{
  "topic": {
    "stem": "MARALIXIBAT_PFIC_REVIEW",
    "name": "Maralixibat in PFIC (MARCH-PFIC + cohort)",
    "ncts": [
      "NCT03905330",
      "NCT02057692"
    ],
    "drug_patterns": [
      "maralixibat"
    ],
    "condition_patterns": [
      "cholestasis",
      "pfic"
    ]
  },
  "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": "NCT03905330",
        "aact_title": "A Study to Evaluate the Efficacy and Safety of Maralixibat in Subjects With Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC)",
        "aact_acronym": "MARCH-PFIC",
        "start_date": "2019-07-09",
        "primary_completion_date": "2022-09-01",
        "aact_intvs": [
          "Maralixibat",
          "Placebo"
        ],
        "aact_conditions": [
          "progressive familial intrahepatic cholestasis (pfic)"
        ],
        "pmid": "38723644",
        "pubmed_year": 2024,
        "pubmed_doi": "10.1016/S2468-1253(24)00080-3",
        "aact_per_arm_counts": {
          "BG000": 47,
          "BG001": 46
        },
        "aact_primary_outcome_measure": "Mean Change in the Average Morning ItchRO(Obs) Severity Score in the Primary Cohort",
        "aact_outcome_count_rows": [
          [
            "OG000",
            "8.0"
          ],
          [
            "OG001",
            "4.0"
          ],
          [
            "OG000",
            "5.0"
          ],
          [
            "OG001",
            "1.0"
          ],
          [
            "OG000",
            "21.0"
          ],
          [
            "OG001",
            "8.0"
          ],
          [
            "OG000",
            "15.0"
          ],
          [
            "OG001",
            "2.0"
          ]
        ],
        "published_effect_type": "MD",
        "published_effect_size": -1.1,
        "published_ci_lower": -1.8,
        "published_ci_upper": -0.3,
        "published_source_snippet": "difference of -1.1 (95% CI -1.8 to -0.3",
        "published_extractor": "rct-extractor-v2"
      }
    },
    {
      "gates": {
        "A_aact_exists": true,
        "B_drug_in_intvs": false,
        "C_condition_in_aact": false,
        "D_pmid_topic_match": false,
        "E_two_arms": true,
        "F_primary_outcome_known": true
      },
      "extracted": {
        "nct": "NCT02057692",
        "aact_title": "Evaluation of LUM001 in the Reduction of Pruritus in Alagille Syndrome",
        "aact_acronym": "ITCH",
        "start_date": "2014-11-24",
        "primary_completion_date": "2016-11-16",
        "aact_intvs": [
          "LUM001",
          "Placebo"
        ],
        "aact_conditions": [
          "alagille syndrome"
        ],
        "pmid": null,
        "aact_per_arm_counts": {
          "BG000": 8,
          "BG001": 11,
          "BG002": 6,
          "BG003": 12
        },
        "aact_primary_outcome_measure": "Change From Baseline to Endpoint (Week 13/Early Termination) in Pruritus",
        "aact_outcome_count_rows": [
          [
            "OG000",
            "1.0"
          ],
          [
            "OG001",
            "7.0"
          ],
          [
            "OG002",
            "9.0"
          ],
          [
            "OG003",
            "5.0"
          ],
          [
            "OG004",
            "12.0"
          ],
          [
            "OG000",
            "0.0"
          ],
          [
            "OG001",
            "1.0"
          ],
          [
            "OG002",
            "0.0"
          ],
          [
            "OG003",
            "0.0"
          ],
          [
            "OG004",
            "0.0"
          ]
        ]
      }
    }
  ],
  "gate_summary": {
    "A_aact_exists": 2,
    "B_drug_in_intvs": 1,
    "C_condition_in_aact": 1,
    "D_pmid_topic_match": 1,
    "E_two_arms": 2,
    "F_primary_outcome_known": 2
  },
  "n_total": 2,
  "n_pass_all": 1,
  "pass_rate": 0.5,
  "verdict": "VIABLE"
}