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Enzyme Replacement Therapy

Pegtarviliase Cohort 1 for Homocystinuria

Phase 1 & 2
Waitlist Available
Led By Reena Sharma, MD
Research Sponsored by Aeglea Biotherapeutics
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Timeline
Screening 3 weeks
Treatment Varies
Follow Up at pre-dose, 1hour, 6hours, 24hours, 48hours, 72hours, 96hours and 120hours
Awards & highlights

Study Summary

This trial will test a new drug for safety and how well it is tolerated in people with a rare genetic disorder that causes high levels of a substance called homocysteine.

Eligible Conditions
  • Homocystinuria

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~at pre-dose, 1hour, 6hours, 24hours, 48hours, 72hours, 96hours and 120hours
This trial's timeline: 3 weeks for screening, Varies for treatment, and at pre-dose, 1hour, 6hours, 24hours, 48hours, 72hours, 96hours and 120hours for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Incidence of treatment-emergent adverse events
Secondary outcome measures
Changes in total plasma homocysteine after treatment with pegtarviliase
Pharmacokinetic Profile of IV pegtarviliase AUC
Pharmacokinetic Profile of IV pegtarviliase Cmax
+7 more

Trial Design

5Treatment groups
Experimental Treatment
Group I: Pegtarviliase Cohort 5Experimental Treatment1 Intervention
Optional cohort for up to 12 subjects ≥12 years of age (≥18 in the US) dosing at Dose E weekly for a total of 13 doses
Group II: Pegtarviliase Cohort 4Experimental Treatment1 Intervention
Planned for 4 subjects ≥12 years of age (≥18 in the US) dosing at Dose D weekly for a total of 4 doses
Group III: Pegtarviliase Cohort 3Experimental Treatment1 Intervention
Planned for 4 subjects ≥12 years of age (≥18 in the US) dosing at Dose C weekly for a total of 4 doses
Group IV: Pegtarviliase Cohort 2Experimental Treatment1 Intervention
Planned for 4 subjects ≥12 years of age dosing at Dose B weekly for a total of 4 doses
Group V: Pegtarviliase Cohort 1Experimental Treatment1 Intervention
Planned for 4 subjects ≥18 years of age dosing at Dose A weekly for a total of 4 doses

Find a Location

Who is running the clinical trial?

Aeglea BiotherapeuticsLead Sponsor
7 Previous Clinical Trials
260 Total Patients Enrolled
Cortney CaudillStudy DirectorAeglea Biotherapeutics
3 Previous Clinical Trials
49 Total Patients Enrolled
Reena Sharma, MDPrincipal InvestigatorNorthern Care Alliance NHS Foundation Trust

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Recent research and studies
clinicaltrials.govStudy
A Multiple Ascending Dose Study of ACN00177 (Pegtarviliase) in Subjects With CBS Deficiency
2021. "A Multiple Ascending Dose Study of ACN00177 (Pegtarviliase) in Subjects With CBS Deficiency". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05154890.
~3 spots leftby May 2025
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