Biotechnology company · RARE

Ultragenyx Pharmaceutical
clinical trial
pipeline

Registry checked

Published

setrusumab is linked to 3 distinct active studies, with 3 Phase 3. Apazunersen Sodium is linked to 3 distinct active studies, with 1 Phase 2, 2 Phase 3.

Phase 3 study NCT06636071 in Osteogenesis Imperfecta lists estimated primary completion in January 2027. Phase 3 study NCT04360265 in Mucopolysaccharidosis IIIA and MPS IIIA lists estimated primary completion in August 2027. These are registry estimates of primary completion, not confirmed results announcements or overall study completion.

Active trial mix

Lead-sponsor scope

19active trials
as lead sponsor
  • Phase 10
  • Phase 23
  • Phase 39
  • Phase 40
  • Phase not recorded7

One highest explicitly recorded phase per active trial. Combined phases use the highest phase. Phase not recorded is shown separately.

Change history

Follow the changes behind the company’s clinical programme.

02 Oct 2026 – 31 Oct 2026 · UTC

Recorded changes

21 trials30 completed days
Recruitment statusPrimary completionEnrolmentFirst observedOther

Verified live registry-derived observations in completed UTC days. Recorded dates are observation dates, not necessarily registry amendment dates. Zero means no eligible observations recorded, not daily monitoring of every trial.

0 archive observations and 0 unverified observations excluded.

View exact daily values and type totals
Recorded observations in completed UTC days
Date (UTC)Recruitment statusPrimary completionEnrolmentFirst observedOtherTotalAffected trials
2026-10-020000000
2026-10-030000000
2026-10-040000000
2026-10-050000000
2026-10-060000000
2026-10-070000000
2026-10-080000000
2026-10-090000000
2026-10-100000000
2026-10-110000000
2026-10-120000000
2026-10-130000000
2026-10-140000000
2026-10-150000000
2026-10-160000000
2026-10-170000221
2026-10-180000000
2026-10-190000000
2026-10-200000000
2026-10-210000000
2026-10-220000000
2026-10-230000000
2026-10-240000000
2026-10-250000000
2026-10-260000000
2026-10-270000000
2026-10-280000000
2026-10-290000000
2026-10-300000000
2026-10-310000000
Type totals0000221 distinct

02 / Drugs & programmes

Explore the pipeline

5 selected programmes

Choose a drug to open its record in the tracker. Sign in to continue.

Apazunersen Sodium

Canonical investigational asset

3
Active trials
0
Recruiting trials
Trial phases
Highest: Phase 3
  • Phase 10
  • Phase 21
  • Phase 32
  • Phase 40
  • Phase not recorded0
Studied condition
Angelman Syndrome
Next primary completion
Not reported
Open drug

setrusumab

Canonical investigational asset

3
Active trials
0
Recruiting trials
Trial phases
Highest: Phase 3
  • Phase 10
  • Phase 20
  • Phase 33
  • Phase 40
  • Phase not recorded0
Studied condition
Osteogenesis Imperfecta
Next primary completion
Jan 2027 · estimated
Open drug

Rebisufligene Etisparvovec

Canonical investigational asset

1
Active trials
1
Recruiting trials
Trial phases
Highest: Phase 3
  • Phase 10
  • Phase 20
  • Phase 31
  • Phase 40
  • Phase not recorded0
Studied condition
MPS IIIA · Mucopolysaccharidosis III · Sanfilippo A
Next primary completion
Not reported
Open drug

Triheptanoin

dojolvi

1
Active trials
0
Recruiting trials
Trial phases
Highest: Phase 3
  • Phase 10
  • Phase 20
  • Phase 31
  • Phase 40
  • Phase not recorded0
Studied condition
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
Next primary completion
Aug 2027 · estimated
Open drug

UX016

Canonical investigational asset

1
Active trials
1
Recruiting trials
Trial phases
Highest: Phase 2
  • Phase 10
  • Phase 21
  • Phase 30
  • Phase 40
  • Phase not recorded0
Studied condition
GNE Myopathy
Next primary completion
Not reported
Open drug

9 of 19 active trials have accepted investigational programme links. Trial context does not establish ownership or complete drug coverage.

5 selected programmes · accepted investigational links cover 9 of 19 active trials · brand names checked where available.

Accepted links do not establish complete intervention mapping or ownership. Programme populations overlap.

Up to six canonical assets identified through accepted investigational links in active trials. Counts describe this trial context and do not establish ownership or a complete corporate drug portfolio. Opening a record requires sign-in and does not start tracking.

