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New treatment option for Neuromuscular Disease
Official title A Phase 3 Study of NTLA-2001 in ATTRv-PN
ClinicalTrials.gov ID: NCT06672237
What this study is testing
What is nexiguran ziclumeran?
nexiguran ziclumeran is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for neuromuscular disease.
Also referred to as NTLA-2001.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This study will be conducted to evaluate the efficacy and safety of a single dose of nexiguran ziclumeran (NTLA-2001) compared to placebo in participants with ATTRv-PN.
- Phase 3: a large, late-stage study
- You might receive a placebo (an inactive treatment) instead of the study drug, decided by chance. You may not know which one you got.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 85
You may be able to join if
- Diagnosis of ATTRv-PN
- Karnofsky Performance Status (KPS) ≥ 60
You likely can't join if
- Other causes of amyloidosis (amyloidosis caused by non-TTR protein)
- Other known causes of sensorimotor or autonomic neuropathy
- Diabetes mellitus
- New York Heart Association Class III or IV heart failure
- Liver failure
- Hepatitis B, hepatitis C or human immunodeficiency virus (HIV) infection
See the full eligibility criteria
- Diagnosis of ATTRv-PN
- Karnofsky Performance Status (KPS) ≥ 60
- Other causes of amyloidosis (amyloidosis caused by non-TTR protein)
- Other known causes of sensorimotor or autonomic neuropathy
- Diabetes mellitus
- New York Heart Association Class III or IV heart failure
- Liver failure
- Hepatitis B, hepatitis C or human immunodeficiency virus (HIV) infection
- Prior receipt of a TTR silencer (Small interfering RNA (siRNA) or Antisense oligonucleotides (ASOs))
- Estimated Glomerular Filtration Rate \< 30 mL/min/1.73 m2
- Unable or unwilling to take vitamin A supplementation for the duration of the study
- History of liver disease
The study team makes the final eligibility decision.
Where it's taking place
- Buenos Aires, Argentina
- San Juan Bautista, Argentina
- Campinas, Brazil
- Ribeirão Preto, Brazil
- Rio de Janeiro, Brazil
- São Paulo, Brazil
- Tlalpan, Mexico
- Singapore, Singapore
- Taipei, Taiwan
- Taoyuan, Taiwan
- Bangkok, Thailand
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years to 85 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Buenos Aires, Argentina; San Juan Bautista, Argentina; Campinas, Brazil; Ribeirão Preto, Brazil; Rio de Janeiro, Brazil; São Paulo, Brazil and 5 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.