4514 (Frontier 2): Mim8 in adults and adolescents with haemophilia A
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A Long-Term Follow-Up Study in Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial
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AFFINE
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BMN 270 Gene Therapy in Severe Haemophilia A Patients with Inhibitors
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BMN 270-301: Gene Therapy Study in Severe Haemophilia A Patients
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Frontier 4
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HAEM 4448
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Haemophilia kids rearfoot motion study
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MO42623 Beyond The Bleed
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R0000-HEMB-2187
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Study of FIX Prophylaxis Replacement Therapy on Haemophilia B Subjects
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UK - EHL Outcome Registry
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The conNeCT study
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UK TTP Registry
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PETS
Treatment stage
Multiple stages
1
Subtype
Antithrombin deficiency
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Treatment stage
Multiple stages
1
Subtype
Haemophilia
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Treatment stage
Multiple stages
12
Subtype
Thrombotic Thrombocytopenic Purpura
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Treatment stage
Multiple stages
2
Subtype
Venous thromboembolism
Count1
Treatment stage
Multiple stages
1
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Population
Description
ECOG
PI - Evelina
PI - KCH
PI - GSTT
PI - PRUH
PI - QEH
PI - UHL
Trial summary
Key Eligibility Criteria
Summary
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Summary
For adults with antiphospholipid antibodies who wish to become pregnant
Randomised trial: Hydroxychloroquine to improve pregnancy outcome in women with Antiphospholipid Antibodies
Not specified
Hunt, B
Patients with antiphospholipid antibodies (aPL) may have an increased risk of having blood clots and women with aPL may have problems during pregnancy. In the presence of aPL blood becomes more ‘sticky’ than usual. aPL occur in about 1% of the population. Knowing that there are 800,000 births a year in the United Kingdom, this means 8,000 pregnancies affected by aPL every year. Women with aPL are more likely to have pregnancy loss and/or small babies and/or pre-eclampsia (high blood pressure and
Women with known aPL (ie. isolated aPL or APS) who are planning pregnancy. aPL are defined by the presence of a positive test for anticardiolipin antibodies (IgG/IgM isotypes > 95th percentile) and/or lupus anticoagulant and/or anti- beta 2 glycoprotein-I (IgG/IgM isotypes > 95th percentile), on two or more consecutive occasions more than 12 weeks apart. The last positive test must be within 12 months of study entry.
For adults with antithrombin deficiency
Phase 3 study: Atenativ in patients with congenital antithrombin deficiency undergoing surgery or delivery
Not specified
Hunt, B
Arm 1 is a treatment arm for any patient with Antithrombin deficiency (ATN def) who is having surgery and needs thrombin to prevent bleeding or clotting issues. Patient will receive compensation for travel and a stipend.
Arm 2 is looking at PK results over a 2 week period of a patient with known ATN def. They are give a dose of the Atenativ and levels are checked daily for 2 weeks. Patients can receive payment and housing due to the extensive number of visits.
Adult male or female patients ≥ 18 and ≤ 80 years of age.
For adults and adolescents with severe haemophilia A
Randomised trial: Mim8 for severe haemophilia A
Not specified
Luo, P
Open-label randomised controlled trial of Mim8 in adult and adolescent patients with severe haemophilia A with or without inhibitors
Male patients with the diagnosis of congenital haemophilia A (FVIII activity < 1%) with or without inhibitors, age 12 years or above at time of signing informed consent.
For patients who have received BMN 270 in a prior BioMarin clinical trial
Long-term follow-up study: BMN 270
Not specified
Dolan, G
A Long-Term Follow-Up Study in Subjects with Severe Hemophilia A Who Received BMN 270, an Adeno-Associated Virus Vector–Mediated Gene Transfer of Human Factor VIII in a Prior BioMarin Clinical Trial
Subjects must have completed their primary treatment study. Subjects may enroll in 270-401 even if they have restarted FVIII prophylaxis or other hemophilia A treatment.
