SPK-8011 for Hemophilia A

Phase-Based Progress Estimates
Virginia Commonwealth University School of Medicine, Richmond, VA
Hemophilia A
SPK-8011 - Genetic
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Study Summary

This study is evaluating whether a gene therapy treatment may help individuals with hemophilia A.

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Treatment Effectiveness

Effectiveness Progress

1 of 3

Other trials for Hemophilia A

Study Objectives

This trial is evaluating whether SPK-8011 will improve 2 primary outcomes and 2 secondary outcomes in patients with Hemophilia A. Measurement will happen over the course of 52 weeks.

52 weeks
Changes from baseline in FVIII activity levels after a single outpatient administration of SPK-8011
Kinetic assessment of SPK-8011 including shedding of vector DNA in bodily fluids
Number of participants requiring a course of steroid therapy for the elevations in liver enzymes
Number of study-related adverse events, including clinically significant abnormal laboratory values

Trial Safety

Safety Progress

1 of 3

Other trials for Hemophilia A

Trial Design

1 Treatment Group

1 of 1
Experimental Treatment

This trial requires 50 total participants across 1 different treatment group

This trial involves a single treatment. SPK-8011 is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 1 & 2 and have already been tested with other people.

All participants who meet the eligibility criteria will receive an outpatient single intravenous (i.v.) administration of SPK-8011.

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 52 weeks
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly 52 weeks for reporting.

Closest Location

Virginia Commonwealth University School of Medicine - Richmond, VA

Eligibility Criteria

This trial is for male patients aged 18 and older. There are 7 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
They have a confirmed diagnosis of hemophilia A, which is evidenced by their medical history and their plasma FVIII activity levels that are ≤ 2% of normal. show original
I have had more than 150 exposure days to FVIII concentrates or cryoprecipitate. show original
Patients who have experienced 10 or more bleeding events over the previous 12 months only if they are receiving on-demand therapy and have a FVIII baseline level of 1-2% of normal are candidates for prophylactic therapy. show original
Do not have any measurable inhibitors to Factor VIII and have not had any prior history of inhibitors to the FVIII protein. show original
Males age18 years or older
I have never had an allergic reaction to a Factor VIII product. show original
if sexually active If you are sexually active, you should agree to use reliable barrier contraception. show original

Patient Q&A Section

What are common treatments for hemophilia a?

"The number of cases of hemophilia A is increasing due to growing incidence and prevalence estimates, changing life-styles, and improving medical knowledge. Consequently, new forms of treatment to improve the quality of life of patients with haemophilia and to improve survival remain required. The availability of purified clotting factors for both prophylaxis and therapeutic purposes and the development of plasma fractionation techniques are two important innovations that have helped to improve survival rates." - Anonymous Online Contributor

Unverified Answer

What is hemophilia a?

"Hemophilia is a genetic disease that creates a severe form of blood clotting disorder. It manifests itself primarily through the skin and joints as bruises and sores. While there is still currently no cure, a vaccine is being researched for people with both sickle cell disease and hemophilia. Without the use of a vaccine, there is no way to prevent a hemophiliacs life from coming to an end.\n\n- http://www.nhs.gov.au/health/education/learn/healthprofessionals/medical_professions.html\n- https://web.archive.org/web/20070717025343/http://www.med." - Anonymous Online Contributor

Unverified Answer

What causes hemophilia a?

"Although the cause of hemophilia is still not well understood, it is now known that a deficiency in the production of clotting factors leads to a hemorrhagic tendency. Patients with hemophilia type A are mostly affected by the most severe form of hemorrhagic disease of the newborn, and they develop hemophilia as a result of inheriting a faulty allele of the factor VIII gene. Those who do not inherit this mutation will have milder bleeding issues. Type B hemophilia, which occurs more frequently, is caused by a mutation in factor IX gene, leading to a deficiency in factor IX, a coagulation factor." - Anonymous Online Contributor

Unverified Answer

What are the signs of hemophilia a?

"The most common symptoms are painful or swollen joints, bone pain and joint instability. Other signs include bleeding into muscle or nose, easy bruising and bleeding from the joints. Bleeding into tendon is rare and is associated with an underlying problem rather than an underlying hemophilia. All patients with an underlying, active bleeding problem should have a bleeding history. In children, the condition can present differently. When a child presents with symptoms of bleeding after an injury, this may be a sign for the presence of hemophilia.\n" - Anonymous Online Contributor

Unverified Answer

How many people get hemophilia a a year in the United States?

"Around 45,000 American boys and girls have hemophilia A; 4 in 10 men and 2 in 10 women who meet the symptoms of hemophilia are asymptomatic. Hemophilia is a rare disorder that the CDC will need to add to its list of recommended childhood diseases." - Anonymous Online Contributor

Unverified Answer

Can hemophilia a be cured?

"We suggest that hemophilia A be treated in a specialty center in the U.S. as soon as possible after contracting the disease since the risk of fatal hemorrhage of the joints is high in a newly diagnosed patient with hemophilia A. The optimal therapy that minimizes the risk of hemorrhage of the joints would be to treat the patient before a major joint is damaged so that surgery may be avoided if it can. These patients should have a life expectancy similar to that of the rest of the population." - Anonymous Online Contributor

Unverified Answer

How serious can hemophilia a be?

"Hemophilia A is an almost non-existent disorder, and there have been no reported cases of fatal hemophilia A for many years because of the availability of anti-hemophilia A drugs and improved monitoring methods. These developments have led to an increased need for new treatments for this rare disorder. As well as the traditional treatments, the use of recombinant coagulation factors, as well as synthetic hemostatic drugs (hemostatic dressings), may be beneficial in some cases. Additionally, specific gene therapy procedures may allow for a cure for hemophilia A by halting the production of the protein the deficient (defective) hemoglobins." - Anonymous Online Contributor

Unverified Answer

Have there been any new discoveries for treating hemophilia a?

"Significant progress has been made in the field, including the development of new recombinant products containing an FVIII peptide. The majority of people with hemophilia now use recombinant products in their treatment and are able to live normal lives. In addition, an effective therapeutic and prophylactic treatment for people with hemophilia B by somatic recombinant retroviral gene therapy is available." - Anonymous Online Contributor

Unverified Answer

What are the latest developments in spk-8011 for therapeutic use?

"spk-8011 should be used as an alternative to conventional drugs used in HA for treatment of haemophilia during surgery and on prophylaxis. In this context, spk-8011, which has some of the advantages inherent in HA, should be used in the shortest time possible to avoid post-transfusion haemorrhaging, thereby achieving maximal benefit." - Anonymous Online Contributor

Unverified Answer

What is the average age someone gets hemophilia a?

"Patients with hemophilia A typically get their diagnosis around age 3, but there is a wide range of age at diagnosis ranging between 1 and 25. There is a small percentage of patients with hemophilia A who do not develop immunothrombocytopenia. The median age of diagnosis was 11.5 years and the average age of development of bleeding symptoms was 15.1 years. The major bleeding episodes for patients with hemophilia were spontaneous. There were no reported bleeding episodes related to dental and orthopedic procedures or minor physical trauma. There are multiple risk factors associated with venous hemorrhage such as cigarette smoking, prior surgery, and previous viral infection, but no known risk factors for minor trauma induced hemorrhage." - Anonymous Online Contributor

Unverified Answer

Who should consider clinical trials for hemophilia a?

"The rationale for clinical trials is that they are justified when a clinical benefit is demonstrated, when the trial compares well to the current standard of care, and when the chance of achieving benefit is high." - Anonymous Online Contributor

Unverified Answer

What are the common side effects of spk-8011?

"Results from a recent paper shows that the common side effects of NVP-SPK-8011 are dose dependent. In addition, there is a clear trend that the lower the dose, the more the observed common side effects. The common side effects showed no gender preference. Some of these common pharmacodynamics side effects are: nausea, fatigue, headache/migraine/sinusitis, and constipation. Results from a recent paper are of great value in the development of new spk-8011 doses and clinical trials." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
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