Trial · NCT04532047
PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)
NCT04532047, Phase 1, Recruiting, University of California, San Francisco
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders). Trials, record NCT04532047. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04532047
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)
- NCT number
- NCT04532047
- Status
- Recruiting
- Phases
- Phase 1
- Enrollment
- 10 participants, estimated
- Lead sponsor
- University of California, San Francisco
- Conditions
- MPS I, MPS II, MPS IVA, MPS VI, Mps VII, Gaucher Disease, Type 2, Gaucher Disease, Type 3, Pompe Disease Infantile-Onset, Wolman Disease
- Interventions
- Aldurazyme (laronidase)
- First posted
- 2020-08-31
- Last updated
- 2026-03-17
Source ClinicalTrials.gov
Registered outcomes6
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of participants with treatment-related adverse events as assessed by CTCAE v5.0. | 6 years | Adverse and serious adverse events including, but not limited to, death within 24 hours after the procedure, stillbirth, death prior to initial hospital discharge,increased response with antibody development above that expected with postnatal ERT, and serious related or serious unexpected adverse events exceeding those expected with the natural history of treated disease during the first five years of life, assessed by CTCAE v5.0. |
| Primary | Number of participants to receive the full initial, weight-based dose of enzyme replacement therapy through the fetal umbilical vein, and subsequent doses throughout the pregnancy. | 6 years | full dose administration compared to the need to halt the intervention prior to administration of a full dose. |
| Primary | Number of participants with the presence and levels of glycosaminoglycans (GAGs) in urine. | 6 years | Laboratory analysis of urine for GAG levels. |
| Primary | The number of participants with improvement or resolution of hydrops (if present). | 6 years | Improvement of hydrops via ultrasound and echocardiogram results (if present). |
| Secondary | Number of participants that show measured levels of antibodies against the enzyme. | 6 years | Laboratory analysis of blood to measure antibody levels. |
| Other | Number of participants that show functional cardiac, growth, mobility, and neurocognitive function. | 6 years | ecogardiogram, skeletal survey, neurocognitve assessments such as Bayley III to assess cardiac, growth, mobility and neurocognitive function. |
Source ClinicalTrials.gov
Outcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
Papers11
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04532047
GET https://conductscience.org/api/trials/v1/NCT04532047/registered