For adult male patients with moderately severe to severe Hemophilia A (FVIII:C≤1%)
Phase 3 open-label study: PF-07055480 (recombinant AAV2/6 human factor VIII gene therapy)
Not specified
Dolan, G
Haemophilia A is caused by a gene that contains a mistake. Because of this, the body does not make enough factor 8 (hereafter referred to as “factor VIII”). Factor VIII is one of the proteins needed for blood to clot. At present, the main therapy options for haemophilia A involve intravenous factor VIII injections. These can be at the time of a bleed (on-demand) or at regular intervals (prophylaxis). The AFFINE clinical study will assess the safety and effectiveness of a study drug to see if a s
Participant must be ≥ 18 and < 65 years of age at the time of signing the informed consent.
For adults with haemophilia A with Active or Prior Inhibitors
Phase 1/2 study: BMN 270, virus vector-mediated gene transfer of Human Factor VIII
Not specified
Neave, L
Haemophilia A (HA) is a genetic bleeding disorder that usually affects males causing increased bleeding in joints and soft tissue with increased risk of death from haemorrhage, bleeding in the brain. For the past decade, patients with HA have received infusions of clotting factor to treat episodes of bleeding. However, patients have developed antibodies to neutralise the effect of the clotting factor treatment leading to an increase in number of bleeding episodes. The alternative treatment for p
Males ≥ 18 years of age with hemophilia A and documented prior residual FVIII activity ≤ 1 IU/dL including, but not limited to, at the time of detected inhibitors, at the time of signing the informed consent.
For adults with haemophilia A, with residual FVIII levels ≤ 1 IU/dL, receiving prophylactic FVIII infusions
Phase 3 open label study: BMN 270, virus vector–mediated gene transfer of Human Factor VIII
Not specified
Dolan, G
A Phase 3 Open-Label, Single-Arm Study To Evaluate The Efficacy and Safety of BMN 270, an Adeno-Associated Virus Vector– Mediated Gene Transfer of Human Factor VIII in Hemophilia A Patients with Residual FVIII Levels ≤ 1 IU/dL Receiving Prophylactic FVIII Infusions
Males ≥ 18 years of age with haemophilia A and residual FVIII levels ≤ 1 IU/dL as evidenced by medical history, at the time of signing the informed consent.
For patients with congenital haemophilia A of any severity
Long term study: extension of Frontier 1, 2, 3, and 5 - Mim8 for haemophilia
Not specified
Luo, P
Open-label long-term safety and efficacy study of Mim8 in patients with Haemophilia A with or without inhibitors
Informed consent obtained before any study-related activities.
For adults with haemophilia
Phase 1/2 dose-escalation study: BMN 270
Not specified
Faruqi, U
A Phase 1/2, Dose-Escalation Safety, Tolerability and Efficacy Study of BMN 270, an Adenovirus-Associated Virus Vector–Mediated Gene Transfer of Human Factor VIII in Patients with Severe Haemophilia A
Not listed
For children between 7 and 11 yo with Haemophilia, with or without ankle bleeds
Identifying discrepancies in rear food movement control using motion capture systems and force plate data
Not specified
Alamelu, J
Haemophilia is a disorder of blood clotting mechanisms almost wholly affecting male children that can cause bleeds to occur inside joints ultimately leading to joint degeneration. This does not happen with all haemophiliacs and is part dependent on how severely they are affected by the condition, but is not wholly explained by the history of bleeding into the joints and it is thought that the onset of degeneration is multifactorial. At present the most commonly affected joint is the ankle. Previ
Group A = haemophilia with ankle bleeds, group B = haemophilia no bleeds, group C = typically developing peers.
For adults and adolescents with severe or moderate haemophilia A without FVIII prophylaxis
Phase 4 open label study: Emicizumab prophylaxis
Not specified
Dolan, G
A MULTICENTER, OPEN-LABEL PHASE IV STUDY TO EVALUATE OVERALL HEALTH, PHYSICAL ACTIVITY, AND JOINT OUTCOMES, IN PARTICIPANTS AGED ≥ 13 AND < 70 YEARS WITH SEVERE OR MODERATE HEMOPHILIA A WITHOUT FVIII INHIBITORS ON EMICIZUMAB PROPHYLAXIS
Signed Informed Consent Form (signed by participant’s legally authorized representative for participants who have not attained the age of majority).
