Enzyme Replacement Therapy for Rare Metabolic Disorder

First-in-human Phase I dose-escalation study of a recombinant enzyme replacement therapy for a rare inherited metabolic enzyme deficiency.

Duration

24 weeks

Visits

14 visits

Compensation

Up to $2,500

Study Overview

This open-label, dose-escalation Phase I study evaluates the safety, tolerability, pharmacokinetics, and preliminary efficacy of a recombinant enzyme replacement therapy in adults with confirmed enzyme deficiency. Participants will receive IV infusions every 2 weeks. Doses will be escalated in cohorts following safety review. Primary endpoint is safety and tolerability through Week 24.

Eligibility Criteria

You May Qualify If:

Adults aged 18–65
Confirmed diagnosis via enzymatic assay and/or genetic testing
Clinically stable at screening
Adequate organ function

You May Not Qualify If:

Prior enzyme replacement therapy within 6 months
Active serious infection
Significant hepatic or renal impairment
Known allergy to recombinant proteins

Study Locations

Tampa Main Office

Apply for This Study

Complete this brief form and our team will contact you within 1 business day.

Questions? Call us directly:

(813) 815-7327

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