A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)

November 25, 2024 updated by: Spark Therapeutics, Inc.

Phase 1/2, Dose-escalation Study to Evaluate the Safety, Tolerability and Efficacy of a Single Intravenous Infusion of SPK-3006 in Adults With Late-onset Pompe Disease

The purpose of this study is to evaluate the safety, tolerability, and efficacy of a single intravenous infusion of SPK-3006 in adults with clinically moderate, late-onset Pompe disease receiving enzyme replacement therapy (ERT). Participants will be treated in sequential, dose-level cohorts.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Active, not recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
4

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Canada
    • British Columbia
      • Vancouver, British Columbia, Canada, V5Z 1M9
        • Vancouver General Hospital
    • Ontario
      • Ottawa, Ontario, Canada, K1Y 4E9
        • The Ottawa Hospital
    • Quebec
      • Montréal, Quebec, Canada, H3A 2B4
        • Montreal Neurological Hospital
  • Denmark
      • Copenhagen, Denmark, 84 2100
        • Rigshospitalet
  • France
      • Angers, France, 49933
        • Centre Hospitalier Universitaire d'Angers
      • Garches, France, 92380
        • CHU Paris IdF Ouest - Hôpital Raymond Poincaré
      • Lille Cedex, France, 59037
        • Centre Hospitalier Regional Universitaire De Lille
      • Marseille, France, 13 13385
        • Assistance Publique Hôpitaux de Marseille
      • Nice, France, 6000
        • Nice University Hospital
  • Germany
      • Munchen, Germany, D08033
        • Friedrich-Baur-Institut Neurologische Klinik Ludwig-Maximilians-Universität München
  • Italy
      • Messina, Italy, 98122
        • Universita Degli Studi Di Messina - Dipartimento di Medicina Clinica e Sperimentale
      • Milano, Italy, 35 20122
        • Universita Degli Studi Di Milano, Laboratorio di Biochimica e Genetica della Malattie Neuromuscolari
      • Naples, Italy, 80138
        • Malattie Metaboliche Universita Degli Studi Di Napoli Federico II
      • Pisa, Italy, 56126
        • UO Neurologia Azienda Ospedaliera Universitaria Pisana
      • Torino, Italy, 10126
        • Azienda Ospedaliera Universitaria Città della Salute e della Scienza di Torino - Neurology, Osp. Molinette
  • Netherlands
      • Rotterdam, Netherlands, 3015 CE
        • Erasmus Medical Center
  • United Kingdom
      • Salford, United Kingdom, M6 8HD
        • Salford Royal MHS Foundation Trust
    • GBR
      • Birmingham, GBR, United Kingdom, B15 2WB
        • New Queen Elizabeth Hospital Birmingham
      • London, GBR, United Kingdom, NW3 2QG
        • The Royal Free London NHS Foundation Trust
  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85013
        • Barrow Neurological Institute
    • California
      • Orange, California, United States, 92868
        • University of California Irvine Health
    • Georgia
      • Atlanta, Georgia, United States, 30329
        • Emory University School of Medicine
    • Kansas
      • Kansas City, Kansas, United States, 66160
        • University of Kansas Medical Center Research Institute
    • Minnesota
      • Minneapolis, Minnesota, United States, 55455
        • University of Minnesota
    • Oregon
      • Portland, Oregon, United States, 97239
        • Oregon Health & Science University
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • UNIVERSITY of PENNSYLVANIA
      • Pittsburgh, Pennsylvania, United States, 15213
        • University of Pittsburgh Medical Center
    • Utah
      • Salt Lake City, Utah, United States, 84108
        • University of Utah
    • Virginia
      • Fairfax, Virginia, United States, 22030
        • Lysosomal and Rare Disorders Research & Treatment Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Provide written informed consent;
  • Males and Females ≥18 years of age with late-onset Pompe disease;
  • Received ERT for at least the previous 24 months
  • Have clinically moderate, late-onset Pompe disease characteristics;
  • Agree to use reliable contraception.

Exclusion Criteria:

  • Active hepatitis B and/or C;
  • Significant underlying liver disease;
  • Human immunodeficiency virus (HIV) infection;
  • Prior hypersensitivity to rhGAA;
  • Pre-existing anti-AAV neutralizing antibody titers;
  • High titer antibody responses to rhGAA;
  • Requires any invasive ventilation or requires noninvasive ventilation while awake and upright;
  • Received any prior vector or gene transfer agent;
  • Active malignancy (except non-melanoma skin cancer);
  • History of liver cancer;
  • Pregnant or nursing women;
  • Any evidence of active infection at the time of SPK-3006 infusion.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: SPK-3006
All participants who meet the eligibility criteria will receive a single intravenous (i.v.) administration of SPK-3006.
Genetic: SPK-3006
adeno-associated viral (AAV) vector

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Number of adverse and serious adverse events (AEs/SAEs), including clinically significant abnormal laboratory values.
Time Frame: Up to 5 years
Adverse events.
Up to 5 years
Occurrence of immune response against AAV capsid
Time Frame: Up to 5 years
Up to 5 years
Occurrence of immune response against GAA transgene
Time Frame: Up to 5 years
Up to 5 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Spark Therapeutics, Inc.

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Principal Investigator: Tahseen Mozaffar, MD, University of California Irvine Health

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
October 1, 2020

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
April 1, 2032

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
April 1, 2032

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
September 16, 2019

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
September 16, 2019

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
September 18, 2019

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
November 27, 2024

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
November 25, 2024

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
November 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • SPK-3006-101
  • 2019-001283-30 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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