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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT05345171
Registration number
NCT05345171
Ethics application status
Date submitted
18/04/2022
Date registered
25/04/2022
Date last updated
3/05/2024
Titles & IDs
Public title
Clinical Study of DTX301 AAV- Mediated Gene Transfer for Ornithine Transcarbamylase(OTC) Deficiency
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Scientific title
A Phase 3, Randomized, Double-blind, Placebo-controlled Study of Adeno-associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Patients With Late-onset OTC Deficiency
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Secondary ID [1]
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2020-003384-25
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Secondary ID [2]
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DTX301-CL301
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Condition category
Condition code
Neurological
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Other neurological disorders
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Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Metabolic and Endocrine
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Metabolic disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Other interventions - DTX301
Other interventions - Placebo
Treatment: Drugs - Oral Corticosteroids
Treatment: Drugs - Placebo for oral corticosteroids
Treatment: Drugs - Sodium Acetate
Experimental: DTX301, Then Placebo - Participants receive single peripheral intravenous (IV) infusion of DTX301 in solution. At week 64, participants receive single peripheral IV infusion of placebo.
Experimental: Placebo, Then DTX301 - Participants receive single peripheral IV infusion of placebo. At week 64, participants receive single peripheral IV infusion of DTX301 in solution.
Other interventions: DTX301
non-replicating, self-complementary recombinant adeno-associated virus serotype 8 (AAV8) vector
Other interventions: Placebo
normal saline infusion
Treatment: Drugs: Oral Corticosteroids
Participants who receive DTX301 solution will receive oral corticosteroids.
Treatment: Drugs: Placebo for oral corticosteroids
Participants who receive Placebo will receive placebo corticosteroids to maintain the study blind
Treatment: Drugs: Sodium Acetate
A tracer for the Ureagenesis Rate Test (URT)
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Intervention code [1]
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Other interventions
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Intervention code [2]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Plasma Ammonia as Measured by 24-hour Ammonia (AUC0-24)
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Assessment method [1]
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Timepoint [1]
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Week 64
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Primary outcome [2]
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Percentage of Participants Who Have Achieved Complete Response
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Assessment method [2]
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Timepoint [2]
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Week 64
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Secondary outcome [1]
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Percentage of Participants Who Have Achieved Complete Response, Response, or No Response
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Assessment method [1]
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Timepoint [1]
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Week 64
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Secondary outcome [2]
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Patient Global Impression of Change (PGIC) Overall Change Score
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Assessment method [2]
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Timepoint [2]
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Week 64
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Secondary outcome [3]
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Rate of Hyperammonemic Crises (HACs) from Baseline to Week 64 Compared to Pre-Enrollment
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Assessment method [3]
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Timepoint [3]
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Pre-enrollment, Baseline, Week 64
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Secondary outcome [4]
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Change from Baseline in Plasma Ammonia (AUC0-24) After DTX301 Exposure
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Assessment method [4]
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Timepoint [4]
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Baseline, Up to Week 64
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Secondary outcome [5]
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Change from Baseline in Plasma Ammonia (AUC0-24)
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Assessment method [5]
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Timepoint [5]
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Baseline, Up to Week 64
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Secondary outcome [6]
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Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, Related TEAEs, Related Serious TEAEs and Adverse Events of Special Interest (AESIs)
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Assessment method [6]
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Timepoint [6]
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Up to Week 324
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Secondary outcome [7]
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Number of Participants With Clinically Significant Changes From Baseline in Laboratory Values, Physical Examination Results, and Vital Sign Measurements
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Assessment method [7]
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Timepoint [7]
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Baseline, Up to Week 324
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Secondary outcome [8]
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Number of Participants With Anti-OTC Antibodies
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Assessment method [8]
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Timepoint [8]
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Up to Week 324
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Eligibility
Key inclusion criteria
Key
- Confirmed clinical diagnosis of late-onset OTC deficiency with historical
documentation by enzymatic (ie, liver biopsy), biochemical (ie, hyperammonemia in the
presence of elevated plasma glutamine, low citrulline, and elevated spot urine orotic
acid), or molecular testing (ie, OTC analysis)
- Free from symptomatic hyperammonemia and has not required emergent active intervention
for hyperammonemia within 4 weeks before screening/baseline
- If on ongoing daily ammonia scavenger therapy, must be at stable daily dose(s) for = 4
weeks prior to screening
- If on a protein-restricted diet, must be on a stable total daily protein intake that
does not vary more than 20% for = 4 weeks prior to screening
- From the time written informed consent through Week 128, females of childbearing
potential and fertile males must consent to use highly effective contraception. If
female, agree not to become pregnant. If male, agree not father a child or donate
sperm
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Minimum age
12
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
- Significant hepatic inflammation or cirrhosis
- Estimated glomerular filtration rate < 60 mL/min/1.73 m2 at screening by the 2021
CKD-EPI creatinine-based formula (Inker et al., 2021) for patients = 18 years of age
or the Schwartz bedside formula (Schwartz and Work, 2009) for patients < 18 years of
age
- Evidence of active hepatitis B virus (HBV) or hepatitis C virus (HCV) infection,
documented by current use of antiviral therapy for HBV or HCV or by hepatitis B
surface antigen (HBsAg) or HCV RNA positivity
- Active infection (viral or bacterial)
- Detectable pre-existing antibodies to the AAV8 capsid
- Presence or history of any condition that, in the view of the Investigator, would
interfere with participation, pose undue risk, or would confound interpretation of
results
- Participation (current or previous) in another gene transfer study
Note: Additional inclusion/exclusion criteria may apply, per protocol
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
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Intervention assignment
Crossover
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
18/10/2022
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
1/12/2028
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Actual
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Sample size
Target
50
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
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Recruitment hospital [1]
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Westmead Hospital - Sydney
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Recruitment postcode(s) [1]
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2145 - Sydney
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Recruitment outside Australia
Country [1]
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United States of America
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Arkansas
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United States of America
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California
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United States of America
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Colorado
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United States of America
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Connecticut
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United States of America
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Florida
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United States of America
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Illinois
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United States of America
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Ohio
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United States of America
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Utah
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Argentina
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Buenos Aires
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Argentina
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Córdoba
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Brazil
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Porto Alegre
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Canada
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Ontario
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France
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Bron
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France
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Paris
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Germany
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Heidelberg
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Italy
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Napoli
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Italy
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Padova
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Italy
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Torino
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Japan
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Kumamoto
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Japan
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Toyoake
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Netherlands
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Rotterdam
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Portugal
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Porto
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Spain
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Barcelona
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Spain
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Santiago De Compostela
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United Kingdom
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State/province [25]
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Birmingham
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Ultragenyx Pharmaceutical Inc
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
The primary objective is to evaluate the efficacy of DTX301 on the improvement of ornithine transcarbamylase (OTC) function by maintaining safe plasma ammonia levels with removal of dietary protein restriction and alternative pathway medication.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT05345171
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Medical Director
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Address
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Ultragenyx Pharmaceutical Inc
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Patients Contact: Trial Recruitment
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Address
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Phone
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1-888-756-8657
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Fax
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Email
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[email protected]
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Contact person for scientific queries
No information has been provided regarding IPD availability
What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results not provided in
https://clinicaltrials.gov/ct2/show/NCT05345171
Download to PDF