Glycogen Storage Disease Type IIIPhase 1/2NCT04990388
Safety, Tolerability, and Pharmacokinetics of UX053 in Patients With Glycogen Storage Disease Type III (GSD III)
A Phase 1/2 study testing UX053, Antipyretic and H2 Blocker in people with Glycogen Storage Disease Type III, run by Ultragenyx Pharmaceutical Inc at 6 sites in 3 countries.
Trial summary
The primary objective of the study is to evaluate the safety of UX053 in adults with Glycogen Storage Disease Type III (GSD III). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Glycogen Storage Disease Type III
- What is being tested?
- UX053, Antipyretic and H2 Blocker
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 9 participants
- Where
- 6 sitesin 3 countries
- Study dates
- 18 Oct 2021to 20 Mar 2023
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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
- Confirmed diagnosis of GSD III by gene sequencing or enzymatic testing
- Alanine aminotransferase at or below 5 times normal during the three months prior to dosing
- Willing and able to comply with standard dietary management of GSD III
- Inclusion Criteria for Participants Rescreening Into OL-RD Cohorts After Treatment with UX053 in SAD Cohort:
- If a significant rise in ALT occurs after the prior dose, ALT should show a decreasing trend toward the subject's baseline value
- Total bilirubin, platelets and international normalized ratio (INR) is within normal limits
Who cannot join
- History of liver transplant or currently awaiting liver transplant
- History of cirrhosis
- Active Hepatitis B or C
- Severe kidney impairment
- History of liver cancer or large liver tumors
- History of any cancer within the past 3 years
Show all 18Show fewer
- Known history of HIV infection
- Known severe allergy to polyethylene glycol (PEG), polysorbate, or mRNA vaccine
- Heart failure that causes marked limitation in physical activity
- Poorly controlled diabetes
- Poorly controlled hypothyroidism
- Treatment with immunosuppressive medications such as those used to treat chronic autoimmune conditions and solid organ transplants
- Pregnant or nursing, or planning to become pregnant during the study
- Exclusion Criteria for Participants Rescreening Into OL-RD Cohorts After Treatment with UX053 in SAD Cohort:
- New or worsening symptoms of liver disease (including new or worsening hepatomegaly) along with any increase in transaminase levels
- Receipt of any blood product administration (eg, packed red blood cells, platelet, FFP) for management of consumptive coagulopathy
- An ALT level that is ≥ 8x ULN and > 2x the participants baseline value in the absence of an alternative explanation
- Note: Additional inclusion/exclusion criteria may apply, per protocol
Trial locations and contacts
6 sites in 3 countries.
6 sites in 3 countries
United States4 sites
Rare Disease Research
Atlanta, Georgia, 30329
University of Texas, Health Science Center of Houston
Houston, Texas, 77030
University of California, Irvine
Orange, California, 92868
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Italy1 site
Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico
Milan, 20122
About this study
Official title: A Phase 1/2 First-in-human, Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Single Ascending Doses and Repeat Doses of UX053 in Patients With GSD III
The primary objective of the study is to evaluate the safety of UX053 in adults with Glycogen Storage Disease Type III (GSD III).
Read the detailed description
This study is a phase 1/2 first-in-human (FIH), study to evaluate the safety, tolerability, and pharmacokinetic (PK) of a single ascending dose (SAD) and repeat doses (RD) of UX053 in patients with GSD III. The SAD cohorts will be open-label (OL). There will be two types of RD cohorts, an open-label (OL-RD) and a randomized, double-blind (DB), and placebo-controlled (DB-RD).
