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Medfølende brug af Concizumab, hvis du har hæmofili

26. oktober 2026 opdateret af: Novo Nordisk A/S

Concizumab Compassionate Use Program for patienter med medfødt hæmofili

The compassionate use programme will give participants concizumab for free, even though it is not yet approved by health authorities. This is because participants need this medicine to treat their haemophilia properly. The programme will check that participants are safe and that the medicine works for them. The programme may last for years. Participants will take one injection under their skin every day. Participants will have 4-5 visits with the study doctor for the first half year. After that they will have 1 visit every half year. At all clinic visits participants will have blood samples taken. Participants will fill in a diary between the visits.

A patient is considered to have completed the programme when any of the following criteria occurred first: 1) when the patient is included in a clinical trial with concizumab or 2) up to 6 months after concizumab is commercially available in the patient's country and approved for the patient (The time span of 6 months should provide ample time for the patient to obtain concizumab commercially) or 3) the sponsor decides to discontinue concizumab clinical development for the patient's population.

Studieoversigt

Status

Ikke længere tilgængelig

Betingelser

Intervention / Behandling

Undersøgelsestype

Udvidet adgang

Udvidet adgangstype

  • Befolkning af middelstørrelse

Kontakter og lokationer

Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.

Studiesteder

  • Bulgarien
      • Plovdiv, Bulgarien, 4002
        • UMHAT Sveti Georgi EAD, Plovdiv, Clinic of Pediatrics
  • Forenede Stater
    • California
      • Los Angeles, California, Forenede Stater, 90027
        • Children's Hospital Los Angeles - Endocrinology
      • San Francisco, California, Forenede Stater, 94158
        • University of California San Francisco UCSF
    • Connecticut
      • Hartford, Connecticut, Forenede Stater, 06106
        • Connecticut Children's Medical Center
    • District of Columbia
      • Washington D.C., District of Columbia, Forenede Stater, 20007
        • Georgetown University Medical Center
    • Georgia
      • Augusta, Georgia, Forenede Stater, 30912
        • Augusta University
      • Savannah, Georgia, Forenede Stater, 31404
        • Memorial Health University Medical Center
    • Idaho
      • Boise, Idaho, Forenede Stater, 83712
        • St. Luke's Mountain States Tumor Institute
    • Illinois
      • Chicago, Illinois, Forenede Stater, 60611
        • Childrens Hospital of Chicago
    • Indiana
      • Indianapolis, Indiana, Forenede Stater, 46260
        • Indiana Hemophilia-Thromb Ctr
    • Louisiana
      • New Orleans, Louisiana, Forenede Stater, 70118
        • Children's Hosp-New Orleans
    • Michigan
      • Detroit, Michigan, Forenede Stater, 48201
        • Children's Hospital of Michigan
    • Mississippi
      • Flowood, Mississippi, Forenede Stater, 39232
        • Southern Specialty Clinic
      • Madison, Mississippi, Forenede Stater, 39110
        • Louisiana Ctr for Adv Med-LCAM
    • Missouri
      • Kansas City, Missouri, Forenede Stater, 64108
        • The Children's Mercy Hospital
    • Nebraska
      • Omaha, Nebraska, Forenede Stater, 68114
        • Children's Nebraska
    • North Carolina
      • Greenville, North Carolina, Forenede Stater, 27834
        • ECU Sickle Cell Comp Clinic
    • Ohio
      • Columbus, Ohio, Forenede Stater, 43205
        • Nationwide Children's Hospital
    • Pennsylvania
      • Hershey, Pennsylvania, Forenede Stater, 17033
        • Penn State Hershey Medical Center
    • South Carolina
      • Greenville, South Carolina, Forenede Stater, 29605
        • BI-LO Chrt Childn's Cancer Ctr
    • Texas
      • Fort Worth, Texas, Forenede Stater, 76104
        • Cook Children's Hospital-Hematology-Oncology
      • Houston, Texas, Forenede Stater, 77030
        • Texas Children's Hospital_Houston
      • Houston, Texas, Forenede Stater, 77030
        • Univ TX Hlth Sci Ctr Houston
    • Virginia
      • Richmond, Virginia, Forenede Stater, 23298-0461
        • Virginia Cwealth Univ Richmond
  • Sverige
      • Solna, Sverige, 171 64
        • Koagulationsmottagningen

Deltagelseskriterier

Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.

Berettigelseskriterier

Aldre berettiget til at studere

  • Barn
  • Voksen
  • Ældre voksen

Tager imod sunde frivillige

Ingen

Køn, der er berettiget til at studere

Alle

Beskrivelse

Inclusion Criteria:

  • Informed consent obtained before any programme-related activities. Programme-related activities are any procedures that are carried out as part of the programme.

  • Patients with congenital haemophilia:

    1. severe haemophilia A (coagulation factor VIII (FVIII) less than 1%) or moderate/severe haemophilia B (coagulation factor IX (FIX) less than or equal to 2%) without inhibitors or
    2. any haemophilia severity with documented history of inhibitors (more than or equal to 0.6 bethesda unit (BU)) who cannot be treated satisfactorily with authorised and marketed medicines (example: due to inhibitors or allergic reactions to factor-containing products, or due to poor venous access), and who are not able to enrol in clinical trials designed to support the development and registration of concizumab medicines (example: due to inhibitors or allergic reactions to factor-containing products, or due to poor venous access) as per investigator and Novo Nordisk assessment.

  • The potential benefit for the individual patient justifies the potential risks of treatment.

Exclusion Criteria:

  • Known or suspected hypersensitivity to investigational medicinal product or related products.
  • Any condition (current or medical history), which in the investigator's or Novo Nordisk's opinion might jeopardise patient's safety or compliance with the protocol.

Studieplan

Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.

Hvordan er undersøgelsen tilrettelagt?

Samarbejdspartnere og efterforskere

Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.

Sponsor

Novo Nordisk A/S

Datoer for undersøgelser

Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.

Datoer for studieregistrering

Først indsendt

4. juni 2021

Først indsendt, der opfyldte QC-kriterier

4. juni 2021

Først opslået (Faktiske)

10. juni 2021

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

28. oktober 2026

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

26. oktober 2026

Sidst verificeret

1. oktober 2026

Mere information

Begreber relateret til denne undersøgelse

Yderligere relevante MeSH-vilkår

Andre undersøgelses-id-numre

  • NN7415-4807

Disse oplysninger blev hentet direkte fra webstedet clinicaltrials.gov uden ændringer. Hvis du har nogen anmodninger om at ændre, fjerne eller opdatere dine undersøgelsesoplysninger, bedes du kontakte register@clinicaltrials.gov. Så snart en ændring er implementeret på clinicaltrials.gov, vil denne også blive opdateret automatisk på vores hjemmeside .

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