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Gene Therapy
SPK-8016 for Hemophilia A
Phase 1 & 2
Waitlist Available
Led By Lindsey George, MD
Research Sponsored by Spark Therapeutics, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up from 28 days post vector administration up to week 52
Awards & highlights
Study Summary
This trial is testing a new drug, SPK-8016, to see if it is safe and effective in treating patients with hemophilia A who also have inhibitors to FVIII.
Eligible Conditions
- Hemophilia A
- Blood Clotting Disorder
- Blood Coagulation Disorders
- Blood Diseases
- Gene Transfer
- Gene Therapy
- Vector-Borne Disease
- Adeno-Associated Virus
- Coagulation Disorders
- Hemorrhagic Disorders
- Genetic Disorders
- Genetic Diseases
- Recombinant
- Inhibitors
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ from 28 days post vector administration up to week 52
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~from 28 days post vector administration up to week 52
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Annualized Infusion Rate
Number of Bleeding Events (Spontaneous and Traumatic) Since 28 Day Post Vector Administration
Number of Participants With Adverse Events (AEs)
+3 moreSecondary outcome measures
Number of Participants With Immune Responses to AAV Capsid Protein and BDD-hFVIII Transgene
Number of Participants With Vector-shedding of SPK-8016 in Bodily Fluids
Time to Achieve Steady-state FVIII Activity Levels
Side effects data
From 2023 Phase 1 & 2 trial • 4 Patients • NCT0373458850%
Lethargy
50%
Weight Increased
50%
Headache
50%
Arthralgia
50%
Oropharyngeal Pain
25%
Vision Blurred
25%
Abdominal Pain Upper
25%
Pharyngeal Erythema
25%
Cushingoid
25%
Diarrhoea
25%
Feeling Hot
25%
Dyspepsia
25%
Food Poisoning
25%
Joint Swelling
25%
Nausea
25%
Contusion
25%
Tooth Fracture
25%
Tooth Loss
25%
Candida Infection
25%
Vomiting
25%
Skin Abrasion
25%
Skin Laceration
25%
Dizziness
25%
Fatigue
25%
Oedema Peripheral
25%
Swelling Face
25%
Back Pain
25%
Drug-Induced Liver Injury
25%
Tooth Abscess
25%
Thermal Burn
25%
Cough
25%
Hypoaesthesia
25%
Rash
25%
Haematemesis
25%
Gastroenteritis Viral
25%
Joint Noise
25%
Muscular Weakness
25%
Pruritus
25%
Nasopharyngitis
25%
Wheezing
25%
Acne
25%
Oral Herpes
25%
Pharyngitis Streptococcal
25%
Limb Crushing Injury
25%
Libido Decreased
25%
Skin Infection
25%
Scratch
25%
Blood Glucose Increased
25%
Hyperglycaemia
25%
Pollakiuria
25%
Anxiety
25%
Skin Irritation
100%
80%
60%
40%
20%
0%
Study treatment Arm
SPK-8016
Trial Design
1Treatment groups
Experimental Treatment
Group I: SPK-8016Experimental Treatment1 Intervention
All participants who meet the eligibility criteria will receive an outpatient single intravenous (i.v.) administration of SPK-8016.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
SPK-8016
2019
Completed Phase 2
~10
Find a Location
Who is running the clinical trial?
Spark Therapeutics, Inc.Lead Sponsor
14 Previous Clinical Trials
430 Total Patients Enrolled
Spark TherapeuticsLead Sponsor
13 Previous Clinical Trials
350 Total Patients Enrolled
Tiffany Chang, MDStudy DirectorSpark Therapeutics, Inc.
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- You have a current hepatitis B or C infection.You have a severe form of hemophilia A with low levels of FVIII activity or frequent bleeding.You have a serious liver disease.You have never had a serious allergic reaction to FVIII or IV immunoglobulin.You have evidence of HIV infection and low CD4 counts, unless you are a stable HIV-positive participant who meets certain criteria.You have antibodies in your blood that react with the AAV-Spark capsid.You have a long-term infection or another long-term illness.You have received a gene therapy or experimental drug in another study within the past year.You have used FVIII concentrates or cryoprecipitates for more than 150 days.You do not have any natural substances in your body that can block the function of FVIII, and you have not shown any signs of not responding to FVIII treatment.
Research Study Groups:
This trial has the following groups:- Group 1: SPK-8016
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Is this trial being run at a large number of clinics in our state?
"There are 9 trial sites operational at the moment, with locations in Peoria, Portland and Philadelphia as well as 6 other cities. If you want to take part in this study, make sure to choose a location close to you to limit travel."
Answered by AI
Are there any unfilled positions in this clinical trial for new participants?
"From the data on clinicaltrials.gov, it can be inferred that this trial is looking for more patients as of 10/28/2022. The listing was first posted on 1/30/2019."
Answered by AI
How many people have signed up to be a part of this research?
"The sponsor, Spark Therapeutics, requires a total of 30 patients that meet the clinical trial's inclusion criteria to proceed with the study. The study will be conducted out of various locations including Illinois Bleeding and Clotting Disorders Institute in Peoria, Illinois and Oregon Health & Science University in Portland, Oregon."
Answered by AI
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