- Conditions
- Glycogen Storage Disease Type III
- Being tested
- UX053, Placebo, Antipyretic, H2 Blocker, H1 Blocker
- Medical subject headings
- Glycogen Storage Disease Type III
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
UX053Biological
mRNA-based biologic
PlaceboOther
consists of the same components as the formulation buffer for UX053
AntipyreticDrug
Also called paracetamol, acetaminophen, ibuprofen
participants will receive oral premedication prior to infusion
H2 BlockerDrug
Also called famotidine
participants will receive oral premedication prior to infusion
H1 BlockerDrug
Also called cetirizine
participants will receive oral premedication prior to infusion
| Study group | Role | What they receive |
|---|---|---|
| UX053 Dose Level 1S ->OL-1R | Experimental | Participants receive a single, peripheral intravenous (IV) infusion of UX053. After completion of the 90-day Follow up Period, participants can enter the open label repeat dose (OL-RD) cohort where they will receive UX053 every 4 weeks (Q4W) for 4 doses. Participants will also receive premedication, consisting of oral paracetamol/acetaminophen or ibuprofen, an H2 blocker, and an H1 blocker. Receives: Biological: UX053; Drug: Antipyretic; Drug: H2 Blocker; Drug: H1 Blocker |
| UX053 Dose Level 2S->OL-2R | Experimental | Participants receive a single, peripheral intravenous (IV) infusion of UX053. After completion of the 90-day Follow up Period, participants can enter the open label repeat dose (OL-RD) cohort where they will receive UX053 every 4 weeks (Q4W) for 4 doses. Participants will also receive premedication, consisting of oral paracetamol/acetaminophen or ibuprofen, an H2 blocker, and an H1 blocker. Receives: Biological: UX053; Drug: Antipyretic; Drug: H2 Blocker; Drug: H1 Blocker |
| UX053 Dose Level 3S->OL-3R | Experimental | Participants receive a single, peripheral intravenous (IV) infusion of UX053. After completion of the 90-day Follow up Period, participants can enter the open label repeat dose (OL-RD) cohort where they will receive UX053 every 4 weeks (Q4W) for 4 doses. Participants will also receive premedication, consisting of oral paracetamol/acetaminophen or ibuprofen, an H2 blocker, and an H1 blocker. Receives: Biological: UX053; Drug: Antipyretic; Drug: H2 Blocker; Drug: H1 Blocker |
| UX053 or Placebo Dose Level DB-1R | Experimental | Participants randomized to receive a single, peripheral IV infusion of UX053 or Placebo every 2 weeks (Q2W) for 5 doses. Participants will also receive premedication, consisting of oral paracetamol/acetaminophen or ibuprofen, an H2 blocker, and an H1 blocker. Receives: Biological: UX053; Other: Placebo; Drug: Antipyretic; Drug: H2 Blocker; Drug: H1 Blocker |
| UX053 Dose Level DB-2R | Experimental | Participants randomized to receive a single, peripheral IV infusion of UX053 or Placebo every 2 weeks (Q2W) for 5 doses. Participants will also receive premedication, consisting of oral paracetamol/acetaminophen or ibuprofen, an H2 blocker, and an H1 blocker. Receives: Biological: UX053; Other: Placebo; Drug: Antipyretic; Drug: H2 Blocker; Drug: H1 Blocker |
| UX053 Dose Level DB-3R | Experimental | Participants randomized to receive a single, peripheral IV infusion of UX053 or Placebo every 2 weeks (Q2W) for 5 doses. Participants will also receive premedication, consisting of oral paracetamol/acetaminophen or ibuprofen, an H2 blocker, and an H1 blocker. Receives: Biological: UX053; Other: Placebo; Drug: Antipyretic; Drug: H2 Blocker; Drug: H1 Blocker |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Deaths, Discontinuations, and/or Dose Changes
An AE is defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. A TEAE is defined as any AE not present prior to the initiation of the drug treatment or any AE already present that worsens in either intensity or frequency following exposure to the drug treatment. An SAE is an AE that meets any of the following criteria in the view of either the Investigator or Ultragenyx: death; life-threatening; inpatient hospitalization or prolongation of existing hospitalization; disability/Incapacity; congenital anomaly/birth defect not present at screening; other important medical events. Severity of events were graded as mild (grade1), moderate (grade 2), severe (grade 3), life-threatening (grade 4), or death (grade 5).
Measured: From first dose of study drug through the end of study (up to Day 90)
Secondary outcomes (7)
Pharmacokinetics (PK) of Amylo-α-1,6-glucosidase 4-alpha-glucanotransferase Messenger Ribonucleic Acid (AGL mRNA) and the Excipient ATX95: Maximum Blood/Plasma Concentration (Cmax)
Measured: Pre-infusion; 1, 3, 4, 4.5, 5 hr (± 10 min), 6 hr (± 20 min), 8, 10 hr (± 30 min), 24 hr (± 2 hr), 96, 168 hr (± 24 hr), 336, 504, 672 hr (± 48 hr) post-start of infusion
PK of AGL mRNA and the Excipient ATX95: Time to Peak Drug Concentration (Tmax)
Measured: Pre-infusion; 1, 3, 4, 4.5, 5 hr (± 10 min), 6 hr (± 20 min), 8, 10 hr (± 30 min), 24 hr (± 2 hr), 96, 168 hr (± 24 hr), 336, 504, 672 hr (± 48 hr) post-start of infusion
PK of AGL mRNA and the Excipient ATX95: Total Drug Exposure to the Last Measurable Concentration (AUC0-last)
Measured: Pre-infusion; 1, 3, 4, 4.5, 5 hr (± 10 min), 6 hr (± 20 min), 8, 10 hr (± 30 min), 24 hr (± 2 hr), 96, 168 hr (± 24 hr), 336, 504, 672 hr (± 48 hr) post-start of infusion
PK of AGL mRNA and the Excipient ATX95: Total Drug Exposure to Infinity (AUC0-inf)
Measured: Pre-infusion; 1, 3, 4, 4.5, 5 hr (± 10 min), 6 hr (± 20 min), 8, 10 hr (± 30 min), 24 hr (± 2 hr), 96, 168 hr (± 24 hr), 336, 504, 672 hr (± 48 hr) post-start of infusion
PK of AGL mRNA and the Excipient ATX95: Elimination Half-life (t½)
Measured: Pre-infusion; 1, 3, 4, 4.5, 5 hr (± 10 min), 6 hr (± 20 min), 8, 10 hr (± 30 min), 24 hr (± 2 hr), 96, 168 hr (± 24 hr), 336, 504, 672 hr (± 48 hr) post-start of infusion
PK of AGL mRNA and the Excipient ATX95: Clearance (CL)
Measured: Pre-infusion; 1, 3, 4, 4.5, 5 hr (± 10 min), 6 hr (± 20 min), 8, 10 hr (± 30 min), 24 hr (± 2 hr), 96, 168 hr (± 24 hr), 336, 504, 672 hr (± 48 hr) post-start of infusion
PK of AGL mRNA and the Excipient ATX95: Volume of Distribution at Steady State (Vss)
Measured: Pre-infusion; 1, 3, 4, 4.5, 5 hr (± 10 min), 6 hr (± 20 min), 8, 10 hr (± 30 min), 24 hr (± 2 hr), 96, 168 hr (± 24 hr), 336, 504, 672 hr (± 48 hr) post-start of infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Aug 2021
- Study start18 Oct 2021
- Primary completion20 Mar 2023
- Study completion20 Mar 2023
Why it stopped: Sponsor decision not related to safety concerns
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Glycogen Storage Disease Type IIIStudies of the same condition, open and under way.
- More trials run by Ultragenyx Pharmaceutical IncThe sponsor's other registered studies.
- 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
- Sequential
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Ultragenyx Pharmaceutical Inc
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04990388
- Sponsor's protocol ID
- UX053-CL101
- Other IDs
- 2021-000903-19
- Status last verified
- Apr 2024
- First submitted
- 26 Jul 2021
- Last updated
- 16 Apr 2024
- Results posted
- Yes, on ClinicalTrials.gov
- 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 16 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2 First-in-human, Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Single Ascending Doses and Repeat Doses of UX053 in Patients With GSD III
- ClinicalTrials.gov ID
- NCT04990388
- Acronym / sponsor ID
- UX053-CL101
- Conditions
- Glycogen Storage Disease Type III
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Sponsor decision not related to safety concerns
- Enrolment
- 9 participants (actual)
- Lead sponsor
- Ultragenyx Pharmaceutical IncIndustry (pharma, biotech, devices)
- Study start
- 18 Oct 2021 (actual)
- Primary completion
- 20 Mar 2023 (actual)
- Study completion
- 20 Mar 2023 (actual)
- First posted
- 4 Aug 2021 (actual)
- Results
- Posted 16 Apr 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The primary objective of the study is to evaluate the safety of UX053 in adults with Glycogen Storage Disease Type III (GSD III).
Detailed description
This study is a phase 1/2 first-in-human (FIH), study to evaluate the safety, tolerability, and pharmacokinetic (PK) of a single ascending dose (SAD) and repeat doses (RD) of UX053 in patients with GSD III. The SAD cohorts will be open-label (OL). There will be two types of RD cohorts, an open-label (OL-RD) and a randomized, double-blind (DB), and placebo-controlled (DB-RD).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 6
| Arm | Type | Receives |
|---|---|---|
| UX053 Dose Level 1S ->OL-1R | Experimental | Biological: UX053, Drug: Antipyretic, Drug: H2 Blocker, Drug: H1 BlockerParticipants receive a single, peripheral intravenous (IV) infusion of UX053. After completion of the 90-day Follow up Period, participants can enter the open label repeat dose (OL-RD) cohort where they will receive UX053 every 4 weeks (Q4W) for 4 doses. Participants will also receive premedication, consisting of oral paracetamol/acetaminophen or ibuprofen, an H2 blocker, and an H1 blocker. |
| UX053 Dose Level 2S->OL-2R | Experimental | Biological: UX053, Drug: Antipyretic, Drug: H2 Blocker, Drug: H1 BlockerParticipants receive a single, peripheral intravenous (IV) infusion of UX053. After completion of the 90-day Follow up Period, participants can enter the open label repeat dose (OL-RD) cohort where they will receive UX053 every 4 weeks (Q4W) for 4 doses. Participants will also receive premedication, consisting of oral paracetamol/acetaminophen or ibuprofen, an H2 blocker, and an H1 blocker. |
| UX053 Dose Level 3S->OL-3R | Experimental | Biological: UX053, Drug: Antipyretic, Drug: H2 Blocker, Drug: H1 BlockerParticipants receive a single, peripheral intravenous (IV) infusion of UX053. After completion of the 90-day Follow up Period, participants can enter the open label repeat dose (OL-RD) cohort where they will receive UX053 every 4 weeks (Q4W) for 4 doses. Participants will also receive premedication, consisting of oral paracetamol/acetaminophen or ibuprofen, an H2 blocker, and an H1 blocker. |
| UX053 or Placebo Dose Level DB-1R | Experimental | Biological: UX053, Other: Placebo, Drug: Antipyretic, Drug: H2 Blocker, Drug: H1 BlockerParticipants randomized to receive a single, peripheral IV infusion of UX053 or Placebo every 2 weeks (Q2W) for 5 doses. Participants will also receive premedication, consisting of oral paracetamol/acetaminophen or ibuprofen, an H2 blocker, and an H1 blocker. |
| UX053 Dose Level DB-2R | Experimental | Biological: UX053, Other: Placebo, Drug: Antipyretic, Drug: H2 Blocker, Drug: H1 BlockerParticipants randomized to receive a single, peripheral IV infusion of UX053 or Placebo every 2 weeks (Q2W) for 5 doses. Participants will also receive premedication, consisting of oral paracetamol/acetaminophen or ibuprofen, an H2 blocker, and an H1 blocker. |
| UX053 Dose Level DB-3R | Experimental | Biological: UX053, Other: Placebo, Drug: Antipyretic, Drug: H2 Blocker, Drug: H1 BlockerParticipants randomized to receive a single, peripheral IV infusion of UX053 or Placebo every 2 weeks (Q2W) for 5 doses. Participants will also receive premedication, consisting of oral paracetamol/acetaminophen or ibuprofen, an H2 blocker, and an H1 blocker. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Confirmed diagnosis of GSD III by gene sequencing or enzymatic testing
- Alanine aminotransferase at or below 5 times normal during the three months prior to dosing
- Willing and able to comply with standard dietary management of GSD III
- Inclusion Criteria for Participants Rescreening Into OL-RD Cohorts After Treatment with UX053 in SAD Cohort:
- If a significant rise in ALT occurs after the prior dose, ALT should show a decreasing trend toward the subject's baseline value
Key exclusion criteria (18 listed)
- History of liver transplant or currently awaiting liver transplant
- History of cirrhosis
- Active Hepatitis B or C
- Severe kidney impairment
- History of liver cancer or large liver tumors
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 |
|---|---|---|
| UX053mRNA-based biologic | Biological | UX053 Dose Level 1S ->OL-1R, UX053 Dose Level 2S->OL-2R, UX053 Dose Level 3S->OL-3R, UX053 Dose Level DB-2R, UX053 Dose Level DB-3R, UX053 or Placebo Dose Level DB-1R |
| Placeboconsists of the same components as the formulation buffer for UX053 | Other | UX053 Dose Level DB-2R, UX053 Dose Level DB-3R, UX053 or Placebo Dose Level DB-1R |
| AntipyreticAlso: paracetamol, acetaminophen, ibuprofenparticipants will receive oral premedication prior to infusion | Drug | UX053 Dose Level 1S ->OL-1R, UX053 Dose Level 2S->OL-2R, UX053 Dose Level 3S->OL-3R, UX053 Dose Level DB-2R, UX053 Dose Level DB-3R, UX053 or Placebo Dose Level DB-1R |
| H2 BlockerAlso: famotidineparticipants will receive oral premedication prior to infusion | Drug | UX053 Dose Level 1S ->OL-1R, UX053 Dose Level 2S->OL-2R, UX053 Dose Level 3S->OL-3R, UX053 Dose Level DB-2R, UX053 Dose Level DB-3R, UX053 or Placebo Dose Level DB-1R |
| H1 BlockerAlso: cetirizineparticipants will receive oral premedication prior to infusion | Drug | UX053 Dose Level 1S ->OL-1R, UX053 Dose Level 2S->OL-2R, UX053 Dose Level 3S->OL-3R, UX053 Dose Level DB-2R, UX053 Dose Level DB-3R, UX053 or Placebo Dose Level DB-1R |
Primary outcome
Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Deaths, Discontinuations, and/or Dose Changes
Time frame: From first dose of study drug through the end of study (up to Day 90)
An AE is defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. A TEAE is defined as any AE not present prior to the initiation of the drug treatment or any AE already present that worsens in either intensity or frequency following exposure to the drug treatment. An SAE is an AE that meets any of the following criteria in the view of either the Investigator or Ultragenyx: death; life-threatening; inpatient hospitalization or prolongation of existing hospitalization; disability/Incapacity; congenital anomaly/birth defect not present at screening; other important medical events. Severity of events were graded as mild (grade1), moderate (grade 2), severe (grade 3), life-threatening (grade 4), or death (grade 5).
