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COMPLETED Phase 1

Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors

NCT03734588 · View on ClinicalTrials.gov ↗

Study Summary

SPK-8016 is in development for the treatment of patients with inhibitors to FVIII. This Phase 1/2, open-label, non-randomized, dose-finding study to evaluate the safety, efficacy, and tolerability of SPK-8016 in adult males with severe hemophilia A and no measurable inhibitor against FVIII.

Interventions

  • GENETIC SPK-8016

Study Locations (11)

Pennsylvania

  • Penn State Health — Hershey
  • Children's Hospital of Philadelphia — Philadelphia
  • Jefferson University Hospitals — Philadelphia
  • Hemophilia Center of Western Pennsylvania — Pittsburgh

California

  • Orthopaedic Institute for Children — Los Angeles

Illinois

  • Illinois Bleeding and Clotting Disorders Institute — Peoria

Michigan

  • University of Michigan — Ann Arbor

Mississippi

  • Mississippi Center for Advanced Medicine — Madison

New York

  • Weill Cornell Medicine — New York

Oregon

  • Oregon Health & Science University — Portland

Virginia

  • Virginia Commonwealth University School of Medicine — Richmond

Trial Details

FieldValue
Enrollment Target 4 participants
Start Date 2019-01-30
Est. Completion 2023-01-19
Phase Phase 1

Sponsor

Spark Therapeutics

6 total trials

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Full Details on ClinicalTrials.gov ↗

What the Registry Record Tells You About NCT03734588

The ClinicalTrials.gov registry entry for NCT03734588 describes a study currently listed as completed. It is categorized as Phase 1, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 4 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Spark Therapeutics, which has 6 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.

The record links to 10 conditions, with Genetic Diseases, Inborn appearing as the primary indexed condition, and to 1 intervention — of which SPK-8016 is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.

Geographic footprint matters for practical reasons: NCT03734588 reports 11 study locations spanning 8 distinct geographic areas — top geographies include Pennsylvania, California, Illinois. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.

Frequently Asked Questions

What is clinical trial NCT03734588 about?

NCT03734588 is a clinical study titled "Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors". SPK-8016 is in development for the treatment of patients with inhibitors to FVIII. This Phase 1/2, open-label, non-randomized, dose-finding study to evaluate the safety, efficacy, and tolerability of SPK-8016 in adult males with severe hemophilia A and no measurable inhibitor against FVIII.

What is the current status of trial NCT03734588?

This trial is currently completed. It is a Phase 1 study. The enrollment target is 4 participants. The study started on 2019-01-30. Estimated completion is 2023-01-19.

What conditions does trial NCT03734588 study?

This clinical trial studies the following conditions: Genetic Diseases, Inborn, Blood Coagulation Disorder, Genetic Diseases, X-Linked, Blood Coagulation Disorders, Inherited, Factor VIII (FVIII). These conditions were identified from the trial registry and reflect the primary focus areas of the research.

What interventions are being tested in trial NCT03734588?

The interventions under investigation include: SPK-8016 (GENETIC). Each intervention is being evaluated for safety and efficacy as part of this clinical study.

Who is sponsoring clinical trial NCT03734588?

This trial is sponsored by Spark Therapeutics, which has 6 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.

Where is trial NCT03734588 being conducted?

This trial has 11 study locations across California, Illinois, Michigan, Mississippi, New York. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.

Related

Data sourced from official U.S. government datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial