Pompe DiseasePhase 1/2NCT02898753
VAL-1221 Delivered Intravenously in Ambulatory and Ventilator-free Participants With Late-Onset Pompe Disease
A Phase 1/2 study testing VAL-1221 and RhGAA in people with Pompe Disease, run by Valerion Therapeutics, LLC at 3 sites in 2 countries.
Trial summary
This Phase I/II open-label, randomized, dose-escalation study will assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of VAL-1221 versus Myozyme®/Lumizyme® in participants with late-onset glycogen storage disease-II (GSD-II) (Pompe disease) Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Pompe Disease
- What is being tested?
- VAL-1221 and RhGAA
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 3 sitesin 2 countries
- Study dates
- 21 Jun 2017to 25 Mar 2020
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
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
- Participant is able and willing to provide informed consent prior to any study procedures are performed
- Diagnosis of GSDII based on one of the following:
- Endogenous cultured skin fibroblast GAA activity less than (<) 40 percent (%) of adult normal level
- Endogenous whole blood or dried blood spot GAA activity in deficiency range
- Genetic analysis showing pathogenic variants in both alleles
- Onset of Pompe disease-related symptoms after 1 year of age
Show all 12Show fewer
- Previously treated with Myozyme or Lumizyme for at least 12 months and on a stable regimen for the past 6 months
- Sexually active participants who are willing to use an acceptable method of contraception (abstinence, oral contraceptives, barrier method with spermicide, surgical sterilization, implanted or injectable contraceptives with a stable dose for at least 1 month prior to Baseline, hormonal intra-uterine device [IUD] inserted at least 1 month prior to Baseline) during the study and for 30 days after completion of treatment
- If participant is female and not considered to be of childbearing potential, she is at least 2 years post-menopause, has undergone a tubal ligation, a total hysterectomy or bilateral oophorectomy
- If participant is female and of childbearing potential, she has a negative serum pregnancy test during screening and Baseline and must be willing to undergo pregnancy testing at specific intervals during the study
- Participant meets at least one of the following criteria: greater than (>) 30% and <80% predicted upright forced volume capacity (FVC) or participant is able to walk >20% but <80% predicted normal on 6-minute walk test with or without use of assistive devices
- Able to comply with protocol requirements
Who cannot join
- Cardiac involvement in first year of life
- Anti-GAA antibody titers >1:51,200 at two time points
- Prior use of chaperone therapy for GSD-II within the last 12 months
- Use of immunosuppressive medication other than glucocorticoids within 6 months prior to study enrollment
- Use of invasive ventilatory assistance other than Bilevel Positive Airway Pressure (BiPAP) at night or during periods of rest
- Has received any investigational medication or has enrolled in any study involving investigational drugs or therapies within 30 days prior to first dose of study drug
Show all 11Show fewer
- Start of or change in usual regimen of albuterol or respiratory muscle training within 30 days prior to first dose of study drug
- History of sensitivity to any of the constituents of the study drug
- Participant is breastfeeding or planning to become pregnant or to breastfeed during the study or is currently breastfeeding
- Participant has a medical condition or circumstance that, in the opinion of the investigator, might compromise the participant's ability to comply with the protocol or the participant's well-being or safety
- Participant has any condition that, in the view of the investigator, places the participant at high risk of poor treatment compliance or of not completing the study
Trial locations and contacts
3 sites in 2 countries.
3 sites in 2 countries
United States2 sites
Duke University Medical Center
Durham, North Carolina, 27710
University of California, Irvine
Orange, California, 92868
United Kingdom1 site
National Hospital for Neurology and Neurosurgery
London, WC1N 3BG
About this study
Official title: A Three-Month, Open-Label, Randomized, Dose-escalation Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of VAL-1221 Versus Myozyme®/Lumizyme® in Patients With Late-Onset GSD-II (Pompe Disease) Followed by Open-Label Treatment With VAL-1221 in All Patients
This Phase I/II open-label, randomized, dose-escalation study will assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of VAL-1221 versus Myozyme®/Lumizyme® in participants with late-onset glycogen storage disease-II (GSD-II) (Pompe disease)
Read the detailed description
Part 1 comprises 3 sequential cohorts of 4 patients each randomized to treatment with either VAL-1221 (at 3, 10, or 30 mg/kg) or positive control (rhGAA). Patients randomized to VAL-1221 will receive 7 intravenous (IV) infusions of VAL-1221 (one infusion every other week) over 12 weeks. Control patients will continue receiving their accustomed dose and regimen of Myozyme®. Part 2 is an uncontrolled extension to evaluate long-term effects of VAL-1221 given by IV infusion once every other week at doses up to 40 mg/kg.
