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New treatment option for Haemophilia A
Official title A Research Study Looking at Long-term Treatment With Mim8 in People With Haemophilia A
ClinicalTrials.gov ID: NCT05685238
What this study is testing
What is Mim8?
Mim8 is an investigational medicine, given as an injectable medicine, being studied as a potential treatment for haemophilia a.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This study is looking at how Mim8 works in people with haemophilia A, who either have inhibitors or do not have inhibitors. Mim8 is a new medicine that will be used to avoid bleeding episodes.
- Phase 3: a large, late-stage study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Adults
You may be able to join if
- Arm 1 \& 2:
- Informed consent obtained before any study related activities. Study related activities are any procedures that are carried out as part of the study...
- Male or female with diagnosis of congenital haemophilia A based on medical records.
- Ongoing participation in study NN7769-4513, NN7769-4514, NN7769-4516, or NN7769-4728 at the time of transfer. Participant should qualify either of...
- Participant from study NN7769-4513, who has participated in the extension part of the study for at least 12 weeks prior to enrolment in study...
You likely can't join if
- Any disorder, except for conditions associated with haemophilia A, which in the investigator's opinion might jeopardise participant's safety or...
- Participant who has discontinued or been withdrawn from studies NN7769-4513, NN7769-4514, NN7769-4516, or NN7769-4728.
- Previous participation in this study. Participation is defined as signed informed consent.
- Female who is pregnant, breast-feeding or intends to become pregnant.
- Female of child-bearing potential and not using a highly effective contraceptive method (highly effective contraceptive measures or as required by...
- Participation (i.e., signed informed consent) in any treatment, clinical study (except from study NN7769-4513, NN7769-4514, NN7769-4516, or...
See the full eligibility criteria
Who can join
- Arm 1 \& 2:
- Informed consent obtained before any study related activities. Study related activities are any procedures that are carried out as part of the study, including activities to determine suitability for the study.
- Male or female with diagnosis of congenital haemophilia A based on medical records.
- Ongoing participation in study NN7769-4513, NN7769-4514, NN7769-4516, or NN7769-4728 at the time of transfer. Participant should qualify either of the following criteria:
- Participant from study NN7769-4513, who has participated in the extension part of the study for at least 12 weeks prior to enrolment in study NN7769-4532, or,
- Participant has completed the end of treatment visit for study NN7769-4514, NN7769-4516 or NN7769-4728.
- Participant and/or participant's parent(s)/participant's Legally acceptable representative (LAR) willingness and ability to comply with scheduled visits and study procedures, including the completion of diary.
What rules you out
- Any disorder, except for conditions associated with haemophilia A, which in the investigator's opinion might jeopardise participant's safety or compliance with the protocol.
- Participant who has discontinued or been withdrawn from studies NN7769-4513, NN7769-4514, NN7769-4516, or NN7769-4728.
- Previous participation in this study. Participation is defined as signed informed consent.
- Female who is pregnant, breast-feeding or intends to become pregnant.
- Female of child-bearing potential and not using a highly effective contraceptive method (highly effective contraceptive measures or as required by local regulation or practice).
- Participation (i.e., signed informed consent) in any treatment, clinical study (except from study NN7769-4513, NN7769-4514, NN7769-4516, or NN7769-4728) of an approved or non-approved investigational medicinal product.
- Any planned major surgery, during part 1 of the study.
- Mental incapacity, unwillingness to cooperate, or a language barrier precluding adequate understanding and cooperation. Arm 3: Inclusion criteria
- Informed consent obtained before any study-related activities. Study-related activities are any procedures that are carried out as part of the study, including activities to determine suitability for the study.
- Male or female with diagnosis of congenital severe haemophilia A (endogenous FVIII activity less than (\<) 1 percentage [%]) with or without FVIII inhibitors based on medical records.
- Aged \<1 year at the time of signing informed consent.
- Body weight greater than or equal to (≥) 3.2 kilograms at the time of signing informed consent.
- previously untreated patients (PUPs) or minimally treated patients (MTPs) (i.e., up to 5 days of exposure to haemophilia-related treatment such as plasma-derived FVIII, recombinant FVIII, fresh frozen plasma...
- Full-term pregnancy (gestational age ≥37 weeks).
- Participant's parent(s)/LAR(s) willingness and ability to comply with scheduled visits and Arm 3 (infant) procedures, including the completion of diary and patient reported outcome (PRO) questionnaire.
- Participants \<3 months of age must show no signs of active intracranial haemorrhage at screening. This is confirmed by cranial ultrasound performed according to local practice and regardless of delivery method.
- Receipt of vitamin K prophylaxis (as per local standard practice).
- Availability of historical results in medical records for:
- activated partial thromboplastin time (aPTT)
- FVIII levels.
- Availability of historical results in medical records or pre-dose sample taken for:
- fibrinogen
- haematology parameters
- biochemistry parameters (aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT), bilirubin and creatinine). Exclusion criteria
- Known or suspected hypersensitivity to trial product or related products.
- Previous participation in study 4532. Participation is defined as signed informed consent.
- Participation (i.e., signed informed consent) in any treatment clinical study with receipt of the last dose within 6 months (or 5 half-lives of the investigational medicinal product, whichever is shorter) before planned...
- Exposure to non-factor haemostatic products for bleeding prophylaxis within 6 months (or 5 half-lives of the medicinal product, whichever is shorter) before planned enrolment.
- Known congenital or acquired coagulation disorders other than haemophilia A.
- Other conditions (e.g., autoimmune disease) or laboratory abnormality that may increase the risk of bleeding or thrombosis, as evaluated by the investigator. Any disorder, except for conditions associated with...
- Lack of adequate parental/legally acceptable representative (LAR) support to enter accurately and timely information regarding treatment and bleeding episodes into an (electronic) diary.
- Previous or current treatment for thromboembolic disease (with the exception of previous catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing) or signs of thromboembolic disease.
- Any planned major surgery, during part 1 of Arm 3 (infant). For definition of major surgery.
- Immune tolerance induction planned to take place after treatment initiation.
- Hepatic dysfunction defined as AST and/or ALT greater than (\>) 3 times the upper limit of normal (ULN) combined with total bilirubin \>1.5 times the ULN.
- Serum creatinine above 1.5 times the ULN.
The study team makes the final eligibility decision.
Where it's taking place
- Los Angeles, California, United States
- Aurora, Colorado, United States
- Miami, Florida, United States
- Tampa, Florida, United States
- Atlanta, Georgia, United States
- Chicago, Illinois, United States
- Indianapolis, Indiana, United States
- Iowa City, Iowa, United States
- Detroit, Michigan, United States
- Cleveland, Ohio, United States
- Dayton, Ohio, United States
- Hershey, Pennsylvania, United States
- Philadelphia, Pennsylvania, United States
- Nashville, Tennessee, United States
- Innsbruck, Austria
- Vienna, Austria
- Brussels, Belgium
- Leuven, Belgium
- Sofia, Bulgaria
- Hamilton, Ontario, Canada
+ 90 more site(s).
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, not specified. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Los Angeles, California, United States; Aurora, Colorado, United States; Miami, Florida, United States; Tampa, Florida, United States; Atlanta, Georgia, United States; Chicago, Illinois, United States and 104 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.