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Search Results within category "Blood Disorders"

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Suggestions within category "Blood Disorders"


5 Study Matches

A Multi-Center, Observational Study in Males with Hemophilia A

SPK-8011-301 is a prospective, multi-center, observational study to collect efficacy and selected safety data on frequency and type of bleeding episodes and FVIII prophylaxis replacement therapy in the usual care setting of adult males with clinically severe hemophilia A (i.e., ≤ 2% IU/dL FVIII activity level), who are negative for neutralizing antibody (Nab) to AAV-Spark200.
Cynthia Campbell-Baird, RN at cbaird@pennstatehealth.psu.edu or 717-531-5777
Male
18 year(s) or older
This study is NOT accepting healthy volunteers
NCT03876301
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Inclusion Criteria:
Clinically severe Hemophilia A
Patients on prophylactic regimen > 2 months
Previous exposure to FVIII therapy
No history of hypersensitivity or anaphylaxis with any FVIII or IV immunoglobulin product
No measurable inhibitor against FVIII
Exclusion Criteria:
Active Hepatitis B or C within past 12 months
Current use of antiviral therapy to treat Hepatitis B or C
Documented significant liver disease
Serological evidence of HIV-1 or HIV-2
Anti-AAV-Spark neutralizing antibody titers >1:1
Blood Disorders
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Hershey, PA

An open-label, multinational study of fitusiran prophylaxis in male pediatric subjects aged 1 to less than 12 years with hemophilia A or B.

A study to confirm the appropriate dose of fitusiran when given to severe Hemophilia A or B patients with inhibitors between the ages of 1 and <12 years old.
Male
Younger than 18 years old
This study is NOT accepting healthy volunteers
NCT03974113
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Inclusion Criteria:
Severe hemophilia A or B (FVIII <1% or FIX ≤2%)
Inhibitory antibodies to FVIII or FIX
Age 1 to <12 years
Exclusion Criteria:
AT activity <60% at screening
Co-existing thrombophilic disorder
Central or peripheral indwelling catheter, with a hx of venous access complications
Anticipated need of surgery during the study
Presence of clinically significant liver disease
Blood Disorders
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Hershey, PA

Gene-transfer, open-label, dose-escalation study of SPK-8011 [recombinant adeno-associated viral vector with B-domain deleted human factor VIII gene] in individuals with hemophilia A

Gene-transfer, open-label, dose-escalation study of SPK-8011 in individuals with hemophilia A. Primary Objective to evaluate the safety, tolerability and efficacy of SPK-8011 a recombinant adeno-associated viral vector with B-domain deleted human factor VIII gene.
Cynthia Campbell-Baird at cbaird@pennstatehealth.psu.edu or 717-531-5777
Male
18 year(s) or older
This study is NOT accepting healthy volunteers
NCT03003533
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Inclusion Criteria:
Hemophilia A
FVIII Level <1% or 1-2% with> 10 bleeds past 52 weeks or 1-2% on prophy treatment
Greater than 150 exposure days to Factor VIII concentrate or cryo
No evidence of Factor VIII Inhibitor
Acceptable lab values as defined by protocol
Exclusion Criteria:
Active Hep B or C &/or current antiviral therapy for Hep B or C
Significant underlying liver disease
HIV-1 or HIV-2 with CD4 count </=l to 200
Other investigational product in the past 12 weeks or gene therapy research within the past 52 weeks
Any other significant condition/disease that in the opinion of the investigator would make the subject unsuuitable
Blood Disorders
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Hershey, PA

Dose-finding study of SPK-8016 gene therapy in patients with hemophilia A to support future evaluations in individuals with FVIII inhibitors.

SPK-8016-101 is a Phase 1/2a, open-label, non-randomized, dose-finding study designed to evaluate the safety, efficacy, and tolerability of SPK-8016 in adult males with clinically severe hemophilia A and no measurable inhibitor against FVIII.
Cynthia Campbell-Baird, RN at cbaird@pennstatehealth.psu.edu or 717-531-5777
Male
18 year(s) or older
This study is NOT accepting healthy volunteers
NCT03734588
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Inclusion Criteria:
Clinically severe Hemophilia A
Previous exposure to FVIII therapy
Have >150 exposure days to any recombinant and/or plasma-derived FVIII concentrates
No history of hypersensitivity or anaphylaxis with any FVIII or IV immunoglobulin product
No measurable inhibitor against FVIII
Exclusion Criteria:
Active Hepatitis B or C
Current use of antiviral therapy to treat Hepatitis B or C
Documented significant liver disease
Serological evidence of HIV-1 or HIV-2
Anti-AAV-Spark neutralizing antibody titers >1:5
Blood Disorders
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Hershey, PA

Phase 3, prospective, multi-center, open label study to investigate safety, immunogenicity, and hemostatic efficacy of PEGylated Factor VIII (BAX 855) in previously untreated patients (PUPs) < 6 years with severe hemophilia A (FVIII < 1%)

Phase 3, prospective, multi-center, open label study to investigate safety, immunogenicity, and hemostatic efficacy of PEGylated Factor VIII (BAX 855) in previously untreated patients (PUPs) < 6 years with severe hemophilia A (FVIII < 1%)
All
Younger than 18 years old
This study is NOT accepting healthy volunteers
NCT02615691
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Inclusion Criteria:
< 6 years old at the time of screening
previously untreated with < 3 EDs to ADVATE, BAX 855 or FFP any time prior to screening
severe hemophilia A (FVIII < 1%)
Exclusion Criteria:
History or current FVIII inhibitory antibodies (≥ 0.6 BU
Diagnosis of an inherited or acquired hemostatic defect other than hemophilia A
Previously treated with cryo, PRBC, platelets or any type of FVIII concentrate other than ADVATE, BAX 855v or FFP
weight is < 5 kg
Platelet count < 100,000/mL; severe chronic hepatic dysfunction or severe renal impairment
Blood Disorders
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Study Locations

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Location
Hershey, PA