c. Platelet count < 100,000 cells/mm³. d. Vitamin A < lower limit of normal (LLN).
e. International normalized ratio (INR) > 2.0 (> 3.5 for participants receiving anticoagulant therapy).
f. Vitamin B12 < LLN.
23. Known allergy to any LNP component, or previous LNP treatment with any serious/severe/life-threatening treatment-related laboratory abnormalities or adverse events (AEs).
24. Unable or unwilling to take vitamin A supplements during the trial.
25. Unable or unwilling to receive any medications specified in the trial protocol, including corticosteroids (e.g., required pre-treatment medication courses, reactive/prophylactic corticosteroids, etc.).
26. The trial administrator believes the expected survival is less than 24 months.
27. History of alcohol or drug abuse within 3 years prior to screening.
28. Any condition, abnormal laboratory test results, or other reason deemed by the trial administrator to potentially adversely affect participant safety, impair trial outcome assessment, or hinder adherence to the trial protocol.
29. Unwilling to comply with the trial procedures outlined in the trial protocol, including follow-up, or unwilling to fully cooperate with the trial administrator's instructions.
30. Unable or unwilling to avoid rosuvastatin use for 7 days prior to and 64 days after administration of the trial intervention.
31. History of MASH (metabolic steatohepatitis).
32. History of autoimmune hepatitis.
1. Other known causes of sensorimotor or autonomic neuropathy (e.g., autoimmune diseases, monogammaglobulinemia, etc.).
2. Amyloidosis attributable to non-TR proteins (e.g., amyloid light chain amyloidosis), or a known history of primary amyloidosis or leptomeningeal amyloidosis.
3. Known diabetes mellitus.
4. Poorly controlled hypothyroidism or hyperthyroidism.
5. New York Heart Association (NYHA) grade III or IV heart failure (HF).
6. A history of myocardial infarction, unstable angina, severe aortic stenosis, cerebrovascular accident or transient ischemic attack, symptomatic peripheral artery disease, pulmonary embolism, or deep vein thrombosis within 3 months prior to the scheduled first interventional treatment. Regarding the above medical history, it must be performed more than 3 months prior to the scheduled first trial intervention, and data must show optimal clinical management and stable condition.
7. Known history of cirrhosis.
8. Known family or personal history of severe or uncontrolled thrombotic disease.
9. Known or suspected systemic viral, parasitic, or fungal infection, or antibiotic treatment for bacterial infection within 14 days prior to the first trial intervention.
10. Active or chronic hepatitis B or C infection, or a positive hepatitis B surface antigen (HBsAg) or hepatitis C virus (HCV) antibody (Ab) test result.
11. History of positive human immunodeficiency virus (HIV) status.
Note: Local HIV testing is only required if mandated by local or national regulations.
12. A history of active malignancy within 3 years prior to screening or during the screening period, except for:
a. Basal cell carcinoma of the skin.
b. Squamous cell carcinoma of the skin that has undergone curative resection.
c. Cervical carcinoma in situ that has received curative treatment.
d. Non-metastatic prostate adenocarcinoma that has been stabilized with hormone therapy by an oncologist, or that can be managed with observation alone as appropriate.
e. Ductal carcinoma in situ of the breast that has undergone radical treatment and for which the participant has received at least 6 months of stable anti-hormone therapy.
f. Melanoma in situ that has been completely resected and for which no cancer cells were found at the tissue margins.
13. Pregnancy or breastfeeding.
14. Having undergone major surgery within 3 months prior to the scheduled initial administration of the trial intervention, or scheduled to undergo major surgery at any time during the trial period.
15. Previous treatment with a TTR gene silencing agent (small interfering RNA, siRNA or antisense oligonucleotide, ASO).
16. Currently using tafamidis, diflunisal, acoramidis, doxycycline, or tauro-ursodesoxycholic acid (TUDCA); if any of these medications have been previously used, a 30-day clearance period must be completed before the first administration of the trial intervention.
17. Received warfarin or heparin/heparin derivative antithrombotic therapy within 14 days prior to the first administration of the trial intervention, or is expected to require warfarin antithrombotic therapy until day 64.
Note: The use of apixaban, dabigatran, edoxaban, or rivaroxaban is permitted, provided that the dose remains stable for 28 days prior to screening, stable during screening, and is expected to remain stable for 90 days after the first administration of the trial intervention.
18. Plans to begin potentially hepatotoxic therapy (e.g., amiodarone) within 64 days of administering the trial intervention.
19. Has received a liver, heart, or other solid organ transplant; a bone marrow transplant; or is expected to receive a transplant within one year prior to screening.
Note: Prior or planned corneal transplants are not excluded.
20. Has received any deactivated, subunit, or messenger ribonucleic acid (mRNA)/lipid nanoparticle (LNP) vaccine within 14 days prior to administering the trial intervention; has received any live vaccine within 28 days prior to administering the trial intervention; or is scheduled to receive any vaccine within 28 days of administering the trial intervention.
21. Received any investigational drug within 6 months prior to the first administration of the investigational intervention.
22. Participants meet any of the following laboratory criteria:
a. Aspartate aminotransferase (AST), alanine aminotransferase (ALT), or total bilirubin > 1.5 × upper limit of normal (ULN) (see the exceptions for Gilbert's syndrome below).
a. For participants with a history of Gilbert's syndrome, total bilirubin > 3.0 × ULN.
b. Estimated glomerular filtration rate (eGFR) < 30 mL/min/1.73 m² as measured by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) formula.
c. Platelet count < 100,000 cells/mm³. d. Vitamin A < lower limit of normal (LLN).
e. International normalized ratio (INR) > 2.0 (> 3.5 for participants receiving anticoagulant therapy).
f. Vitamin B12 < LLN.
23. Known allergy to any LNP component, or previous LNP treatment with any serious/severe/life-threatening treatment-related laboratory abnormalities or adverse events (AEs).
24. Unable or unwilling to take vitamin A supplements during the trial.
25. Unable or unwilling to receive any medications specified in the trial protocol, including corticosteroids (e.g., required pre-treatment medication courses, reactive/prophylactic corticosteroids, etc.).
26. The trial administrator believes the expected survival is less than 24 months.
27. History of alcohol or drug abuse within 3 years prior to screening.
28. Any condition, abnormal laboratory test results, or other reason deemed by the trial administrator to potentially adversely affect participant safety, impair trial outcome assessment, or hinder adherence to the trial protocol.
29. Unwilling to comply with the trial procedures outlined in the trial protocol, including follow-up, or unwilling to fully cooperate with the trial administrator's instructions.
30. Unable or unwilling to avoid rosuvastatin use for 7 days prior to and 64 days after administration of the trial intervention.
31. History of MASH (metabolic steatohepatitis).
32. History of autoimmune hepatitis.
The Estimated Number of Participants
-
Taiwan
12 participants
-
Global
60 participants
