Pompe DiseasePhase 1/2NCT04093349
A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)
A Phase 1/2 study testing SPK-3006 in people with Pompe Disease and Pompe Disease (Late-onset), run by Spark Therapeutics, Inc. at 29 sites in 8 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Pompe Disease, Pompe Disease (Late-onset), Glycogen Storage Disease Type 2
- What is being tested?
- SPK-3006
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 29 sitesin 8 countries
- Study dates
- 1 Oct 2020to Apr 2032
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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;
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- 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.
Trial locations and contacts
29 sites in 8 countries.
29 sites in 8 countries
United States10 sites
Emory University School of Medicine
Atlanta, Georgia, 30329
Lysosomal and Rare Disorders Research & Treatment Center
Fairfax, Virginia, 22030
University of Kansas Medical Center Research Institute
Kansas City, Kansas, 66160
University of Minnesota
Minneapolis, Minnesota, 55455
University of California Irvine Health
Orange, California, 92868
University of Pennsylvania
Philadelphia, Pennsylvania, 19104
Barrow Neurological Institute
Phoenix, Arizona, 85013
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15213
Oregon Health & Science University
Portland, Oregon, 97239
University of Utah
Salt Lake City, Utah, 84108
France5 sites
Centre Hospitalier Universitaire d'Angers
Angers, 49933
CHU Paris IdF Ouest - Hôpital Raymond Poincaré
Garches, 92380
Centre Hospitalier Régional Universitaire de Lille
Lille, 59037
Assistance Publique Hôpitaux de Marseille
Marseille, 13 13385
Nice University Hospital
Nice, 6000
Italy5 sites
Universita Degli Studi Di Messina - Dipartimento di Medicina Clinica e Sperimentale
Messina, 98122
Universita Degli Studi Di Milano, Laboratorio di Biochimica e Genetica della Malattie Neuromuscolari
Milan, 35 20122
Malattie Metaboliche Universita Degli Studi Di Napoli Federico II
Naples, 80138
UO Neurologia Azienda Ospedaliera Universitaria Pisana
Pisa, 56126
Azienda Ospedaliera Universitaria Città della Salute e della Scienza di Torino - Neurology, Osp. Molinette
Torino, 10126
Canada3 sites
Montreal Neurological Hospital
Montreal, Quebec, H3A 2B4
The Ottawa Hospital
Ottawa, Ontario, K1Y 4E9
Vancouver General Hospital
Vancouver, British Columbia, V5Z 1M9
United Kingdom3 sites
New Queen Elizabeth Hospital Birmingham
Birmingham, GBR, B15 2WB
The Royal Free London NHS Foundation Trust
London, GBR, NW3 2QG
Salford Royal MHS Foundation Trust
Salford, M6 8HD
Germany1 site
Friedrich-Baur-Institut Neurologische Klinik Ludwig-Maximilians-Universität München
München, D08033
About this study
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
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.
- Conditions
- Pompe Disease, Pompe Disease (Late-onset), Glycogen Storage Disease Type 2, Glycogen Storage Disease Type II, LOPD, Lysosomal Storage Diseases
- Being tested
- SPK-3006
- Medical subject headings
- Glycogen Storage Disease Type II, Lysosomal Storage Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SPK-3006Genetic
adeno-associated viral (AAV) vector
| Study group | Role | What they receive |
|---|---|---|
| SPK-3006 | Experimental | All participants who meet the eligibility criteria will receive a single intravenous (i.v.) administration of SPK-3006. Receives: Genetic: SPK-3006 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of adverse and serious adverse events (AEs/SAEs), including clinically significant abnormal laboratory values.
Adverse events.
Measured: Up to 5 years
Occurrence of immune response against AAV capsid
Measured: Up to 5 years
Occurrence of immune response against GAA transgene
Measured: Up to 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Sep 2019
- Study start1 Oct 2020
- Primary completionApr 2032 (estimated)
- Study completionApr 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pompe DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- Spark Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Tahseen Mozaffar, MD, Principal investigator, University of California Irvine Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04093349
- Sponsor's protocol ID
- SPK-3006-101
- Other IDs
- 2019-001283-30, 2019
- Status last verified
- Nov 2024
- First submitted
- 16 Sep 2019
- Last updated
- 27 Nov 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Nov 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT04093349
- Acronym / sponsor ID
- SPK-3006-101
- Conditions
- Pompe Disease, Pompe Disease (Late-onset), Glycogen Storage Disease Type 2, Glycogen Storage Disease Type II, LOPD, Lysosomal Storage Diseases
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 4 participants (actual)
- Lead sponsor
- Spark Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 1 Oct 2020 (actual)
- Primary completion
- Apr 2032 (estimated)
- Study completion
- Apr 2032 (estimated)
- First posted
- 18 Sep 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| SPK-3006 | Experimental | Genetic: SPK-3006All participants who meet the eligibility criteria will receive a single intravenous (i.v.) administration of SPK-3006. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (12 listed)
- 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;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SPK-3006adeno-associated viral (AAV) vector | Genetic | SPK-3006 |
Primary outcome
Number of adverse and serious adverse events (AEs/SAEs), including clinically significant abnormal laboratory values.
Time frame: Up to 5 years
Adverse events.
Occurrence of immune response against AAV capsid
Time frame: Up to 5 years
Occurrence of immune response against GAA transgene
Time frame: Up to 5 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Nov 2024.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Tahseen Mozaffar, MD principal investigator
University of California Irvine Health
29 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing SPK-3006 in people with Pompe Disease and Pompe Disease (Late-onset), run by Spark Therapeutics, Inc. at 29 sites in 8 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 27 Nov 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 29 sites in United States, France, Italy, Canada, United Kingdom and 3 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of adverse and serious adverse events (AEs/SAEs), including clinically significant abnormal laboratory values. (time frame: Up to 5 years). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Nov 2024.