- Conditions
- Pompe Disease
- Being tested
- VAL-1221, RhGAA
- Keywords
- GSD-II, Pompe Disease, Glycogen Storage Diseases, GAA
- Medical subject headings
- Glycogen Storage Disease Type II, Glycogen Storage Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VAL-1221Drug
VAL-1221 3, 10, or 30 mg/kg as per the dose and schedule specified in the arm group description
RhGAADrug
Also called Myozyme, Lumizyme
Active comparator
| Study group | Role | What they receive |
|---|---|---|
| VAL-1221 3 mg/kg | Experimental | Part 1: Participants will receive VAL-1221 3 mg/kg IV infusion every other week for 12 weeks, inclusive, for a total of 7 infusions.
Part 2: Participants from Part 1 of the study who were randomized to VAL-1221 3 mg/kg can enter Part 2 of the study and receive VAL-1221 at a dose of 3 mg/kg IV infusion every other week. The dose can be increased by the Investigator to 10 mg/kg and further to 30 mg/kg (after at least 12 weeks of dosing at 10 mg/kg), depending upon the pharmacodynamics, efficacy, and safety data. Receives: Drug: VAL-1221 |
| VAL-1221 10 mg/kg | Experimental | Part 1: Participants will receive VAL-1221 10 mg/kg IV infusion every other week for 12 weeks, inclusive, for a total of 7 infusions.
Part 2: Participants from Part 1 of the study who were randomized to VAL-1221 10 mg/kg can enter Part 2 of the study and receive VAL-1221 at a dose of 10 mg/kg IV infusion every other week. The dose can be increased by the Investigator to 30 mg/kg IV infusion, depending upon the pharmacodynamics, efficacy, and safety data. Receives: Drug: VAL-1221 |
| VAL-1221 30 mg/kg | Experimental | Part 1: Participants will receive VAL-1221 30 mg/kg IV every other week for 12 weeks, inclusive, for a total of 7 infusions.
Part 2: Participants from Part 1 of the study who were randomized to VAL-1221 30 mg/kg can enter Part 2 of the study and receive VAL-1221 at a dose of 30 mg/kg IV inufsion every other week. Receives: Drug: VAL-1221 |
| rhGAA | Active comparator | Part 1: Participants will be maintained on their current dose and regimen of Myozyme or Lumizyme.
Part 2: Participants from Part 1 of the study who were randomized to rhGAA can enter Part 2 of the study and receive VAL-1221 either 3 mg/kg, 10 mg/kg, or 30 mg/kg (based on the dose of VAL-1221 in respective cohorts to which they were randomized in Part 1) IV infusion every other week. Receives: Drug: VAL-1221; Drug: RhGAA |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Treatment-related Treatment-emergent Adverse Events (TEAEs)
Measured: Baseline of Study Part 1 though Month 24 of Study Part 2
Number of Participants With Infusion-Associated Reactions to VAL-1221
Measured: Baseline of Study Part 1 though Month 24 of Study Part 2
Percentage of Participants with Anti-VAL-1221 Antibodies
Measured: Baseline of Study Part 1 though Month 24 of Study Part 2
Percentage of Participants with GAA Antibodies
Measured: Baseline of Study Part 1 though Month 24 of Study Part 2
Secondary outcomes (11)
Pharmacokinetics of VAL-1221 in Plasma: Maximum Observed Concentration (Cmax)
Measured: Part 1: Pre-infusion, end of infusion (EOI) (infusion duration 1 mg/kg/hour), at 0.25, 0.5, 1, 2, 4, 12, 24, 48 and 336 hours after EOI on Days 1, 2, 3 and Week 12; Part 2: Pre-infusion on Weeks 12, 14, 16, 18, 20, 22, Months 12, 24, 36
Pharmacokinetics of VAL-1221 in Plasma: Time to Reach Maximum Concentration (Tmax)
Measured: Part 1: Pre-infusion, end of infusion (EOI) (infusion duration 1 mg/kg/hour), at 0.25, 0.5, 1, 2, 4, 12, 24, 48 and 336 hours after EOI on Days 1, 2, 3 and Week 12; Part 2: Pre-infusion on Weeks 12, 14, 16, 18, 20, 22, Months 12, 24, 36