For patients with haemophilia B
Disease characteristic evaluation in patients receiving prophylaxis with standard of care fix replacement therapy
Not specified
Dolan, G
A PROSPECTIVE STUDY TO EVALUATE DISEASE CHARACTERISTICS IN HEMOPHILIA B PARTICIPANTS RECEIVING PROPHYLAXIS WITH STANDARD OF CARE FIX REPLACEMENT THERAPY
None listed
For adults with moderately severe to severe adult haemophilia B (FIX:C≤2%) who are negative for neutralising antibodies (NAB) to adeno-associated virus vector (AAV)-spark100
Open-label, non-investigational product study: Factor IX (FIX) prophylaxis replacement therapy i
Not specified
Dolan, G
This study is being carried out to assess how effective current factor IX prophylaxis replacement therapy is in treating haemophilia B and also to determine if the participant’s body produces antibodies to a particular virus, specifically AAV-Spark 100. Patients with haemophilia B have defective factor IX genes. Gene therapy works by using a tool called a “vector” to transfer a normal or “working” factor IX gene into patient’s liver cells where factor IX is usually processed and excreted. The vi
Evidence of a personally signed and dated informed consent document indicating that the participant has been informed of all pertinent aspects of the study.
For patients with haemophilia A or B being considered for use of extended half life clotting factor concentrate
Research database: real-world outcomes with extended half-life concentrates for haemophilia A and B
Not specified
Dolan, G
Severe haemophilia A and B (SHA, SHB) are inherited bleeding disorders affecting male patients and are characterised by low levels of circulating clotting factors VIII and IX respectively. Clinically low levels present with multiple recurrent bleeds into joints and muscle from the first couple of years of life. In addition patients may present with spontaneous and potentially fatal bleeding into any organ. The mainstay of treatment is replacement with the missing factor in the form of intravenou
Patients with Haemophilia A or B requiring replacement therapy
For adults with acute episode TTP
Questionnaire-based observational study
Not specified
Doyle, A
Thrombotic thrombocytopenic purpura (TTP) is a rare condition, which has a very high risk of death if not recognised and given immediate treatment. TTP is caused by a very low level of an enzyme in the body, called ADAMTS13. A lack of ADAMTS13 causes multiple small clots to form around the body which can disrupt the blood flow to important organs. Although survival has improved significantly, it is now being recognised that patients with TTP may suffer with longer term complications as a result
Acute episode TTP:
For children and adults with Thrombotic Thrombocytopenic Purpura
Research database and biobank
Not specified
Doyle, A
Thrombotic thrombocytopenic purpura is a rare sudden onset, life threatening illness that typically affects women between the age of 30 to 40 years, although can present at any age from birth to 80 years. The majority of cases are due to antibodies against the missing enzyme, ADAMTS 13. Lack of this enzyme results in small thrombi in all the body organs. Even with treatment, specifically plasma exchange, 15-20% of patients die. In the South East of England, we have presented a registry of patien
Patients with a clinical diagnosis of acute TTP, defined by thrombocytopenia, MAHA which may be associated with clinical evidence of organ compromise.
For adults with venous thromboembolism undergoing surgery
Randomised trial: graduated compression stockings to prevent venous thromboembolism in low-risk surgical patients
Not specified
Hunt, B
Hospital acquired thrombosis describes blood clots that form in the legs and lungs after someone is treated in hospital. Clots in the leg can cause swelling, pain and other problems. If a clot in the leg travels to the lungs, it may be life threatening. Having surgery increases the risk of developing blood clots. People having short-stay surgery (who either go home the same day or who stay overnight but go home shortly afterwards) are at a much lower risk of developing a blood clot than those wh
Adults (18-59 years of age) scheduled to undergo a surgical procedure with a hospital stay < 48 hours.