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Gene Therapy

Gene Transfer with SPK-8011 for Hemophilia A

Q: In how many different medical clinics is this clinical study being performed today?

<p>In addition to Richmond, <a href="https://www.withpower.com/clinical-trials/virginia">Virginia</a>'s Virginia Commonwealth University School of Medicine and Gainesville, <a href="https://www.withpower.com/clinical-trials/florida">Florida</a>'s University of Florida Health, this clinical trial is also enrolling patients at <a href="https://www.withpower.com/clinical-trials/oregon">Oregon</a> Health &#x26; Science University in Portland. There are a total of 11 enrolment locations.</p>

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 52 weeks

Awards & highlights

Study Summary

This trial is testing a new gene therapy treatment for hemophilia A. The goal is to see if it is safe and effective.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 52 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~52 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and 52 weeks for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Changes from baseline in FVIII activity levels after a single outpatient administration of SPK-8011

Number of study-related adverse events, including clinically significant abnormal laboratory values

Secondary outcome measures

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

Trial Design

1Treatment groups

Experimental Treatment

Group I: SPK-8011Experimental Treatment1 Intervention

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

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

SPK-8011

2017

Completed Phase 2

~30

Do I qualify?Apply below to find out

Find a Location

Who is running the clinical trial?

Spark Therapeutics, Inc.Lead Sponsor

14 Previous Clinical Trials

409 Total Patients Enrolled

6 Trials studying Hemophilia A

146 Patients Enrolled for Hemophilia A

Spark TherapeuticsLead Sponsor

13 Previous Clinical Trials

329 Total Patients Enrolled

5 Trials studying Hemophilia A

66 Patients Enrolled for Hemophilia A

Lindsey George, MDPrincipal InvestigatorChildren's Hospital of Philadelphia

1 Previous Clinical Trials

4 Total Patients Enrolled

1 Trials studying Hemophilia A

4 Patients Enrolled for Hemophilia A

Media Library

SPK-8011 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT03003533 — Phase 1 & 2

Hemophilia A Research Study Groups: SPK-8011

Hemophilia A Clinical Trial 2023: SPK-8011 Highlights & Side Effects. Trial Name: NCT03003533 — Phase 1 & 2

SPK-8011 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03003533 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are recruitment efforts for this research still underway?

"Yes, this information is accurate. The clinical trial in question was most recently updated on 7/15/2022 and it is actively recruiting patients who meet the study's requirements."

Answered by AI

In how many different medical clinics is this clinical study being performed today?

"In addition to Richmond, Virginia's Virginia Commonwealth University School of Medicine and Gainesville, Florida's University of Florida Health, this clinical trial is also enrolling patients at Oregon Health & Science University in Portland. There are a total of 11 enrolment locations."

Answered by AI

How many individuals are being asked to take part in this experiment?

"That is correct. The website clinicaltrials.gov has information stating that this particular trial is looking for patients right now. This trial was first posted on 1/26/2017, and the most recent update was on 7/15/2022. They are recruiting 50 participants from 11 locations in total."

Answered by AI

Recent research and studies

Molecular Therapy - Methods & Clinical DevelopmentJournal

Site-directed Mutagenesis Improves the Transduction Efficiency of Capsid Library-derived Recombinant AAV Vectors

Ran, Gai, Xiao Chen, Yilin Xie, Qingyun Zheng, Jinyan Xie, Chenghui Yu, Nikea Pittman, et al.. 2020. “Site-directed Mutagenesis Improves the Transduction Efficiency of Capsid Library-derived Recombinant AAV Vectors”. Molecular Therapy - Methods & Clinical Development. Elsevier BV. doi:10.1016/j.omtm.2020.03.007.

New England Journal of MedicineJournal

Multiyear Factor VIII Expression After AAV Gene Transfer for Hemophilia A

George, Lindsey A., Paul E. Monahan, M. Elaine Eyster, Spencer K. Sullivan, Margaret V. Ragni, Stacy E. Croteau, John E.J. Rasko, et al.. 2021. “Multiyear Factor VIII Expression After AAV Gene Transfer for Hemophilia A”. New England Journal of Medicine. Massachusetts Medical Society. doi:10.1056/nejmoa2104205.

clinicaltrials.govStudy

A Gene Transfer Study for Hemophilia A

2017. "A Gene Transfer Study for Hemophilia A". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03003533.