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RESOLUTESM is a Phase 1/2 Investigational Gene Transfer Study for Adults with Late-Onset Pompe Disease (LOPD)

UPDATE: Trial Enrollment Resumed August 2020

After careful review of the emerging epidemiological data and ongoing mitigation measures to prevent the spread of COVID-19 (SARS-CoV-2), and in consultation with the RESOLUTE Steering Committee, Spark Therapeutics restarted enrollment into RESOLUTE in August 2020.

Safety Measures Implemented

The following safety measures will be implemented into RESOLUTE to help minimize the risk of COVID-19 (SARS-CoV-2) exposure to patients and their caregivers:


All patients and their caregivers must follow risk mitigation guidelines: face masks, social distancing, hand washing, etc.


Screening all patients for COVID-19 (SARS-CoV-2) active virus infection during the screening period of the study, prior to investigational product candidate SPK-3006 infusion, and after receiving SPK-3006 infusion.


Additional safety measures may be initiated, as per the judgment of the Investigator, per institutional guideline and in consultation with the Sponsor Medical Monitor.

Decision Details: On 16 March 2020, Spark Therapeutics voluntarily suspended enrollment into RESOLUTE out of an abundance of caution for the health and safety of patients with Pompe Disease, who may have compromised respiratory function, to minimize their risk of exposure to COVID-19 (SARS-CoV-2), as a result of traveling to the investigational sites.

This decision was based on:
The World Health Organization (WHO) declaration of a global pandemic on 11 March 2020, in response to the COVID-19 (SARS-CoV-2) outbreak.
The CDC guidelines, which consider patients with serious chronic medical conditions, such as lung disease (e.g. Pompe disease), an at-risk population for more severe cases of COVID-19 (SARS-CoV-2).

RESOLUTE at a Glance

Late-onset Pompe disease (current)
20 out of 20 (100%)
October 2020
October 2023
NCT04093349 ( | SPK-3006-101 | 2019-001283-30 (EudraCT Number)

What is Pompe Disease?

Pompe disease is a lysosomal storage disorder and neuromuscular disease, with systemic, multi-organ manifestations resulting from mutations in the gene encoding acid alpha-glucosidase (GAA). The disease is oftentimes life-limiting and can be fatal. Mutations in the gene prevent the GAA enzyme from breaking down the energy storage molecule, glycogen, effectively, which allows it to build up to toxic levels in lysosomes, common structures known as organelles found in cells in the body. This can damage organs and tissues throughout the body, particularly in muscles, causing Pompe disease.


RESOLUTE is a clinical study of an investigational gene therapy (also referred to as gene transfer) called SPK-3006. It is being studied as a potential treatment for adults living with LOPD. Different doses of SPK-3006 are being studied with the goal of finding which dose, if any, may be safe and effective. Each participant will receive one of the doses, one time.

Questions? Contact us at

Is this study right for me?

Age: 18+ | Genders: Male and Female

Accepts Healthy Volunteers: No

Entry Criteria
The RESOLUTE clinical trial is recruiting men and women who have LOPD. In order to be considered for the trial, participants must:

  • have clinical symptoms of LOPD affecting daily living and have received Enzyme Replacement Therapy (ERT) for at least the past 24 months (2 years)
  • agree to use reliable birth control
  • not have any liver disease
  • have not recieved any gene therapy before

This is not a complete list of requirements to be considered for participation in the RESOLUTE clinical trial. For more details, please speak with your physician, visit, or contact us at

What should I expect if I participate in this clinical study?

Participating in this clinical study requires a variety of tests and you will be monitored throughout the study. Through these medical tests, the research team will learn how your body responds to the investigational gene therapy, the safety of the gene therapy, and how well the gene therapy may be working in your body.

There are 3 defined periods in the study:

  1. Screening period: when participant eligibility is determined
  2. Dosing day: a one-time IV infusion of investigational SPK-3006
  3. Follow-up: physical examination and functional testing is conducted for about 52 weeks after receiving investigational SPK-3006

Due to COVID-19 (SARS-CoV-2), the schedule of assessments currently included in the study protocol has been carefully evaluated to minimize visits to study sites. Whenever possible, certain study assessments take place in the participant’s home to reduce the burden of participation in the study.

Long term follow up in gene therapy clinical studies:

While the first phase of a gene therapy study may last a year or so, gene therapy studies often involve long-term follow-up over several years. Long-term follow-up is critical to understanding the potential benefits and risks of investigational gene therapy over time. Given that gene therapy research is relatively new, there is still much to learn about the long-term effects of investigational gene therapy, including how long a treatment may be considered effective and if there are any side effects that occur later on. Long-term follow up of clinical study participants provides an important pathway to that understanding. When deciding if participation in an investigational gene therapy clinical study is right for you, the expected long-term follow-up will be important for you to consider.

Where are the study locations?

Clinical study sites are active and enrolling. If you are interested in learning more, and to view current site locations, please visit the RESOLUTE study listing on

What is investigational gene therapy?

Gene therapy is a potential approach to treating or preventing genetic diseases. The goal of gene therapy is to address a genetic disease at its source—the gene. This can be done by modifying (changing) genes or creating new functional genes in a laboratory and delivering them to specific cells in the body.

Scientists have been investigating gene therapy for more than 50 years, and the science around gene therapy continues to evolve. To date, more than 2,600 gene therapy clinical trials are planned, ongoing, or have been completed for different genetic diseases.

Gene therapy is an approach being investigated for use in Pompe disease. This approach introduces a new functional copy of a gene with the aim of restoring or enhancing its original function. It is sometimes referred to as gene augmentation, gene transfer, or gene replacement. View the video to learn more about one approach to gene therapy for Pompe disease.