Pharmacokinetics of VAL-1221 in Plasma: Area Under the Concentration Time Curve (AUC)
Measured: Part 1: Pre-infusion, end of infusion (EOI) (infusion duration 1 mg/kg/hour), at 0.25, 0.5, 1, 2, 4, 12, 24, 48 and 336 hours after EOI on Days 1, 2, 3 and Week 12; Part 2: Pre-infusion on Weeks 12, 14, 16, 18, 20, 22, Months 12, 24, 36
Pharmacokinetics of VAL-1221 in Plasma: Terminal Elimination Half-Life ( t1/2)
Measured: Part 1: Pre-infusion, end of infusion (EOI) (infusion duration 1 mg/kg/hour), at 0.25, 0.5, 1, 2, 4, 12, 24, 48 and 336 hours after EOI on Days 1, 2, 3 and Week 12; Part 2: Pre-infusion on Weeks 12, 14, 16, 18, 20, 22, Months 12, 24, 36
Pharmacokinetics of VAL-1221 in Plasma: Apparent Total Body Clearance (CL)
Measured: Part 1: Pre-infusion, end of infusion (EOI) (infusion duration 1 mg/kg/hour), at 0.25, 0.5, 1, 2, 4, 12, 24, 48 and 336 hours after EOI on Days 1, 2, 3 and Week 12; Part 2: Pre-infusion on Weeks 12, 14, 16, 18, 20, 22, Months 12, 24, 36
Pharmacokinetics of VAL-1221 in Plasma: Apparent Volume (V)
Measured: Part 1: Pre-infusion, end of infusion (EOI) (infusion duration 1 mg/kg/hour), at 0.25, 0.5, 1, 2, 4, 12, 24, 48 and 336 hours after EOI on Days 1, 2, 3 and Week 12; Part 2: Pre-infusion on Weeks 12, 14, 16, 18, 20, 22, Months 12, 24, 36
Change from Baseline in Urinary Hexose Tetrasaccharide (hex4) Excretion at Week 12, Months 6, 9, and 12
Measured: Baseline of Study Part 1, Week 12 of Study Part 1, Months 12, 24, and 36 of Study Part 2
Change from Baseline in Serum Creatine Kinase (CK) at Week 12, Months 6, 9, and 12
Measured: Baseline of Study Part 1, Week 12 of Study Part 1, Months 12, 24, and 36 of Study Part 2
Change from Baseline in the Amount of Acid Alpha Glucosidase (GAA) Activity Present in Muscle at Week 12
Measured: Baseline of Study Part 1, Week 12 of Study Part 1
Change from Baseline in the Muscle Glycogen Content at Week 12
Measured: Baseline of Study Part 1, Week 12 Study Part 1
Change from Baseline in Creatinine Excretion at Months 6, 9, and 12
Measured: Baseline of Study Part 1, Months 12, 24, 36 of Study Part 2
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Sep 2016 (estimated)
- Study start21 Jun 2017
- Primary completion25 Mar 2020
- Study completion25 Mar 2020
Why it stopped: Company closure due to lack of funding
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
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Valerion Therapeutics, LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Hal Landy, MD, Study director, Valerion Therapeutics, LLC
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02898753
- Sponsor's protocol ID
- VAL1221-201-16
- Status last verified
- May 2020
- First submitted
- 8 Sep 2016
- Last updated
- 2 Jun 2020
- 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 2 Jun 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Three-Month, Open-Label, Randomized, Dose-escalation Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of VAL-1221 Versus Myozyme®/Lumizyme® in Patients With Late-Onset GSD-II (Pompe Disease) Followed by Open-Label Treatment With VAL-1221 in All Patients
- ClinicalTrials.gov ID
- NCT02898753
- Acronym / sponsor ID
- VAL1221-201-16
- Conditions
- Pompe Disease
- Keywords
- GSD-II, Pompe Disease, Glycogen Storage Diseases, GAA
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Company closure due to lack of funding
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Valerion Therapeutics, LLCIndustry (pharma, biotech, devices)
- Study start
- 21 Jun 2017 (actual)
- Primary completion
- 25 Mar 2020 (actual)
- Study completion
- 25 Mar 2020 (actual)
- First posted
- 13 Sep 2016 (estimated)
- 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.
