deltatrials
Terminated PHASE1/PHASE2 INTERVENTIONAL 5-arm NCT04581785

Gene Therapy With hLB-001 in Pediatric Patients With Severe Methylmalonic Acidemia (SUNRISE)

A Phase 1/2 Open-label Clinical Study of hLB-001 Gene Therapy in Pediatric Patients With Methylmalonic Acidemia Characterized by MMUT Mutations

Sponsor: Alexion Pharmaceuticals, Inc.

Conditions Methylmalonic Acidemia
Interventions hLB-001
Updated 14 times since 2020 Last updated: Feb 21, 2024 Started: May 29, 2021 Primary completion: Jan 10, 2023 Completion: Jan 10, 2023
This information is for research purposes only and is not medical advice. Consult a healthcare provider before making any medical decision.

Terminated

Due to low likelihood of clinical benefit in treated participants.

Other terminated trials from Alexion Pharmaceuticals, Inc.

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Listed as NCT04581785, this PHASE1/PHASE2 trial focuses on Methylmalonic Acidemia and remains terminated or withdrawn. Sponsored by Alexion Pharmaceuticals, Inc., it has been updated 14 times since 2021, reflecting substantial change activity. This study adds to the evidence base for this therapeutic area through structured, versioned documentation.

Status Flow

~Nov 2020 – ~Jan 2021 · 2 months · monthly snapshot~Jan 2021 – ~Mar 2021 · 59 days · monthly snapshot~Mar 2021 – ~May 2021 · 2 months · monthly snapshot~May 2021 – ~Sep 2021 · 4 months · monthly snapshot~Sep 2021 – ~Sep 2022 · 12 months · monthly snapshot~Sep 2022 – ~Jan 2023 · 4 months · monthly snapshot~Jan 2023 – ~Mar 2023 · 59 days · monthly snapshot~Mar 2023 – ~Apr 2023 · 31 days · monthly snapshot~Apr 2023 – ~May 2023 · 30 days · monthly snapshot~May 2023 – ~Dec 2023 · 7 months · monthly snapshot~Dec 2023 – ~Mar 2024 · 3 months · monthly snapshot~Mar 2024 – ~Jul 2024 · 4 months · monthly snapshot~Jul 2024 – ~Sep 2024 · 2 months · monthly snapshot~Sep 2024 – present · 22 months · monthly snapshot

Change History

14 versions recorded

  1. Sep 2024 — Present [monthly]

    Terminated PHASE1/PHASE2

  2. Jul 2024 — Sep 2024 [monthly]

    Terminated PHASE1/PHASE2

    Phase: PHASE1_PHASE2 → PHASE1/PHASE2

  3. Mar 2024 — Jul 2024 [monthly]

    Terminated PHASE1_PHASE2

  4. Dec 2023 — Mar 2024 [monthly]

    Terminated PHASE1_PHASE2

  5. May 2023 — Dec 2023 [monthly]

    Terminated PHASE1_PHASE2

    Status: Active Not Recruiting → Terminated

▶ Show 9 earlier versions

  1. Apr 2023 — May 2023 [monthly]

    Active Not Recruiting PHASE1_PHASE2

  2. Mar 2023 — Apr 2023 [monthly]

    Active Not Recruiting PHASE1_PHASE2

    Status: Recruiting → Active Not Recruiting

  3. Jan 2023 — Mar 2023 [monthly]

    Recruiting PHASE1_PHASE2

  4. Sep 2022 — Jan 2023 [monthly]

    Recruiting PHASE1_PHASE2

  5. Sep 2021 — Sep 2022 [monthly]

    Recruiting PHASE1_PHASE2

  6. May 2021 — Sep 2021 [monthly]

    Recruiting PHASE1_PHASE2

  7. Mar 2021 — May 2021 [monthly]

    Recruiting PHASE1_PHASE2

    Status: Not Yet Recruiting → Recruiting

  8. Jan 2021 — Mar 2021 [monthly]

    Not Yet Recruiting PHASE1_PHASE2

  9. Nov 2020 — Jan 2021 [monthly]

    Not Yet Recruiting PHASE1_PHASE2

    First recorded

Eligibility Summary

No eligibility information available.

Contact Information

Sponsor contact:
  • Alexion Pharmaceuticals, Inc.
  • LogicBio Therapeutics, Inc
Data source: Alexion Pharmaceuticals, Inc.

For direct contact, visit the study record on ClinicalTrials.gov .

Study Locations