deltatrials
Recruiting PHASE1 INTERVENTIONAL 1-arm NCT04532047

PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders) (PEARL)

Sponsor: Duke University

Conditions Gaucher Disease, Type 2 Gaucher Disease, Type 3 MPS I MPS II MPS IVA MPS VI Mps VII Pompe Disease Infantile-Onset Wolman Disease
Interventions Aldurazyme (laronidase)
Updated 13 times since 2020 Last updated: Mar 6, 2025 Started: Jul 1, 2021 Primary completion: Jul 31, 2031 Completion: Jul 31, 2032
This information is for research purposes only and is not medical advice. Consult a healthcare provider before making any medical decision.

A PHASE1 clinical study on Gaucher Disease, Type 2 and Gaucher Disease, Type 3, this trial is actively recruiting participants. The trial is conducted by Duke University and has accumulated 13 data snapshots since 2021. Longitudinal tracking of this trial contributes to a broader understanding of treatment development timelines.

Status Flow

~Sep 2020 – ~Nov 2020 · 2 months · monthly snapshot~Nov 2020 – ~Jan 2021 · 2 months · monthly snapshot~Jan 2021 – ~Feb 2021 · 31 days · monthly snapshot~Feb 2021 – ~Apr 2021 · 59 days · monthly snapshot~Apr 2021 – ~May 2021 · 30 days · monthly snapshot~May 2021 – ~May 2022 · 12 months · monthly snapshot~May 2022 – ~Sep 2022 · 4 months · monthly snapshot~Sep 2022 – ~Oct 2023 · 13 months · monthly snapshot~Oct 2023 – ~Jul 2024 · 9 months · monthly snapshot~Jul 2024 – ~Sep 2024 · 2 months · monthly snapshot~Sep 2024 – ~Feb 2025 · 5 months · monthly snapshot~Feb 2025 – ~Apr 2025 · 59 days · monthly snapshot~Apr 2025 – present · 14 months · monthly snapshot

Change History

13 versions recorded

  1. Apr 2025 — Present [monthly]

    Recruiting PHASE1

  2. Feb 2025 — Apr 2025 [monthly]

    Recruiting PHASE1

  3. Sep 2024 — Feb 2025 [monthly]

    Recruiting PHASE1

  4. Jul 2024 — Sep 2024 [monthly]

    Recruiting PHASE1

  5. Oct 2023 — Jul 2024 [monthly]

    Recruiting PHASE1

▶ Show 8 earlier versions

  1. Sep 2022 — Oct 2023 [monthly]

    Recruiting PHASE1

  2. May 2022 — Sep 2022 [monthly]

    Recruiting PHASE1

  3. May 2021 — May 2022 [monthly]

    Recruiting PHASE1

    Status: Not Yet Recruiting → Recruiting

  4. Apr 2021 — May 2021 [monthly]

    Not Yet Recruiting PHASE1

  5. Feb 2021 — Apr 2021 [monthly]

    Not Yet Recruiting PHASE1

  6. Jan 2021 — Feb 2021 [monthly]

    Not Yet Recruiting PHASE1

  7. Nov 2020 — Jan 2021 [monthly]

    Not Yet Recruiting PHASE1

  8. Sep 2020 — Nov 2020 [monthly]

    Not Yet Recruiting PHASE1

    First recorded

Eligibility Summary

No eligibility information available.

Contact Information

Sponsor contact:
  • Duke University
  • University of California, San Francisco
Data source: University of California, San Francisco

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

Study Locations