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

  • End date
    Oct 25, 2023
  • participants needed
  • sponsor
    Spark Therapeutics
Updated on 16 January 2022
replacement therapy
gene therapy


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.

Condition LOPD, Pompe Disease, Glycogen storage disease type II, Lysosomal Storage Disease
Treatment SPK-3006
Clinical Study IdentifierNCT04093349
SponsorSpark Therapeutics
Last Modified on16 January 2022


Yes No Not Sure

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
Clear my responses

How to participate?

Step 1 Connect with a study center
What happens next?
  • You can expect the study team to contact you via email or phone in the next few days.
  • Sign up as volunteer to help accelerate the development of new treatments and to get notified about similar trials.

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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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