{
  "topic": {
    "stem": "VOXELOTOR_SCD_AUTO_2",
    "name": "Voxelotor in SCD (broader)",
    "ncts": [
      "NCT03036813",
      "NCT04724421",
      "NCT05561140",
      "NCT02850406",
      "NCT03573882",
      "NCT05228834",
      "NCT04581356",
      "NCT04930445"
    ],
    "drug_patterns": [
      "voxelotor"
    ],
    "condition_patterns": [
      "sickle"
    ]
  },
  "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": "NCT03036813",
        "aact_title": "Study to Evaluate the Effect of Voxelotor Administered Orally to Patients With Sickle Cell Disease (GBT_HOPE)",
        "aact_acronym": "GBT_HOPE",
        "start_date": "2016-12-31",
        "primary_completion_date": "2019-10-08",
        "aact_intvs": [
          "voxelotor",
          "placebo"
        ],
        "aact_conditions": [
          "sickle cell disease"
        ],
        "pmid": "33838113",
        "pubmed_year": 2021,
        "pubmed_doi": "10.1016/S2352-3026(21)00059-4",
        "aact_per_arm_counts": {
          "BG000": 92,
          "BG001": 90,
          "BG002": 92
        },
        "aact_primary_outcome_measure": "Number of Participants With Increase in Hb >1 g/dL From Baseline to Week 24",
        "aact_outcome_count_rows": [
          [
            "OG000",
            "30.0"
          ],
          [
            "OG001",
            "46.0"
          ],
          [
            "OG002",
            "6.0"
          ]
        ]
      }
    },
    {
      "gates": {
        "A_aact_exists": true,
        "B_drug_in_intvs": true,
        "C_condition_in_aact": true,
        "D_pmid_topic_match": false,
        "E_two_arms": false,
        "F_primary_outcome_known": false
      },
      "extracted": {
        "nct": "NCT04724421",
        "aact_title": "Expanded Access Protocol for Adults and Pediatric Patients With Sickle Cell Disease Who Have No Alternative Treatment Options",
        "aact_acronym": "",
        "start_date": "",
        "primary_completion_date": "",
        "aact_intvs": [
          "voxelotor"
        ],
        "aact_conditions": [
          "sickle cell disease"
        ],
        "pmid": null,
        "aact_per_arm_counts": {},
        "aact_primary_outcome_measure": "",
        "aact_outcome_count_rows": []
      }
    },
    {
      "gates": {
        "A_aact_exists": true,
        "B_drug_in_intvs": true,
        "C_condition_in_aact": true,
        "D_pmid_topic_match": false,
        "E_two_arms": true,
        "F_primary_outcome_known": true
      },
      "extracted": {
        "nct": "NCT05561140",
        "aact_title": "Resolution of Sickle Cell Leg Ulcers With Voxelotor",
        "aact_acronym": "RESOLVE",
        "start_date": "2022-05-30",
        "primary_completion_date": "2024-06-26",
        "aact_intvs": [
          "voxelotor oral tablet",
          "placebo"
        ],
        "aact_conditions": [
          "leg ulcers",
          "sickle cell disease"
        ],
        "pmid": null,
        "aact_per_arm_counts": {
          "BG000": 45,
          "BG001": 43
        },
        "aact_primary_outcome_measure": "Percentage of Participants Who Achieved Resolution of the Target Ulcers by Week 12 in Randomized Treatment Period",
        "aact_outcome_count_rows": [
          [
            "OG000",
            "6.7"
          ],
          [
            "OG001",
            "7.0"
          ],
          [
            "OG000",
            "24.4"
          ],
          [
            "OG001",
            "32.6"
          ]
        ]
      }
    },
    {
      "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": "NCT02850406",
        "aact_title": "Study to Evaluate the Effect of GBT440 in Pediatrics With Sickle Cell Disease",
        "aact_acronym": "HOPE-KIDS",
        "start_date": "2016-07-05",
        "primary_completion_date": "2023-10-02",
        "aact_intvs": [
          "voxelotor"
        ],
        "aact_conditions": [
          "sickle cell disease"
        ],
        "pmid": "35451176",
        "pubmed_year": 2022,
        "pubmed_doi": "10.1002/pbc.29716",
        "aact_per_arm_counts": {
          "BG000": 7,
          "BG001": 6,
          "BG002": 25,
          "BG003": 15,
          "BG004": 51,
          "BG005": 11,
          "BG006": 9,
          "BG007": 23
        },
        "aact_primary_outcome_measure": "Part A: Maximum Concentration (Cmax) of Voxelotor in Whole Blood",
        "aact_outcome_count_rows": [
          [
            "OG000",
            "9.0"
          ],
          [
            "OG001",
            "20.0"
          ],
          [
            "OG000",
            "6.0"
          ],
          [
            "OG001",
            "12.0"
          ],
          [
            "OG000",
            "6.0"
          ],
          [
            "OG001",
            "14.0"
          ],
          [
            "OG000",
            "3.0"
          ],
          [
            "OG001",
            "9.0"
          ],
          [
            "OG000",
            "96.3"
          ],
          [
