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New treatment option for MPS I
Official title PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)
ClinicalTrials.gov ID: NCT04532047
What this study is testing
What is Aldurazyme (laronidase)?
Aldurazyme (laronidase) is an investigational medicine, being studied as a potential treatment for mps i.
Also referred to as Elaprase (idursulfase), Vimizim (elosulfase alfa).
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- For detailed information, please view our study website: https://pearltrial.ucsf.edu/ The investigators aims to determine the the maternal and fetal safety and feasibility of in utero fetal enzyme replacement therapy in fetuses with Lysosomal Storage Diseases.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 50, women only
You may be able to join if
- Live male or female fetuses at 18 0/7 weeks to 34 6/7 weeks gestation
- Diagnosis of one of the 8 included LSDs in utero by genetic or enzymatic analyses performed on amniotic fluid, fetal blood, placental tissue, or...
- Pregnant women age 18 years to 50 years, carrying a live male or female fetus at 18 0/7 weeks to 34 6/7 weeks gestation
- Identified through the above listed means to be carrying a fetus with an LSD.
- Ability to give written informed consent and comply with the requirements of the study.
You likely can't join if
- Fetuses with a concurrent severe structural anomaly
- Fetuses with an additional pathogenic genetic variant not related to the underlying LSD that contribute a significant risk of morbidity or mortality...
- Women with one or more significant comorbidities that would preclude fetal intervention including, but not limited to:
- inability to complete the procedure secondary to maternal body habitus or placental location
- significant cardiopulmonary disease
- mirror syndrome
See the full eligibility criteria
- Live male or female fetuses at 18 0/7 weeks to 34 6/7 weeks gestation
- Diagnosis of one of the 8 included LSDs in utero by genetic or enzymatic analyses performed on amniotic fluid, fetal blood, placental tissue, or other samples through chorionic villus sampling (CVS), amniocentesis...
- Pregnant women age 18 years to 50 years, carrying a live male or female fetus at 18 0/7 weeks to 34 6/7 weeks gestation
- Identified through the above listed means to be carrying a fetus with an LSD.
- Ability to give written informed consent and comply with the requirements of the study.
- Fetuses with a concurrent severe structural anomaly
- Fetuses with an additional pathogenic genetic variant not related to the underlying LSD that contribute a significant risk of morbidity or mortality. Hydrops fetalis will not be an exclusion criterion because ERT has...
- Women with one or more significant comorbidities that would preclude fetal intervention including, but not limited to:
- inability to complete the procedure secondary to maternal body habitus or placental location
- significant cardiopulmonary disease
- mirror syndrome
- end organ failure
- altered mental status
- placental abruption
- active preterm labor
- preterm premature rupture of membranes.
- Mother will require therapeutic dosing of anticoagulation within 24 hours prior to or following the intervention.
The study team makes the final eligibility decision.
Where it's taking place
- San Francisco, California, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling female, 18 years to 50 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include San Francisco, California, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.