7 secondary outcomes
Pharmacokinetics (PK) of Amylo-α-1,6-glucosidase 4-alpha-glucanotransferase Messenger Ribonucleic Acid (AGL mRNA) and the Excipient ATX95: Maximum Blood/Plasma Concentration (Cmax)
Time frame: Pre-infusion; 1, 3, 4, 4.5, 5 hr (± 10 min), 6 hr (± 20 min), 8, 10 hr (± 30 min), 24 hr (± 2 hr), 96, 168 hr (± 24 hr), 336, 504, 672 hr (± 48 hr) post-start of infusion
PK of AGL mRNA and the Excipient ATX95: Time to Peak Drug Concentration (Tmax)
Time frame: Pre-infusion; 1, 3, 4, 4.5, 5 hr (± 10 min), 6 hr (± 20 min), 8, 10 hr (± 30 min), 24 hr (± 2 hr), 96, 168 hr (± 24 hr), 336, 504, 672 hr (± 48 hr) post-start of infusion
PK of AGL mRNA and the Excipient ATX95: Total Drug Exposure to the Last Measurable Concentration (AUC0-last)
Time frame: Pre-infusion; 1, 3, 4, 4.5, 5 hr (± 10 min), 6 hr (± 20 min), 8, 10 hr (± 30 min), 24 hr (± 2 hr), 96, 168 hr (± 24 hr), 336, 504, 672 hr (± 48 hr) post-start of infusion
PK of AGL mRNA and the Excipient ATX95: Total Drug Exposure to Infinity (AUC0-inf)
Time frame: Pre-infusion; 1, 3, 4, 4.5, 5 hr (± 10 min), 6 hr (± 20 min), 8, 10 hr (± 30 min), 24 hr (± 2 hr), 96, 168 hr (± 24 hr), 336, 504, 672 hr (± 48 hr) post-start of infusion
PK of AGL mRNA and the Excipient ATX95: Elimination Half-life (t½)
Time frame: Pre-infusion; 1, 3, 4, 4.5, 5 hr (± 10 min), 6 hr (± 20 min), 8, 10 hr (± 30 min), 24 hr (± 2 hr), 96, 168 hr (± 24 hr), 336, 504, 672 hr (± 48 hr) post-start of infusion
PK of AGL mRNA and the Excipient ATX95: Clearance (CL)
Time frame: Pre-infusion; 1, 3, 4, 4.5, 5 hr (± 10 min), 6 hr (± 20 min), 8, 10 hr (± 30 min), 24 hr (± 2 hr), 96, 168 hr (± 24 hr), 336, 504, 672 hr (± 48 hr) post-start of infusion
PK of AGL mRNA and the Excipient ATX95: Volume of Distribution at Steady State (Vss)
Time frame: Pre-infusion; 1, 3, 4, 4.5, 5 hr (± 10 min), 6 hr (± 20 min), 8, 10 hr (± 30 min), 24 hr (± 2 hr), 96, 168 hr (± 24 hr), 336, 504, 672 hr (± 48 hr) post-start of infusion
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Apr 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Ultragenyx Patient Advocacy/GSD III Disease InformationLink provided by the sponsor.
- 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.
Medical Director study director
Ultragenyx Pharmaceutical Inc
6 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing UX053, Antipyretic and H2 Blocker in people with Glycogen Storage Disease Type III, run by Ultragenyx Pharmaceutical Inc at 6 sites in 3 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 6 inclusion and 18 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 16 Apr 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in United States, Italy, Spain. 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?
Yes. Results are posted in the registry (first posted 16 Apr 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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 Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Deaths, Discontinuations, and/or Dose Changes (time frame: From first dose of study drug through the end of study (up to Day 90)). 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 16 Apr 2024.