This Phase I/II open-label, randomized, dose-escalation study will assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of VAL-1221 versus Myozyme®/Lumizyme® in participants with late-onset glycogen storage disease-II (GSD-II) (Pompe disease)
Detailed description
Part 1 comprises 3 sequential cohorts of 4 patients each randomized to treatment with either VAL-1221 (at 3, 10, or 30 mg/kg) or positive control (rhGAA). Patients randomized to VAL-1221 will receive 7 intravenous (IV) infusions of VAL-1221 (one infusion every other week) over 12 weeks. Control patients will continue receiving their accustomed dose and regimen of Myozyme®. Part 2 is an uncontrolled extension to evaluate long-term effects of VAL-1221 given by IV infusion once every other week at doses up to 40 mg/kg.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| VAL-1221 3 mg/kg | Experimental | Drug: VAL-1221Part 1: Participants will receive VAL-1221 3 mg/kg IV infusion every other week for 12 weeks, inclusive, for a total of 7 infusions. Part 2: Participants from Part 1 of the study who were randomized to VAL-1221 3 mg/kg can enter Part 2 of the study and receive VAL-1221 at a dose of 3 mg/kg IV infusion every other week. The dose can be increased by the Investigator to 10 mg/kg and further to 30 mg/kg (after at least 12 weeks of dosing at 10 mg/kg), depending upon the pharmacodynamics, efficacy, and safety data. |
| VAL-1221 10 mg/kg | Experimental | Drug: VAL-1221Part 1: Participants will receive VAL-1221 10 mg/kg IV infusion every other week for 12 weeks, inclusive, for a total of 7 infusions. Part 2: Participants from Part 1 of the study who were randomized to VAL-1221 10 mg/kg can enter Part 2 of the study and receive VAL-1221 at a dose of 10 mg/kg IV infusion every other week. The dose can be increased by the Investigator to 30 mg/kg IV infusion, depending upon the pharmacodynamics, efficacy, and safety data. |
| VAL-1221 30 mg/kg | Experimental | Drug: VAL-1221Part 1: Participants will receive VAL-1221 30 mg/kg IV every other week for 12 weeks, inclusive, for a total of 7 infusions. Part 2: Participants from Part 1 of the study who were randomized to VAL-1221 30 mg/kg can enter Part 2 of the study and receive VAL-1221 at a dose of 30 mg/kg IV inufsion every other week. |
| rhGAA | Active comparator | Drug: VAL-1221, Drug: RhGAAPart 1: Participants will be maintained on their current dose and regimen of Myozyme or Lumizyme. Part 2: Participants from Part 1 of the study who were randomized to rhGAA can enter Part 2 of the study and receive VAL-1221 either 3 mg/kg, 10 mg/kg, or 30 mg/kg (based on the dose of VAL-1221 in respective cohorts to which they were randomized in Part 1) IV infusion every other week. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- Participant is able and willing to provide informed consent prior to any study procedures are performed
- Diagnosis of GSDII based on one of the following:
- Onset of Pompe disease-related symptoms after 1 year of age
- Previously treated with Myozyme or Lumizyme for at least 12 months and on a stable regimen for the past 6 months
- Sexually active participants who are willing to use an acceptable method of contraception (abstinence, oral contraceptives, barrier method with spermicide, surgical sterilization, implanted or injectable contraceptives with a stable dose for at least 1 month prior to Baseline, hormonal intra-uterine device [IUD] inserted at least 1 month prior to Baseline) during the study and for 30 days after completion of treatment
Key exclusion criteria (11 listed)
- Cardiac involvement in first year of life
- Anti-GAA antibody titers >1:51,200 at two time points
- Prior use of chaperone therapy for GSD-II within the last 12 months
- Use of immunosuppressive medication other than glucocorticoids within 6 months prior to study enrollment
- Use of invasive ventilatory assistance other than Bilevel Positive Airway Pressure (BiPAP) at night or during periods of rest
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 |
|---|---|---|
| VAL-1221VAL-1221 3, 10, or 30 mg/kg as per the dose and schedule specified in the arm group description | Drug | VAL-1221 10 mg/kg, VAL-1221 3 mg/kg, VAL-1221 30 mg/kg, rhGAA |
| RhGAAAlso: Myozyme, LumizymeActive comparator | Drug | rhGAA |
Primary outcome
Number of Treatment-related Treatment-emergent Adverse Events (TEAEs)
