Hemophilia A

Study of a pd vWF/FVIII, Biostate®, in Subjects With Haemophilia A

NCT00879541 | PHASE 2 | INTERVENTIONAL

The aim of this study are to * assess the efficacy of Biostate® \[Study Product (SP)\] in subjects with Haemophilia A * compare the pharmacokinetics of Biostate® \[SP\] with the previously marketed product Biostate® (here referred to as Biostate® \[Reference Product (RP)\]). This study is divided into 3 parts: Part 1: Cross-over pharmacokinetic (PK) component. PK subjects will be randomised to determine the order in which they receive the two study products. This part of the study is double-blinded. Part 2: Efficacy component. All subjects will receive Biostate® \[SP\] as required to manage their haemophilia condition for an estimated period of 6 months (or minimum of 50 exposure days) to assess efficacy and safety of the product. This part of the study is open-label. Part 3: Repeat pharmacokinetic assessment. Subjects who participated in Part 1 (PK component) will undergo a repeat PK assessment on Day 180 following administration of Biostate® \[SP\].

14 Sites

81 Participants

Recruiting

12 Years to 17 Years

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Study Site

Plovdiv,Bulgaria

Study Site

Sofia,Bulgaria

Study Site

Varna,Bulgaria

Study Site

Skopje,Macedonia, The Former Yugoslav Republic of

Study Site

Bialystok,Poland

Study Site

Gdansk,Poland

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Krakow,Poland

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Lublin,Poland

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Poznan,Poland

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Warszawa,Poland

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Wroclaw,Poland

Study Site

Barnaul,Russian Federation

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Kirov,Russian Federation

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Moscow,Russian Federation

Study Eligibility Criteria

Inclusion Criteria

* Diagnosed with Haemophilia A with ≤ 1% Factor VIII (FVIII) levels in the absence of factor replacement
* Evidence of vaccination against hepatitis A and B (or presence of antibodies against hepatitis A and B due to either a previous infection or prior immunisation) within 10 years prior to Day 1 documented in the medical notes
* At least 150 days of prior exposure to a FVIII replacement product
* Written informed consent given
Exclusion Criteria (for participation in the pharmacokinetic (PK) component):
* Active bleeding
* Body weight \> 100 kg
Exclusion Criteria (for all subjects):
* Receipt of an infusion of any FVIII product, cryoprecipitate, whole blood, plasma, or desmopressin acetate (DDAVP) in the 4 days prior to Day 1
* Known history of FVIII inhibitors, or FVIII inhibitor level \> 0.6 Bethesda Units (BU) at screening
* Receipt of aspirin or other Non-Steroidal Anti-Inflammatory Drugs (NSAIDs) within 7 days of administration of study product.
* CD4 lymphocytes \< 200/µL. Subjects wo are HIV-1 positive may be considered for the study if viral load ≤ 200 particles/µL at screening and all other eligibility criteria are met.
* Impaired liver function ie. bilirubin \>1.5 x upper limit of normal (ULN) and/or AST/ALT \> 2.5 x ULN at screening.
* Acute or chronic medical condition, other than haemophilia A, which may, in the opinion of the Investigator, affect the conduct of the study
* von Willebrand Disease (VWD) with Von Willebrand Factor:Ristocetin Cofactor (vWF:RCo) level \< 50 IU/dL at screening
* Evidence or a history (within the previous 12 months) of abuse of any drug substance, licit or illicit
* Known or suspected hypersensitivity or previous evidence of severe side effects to Biostate®, FVIII concentrates or human albumin
* Participation in a clinical study or use of an investigational compound (e.g. a new chemical entity not approved for clinical use) in the 3 months preceding the first day of study drug administration, or plans to enter such a study during the study period
* Not willing and/or not able to comply with study requirements

Exclusion Criteria

* Pseudohyperkalemia due to hemolysis or to abnormally high numbers of platelets (\>500,000/mm3), leukocytes (\>70,000/mm3), or erythrocytes (hematocrit \>55%) at Screening based on results obtained locally
* Evidence of potassium-related electrocardiogram (ECG) changes at Screening
* Any of the following kidney conditions: maintenance hemodialysis or peritoneal dialysis, renal artery stenosis, and acute kidney injury or a history of acute renal insufficiency in the past 3 months
* Severe disorder of stomach or intestines including surgery that could affect gastrointestinal transit of the drug
* Increased liver enzymes (ALT, AST \> 3 times upper limit of normal) at Screening
* Active cancer, currently on cancer treatment or history of cancer in the past 2 years (except for non-melanoma skin cancer)
* Heart or liver transplant, or anticipated need for transplant during the study treatment period including a scheduled kidney transplant recipient. (Note: patients currently on a kidney transplant wait list are not excluded unless there is an identified donor).
* Alcohol abuse or substance use disorder within 1 year of Screening
* Subjects currently being treated with or having taken any one of the following medications (includes resins) in the 7 days prior to Screening: sodium or calcium polystyrene sulfonate, drospirenone
* Use of certain medications that can affect blood potassium levels if doses have not been stable for at least 14 days prior to Screening or if doses are anticipated to change during the 14-day PD / Dose Finding Phase
* Use of investigational product within 30 days of screening or within 5 half-lives, whichever is longer
* Known hypersensitivity to patiromer or its components
* In the opinion of the Investigator, any medical condition, uncontrolled systemic disease, or serious intercurrent illness that would significantly decrease study compliance or jeopardize the safety of the subject or potentially affect the quality of the data

Additional Studies

Additional studies can be found at ClinicalTrials.gov