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

NCT03734588 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 4

Last updated 2024-02-23

Study results available
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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.

Conditions

  • Adeno-Associated Virus (AAV)
  • Blood Coagulation Disorder
  • Blood Coagulation Disorders, Inherited
  • Coagulation Protein Disorders
  • Factor VIII (FVIII)
  • Factor VIII (FVIII) Deficiency
  • Factor VIII (FVIII) Gene
  • Factor VIII (FVIII) Protein
  • Genetic Diseases, Inborn
  • Genetic Diseases, X-Linked
  • Gene Therapy
  • Gene Transfer
  • Hematologic Diseases
  • Hemorrhagic Disorders
  • Recombinant
  • Vector
  • Inhibitors

Interventions

GENETIC

SPK-8016

adeno-associated viral vector

Sponsors & Collaborators

  • Spark Therapeutics, Inc.

    lead INDUSTRY

Principal Investigators

  • Tiffany Chang, MD · Spark Therapeutics, Inc.

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-01-30
Primary Completion
2020-10-14
Completion
2023-01-19
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03734588 on ClinicalTrials.gov