A Gene Transfer Study for Hemophilia A

Status Completed

Clinical Trial 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.

Clinical Trial 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 so that they can produce factor VIII protein. ;

Study Design

Related Conditions & MeSH terms

Hemophilia A

Clinical Trial Details

NCT number	NCT03003533
Study type	Interventional
Source	Spark Therapeutics
Contact
Status	Completed
Phase	Phase 1/Phase 2
Start date	January 26, 2017
Completion date	December 5, 2023

View Details

See also
Status	Clinical Trial	Phase
Completed	`NCT03834727` - Characterizing the Impact and Treatment of Reproductive Tract Bleeding on Women and Post-menarchal Girls With Bleeding Disorders
Completed	`NCT03191799` - A Study to Evaluate the Safety and Tolerability of Prophylactic Emicizumab in Hemophilia A Patients With Inhibitors	Phase 3
Completed	`NCT01599819` - BAX 855 Dose-Escalation Safety Study	Phase 1
Terminated	`NCT04541628` - Safety & Efficacy of Encapsulated Allogeneic FVIII Cell Therapy in Haemophilia A	Phase 1/Phase 2
Completed	`NCT02847637` - A Clinical Trial to Evaluate Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Participants Without Inhibitors	Phase 3
Completed	`NCT04072237` - Study of Coagulation Faction VIIa Variant Marzeptacog Alfa (Activated) in Adult Subjects With Hemophilia	Phase 1
Completed	`NCT04085458` - Study to Gain More Information on How Safe and Effective Jivi Works in Patients With Severe Hemophilia A (Post-marketing Investigation)	Phase 4
Completed	`NCT04565236` - A Post Approval Commitment Study to Gain More Information on How Safe and Effective KOVALTRY is in Chinese Children, Adolescents /Adults With Severe Hemophilia A	Phase 4
Recruiting	`NCT05987449` - A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A	Phase 1/Phase 2
Active, not recruiting	`NCT04621916` - Preventing Inhibitor Recurrence Indefinitely	Phase 4
Not yet recruiting	`NCT02888223` - Pharmacokinetic Study of SCT800 in Previously Treated Patients With Hemophilia A	Phase 1
Completed	`NCT02528968` - National Study of a Pharmacokinetic-Focused Educational Package for Patients With Severe Haemophilia A	N/A
Completed	`NCT02225483` - Phenotypic Heterogeneity in Hemophilia A: An Investigation of the Role of Platelet Function	N/A
Completed	`NCT02199717` - An Institutional Pilot Study to Investigate Physical Activity Patterns in Boys With Hemophilia	N/A
Completed	`NCT01217255` - Comparing the Burden of Illness of Hemophilia in the Developing and the Developed World
Terminated	`NCT00995046` - Individually Tailored Prophylaxis in Patients With Severe Hemophilia A	N/A
Completed	`NCT00969319` - Effekt-2 - Efficacy and Safety of Long-term Treatment With KOGENATE® FS in Latin America	N/A
Completed	`NCT00868530` - Study Evaluating On-Demand Treatment Of Xyntha In Chinese Subjects	Phase 3
Completed	`NCT00839202` - Activity and Content of Factor VIII (FVIII) in Human Plasma: The Assessment of a Novel Immunoassay	N/A
Completed	`NCT00629837` - Pharmacokinetics and Safety of a Single Intravenous Infusion of BAY 79-4980	Phase 1