A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)
Public ClinicalTrials.gov record NCT04093349. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
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
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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 identification
- NCT ID
- NCT04093349
- Recruitment status
- Active, not recruiting
- Study type
- Interventional
- Phase
- Phase 1, Phase 2
- Enrollment
- 4 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 18 Years and older
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Oct 1, 2020 (Actual)
- Primary completion
- Apr 2032 (Estimated)
- Completion
- Apr 2032 (Estimated)
- Last update posted
- Nov 27, 2024
2020 – 2032
United States locations
- U.S. sites
- 10
- U.S. states
- 9
- U.S. cities
- 10
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Barrow Neurological Institute | Phoenix | Arizona | 85013 | — |
| University of California Irvine Health | Orange | California | 92868 | — |
| Emory University School of Medicine | Atlanta | Georgia | 30329 | — |
| University of Kansas Medical Center Research Institute | Kansas City | Kansas | 66160 | — |
| University of Minnesota | Minneapolis | Minnesota | 55455 | — |
| Oregon Health & Science University | Portland | Oregon | 97239 | — |
| University of Pennsylvania | Philadelphia | Pennsylvania | 19104 | — |
| University of Pittsburgh Medical Center | Pittsburgh | Pennsylvania | 15213 | — |
| University of Utah | Salt Lake City | Utah | 84108 | — |
| Lysosomal and Rare Disorders Research & Treatment Center | Fairfax | Virginia | 22030 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 19 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT04093349, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Nov 27, 2024 · Synced Oct 27, 2026
Related: full search, browse by condition, browse by drug or therapy, browse by sponsor, browse by U.S. city.
Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT04093349 live on ClinicalTrials.gov.