A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B

Study acronym: BASIS KIDS
StatusRecruiting
PhasePhase 3
SponsorPfizer
Started

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Record History

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  1. Minor Eligibility criteria expanded, about 259 words
    • Site details revised at 19 of 63 sites
  2. Notable Primary endpoints: details revised at 1 of 8 entries
    • Study sites: 64 to 63
    • Eligibility criteria expanded, about 29 words
    • Secondary endpoints: details revised at 1 of 10 entries
  3. Minor Trial sites expanded: 34 → 64 locationsv35
  4. Minor Trial sites reduced: 66 → 34 locationsv34
  5. Minor Study sites: 60 to 66v33
Show 31 earlier versionsHide 31 earlier versions
  1. Minor Study sites: 69 to 60v32
  2. Minor Study sites: 72 to 69v31
  3. Minor Site details revised at 1 of 72 sitesv30
  4. Minor Study sites: 69 to 72v29
  5. Minor Study sites: 65 to 69v28
  6. Minor Study sites: 63 to 65
    • Eligibility criteria expanded, about 4 words
    v27
  7. Minor Study sites: 62 to 63v26
  8. Minor Study sites: 61 to 62v25
  9. Minor Study sites: 59 to 61v24
  10. Minor Study sites: 58 to 59
    • Eligibility criteria expanded, about 58 words
    v23
  11. Minor Study sites: 53 to 58v22
  12. Minor Study sites: 44 to 53v21
  13. Minor Study sites: 39 to 44v20
  14. Minor Study sites: 36 to 39v19
  15. Minor Site details revised at 1 of 36 sitesv18
  16. Minor Study sites: 34 to 36v17
  17. Minor Study sites: 33 to 34v16
  18. Minor Study sites: 32 to 33v15
  19. Minor Study sites: 29 to 32v14
  20. Minor Study sites: 20 to 29v13
  21. Minor Study sites: 19 to 20
    • Eligibility criteria expanded, about 26 words
    v12
  22. Minor Study sites: 17 to 19v11
  23. Minor Study sites: 14 to 17v10
  24. Minor Study sites: 13 to 14v9
  25. Minor Study sites: 12 to 13v8
  26. Minor Study sites: 6 to 12v7
  27. Minor Study sites: 4 to 6v6
  28. Minor Study sites: 3 to 4v5
  29. Minor Study sites: 2 to 3v4
  30. Notable Trial status: Not Yet Recruiting to Recruiting
    • Study sites: 0 to 2
    • Start date changed from estimate of Dec 5, 2022 to Dec 9, 2022
    v3
  31. Minor Start date: Nov 15, 2022 to Dec 5, 2022v2
  1. Study First Submitted
  • Notable: the trial’s plan changed such as a big move in its timeline or size, a new primary endpoint, sponsor or phase, or recruiting opened or resumed.
  • Minor: routine progress and housekeeping, such as sites added or enrollment closed.
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The purpose of this clinical trial is to learn about the safety and effects of the study medicine (called marstacimab) for the potential treatment of hemophilia in pediatric patients. This study will enroll pediatric participants from ages 1 to 17 years in a sequential manner. The study opened the enrollment to adolescent participants aged 12 to 17 years first. Then children aged 6 to 11 years erere permitted to enroll. Lastly, children aged 1 to 5 years are permitted to enroll. This study will enroll male participants who: * have severe Hemophilia A or moderately severe to severe Hemophilia B (with or without inhibitors) * have accurate historical records documenting all factor VIII, factor IX, or bypass agent infusions and hemophilia bleed events for at least 1 year prior to entering the study for participants from 6 to 17 years of age, and at least 3 months prior to entering the study for participants from 1 to 5 years of age * if a non-inhibitor patient, must be on a stable routine prophylaxis regimen with factor VIII or factor IX replacement products for at least 12 months prior to study entry for participants from 6 to 17 years of age, and least 3 months for participants aged from 1 to 5 years of age * if an inhibitor patient, must be on an on-demand bypass treatment regimen during the 12 months prior to study entry for participants from 6 to 17 years of age, and during the 3 months prior to study entry for participants aged from 1 to 5 years of age All participants in this study will receive marstacimab to use prophylactically. Marstacimab will be given once a week as a subcutaneous (under the skin) shot. The first dose of marstacimab will be given at the study site by the study site staff. During the 12-month treatment period, weekly doses of marstacimab can be given at home, or if preferred, the doses may be given by the study site staff. To help us determine if the study medicine is safe and effective, we will compare participant experiences when they are taking the study medicine to a historical period when they were not. Researchers want to see if the study medicine works to prevent the bleeding episodes commonly experienced by patients with Hemophilia. Participants will be in this study for about 14 months (approximately 1 month in a Screening period, 12 months receiving treatment, and 1 month in a follow-up period) during which they will visit the study site at least 10 times. If preferred, and if local regulations allow it, 2 of the study visits can be completed at the participant's home instead of at the study site. There will also be 6 scheduled telephone calls approximately every 2 months.
Trial Details
NCT Number NCT05611801
Lead Sponsor Pfizer
Conditions
(from sponsor)
Hemophilia A, Hemophilia B
Related terms Hemophilia A, Hemophilia B, Hemorrhage
Enrollment 100 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov