Active not recruitingOBSERVATIONAL

Long-Term Safety and Efficacy of Spark-Sponsored Gene Therapies in Males With Hemophilia A

This research study is checking in on men with haemophilia A who previously had a special treatment called gene therapy (either SPK-8011 or SPK-8016) in another study. The main goal is to understand if these treatments are safe over a long time and how well they continue to work to help manage their haemophilia. Researchers will follow these men for up to 10 years after their initial treatment to gather important information. This helps us learn more about new ways to treat haemophilia A and ensures that these therapies are a good option for patients in the future.

At a glance

Status

Active not recruiting

Sponsor

Spark Therapeutics, Inc.

Enrolment target

Start

14 Aug 2018

Estimated completion

01 Dec 2032

Hemophilia A

What is this study about?

Haemophilia A is a condition where your blood doesn't clot properly, which can lead to bleeding problems. Gene therapy is a new type of treatment that aims to correct the genetic fault that causes haemophilia A, potentially offering a long-lasting solution.

This particular study is a follow-up for men who have already received one of two specific gene therapies, called SPK-8011 or SPK-8016, in a previous study. Think of it like a long-term check-up. The researchers want to keep a close eye on these men to see how they are doing over many years. They are specifically interested in understanding if the treatment remains safe and effective in the long run.

By following these participants for a total of up to 10 years after their initial treatment, scientists can collect valuable information. This helps them understand the lasting effects of these new gene therapies, ensuring that they provide a safe and helpful way to manage haemophilia A for patients.

Key takeaways

This study is for adult men with haemophilia A.
It's a long-term follow-up for gene therapy treatments SPK-8011 or SPK-8016.
The goal is to check long-term safety and how well the treatment continues to work.
Participants will be followed for up to 10 years after their initial treatment.
No new medication will be given in this study.
Your willingness to attend appointments is important for participation.

Who may be eligible?

This study is only for adult men who have haemophilia A and have already taken part in a previous Spark-sponsored study where they received one dose of either SPK-8011 or SPK-8016 gene therapy. You must have received this particular treatment in one of those earlier studies to be considered.

It's very important that you understand what the study involves, including any potential benefits or risks, before you agree to take part. You'll need to give your signed permission, known as informed consent, before any study checks begin.

Unfortunately, you wouldn't be able to join this study if you are not able or willing to attend all the scheduled appointments and follow the study's instructions. This is because regular check-ups are crucial for gathering the necessary long-term information.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you an adult man (18 years or older)?
Do you have haemophilia A?
Did you receive a single dose of SPK-8011 or SPK-8016 gene therapy in a previous Spark study?
Are you able and willing to attend all scheduled study appointments for up to 10 years?
Are you willing to sign a consent form after understanding the study?

Answer every question to see your result.

What does participation involve?

If you join this study, it means you'll be part of a long-term follow-up. This involves regular visits to the clinic so that researchers can monitor your health and the effects of the gene therapy you received. These visits will include various assessments, such as blood tests or other checks, to see how your body is responding and how well the treatment is working. The study will not involve any new medication; it's about watching the long-term effects of the gene therapy you've already had.

You will be followed for a total of up to 10 years from the time you first received your gene therapy dose in the previous study. This means the study duration for you will vary depending on how long ago you had your initial treatment. It's important to be willing and able to attend all these scheduled check-ups throughout this period.

Potential risks and benefits

As this is a follow-up study, you won't be receiving new treatment, but monitoring for any long-term effects. The potential benefits include contributing to important knowledge about the long-term safety and effectiveness of gene therapy for haemophilia A, which could help future patients. There are always potential risks with any medical study, even follow-up, such as discomfort from blood draws or the time commitment for visits. You will be told about any known risks during the consent process. Remember, you can choose to leave the study at any time, for any reason, without it affecting your medical care.

Locations (12)

Boston Children's Hospital
Verified postcode
Boston, United States
Mississippi Center for Advanced Medicine
Verified postcode
Madison, United States
Truman Medical Centers
Verified postcode
Kansas City, United States
Oregon Health & Science University
Verified postcode
Portland, United States
Pennsylvania State University Milton S. Hershey Medical Center
Verified postcode
Hershey, United States
The Children's Hospital of Philadelphia
Verified postcode
Philadelphia, United States
Thomas Jefferson University
Verified postcode
Philadelphia, United States
Hemophilia Center of Western Pennsylvania
Verified postcode
Pittsburgh, United States
Virginia Commonwealth University
Verified postcode
Richmond, United States
Royal Prince Alfred Hospital Department of Cell & Molecular Therapies
Verified postcode
Sydney, Australia
The Alfred Hospital & Monash Medical Centre
Verified postcode
Melbourne, Australia
St. Michael's Hospital
Verified postcode
Toronto, Canada

Common questions

What is gene therapy?

Gene therapy is a treatment that aims to fix the root cause of a disease, like haemophilia A, by introducing new genetic material into the body.

Do I get a new treatment in this study?

No, this study is only for following up on the gene therapy you received in a previous study. You will not get a new dose of medication.

How long will I be in this study?

You will be followed for a total of up to 10 years from when you first received your gene therapy dose.

Why is this study important?

This study helps us understand how safe and effective these gene therapies are over a long period, which is crucial for future treatments for haemophilia A.

Can I leave the study if I change my mind?

Yes, you can withdraw from the study at any time without any reason, and it won't affect your medical care.