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SOCIETY FOR INHERITED METABOLIC DISORDERS |
You are invited to a
virtual information session regarding Prenatal Enzyme Replacement Therapy for
Lysosomal Storage Diseases scheduled for Thursday, April 27, 2023,
8:00-9:30 AM PDT (check
your time zone).
We will present
preliminary results from a phase 1 clinical trial (NCT04532047)
to treat eight lysosomal storage diseases prenatally by infusing the fetus with
weight-adjusted doses of recombinant enzymes currently approved for use in
pediatric patients.
Conditions include
MPS 1, 2, 4a, 6, 7; Gaucher Disease Type 2 and 3; Infantile-onset Pompe Disease;
and Wolman Disease.
In this session,
Dr. Tippi MacKenzie, Professor of Surgery at the University of California, San
Francisco, and colleagues from UCSF will discuss the following:
·
Preclinical studies for in utero enzyme
replacement therapy (IUERT)
·
Rationale and Investigational New Drug
approval for IUERT
·
Inclusion/exclusion criteria and the role
of the Enrollment Advisory Board
·
Clinical protocol and care of patients
through the trial
·
Case review of the first patient treated
using the IUERT clinical protocol (Infantile-onset Pompe Disease)
·
Preliminary information on two additional
patients treated with IUERT
·
Considerations for in utero infusions of
ERT via the umbilical vein
·
Considerations for postnatal care,
including clinical trials of gene therapies
Register
and join us on April 27 to learn about
this innovative approach to treat lysosomal storage diseases before birth.
Please share
this announcement with colleagues and
patients who might be interested in learning more. All are welcome to
attend.
Best regards,
Tippi MacKenzie, MD
Professor of Surgery
Benioff UCSF Professor in Children’s Health
Director, Eli and Edythe Broad Center of Regeneration Medicine and
Stem Cell Research at UCSF
Co-Director, Center for Maternal-Fetal Precision Medicine
John G. Bowes Distinguished Professor in Stem Cell and Tissue
Biology
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Clinical Trial Referrals
NCT04532047: In Utero Enzyme Replacement Therapy for Lysosomal
Storage Diseases (IUERT) We anticipate that we will
identify patients in families that already know their carrier status.
Please contact us if you identify a pregnancy affected by MPS 1, 2, 4a,
6, 7; Gaucher Disease Type 2, 3; Infantile-onset Pompe Disease; or
Wolman Disease; or a family that is considering a repeat pregnancy. Funds are available to
host families in San Francisco and to cover the research costs of the
trial. We anticipate many costs will be covered by insurance as
well. To refer a patient, contact
the study team at
fetaltreatmentcenter@ucsf.edu
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