SNPMiner Trials by Shray Alag


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Report for Clinical Trial NCT04201418

Developed by Shray Alag, 2020.
SNP Clinical Trial Gene

A Phase 4 Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of Hereditary Transthyretin-Mediated (hATTR) Amyloidosis With a V122I or T60A Mutation

To evaluate the effectiveness of patisiran in patients with hATTR amyloidosis with polyneuropathy who have a V122I or T60A mutation.

NCT04201418 Hereditary Transthyretin-mediated (hATTR) Amyloidosis Polyneuropathy
MeSH:Polyneuropathies Amyloidosis
HPO:Amyloidosis Motor polyneuropathy Polyneuropathy

1 Interventions

Name: Patisiran

Description: Patisiran lipid complex injection, for intravenous use
Type: Drug
Group Labels: 1

Patisiran


Primary Outcomes

Description: PND Scores: Stage 0=No symptoms, Stage 1=Sensory disturbances but preserved walking capability, Stage 2=Impaired walking capacity, but ability to walk without a stick or crutches, Stage 3A/B=Walking with the help of 1 or 2 sticks or crutches, Stage 4=confined to wheel chair or bedridden.

Measure: Percentage of Participants with Stable or Improved Polyneuropathy Disability (PND) Score at 12 Months Relative to Baseline

Time: Baseline, Month 12

Time Perspective: Prospective

Cohort


There are 2 SNPs

SNPs


1 T60A

A Phase 4 Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of Hereditary Transthyretin-Mediated (hATTR) Amyloidosis With a V122I or T60A Mutation. --- V122I --- --- T60A ---

A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of hATTR Amyloidosis With a V122I or T60A Mutation To evaluate the effectiveness of patisiran in patients with hATTR amyloidosis with polyneuropathy who have a V122I or T60A mutation. --- V122I --- --- T60A ---

A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of hATTR Amyloidosis With a V122I or T60A Mutation To evaluate the effectiveness of patisiran in patients with hATTR amyloidosis with polyneuropathy who have a V122I or T60A mutation. --- V122I --- --- T60A --- --- V122I --- --- T60A ---

PND Scores: Stage 0=No symptoms, Stage 1=Sensory disturbances but preserved walking capability, Stage 2=Impaired walking capacity, but ability to walk without a stick or crutches, Stage 3A/B=Walking with the help of 1 or 2 sticks or crutches, Stage 4=confined to wheel chair or bedridden.. Inclusion Criteria: - Diagnosed with hATTR amyloidosis with polyneuropathy, with a documented V122I or T60A mutation - PND score of I-IIIB at the baseline visit - Naive to patisiran treatment at the time of enrollment with intention to initiate treatment with patisiran. --- V122I --- --- T60A ---

Exclusion Criteria: - New York Heart Association (NYHA) heart failure classification ≥3 - Karnofsky Performance Status (KPS) <60% - Unstable congestive heart failure (CHF) - Known primary amyloidosis (AL) or leptomeningeal amyloidosis - Prior major organ transplant - Previously received patisiran - Previous treatment with a TTR silencing therapy Inclusion Criteria: - Diagnosed with hATTR amyloidosis with polyneuropathy, with a documented V122I or T60A mutation - PND score of I-IIIB at the baseline visit - Naive to patisiran treatment at the time of enrollment with intention to initiate treatment with patisiran. --- V122I --- --- T60A ---


2 V122I

A Phase 4 Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of Hereditary Transthyretin-Mediated (hATTR) Amyloidosis With a V122I or T60A Mutation. --- V122I ---

A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of hATTR Amyloidosis With a V122I or T60A Mutation To evaluate the effectiveness of patisiran in patients with hATTR amyloidosis with polyneuropathy who have a V122I or T60A mutation. --- V122I ---

A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of hATTR Amyloidosis With a V122I or T60A Mutation To evaluate the effectiveness of patisiran in patients with hATTR amyloidosis with polyneuropathy who have a V122I or T60A mutation. --- V122I --- --- T60A --- --- V122I ---

PND Scores: Stage 0=No symptoms, Stage 1=Sensory disturbances but preserved walking capability, Stage 2=Impaired walking capacity, but ability to walk without a stick or crutches, Stage 3A/B=Walking with the help of 1 or 2 sticks or crutches, Stage 4=confined to wheel chair or bedridden.. Inclusion Criteria: - Diagnosed with hATTR amyloidosis with polyneuropathy, with a documented V122I or T60A mutation - PND score of I-IIIB at the baseline visit - Naive to patisiran treatment at the time of enrollment with intention to initiate treatment with patisiran. --- V122I ---

Exclusion Criteria: - New York Heart Association (NYHA) heart failure classification ≥3 - Karnofsky Performance Status (KPS) <60% - Unstable congestive heart failure (CHF) - Known primary amyloidosis (AL) or leptomeningeal amyloidosis - Prior major organ transplant - Previously received patisiran - Previous treatment with a TTR silencing therapy Inclusion Criteria: - Diagnosed with hATTR amyloidosis with polyneuropathy, with a documented V122I or T60A mutation - PND score of I-IIIB at the baseline visit - Naive to patisiran treatment at the time of enrollment with intention to initiate treatment with patisiran. --- V122I ---



HPO Nodes


HPO: