NCT06703606Toboroz1. fázis
A Study to Learn About How Changing Therapy From Emicizumab to Marstacimab Affects People With the Severe Hemophilia A.
A PHASE 1B OPEN-LABEL NON-RANDOMIZED STUDY TO ASSESS THE SAFETY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF MARSTACIMAB TREATMENT FOLLOWING THE DISCONTINUATION OF EMICIZUMAB THERAPY IN ADOLESCENT AND ADULT PARTICIPANTS WITH SEVERE HEMOPHILIA A WITHOUT INHIBITORS.
Forrás: ClinicalTrials.gov · Utoljára frissítve: 2026. okt. 15.
Rövid összefoglaló (regiszter, angolul)
The purpose of the study is to learn about safety, how the body processes marstacimab and how it works in patients with severe hemophilia A without inhibitors. Hemophilia A is rare bleeding disorder where the blood doesn't clot normally. This causes a person to bleed a lot, even from a small cut. These patients who are on emicizumab medicine for routine prophylaxis for at least 6 months, and desire to switch to marstacimab medicine. Inhibitors are antibodies that the immune system develops because it sees the infused clotting factor as a foreign substance that needs to be destroyed. Antibodies are proteins that eat up the activated factor before it has time to stop the bleeding. Prophylaxis are preventive medicines. This study is seeking for participants: * with severe Hemophilia A withouth inhibitors who are on emicizumab treatment for at least 6 months. * must be 12 to less than 75 years old * must have a body weight of at least 35 kilograms. The results from this study will serve as a guide to doctors and their hemophilia A patients who will change their medicines in the real-world clinical setting. Patients who can take part in the study will receive marstacimab medicine as weekly injections under the skin of 150 milligrams for 4 months. Study treatment with marstacimab will be initiated no earlier than 14 days after last dose of emicizumab. The study can last up to 6 months. The sponsor will provide marstacimab. Patients will continue their usual treatment with the infused clotting factor for their bleeds when taking part in the study. Roll-over into an optional study treatment extension period will be available to participants who wish to continue prophylaxis with marstacimab in countries where it is not commercially available.
- Állapot (regiszter)
- Toboroz
- Fázis (regiszter)
- 1. fázis
- Vizsgálattípus
- Beavatkozással járó
- Résztvevők száma
- 15 (tervezett)
- Első benyújtás
- 2024. nov. 21.
- Kezdés
- 2025. máj. 13.
- Elsődleges befejezés
- 2027. máj. 5.
- Fő szponzor
- Pfizer
- Betegségek
- Hemofília
- NCT-szám
- NCT06703606
Mit vállalsz
- Vizsgálat típusa: Beavatkozással járó · 1. fázis
- Tervezett résztvevők: 15
- 2025. máj. 13. és az elsődleges befejezés (2027. máj. 5.) között
- 10 helyszín, 3 országban
- Vizitek és vizsgálatok: kérdezd a vizsgálat csapatát, a regiszter nem sorolja fel őket
A(z) ClinicalTrials.gov regisztermezőiből összeállítva. A vizsgálat felépítését írja le.
Ki nem vehet részt
Szó szerint a(z) ClinicalTrials.gov forrásból, témák szerint csoportosítva. Hogy ki vehet részt, azt csak a vizsgálat csapata dönti el.
Korábbi kezelés
- Previous or current treatment for or history of coronary artery diseases, venous or arterial thrombosis, or ischemic disease.
- Current use of any prohibited concomitant medication(s) or unwillingness or inability to use a required concomitant medication(s).
- Previous administration of an investigational product (drug or vaccine) within 30 days or 5 half-lives preceding the first dose of study intervention used in this study (whichever is longer). Participation in studies of other investigational products (drug or vaccine) at any time during participation in this study.
- Known planned surgical procedure.
Más betegség
- Hypersensitivity or allergic reaction to hamster protein or other components of the study intervention.
Laborértékek
- Any medical or psychiatric condition including recent (within the past year) or active suicidal ideation/behavior or laboratory abnormality that may increase the risk of study participation or, in the investigator's judgment, make the participant inappropriate for the study.
- Platelet count <100,000/μl or hemoglobin <10 g/dL.
- Clinically significant renal or hepatic function abnormality based on laboratory results at screening, or known kidney or liver disease.
- CD4 cell count ≤200/μl if HIV positive.
Egyéb
- Known hemostatic defect other than hemophilia A.
- Screening 12-lead ECG that demonstrates clinically significant abnormalities that, in the opinion of the investigator, may affect participant safety or interpretation of study results.
- Investigator site staff directly involved in the conduct of the study and their family members, site staff otherwise supervised by the investigator, and sponsor and sponsor-delegate employees directly involved in the conduct of the study and their family members.
Kiket keres a vizsgálat
- Male and 12 to <75 years of age with a minimum body weight of 35 kg at the time of signing the informed consent.
- Diagnosis of severe hemophilia A (FVIII activity <1%) without inhibitors.
- On emicizumab therapy at a standard clinical dose for ≥6 months.
Életkor a regiszterben: 12 év – 74 év · Nem: csak férfiak · Egészséges önkénteseket nem fogad
Helyszínek (10)
Helyszínek a regiszter sorrendjében. Ha a profilodba mented a városodat, távolság szerint rendeződnek.
| Helyszín | Város | Ország | Helyszín állapota |
|---|---|---|---|
| K J Somaiya Hospital & Research Centre | Mumbai | India | Toboroz |
| Nirmal Hospital Pvt Ltd. | Surat | India | Toboroz |
| Ege University Faculty of Medicine Dean's Office | Bornova | Törökország | Toboroz |
| Istanbul Universitesi Tıp Fakultesi Onkoloji Enstitusu | Fatih | Törökország | Toboroz |
| ECU Health Medical Center | Greenville | Egyesült Államok | Toboroz |
| Brody School of Medicine at East Carolina University | Greenville | Egyesült Államok | Toboroz |
| Altman Clinical and Translational Research Institute | La Jolla | Egyesült Államok | Toborzás még nem indult |
| Altman Clinical and Translational Research Institute-IDS | La Jolla | Egyesült Államok | Toborzás még nem indult |
| Orthopaedic Institute for Children/Orthopaedic Hemophilia Treatment Center | Los Angeles | Egyesült Államok | Toboroz |
| UCSD Center for Bleeding and Clotting Disorders | San Diego | Egyesült Államok | Toborzás még nem indult |
Ez az oldal tájékoztatás, nem orvosi tanács. Bármely vizsgálatról az orvosoddal beszélj.