03 / Clinical trial timeline

Ultragenyx Pharmaceutical trials in progress

Registry start → study completion

◇ Recorded observation│ Primary completion milestoneHatched ends retain month / year uncertaintyHover, focus or tap an observation
Trial / programme
6 selected Phase 3 / 2 trials
2021202220232024202520262027202820292030
Setrusumab in Pediatric Japanese Subjects With Osteogenesis Imperfecta
setrusumab

NCT06636071

Phase 3 · Active, not recruiting

Trial source and qualifications

Setrusumab in Pediatric Japanese Subjects With Osteogenesis Imperfecta

Duration ends at independently recorded study completion. Registry primary completion, not a confirmed results announcement. Observation markers describe recorded observations, not scheduled completion dates. Up to three newest eligible verified observations from a bounded trailing 24-month candidate window are shown, not full history. 3 inspected observations were not displayed.

3 candidate observations · 0 shown · 0 recorded after primary completion · 3 unverified · 0 invalid dates · 0 before supported start · 0 outside horizon · 0 excluded by display cap · source retrieval not capped.

ClinicalTrials.gov source

3 inspected observations omitted. At most three verified observations are shown, not full history.

    Start: 25 Oct 2024 · actual
    Primary completion: Jan 2027 · estimated
    Study completion: Jan 2028 · estimated

    2021202220232024202520262027202820292030
    Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA
    Asset mapping not recorded

    NCT04360265

    Phase 3 · Enrolling by invitation

    Trial source and qualifications

    Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA

    Duration ends at independently recorded study completion. Source date qualifications: start-clipped-at-horizon. Registry primary completion, not a confirmed results announcement. Observation markers describe recorded observations, not scheduled completion dates. Up to three newest eligible verified observations from a bounded trailing 24-month candidate window are shown, not full history. 3 inspected observations were not displayed.

    Start clipped at the display horizon.

    3 candidate observations · 0 shown · 0 recorded after primary completion · 3 unverified · 0 invalid dates · 0 before supported start · 0 outside horizon · 0 excluded by display cap · source retrieval not capped.

    ClinicalTrials.gov source

    3 inspected observations omitted. At most three verified observations are shown, not full history.

      Start: 28 Sept 2020 · actual
      Primary completion: Aug 2027 · estimated
      Study completion: Aug 2027 · estimated

      2021202220232024202520262027202820292030
      A Study to Determine the Effect of Triheptanoin Compared With Even-Chain MCT on MCEs in Pediatric Patients With LC-FAOD
      Triheptanoin

      NCT05933200

      Phase 3 · Active, not recruiting

      Trial source and qualifications

      A Study to Determine the Effect of Triheptanoin Compared With Even-Chain MCT on MCEs in Pediatric Patients With LC-FAOD

      Duration ends at independently recorded study completion. Registry primary completion, not a confirmed results announcement. Observation markers describe recorded observations, not scheduled completion dates. Up to three newest eligible verified observations from a bounded trailing 24-month candidate window are shown, not full history. 5 inspected observations were not displayed.

      5 candidate observations · 0 shown · 0 recorded after primary completion · 5 unverified · 0 invalid dates · 0 before supported start · 0 outside horizon · 0 excluded by display cap · source retrieval not capped.

      ClinicalTrials.gov source

      5 inspected observations omitted. At most three verified observations are shown, not full history.

        Start: 28 Feb 2023 · actual
        Primary completion: Aug 2027 · estimated
        Study completion: Aug 2027 · estimated

        2021202220232024202520262027202820292030
        Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency
        Asset mapping not recorded

        NCT05345171

        Phase 3 · Active, not recruiting

        Trial source and qualifications

        Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency

        Duration ends at independently recorded study completion. Source date qualifications: end-clipped-at-horizon. Registry primary completion, not a confirmed results announcement. Observation markers describe recorded observations, not scheduled completion dates. Up to three newest eligible verified observations from a bounded trailing 24-month candidate window are shown, not full history. 5 inspected observations were not displayed.

        End clipped at the display horizon.

        5 candidate observations · 0 shown · 0 recorded after primary completion · 5 unverified · 0 invalid dates · 0 before supported start · 0 outside horizon · 0 excluded by display cap · source retrieval not capped.

        ClinicalTrials.gov source

        5 inspected observations omitted. At most three verified observations are shown, not full history.

          Start: 18 Oct 2022 · actual
          Primary completion: Sept 2027 · estimated
          Study completion: Mar 2031 · estimated

          2021202220232024202520262027202820292030
          First-in-human Study of UX016 in GNEM
          UX016

          NCT07511556

          Phase 2 · Recruiting

          Trial source and qualifications

          First-in-human Study of UX016 in GNEM

          Duration ends at independently recorded study completion. Registry primary completion, not a confirmed results announcement. Observation markers describe recorded observations, not scheduled completion dates. Up to three newest eligible verified observations from a bounded trailing 24-month candidate window are shown, not full history. 2 inspected observations were not displayed.

          2 candidate observations · 0 shown · 0 recorded after primary completion · 1 unverified · 0 invalid dates · 1 before supported start · 0 outside horizon · 0 excluded by display cap · source retrieval not capped.