            "OG001",
            "100.0"
          ]
        ]
      }
    },
    {
      "gates": {
        "A_aact_exists": true,
        "B_drug_in_intvs": true,
        "C_condition_in_aact": true,
        "D_pmid_topic_match": false,
        "E_two_arms": true,
        "F_primary_outcome_known": true
      },
      "extracted": {
        "nct": "NCT03573882",
        "aact_title": "Study to Assess the Effect of Long-term Treatment With Voxelotor in Participants Who Have Completed Treatment in Study GBT440-031",
        "aact_acronym": "034OLE",
        "start_date": "2018-06-06",
        "primary_completion_date": "2024-11-12",
        "aact_intvs": [
          "voxelotor"
        ],
        "aact_conditions": [
          "sickle cell disease"
        ],
        "pmid": null,
        "aact_per_arm_counts": {
          "BG000": 62,
          "BG001": 58,
          "BG002": 58
        },
        "aact_primary_outcome_measure": "Number of Participants With Sickle Cell Disease (SCD)-Related Treatment Emergent Adverse Events (TEAEs)",
        "aact_outcome_count_rows": [
          [
            "OG001",
            "42.0"
          ],
          [
            "OG000",
            "47.0"
          ],
          [
            "OG002",
            "42.0"
          ],
          [
            "OG000",
            "59.0"
          ],
          [
            "OG001",
            "49.0"
          ],
          [
            "OG002",
            "52.0"
          ],
          [
            "OG000",
            "39.0"
          ],
          [
            "OG001",
            "33.0"
          ],
          [
            "OG002",
            "22.0"
          ],
          [
            "OG000",
            "18.0"
          ]
        ]
      }
    },
    {
      "gates": {
        "A_aact_exists": true,
        "B_drug_in_intvs": true,
        "C_condition_in_aact": true,
        "D_pmid_topic_match": false,
        "E_two_arms": false,
        "F_primary_outcome_known": true
      },
      "extracted": {
        "nct": "NCT05228834",
        "aact_title": "Voxelotor Neurocognitive Function Study",
        "aact_acronym": "",
        "start_date": "2022-06-24",
        "primary_completion_date": "2022-09-30",
        "aact_intvs": [
          "voxelotor only product in oral dose form",
          "placebo"
        ],
        "aact_conditions": [
          "sickle cell disease"
        ],
        "pmid": null,
        "aact_per_arm_counts": {},
        "aact_primary_outcome_measure": "Change From Baseline in Executive Abilities Composite Score Assessed Using NIH Toolbox Cognition Module At Week 12",
        "aact_outcome_count_rows": []
      }
    },
    {
      "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": "NCT04581356",
        "aact_title": "Voxelotor Sickle Cell Exercise Study",
        "aact_acronym": "",
        "start_date": "2020-09-21",
        "primary_completion_date": "2021-10-25",
        "aact_intvs": [
          "voxelotor"
        ],
        "aact_conditions": [
          "sickle cell anemia"
        ],
        "pmid": "37247190",
        "pubmed_year": 2023,
        "pubmed_doi": "10.1002/pbc.30423",
        "aact_per_arm_counts": {},
        "aact_primary_outcome_measure": "Peak oxygen consumption (VO2)",
        "aact_outcome_count_rows": []
      }
    },
    {
      "gates": {
        "A_aact_exists": true,
        "B_drug_in_intvs": true,
        "C_condition_in_aact": true,
        "D_pmid_topic_match": false,
        "E_two_arms": false,
        "F_primary_outcome_known": true
      },
      "extracted": {
        "nct": "NCT04930445",
        "aact_title": "Oxbryta® Product Registry An Observational Study Designed to Evaluate the Effect of Oxbryta in Individuals With SCD",
        "aact_acronym": "PROSPECT",
        "start_date": "2022-02-04",
        "primary_completion_date": "2024-10-10",
        "aact_intvs": [
          "oxbryta® (voxelotor) 500mg tablets"
        ],
        "aact_conditions": [
          "sickle cell disease"
        ],
        "pmid": null,
        "aact_per_arm_counts": {
          "BG000": 265
        },
        "aact_primary_outcome_measure": "Change in Hemoglobin From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta",
        "aact_outcome_count_rows": [
          [
            "OG000",
            "0.0"
          ],
          [
            "OG000",
            "0.0"
          ],
          [
            "OG000",
            "236.0"
          ],
          [
            "OG000",
            "17.0"
          ],
          [
            "OG000",
            "4.0"
          ],
          [
            "OG000",
            "3.0"
          ],
          [
            "OG000",
            "0.0"
          ],
          [
            "OG000",
            "0.0"
          ],
          [
            "OG000",
            "0.0"
          ],
          [
            "OG000",
            "0.0"
          ]
        ]
      }
    }
  ],
  "n_total": 8,
  "n_pass_all": 2,
  "verdict": "VIABLE",
  "pass_rate": 0.25
}