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Enzyme Replacement Therapy

In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases (IUERT Trial)

Phase 1

Recruiting

Led By Tippi MacKenzie, MD

Research Sponsored by University of California, San Francisco

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

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

Live male or female fetuses at 18 0/7 weeks to 34 6/7 weeks gestation

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 6 years

Awards & highlights

IUERT Trial Summary

This trial will test if a baby with a Lysosomal Storage Disease can be safely treated in the womb.

Who is the study for?

This trial is for pregnant women aged 18-50 with fetuses diagnosed with certain Lysosomal Storage Diseases (LSDs) between 18 to nearly 35 weeks of gestation. Candidates must be able to consent and follow study requirements. Exclusions include mothers needing anticoagulation around the time of treatment, additional fetal genetic risks, or significant maternal health issues.Check my eligibility

What is being tested?

The trial tests the safety and feasibility of administering Aldurazyme (laronidase), an enzyme replacement therapy, directly to a fetus in utero when diagnosed with LSDs like Gaucher Disease or Pompe Disease.See study design

What are the potential side effects?

While specific side effects for in utero treatment are not detailed here, Aldurazyme may generally cause allergic reactions, breathing difficulties, fever, and blood pressure changes in other contexts.

IUERT Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am a pregnant woman aged 18-50, between 18 to 34 weeks of pregnancy.

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I am pregnant with a living fetus between 18 to 34 weeks.

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My unborn baby has been diagnosed with a lysosomal storage disorder.

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My unborn child has been diagnosed with a lysosomal storage disorder.

IUERT Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 6 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~6 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and 6 years for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

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.

Number of participants with the presence and levels of glycosaminoglycans (GAGs) in urine.

Number of participants with treatment-related adverse events as assessed by CTCAE v5.0.

+1 more

Secondary outcome measures

Number of participants that show measured levels of antibodies against the enzyme.

Other outcome measures

Number of participants that show functional cardiac, growth, mobility, and neurocognitive function.

IUERT Trial Design

1Treatment groups

Experimental Treatment

Group I: Experimental: in utero enzyme replacement therapyExperimental Treatment1 Intervention

ERT will be delivered in utero. Typically, the target of the procedure to administer in utero ERT will be the umbilical vein near the insertion of the umbilical cord into the placenta. The dose of the ERT will be dependent on the specific disease process and enzyme being replaced, and the estimated weight of the fetus. The dosage will be the same as the recommended weight-based postnatal dosing, adjusted for estimated fetal weight. IUERT will be repeated every 2-4 weeks, which is an interval consistent with the standard of care for IUTs (every 2-4 weeks) to avoid excessive access through the umbilical vein. This interval is also consistent with the half-life of each relevant enzyme.

0 / 4Eligibility criteria met

Find a Location

Who is running the clinical trial?

University of California, San FranciscoLead Sponsor

2,506 Previous Clinical Trials

15,238,545 Total Patients Enrolled

Duke UniversityOTHER

2,363 Previous Clinical Trials

3,420,506 Total Patients Enrolled

Tippi MacKenzie, MDPrincipal InvestigatorUniversity of California, San Francisco

Media Library

Aldurazyme (laronidase) (Enzyme Replacement Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT04532047 — Phase 1

Myocardial Perfusion Imaging Research Study Groups: Experimental: in utero enzyme replacement therapy

Myocardial Perfusion Imaging Clinical Trial 2023: Aldurazyme (laronidase) Highlights & Side Effects. Trial Name: NCT04532047 — Phase 1

Aldurazyme (laronidase) (Enzyme Replacement Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04532047 — Phase 1

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is the age criterion for this research trial inclusive of those aged 70 or higher?

"The minimum age for this trial is 18 years old, and the maximum age requirement is 50. However, there are 26 studies available to those that fall below the designated threshold, as well as 28 trials open to patients above 65."

Answered by AI

Who is eligible to be a participant in this experiment?

"This clinical trial seeks to enroll 10 individuals, aged 18-50 years old, with myocardial perfusion imaging. Notably, pregnant women of a similar age range carrying a live fetus between 18 0/7 and 34 6/7 weeks gestation will also be considered for the study."

Answered by AI

Are there possible adverse effects to using Aldurazyme (laronidase) therapeutically?

"Minimal clinical safety data exists for Aldurazyme (laronidase), thus our team has allocated a score of 1 on the risk scale."

Answered by AI

How many participants are being monitored in this clinical experiment?

"Affirmative. According to information hosted on clinicaltrials.gov, this medical study was first broadcasted on July 1st 2021 and has since been updated as recently as September 16th 2022. It is looking for 10 individuals from one centre."

Answered by AI

Is enrollment available for this investigation?

"Data sourced from clinicaltrials.gov confirms that the trial is still enlisting participants and was most recently updated on September 16th 2022, after being posted to the site on July 1st 2021."

Answered by AI

Has something like this been tried before?

"Presently, 11 clinical trials of Aldurazyme (laronidase) are in progress across 61 cities and 33 countries. The earliest such trial was run a decade ago by Shire Corporation, involving 25 participants through Phase 1 & 2 assessments. Subsequently, 61 studies have been finalized since then."

Answered by AI

What indications are most commonly associated with Aldurazyme (laronidase) therapy?

"The efficacy of Aldurazyme (laronidase) in managing the manifestations of Hurler-Scheie Syndrome, Mucopolysaccharidosis VII on the Central Nervous System, and Hurler's Syndrome remains to be established."

Answered by AI

Has Aldurazyme (laronidase) been tested before in other scientific studies?

"Currently, 11 medical studies for Aldurazyme (laronidase) are in progress with 4 of them being in the third phase. The majority of these trials focus on Durham, North carolina however there are 169 other sites running tests as well."

Answered by AI

Recent research and studies

Science Translational MedicineJournal

Tolerance Induction and Microglial Engraftment After Fetal Therapy Without Conditioning in Mice with Mucopolysaccharidosis Type VII

Nguyen, Quoc-Hung, Russell G. Witt, Bowen Wang, Carlo Eikani, Jeremy Shea, Lucas K. Smith, Gabrielle Boyle, et al.. 2020. “Tolerance Induction and Microglial Engraftment After Fetal Therapy Without Conditioning in Mice with Mucopolysaccharidosis Type VII”. Science Translational Medicine. American Association for the Advancement of Science (AAAS). doi:10.1126/scitranslmed.aay8980.

JamaJournal

Prevalence of Lysosomal Storage Disorders

Meikle, Peter J.. 1999. “Prevalence of Lysosomal Storage Disorders”. Jama. American Medical Association (AMA). doi:10.1001/jama.281.3.249.

Nature Reviews Disease PrimersJournal

Author Correction: Lysosomal Storage Diseases

Platt, Frances M., Alessandra d’Azzo, Beverly L. Davidson, Elizabeth F. Neufeld, and Cynthia J. Tifft. 2018. “Author Correction: Lysosomal Storage Diseases”. Nature Reviews Disease Primers. Springer Science and Business Media LLC. doi:10.1038/s41572-018-0037-0.

Clinical GeneticsJournal