          ClinicalTrials.gov source

          2 inspected observations omitted. At most three verified observations are shown, not full history.

            Start: Oct 2026 · estimated
            Primary completion: Dec 2028 · estimated
            Study completion: Dec 2028 · estimated

            2021202220232024202520262027202820292030
            Long-term Extension of GTX-102 in Angelman Syndrome
            Apazunersen Sodium

            NCT06415344

            Phase 3 · Active, not recruiting

            Trial source and qualifications

            Long-term Extension of GTX-102 in Angelman Syndrome

            Duration ends at independently recorded study completion. Registry primary completion, not a confirmed results announcement. Observation markers describe recorded observations, not scheduled completion dates. Up to three newest eligible verified observations from a bounded trailing 24-month candidate window are shown, not full history. 1 inspected observations were not displayed.

            2 candidate observations · 1 shown · 0 recorded after primary completion · 1 unverified · 0 invalid dates · 0 before supported start · 0 outside horizon · 0 excluded by display cap · source retrieval not capped.

            ClinicalTrials.gov source

            1 inspected observations omitted. At most three verified observations are shown, not full history.

            • status flip · Recorded 2026-09-09 · Verified live registry-derived observation. Recorded date is not necessarily the registry amendment date. Observations outside the duration bar do not extend the study.

            Start: 31 Jul 2024 · actual
            Primary completion: Feb 2029 · estimated
            Study completion: Feb 2029 · estimated

            2021202220232024202520262027202820292030

            Up to six active Phase 2/3 trials with upcoming estimated primary-completion intervals, ordered by nearest interval, highest phase and NCT. Historical dates never pad this selection. Ten calendar years are shown. Dates retain registry precision and independent date types.

            6 selected of 9 eligible trials · 0 without supported dates · 0 omitted for contradictory chronology. Excluded: 3 actual primary completions, 0 untyped primary completions, 0 past estimates, 0 outside the horizon, 0 contradictory dates.

            04 / Therapeutic focus

            Where the activity is

            4

            active trials in Genetic & Rare Disease

            Primary therapeutic groups are indexed enrichment. Unmapped trials and healthy volunteers remain in the active-trial denominator.

            Active trials by primary therapeutic area

            • Genetic & Rare Disease4 trials
            • Metabolic/Endocrine/Nutrition3 trials
            • Pain & Musculoskeletal3 trials
            • Unmapped9 trials
            • Healthy volunteers0 trials

            Each active trial counts once. Full bar equals all active trials in this lead-sponsor scope. This grouping does not describe commercial strategy.

            06 / Related companies

            All companies

            No supported overlap was identified in the selected programme context. Based on the selected programmes and indexed lead-sponsor records, not a complete ranking or a competitive/ownership assessment.

            Sources, scope and methodology

            ClinicalTrials.gov lead-sponsor scope

            Canonical current-owner attribution covers 63 distinct NCT records attributed through the canonical sponsor map. Included canonical entities: Ultragenyx Pharmaceutical Inc. Phrases retrieve candidates only, then verbatim lead-sponsor attribution determines inclusion.

            ClinicalTrials.gov

            Attribution coverage

            63 distinct registry candidates produced 63 accepted studies. 0 mapped to other owners, 0 lacked a canonical mapping and 0 fell outside an operating subset. Accepted mappings include 63 unverified, 0 identity-only and 0 device-tagged trial memberships. Mapping flags describe evidence quality, not a separate ownership authority.

            Indexed enrichment

            42 scoped records have indexed enrichment and 21 do not. Missing enrichment does not remove a trial from cohort counts.

            Scope observation

            The successful attribution check completed at 2026-11-01T01:19:50.217Z. This is a dated canonical-attribution snapshot. Static pages normally regenerate after 24 hours, but cached HTML can outlive that interval when regeneration fails. Map changes affect the next successful regeneration. This timestamp is separate from individual registry-update dates. Snapshot a16b4598e6c1bc68ef3ed68a273a7f241dd860d2a324d216be93c367b0c1b45c.

            Active means recruiting, not yet recruiting, active not recruiting or enrolling by invitation. Recruiting counts recruiting only. Trial counts describe lead sponsorship under the published canonical-attribution policy. Identified programmes and therapeutic groups depend on available enrichment. Drug, device and other clinical studies may be included. This is the mapped company scope, not an exhaustive corporate trial census.

            Dated canonical-attribution snapshot: 63 mapped lead-sponsored clinical studies across all statuses. 42 have indexed enrichment, 21 do not. Drug, device and other studies may be represented. Sponsor tags do not classify trial interventions. This is the mapped company scope, not an exhaustive corporate trial census. Recorded changes may reflect observation or archive ingestion rather than a new registry amendment.

            Keep following
            what matters.

            Choose the drugs, companies or disease areas you follow. Bring trial changes and upcoming readouts into one watch.

            Build your watch

            Choose what to track in the watch builder.