A Phase 3 Study of NTLA-2001 in ATTRv-PN
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.
Study details:
This is a multinational, multicenter, double-blind, placebo-controlled study in approximately 50 participants, who will be randomized 1:1 to receive a single infusion of either nexiguran ziclumeran or placebo. To ensure all participants will have the potential to receive nexiguran ziclumeran, participants will have the option to cross over to the opposite study arm at Month 12 or Month 18, depending on study criteria.
Eligibility criteria
Researchers look for people who fit a certain description, called eligibility criteria. See if you qualify.
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
Eligibility
Age eligible for study : NA
Healthy volunteers accepted : No
Gender eligible for study: ALL
Things to know
Study dates
Study start:
Primary completion:
Study completion finish:
Study type
TREATMENT
Phase
- PHASE3
Study ID
NCT06672237
Intervention or treatment
BIOLOGICAL: nexiguran ziclumeran
DRUG: Normal Saline as Placebo
Conditions
- Neuromuscular Disease
- Neuromuscular Diseases (NMD)
- Neurodegenerative Disease
- Neurodegenerative Disease, Hereditary
- Neurodegenerative Diseases
- Neuromuscular Diseases
- Nerve Disorders
- Nervous System Disease
- Nervous System Diseases
- Genetic Disease, Inborn
- Amyloidosis, Familial
- Amyloidosis, Hereditary
- Amyloidosis
- Polyneuropathies
- Amyloid Neuropathies
- Amyloid Neuropathies, Familial
- Peripheral Nervous System Disease
- Peripheral Nervous System Diseases
- Metabolism, Inborn Errors
- Metabolic Diseases
You may be eligible to participate in this trial based on your search. Check eligibility by answering some questions.
Research sites nearby
Closest Location:
Westmead Hospital
Select from list below to view details:
Westmead Hospital
Westmead, Australia
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Who is running the clinical trial?
Intellia Therapeutics
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
| Participant Group/Arm | Intervention/Treatment |
|---|---|
EXPERIMENTAL: nexiguran ziclumeran
| Biological: nexiguran ziclumeran |
PLACEBO_COMPARATOR: Normal Saline
| Drug: Normal Saline as Placebo |
What is the study measuring?
Primary Outcome
| Measure | Description | Time Frame |
|---|---|---|
| Modified Neuropathy Impairment Score +7 (mNIS+7) | Not Specified | 18 Months |
| Serum TTR | Not Specified | 29 Days |
Secondary Outcome
| Measure | Description | Time Frame |
|---|---|---|
| Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QoL-DN) Questionnaire | Not Specified | 18 Months |
| Modified Body Mass Index (mBMI) | Not Specified | 18 Months |
| Serum TTR | Not Specified | 18 Months |
References
Clinical Trials Gov: A Phase 3 Study of NTLA-2001 in ATTRv-PNOther trials to consider
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