{
  "topic": {
    "stem": "TOFERSEN_SOD1_ALS_REVIEW",
    "name": "Tofersen in SOD1+ ALS",
    "ncts": [
      "NCT02623699",
      "NCT04856982"
    ],
    "drug_patterns": [
      "tofersen"
    ],
    "condition_patterns": [
      "amyotrophic",
      "lateral",
      "sclerosis"
    ]
  },
  "trials": [
    {
      "gates": {
        "A_aact_exists": true,
        "B_drug_in_intvs": true,
        "C_condition_in_aact": true,
        "D_pmid_topic_match": true,
        "E_two_arms": true,
        "F_primary_outcome_known": true
      },
      "extracted": {
        "nct": "NCT02623699",
        "aact_title": "An Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of BIIB067 (Tofersen) in Adults With Inherited Amyotrophic Lateral Sclerosis (ALS)",
        "aact_acronym": "VALOR (Part C)",
        "start_date": "2016-01-20",
        "primary_completion_date": "2021-07-16",
        "aact_intvs": [
          "Tofersen",
          "Placebo"
        ],
        "aact_conditions": [
          "amyotrophic lateral sclerosis"
        ],
        "pmid": "41661214",
        "pubmed_year": 2026,
        "pubmed_doi": "10.1001/jamaneurol.2025.4946",
        "aact_per_arm_counts": {
          "BG000": 5,
          "BG001": 3,
          "BG002": 3,
          "BG003": 3,
          "BG004": 6,
          "BG005": 12,
          "BG006": 9,
          "BG007": 8,
          "BG008": 9,
          "BG009": 10,
          "BG010": 36,
          "BG011": 72
        },
        "aact_primary_outcome_measure": "Parts A and B: Number of Participants Experiencing Adverse Events (AEs) and Serious Adverse Events (SAEs)",
        "aact_outcome_count_rows": [
          [
            "OG001",
            "13.0"
          ],
          [
            "OG006",
            "10.0"
          ],
          [
            "OG000",
            "2.0"
          ],
          [
            "OG001",
            "2.0"
          ],
          [
            "OG002",
            "3.0"
          ],
          [
            "OG003",
            "3.0"
          ],
          [
            "OG004",
            "6.0"
          ],
          [
            "OG005",
            "12.0"
          ],
          [
            "OG007",
            "9.0"
          ],
          [
            "OG007",
            "1.0"
          ]
        ]
      }
    },
    {
      "gates": {
        "A_aact_exists": true,
        "B_drug_in_intvs": true,
        "C_condition_in_aact": true,
        "D_pmid_topic_match": true,
        "E_two_arms": false,
        "F_primary_outcome_known": true
      },
      "extracted": {
        "nct": "NCT04856982",
        "aact_title": "A Study of BIIB067 (Tofersen) Initiated in Clinically Presymptomatic Adults With a Confirmed Superoxide Dismutase 1 Mutation",
        "aact_acronym": "ATLAS",
        "start_date": "2021-05-17",
        "primary_completion_date": "2027-08-07",
        "aact_intvs": [
          "Tofersen",
          "Placebo"
        ],
        "aact_conditions": [
          "amyotrophic lateral sclerosis associated with a sod1 gene mutation"
        ],
        "pmid": "35585374",
        "pubmed_year": 2022,
        "pubmed_doi": "10.1007/s13311-022-01237-4",
        "aact_per_arm_counts": {},
        "aact_primary_outcome_measure": "Parts B and C: Percentage of Participants with Emergence of Clinically Manifest ALS Within 24 Months of Part B Baseline",
        "aact_outcome_count_rows": []
      }
    }
  ],
  "gate_summary": {
    "A_aact_exists": 2,
    "B_drug_in_intvs": 2,
    "C_condition_in_aact": 2,
    "D_pmid_topic_match": 2,
    "E_two_arms": 1,
    "F_primary_outcome_known": 2
  },
  "n_total": 2,
  "n_pass_all": 1,
  "pass_rate": 0.5,
  "verdict": "VIABLE"
}