Ultragenyx Pharmaceutical Inc. — Sponsor Intelligence | 14 EU Trials

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14 Registered trials

8 Recruiting

6 Completed trials

9 Countries active

19 Unique IMPs

100% Results reported

Phase III

Phase I/II

Phase I (FiH)

Phase III: 7 Phase II/III: 1 Phase II: 1 Phase I/II: 2 Phase III/IV: 1 Phase I (FiH): 2

Oversight events:

1 serious breach 1 corrective measure across 2 trials

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100%

6 of 6 completed trials have published summary results (EU CTR Article 37 — 12-month obligation).

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Top indications

Angelman's syndrome 2 Osteogenesis imperfecta 2 Mucopolysaccharidosis III 2 Fatty acid oxidation disorder 1 Angelman´s syndrome 1

Top IMPs

PARIGLASGENE BRECAPARVOVEC 3 PREDNISOLONE 3 SODIUM DIHYDROGEN PHOSPHATE DIHYDRATE, DISODIUM PHOSPHATE, POTASSIUM CHLORIDE, SODIUM CHLORIDE, CALCIUM CHLORIDE DIHYDRATE, MAGNESIUM SULFATE HEPTAHYDRATE 3 APAZUNERSEN 2 AVALOTCAGENE ONTAPARVOVEC 2 REBISUFLIGENE ETISPARVOVEC 2 SETRUSUMAB 2 2'-O, 4'-C-METHYLENE-P-THIO-ADENYLYL-(3'->5')-2'-O, 4'-C-METHYLENE-P-THIOGUANYLYL-(3'->5')-2'-O, 4'-C-METHYLENE-P-THIO-ADENYLYL-(3'->5')-2'-DEOXY-P-THIOADENYLYL-(3'->5')-2'-DEOXY-P-THIO-THYMIDYLYL-(3'->5')-2'-DEOXY-P-THIO-GUANYLYL-(3'->5')-2'-DEOXY-P-THIO-GUANYLYL-(3'->5')-2'-DEOXY-P-THIO-CYTIDYLYL-(3'->5')-2'-DEOXY-P-THIO-ADENYLYL-(3'->5')-2'-DEOXY-P-THIO-CYTIDYLYL-(3'->5')-2'-DEOXY-P-THIO-ADENYLYL-(3'->5')-2'-DEOXY-P-THIO-THYMIDYLYL-(3'->5')-2'-DEOXY-P-THIO-CYTIDYLYL-(3'->5')-2'-DEOXY-P-THIO-THYMIDYLYL-(3'->5')-2'-O, 4'-C-METHYLENE-5-METHYL-P-THIO-CYTIDYLYL-(3'->5')-2'-O, 4'-C-METHYLENE-5-METHYL-P-THIO-URIDYLYL-(3'->5')-2'-O, 4'-C-METHYLENE-5-METHYL-P-THIO-URIDYLYL-(3'->5')-2'-O, 4'-C-METHYLENEGUANOSINE 1 ADENO-ASSOCIATED VIRAL VECTOR SEROTYPE 9 ENCODING HUMAN ATP7B 1 CALCIUM CHLORIDE, GLUCOSE, ANHYDROUS, SODIUM HYDROGEN CARBONATE, POTASSIUM CHLORIDE, SODIUM CHLORIDE, SODIUM PHOSPHATE, MAGNESIUM SULFATE 1 NERIDRONATE SODIUM 1 PAMIDRONATE DISODIUM 1

All trials

14 total

Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With AS (Aspire) Recruiting

2024-512600-19-00 Phase III 4 countries 2'-O, 4'-C-METHYLENE-P-THIO-ADENYLYL-(3'->5')-2'-O, 4'-C-METHYLENE-P-THIOGUANYLY…
Setrusumab vs Bisphosphonates in Pediatric Subjects with Osteogenesis Imperfecta (Cosmic) Recruiting

2023-504196-24-00 Phase III 5 countries NERIDRONATE SODIUM; PAMIDRONATE DISODIUM; SETRUSUMAB; ZOLEDRONIC ACID
A Phase 1/2/3 Study of UX701 Gene Therapy in Adults with Wilson Disease”. Recruiting

2022-502873-40-00 Phase I (FiH) 5 countries ADENO-ASSOCIATED VIRAL VECTOR SEROTYPE 9 ENCODING HUMAN ATP7B; PREDNISOLONE
A Safety and Efficacy Study of GTX-102 in Subjects with Deletion- or Nondeletion-type Angelman Syndrome (Aurora) Recruiting

2024-519393-39-00 Phase II 3 countries APAZUNERSEN; SODIUM DIHYDROGEN PHOSPHATE DIHYDRATE, DISODIUM PHOSPHATE, POTASSIU…
GSDIa Disease Monitoring Program Recruiting

2023-510219-20-00 Phase III/IV 5 countries PARIGLASGENE BRECAPARVOVEC
A Long-term Extension Trial Investigating the Safety and Efficacy of GTX-102 in Patients with Angelman Syndrome Recruiting

2024-510917-14-00 Phase III 3 countries APAZUNERSEN; CALCIUM CHLORIDE, GLUCOSE, ANHYDROUS, SODIUM HYDROGEN CARBONATE, PO…
A Long-term Follow-up Study of Patients with MPS IIIA from Gene Therapy Clinical Trials Involving the Administration of ABO-102 (scAAV9.U1a.… Recruiting

2023-510392-66-00 Phase III 1 country REBISUFLIGENE ETISPARVOVEC
Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH for Mucopolysaccharidosis (MPS) IIIA Recruiting

2023-510032-37-00 Phase I (FiH) 1 country REBISUFLIGENE ETISPARVOVEC
A study to test product setrusumab in subjects with brittle bone syndrome. Ended

2024-510919-29-00 Phase II/III 6 countries SETRUSUMAB
A Study to Determine the Effect of Triheptanoin Compared with Even-chain MCT on MCEs in Pediatric Patients with LC-FAOD Ended

2023-509809-76-00 Phase III 4 countries TRIHEPTANOIN
A clinical study of a virus transferring the gene for human Ornithine Transcarbamylase (OTC) in patients 12 years of age and older with late… Ended

2024-514337-38-00 Phase III 6 countries AVALOTCAGENE ONTAPARVOVEC; PREDNISOLONE; SODIUM ACETATE (1-13C)
A clinical study to learn about the effects of a virus that transfers the gene for human Ornithine Transcarbamylase (OTC) in adults with lat… Ended

2022-501146-30-00 Phase I/II 2 countries AVALOTCAGENE ONTAPARVOVEC
An Phase 3 clinical study comparing a vector transferring the gene for Glucose-6-Phosphatase with placebo in adults with Glycogen Storage Di… Ended

2023-508750-25-00 Phase III 5 countries PARIGLASGENE BRECAPARVOVEC; PREDNISOLONE
A clinical study to learn about the effects of a virus that transfers the gene for Glucose- 6-Phosphatase (G6Pase) in adults with Glycogen S… Ended

2023-504004-29-00 Phase I/II 2 countries PARIGLASGENE BRECAPARVOVEC

Showing 1–14 of 14 trials

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