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A Gene Transfer Study for Hemophilia A

2016-12-28 23:38:21 | BioPortfolio

Summary

This clinical research study is being conducted by Spark Therapeutics, Inc. to determine the safety and efficacy of the factor VIII gene transfer treatment with SPK-8011 in individuals with hemophilia A.

Description

Hemophilia A is a condition in which blood is unable to clot effectively. It is caused by a mutation or deletion in the gene that is responsible for producing blood-clotting factor VIII protein. Individuals with hemophilia A suffer from repeated bleeding episodes, often into the joints, which can cause chronic joint disease and sometime results in death due to the inability of the blood to clot efficiently. This chronic joint disease can have significant physical, psychosocial, and quality-of-life effects, including financial burden. The current treatment is intravenous (i.v.) injections of factor VIII protein products, either 2-3 times weekly or in response to bleeding.

Recent preliminary clinical data of a hemophilia B gene transfer study (which is also being conducted by Spark Therapeutics) shows all study participants achieving therapeutic factor IX activity levels (average of maintaining factor IX activity levels around 30% of normal with no confirmed bleeds, after receiving Spark gene transfer, with the approach of using the novel bio-engineered recombinant adeno-associated viral (rAAV) vector carrying a high specific activity of a factor IX gene. The approach being tested in this clinical research study uses a further modified novel AAV vector (with a stronger attraction to the human liver) to deliver the human factor VIII (hFVIII) gene into liver cells where FVIII protein is normally made.

Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Conditions

Hemophilia A

Intervention

SPK-8011

Location

University of California Davis - Hemostasis and Thrombosis Center
Sacramento
California
United States
94817

Status

Recruiting

Source

Spark Therapeutics

Results (where available)

View Results

Links

Published on BioPortfolio: 2016-12-28T23:38:21-0500

Clinical Trials [213 Associated Clinical Trials listed on BioPortfolio]

Long-Term Safety and Efficacy of SPK-8011 in Males With Hemophilia A

This long-term follow-up study will continue to evaluate the long-term safety and efficacy of SPK-8011 in males with hemophilia A, who have received a single intravenous administration of ...

ATHN 7: Hemophilia Natural History Study

This is a real-world study of the safety of the treatments used for people with hemophilia. The study will follow people with hemophilia A or B from across the country for about 4 years as...

Study Evaluating ReFacto in Hemophilia A

To identify the causative mutations in previously untreated patients with hemophilia A enrolled in the ReFacto® clinical safety and efficacy study CTN 93-R833-0XX/C9741-28, using two esta...

Females With Severe or Moderate Hemophilia A or B: A Multi-Center Study

To collect and analyze data on females with hemophilia so as to better define the difference between the study population and the male population with hemophilia.

Covalent Tolerance Induction to Factor VIII-Prediction of Inhibitors in Hemophilia

To correlate the HLA type and genetic defect with hemophilia A.

PubMed Articles [99 Associated PubMed Articles listed on BioPortfolio]

Inhibitors in Hemophilia B.

Hemophilia B (HB) is an X-linked bleeding disorder caused by deficiency of factor IX (FIX). Patients with the severe form (FIX

rFIXFc for Immune Tolerance Induction in a Severe Hemophilia B Patient with an Inhibitor and Prior History of ITI Related Nephrotic Syndrome.

A Retrospective Analysis of Intracranial Hemorrhage in Children with Hemophilia A.

To investigate the incidence, risk factors, clinical manifestations and prognosis of intracranial hemorrhage (ICH) in children with hemophilia A in a center of China, we conducted a retrospective anal...

Prevalence of factor VIII inhibitors among Afghan patients with hemophilia A: a first report.

: Prevalence of inhibitors in Afghan hemophilia patients has not been reported previously. Our aim was to determine the prevalence of factor VIII inhibitors among hemophilia A patients from the Kabul ...

Reliability and Validity of Patient-Reported Outcome Instruments in US Adults with Hemophilia B and Caregivers in the B-HERO-S Study.

To assess the reliability and validity of 6 patient-reported outcomes (PRO) instruments for evaluating health-related quality of life in adults with mild-severe hemophilia B and caregivers of children...

Medical and Biotech [MESH] Definitions

A deficiency of blood coagulation factor IX inherited as an X-linked disorder. (Also known as Christmas Disease, after the first patient studied in detail, not the holy day.) Historical and clinical features resemble those in classic hemophilia (HEMOPHILIA A), but patients present with fewer symptoms. Severity of bleeding is usually similar in members of a single family. Many patients are asymptomatic until the hemostatic system is stressed by surgery or trauma. Treatment is similar to that for hemophilia A. (From Cecil Textbook of Medicine, 19th ed, p1008)

The classic hemophilia resulting from a deficiency of factor VIII. It is an inherited disorder of blood coagulation characterized by a permanent tendency to hemorrhage.

A hereditary deficiency of blood coagulation factor XI (also known as plasma thromboplastin antecedent or PTA or antihemophilic factor C) resulting in a systemic blood-clotting defect called hemophilia C or Rosenthal's syndrome, that may resemble classical hemophilia.

Bleeding into the joints. It may arise from trauma or spontaneously in patients with hemophilia.

Stable blood coagulation factor involved in the intrinsic pathway. The activated form XIa activates factor IX to IXa. Deficiency of factor XI is often called hemophilia C.

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