Evaluating NTLA-2001 for treating hereditary amyloidosis-related neuropathy
MAGNITUDE-2: A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of NTLA-2001 in Participants With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN)
PHASE3 · Intellia Therapeutics · NCT06672237
This study is testing if a new treatment called NTLA-2001 can help people with hereditary amyloidosis-related neuropathy feel better compared to a placebo.
Quick facts
| Phase | PHASE3 |
|---|---|
| Study type | Interventional |
| Enrollment | 60 (estimated) |
| Ages | 18 Years to 85 Years |
| Sex | All |
| Sponsor | Intellia Therapeutics (industry) |
| Locations | 14 sites (Buenos Aires and 13 other locations) |
| Trial ID | NCT06672237 on ClinicalTrials.gov |
What this trial studies
This Phase 3 study aims to assess the efficacy and safety of a single dose of nexiguran ziclumeran (NTLA-2001) compared to a placebo in patients diagnosed with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN). The study will involve approximately 50 participants who will be randomly assigned to receive either the treatment or a placebo in a double-blind manner. Participants will have the option to switch to the other treatment arm after 12 or 18 months, ensuring access to the investigational drug for all. The study is designed to provide robust data on the potential benefits of NTLA-2001 for this condition.
Who should consider this trial
Good fit: Ideal candidates for this study are individuals diagnosed with ATTRv-PN and a Karnofsky Performance Status of 60 or higher.
Not a fit: Patients with other causes of amyloidosis or significant comorbidities such as advanced heart failure or liver failure may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly improve the quality of life and neurological function for patients suffering from ATTRv-PN.
How similar studies have performed: Previous studies have shown promise in targeting TTR amyloidosis, suggesting that this approach may yield beneficial results.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Diagnosis of ATTRv-PN * Karnofsky Performance Status (KPS) ≥ 60 Exclusion Criteria: * 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
Where this trial is running
Buenos Aires and 13 other locations
- Hospital Británico de Buenos Aires — Buenos Aires, Argentina (RECRUITING)
- Hospital Italiano de Buenos Aires (HIBA) — Buenos Aires, Argentina (RECRUITING)
- Hospital El Cruce — San Juan Bautista, Argentina (RECRUITING)
- Hospital das Clinicas da Universidade Estadual de Campinas (UNICAMP) — Campinas, Brazil (RECRUITING)
- HCFMRP - Hospital das Clinicas da Faculdade de Medicina de Ribeirao Preto — Ribeirão Preto, Brazil (RECRUITING)
- Instituto de Educacao, Pesquisa e Gestao em Saude — Rio de Janeiro, Brazil (RECRUITING)
- PSEG Centro de Pesquisa Clinica S.A. — São Paulo, Brazil (RECRUITING)
- National Institute of Medical Sciences and Nutrition - Salvador Zubiran (INCMNSZ) — Tlalpan, Mexico (RECRUITING)
- National University Hospital — Singapore, Singapore (RECRUITING)
- Singapore General Hospital — Singapore, Singapore (RECRUITING)
- National Taiwan University Hospital — Taipei, Taiwan (ACTIVE_NOT_RECRUITING)
- Taipei Veterans General Hospital — Taipei, Taiwan (ACTIVE_NOT_RECRUITING)
- Chang Gung Medical Foundation - Linkou Branch — Taoyuan, Taiwan (ACTIVE_NOT_RECRUITING)
- Siriraj Hospital — Bangkok, Thailand (RECRUITING)
Study contacts
- Study coordinator: Trial Manager at Intellia
- Email: medicalinformation@intelliatx.com
- Phone: 1-857-285-6200
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions: Neuromuscular Disease, Neuromuscular Diseases, Neurodegenerative Disease, Neurodegenerative Disease, Hereditary, Neurodegenerative Diseases, Nerve Disorders, Nervous System Disease, Nervous System Diseases