Time frame: Baseline of Study Part 1 though Month 24 of Study Part 2
Number of Participants With Infusion-Associated Reactions to VAL-1221
Time frame: Baseline of Study Part 1 though Month 24 of Study Part 2
Percentage of Participants with Anti-VAL-1221 Antibodies
Time frame: Baseline of Study Part 1 though Month 24 of Study Part 2
Percentage of Participants with GAA Antibodies
Time frame: Baseline of Study Part 1 though Month 24 of Study Part 2
11 secondary outcomes
Pharmacokinetics of VAL-1221 in Plasma: Maximum Observed Concentration (Cmax)
Time frame: Part 1: Pre-infusion, end of infusion (EOI) (infusion duration 1 mg/kg/hour), at 0.25, 0.5, 1, 2, 4, 12, 24, 48 and 336 hours after EOI on Days 1, 2, 3 and Week 12; Part 2: Pre-infusion on Weeks 12, 14, 16, 18, 20, 22, Months 12, 24, 36
Pharmacokinetics of VAL-1221 in Plasma: Time to Reach Maximum Concentration (Tmax)
Time frame: Part 1: Pre-infusion, end of infusion (EOI) (infusion duration 1 mg/kg/hour), at 0.25, 0.5, 1, 2, 4, 12, 24, 48 and 336 hours after EOI on Days 1, 2, 3 and Week 12; Part 2: Pre-infusion on Weeks 12, 14, 16, 18, 20, 22, Months 12, 24, 36
Pharmacokinetics of VAL-1221 in Plasma: Area Under the Concentration Time Curve (AUC)
Time frame: Part 1: Pre-infusion, end of infusion (EOI) (infusion duration 1 mg/kg/hour), at 0.25, 0.5, 1, 2, 4, 12, 24, 48 and 336 hours after EOI on Days 1, 2, 3 and Week 12; Part 2: Pre-infusion on Weeks 12, 14, 16, 18, 20, 22, Months 12, 24, 36
Pharmacokinetics of VAL-1221 in Plasma: Terminal Elimination Half-Life ( t1/2)
Time frame: Part 1: Pre-infusion, end of infusion (EOI) (infusion duration 1 mg/kg/hour), at 0.25, 0.5, 1, 2, 4, 12, 24, 48 and 336 hours after EOI on Days 1, 2, 3 and Week 12; Part 2: Pre-infusion on Weeks 12, 14, 16, 18, 20, 22, Months 12, 24, 36
Pharmacokinetics of VAL-1221 in Plasma: Apparent Total Body Clearance (CL)
Time frame: Part 1: Pre-infusion, end of infusion (EOI) (infusion duration 1 mg/kg/hour), at 0.25, 0.5, 1, 2, 4, 12, 24, 48 and 336 hours after EOI on Days 1, 2, 3 and Week 12; Part 2: Pre-infusion on Weeks 12, 14, 16, 18, 20, 22, Months 12, 24, 36
Pharmacokinetics of VAL-1221 in Plasma: Apparent Volume (V)
Time frame: Part 1: Pre-infusion, end of infusion (EOI) (infusion duration 1 mg/kg/hour), at 0.25, 0.5, 1, 2, 4, 12, 24, 48 and 336 hours after EOI on Days 1, 2, 3 and Week 12; Part 2: Pre-infusion on Weeks 12, 14, 16, 18, 20, 22, Months 12, 24, 36
Change from Baseline in Urinary Hexose Tetrasaccharide (hex4) Excretion at Week 12, Months 6, 9, and 12
Time frame: Baseline of Study Part 1, Week 12 of Study Part 1, Months 12, 24, and 36 of Study Part 2
Change from Baseline in Serum Creatine Kinase (CK) at Week 12, Months 6, 9, and 12
Time frame: Baseline of Study Part 1, Week 12 of Study Part 1, Months 12, 24, and 36 of Study Part 2
Change from Baseline in the Amount of Acid Alpha Glucosidase (GAA) Activity Present in Muscle at Week 12
Time frame: Baseline of Study Part 1, Week 12 of Study Part 1
Change from Baseline in the Muscle Glycogen Content at Week 12
Time frame: Baseline of Study Part 1, Week 12 Study Part 1
Change from Baseline in Creatinine Excretion at Months 6, 9, and 12
Time frame: Baseline of Study Part 1, Months 12, 24, 36 of Study Part 2
Results and safety
The study was stopped early (reason given: Company closure due to lack of funding). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Jun 2020.
- 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.
Hal Landy, MD study director
Valerion Therapeutics, LLC
3 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing VAL-1221 and RhGAA in people with Pompe Disease, run by Valerion Therapeutics, LLC at 3 sites in 2 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 12 inclusion and 11 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: stopped early, as of the sponsor's last update on 2 Jun 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States, United Kingdom. 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?
The study was stopped early (reason given: Company closure due to lack of funding). Results are not available in the source record.
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 Treatment-related Treatment-emergent Adverse Events (TEAEs) (time frame: Baseline of Study Part 1 though Month 24 of Study Part 2). The study lists 4 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 2 Jun 2020.