Safety, Tolerability, and Pharmacokinetics of UX053 in Patients With Glycogen Storage Disease Type III (GSD III)
NCT ID: NCT04990388
Last Updated: 2024-04-16
Study Results
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View full resultsBasic Information
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TERMINATED
PHASE1/PHASE2
9 participants
INTERVENTIONAL
2021-10-18
2023-03-20
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
SEQUENTIAL
TREATMENT
QUADRUPLE
Study Groups
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UX053 Dose Level 1S ->OL-1R
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.
UX053
mRNA-based biologic
Antipyretic
participants will receive oral premedication prior to infusion
H2 Blocker
participants will receive oral premedication prior to infusion
H1 Blocker
participants will receive oral premedication prior to infusion
UX053 Dose Level 2S->OL-2R
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.
UX053
mRNA-based biologic
Antipyretic
participants will receive oral premedication prior to infusion
H2 Blocker
participants will receive oral premedication prior to infusion
H1 Blocker
participants will receive oral premedication prior to infusion
UX053 Dose Level 3S->OL-3R
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.
UX053
mRNA-based biologic
Antipyretic
participants will receive oral premedication prior to infusion
H2 Blocker
participants will receive oral premedication prior to infusion
H1 Blocker
participants will receive oral premedication prior to infusion
UX053 or Placebo Dose Level DB-1R
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.
UX053
mRNA-based biologic
Placebo
consists of the same components as the formulation buffer for UX053
Antipyretic
participants will receive oral premedication prior to infusion
H2 Blocker
participants will receive oral premedication prior to infusion
H1 Blocker
participants will receive oral premedication prior to infusion
UX053 Dose Level DB-2R
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.
UX053
mRNA-based biologic
Placebo
consists of the same components as the formulation buffer for UX053
Antipyretic
participants will receive oral premedication prior to infusion
H2 Blocker
participants will receive oral premedication prior to infusion
H1 Blocker
participants will receive oral premedication prior to infusion
UX053 Dose Level DB-3R
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.
UX053
mRNA-based biologic
Placebo
consists of the same components as the formulation buffer for UX053
Antipyretic
participants will receive oral premedication prior to infusion
H2 Blocker
participants will receive oral premedication prior to infusion
H1 Blocker
participants will receive oral premedication prior to infusion
Interventions
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UX053
mRNA-based biologic
Placebo
consists of the same components as the formulation buffer for UX053
Antipyretic
participants will receive oral premedication prior to infusion
H2 Blocker
participants will receive oral premedication prior to infusion
H1 Blocker
participants will receive oral premedication prior to infusion
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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
* 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
Exclusion Criteria
* 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
* 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
* 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
18 Years
ALL
No
Sponsors
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Ultragenyx Pharmaceutical Inc
INDUSTRY
Responsible Party
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Principal Investigators
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Medical Director
Role: STUDY_DIRECTOR
Ultragenyx Pharmaceutical Inc
Locations
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University of California, Irvine
Orange, California, United States
Rare Disease Research
Atlanta, Georgia, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
University of Texas, Health Science Center of Houston
Houston, Texas, United States
Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico
Milan, , Italy
Hospital Universitario 12 de Octubre
Madrid, , Spain
Countries
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Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Related Links
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Ultragenyx Patient Advocacy/GSD III Disease Information
Other Identifiers
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2021-000903-19
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
UX053-CL101
Identifier Type: -
Identifier Source: org_study_id
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