A Multiple Ascending Dose Study of ACN00177 (Pegtarviliase) in Subjects With CBS Deficiency

Stopped early · Phase 1/Phase 2

Conditions studied: Homocystinuria Due to Cystathionine Beta-Synthase Deficiency

In brief

The purpose of this study is to evaluate the safety and tolerability of pegtarviliase in approximately 36 subjects with homocystinuria due to CBS deficiency.

Key facts

Study ID

NCT05154890

Run by

Aeglea Biotherapeutics

People needed

13

Starts

2021-05-13

Expected to finish

2023-04-21

Last updated by the study team

2023-07-27

Who can join

Age: 12 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Diagnosis of homocystinuria due to CBS deficiency
• Capable of providing signed informed consent/assent and to comply with all study related procedures
• Is ≥12 years of age (≥18 in the US) at the time of signing the informed consent/assent
• Plasma tHcy ≥50 µM (rounded to the nearest whole number) and documentation of previous tHcy ≥80 µM
• Female subjects of child-bearing potential must have a negative serum pregnancy test during the screening period and a negative urine pregnancy test prior to dosing on the first day of treatment
• If the subject (male or female) is engaging in sexual activity, he/she must be unable to become pregnant/cause pregnancy or must agree to use highly effective contraception
• Subjects receiving pyridoxine and/or betaine must be on the same dose of the medication(s) for at least 6 weeks prior to the first administration of study drug and be willing and able to remain on a stable dose for the duration of the study. Similarly, those on prescribed dietary therapy must be on a consistent dietary regimen for at least 6 weeks prior to study drug and should maintain this regimen for the duration of the study

You may not qualify if…

• Other medical conditions or co-morbidity(ies) that, in the opinion of the investigator, would put the subject at increased medical risk or interfere with study compliance or data interpretation (eg, severe intellectual disability that precludes completion of the required study assessments)
• Currently participating in another therapeutic clinical study or has received any investigational agent within 30 days or 5 half-lives, whichever is longer, prior to the first dose of study drug in this study
• Surgery requiring general anesthesia within 8 weeks prior to the first dose of study drug or planned surgery druing the treatment period
• Active infection requiring anti-infective therapy <2 weeks prior to the first dose of study drug in this study; anti-infective therapy that completes ≥2 weeks prior to first dose of study drug is acceptable
• Pregnant or nursing
• Females of child-bearing potential who are using or plan to use estrogen-containing contraception during the study (unless the subject currently using estrogen-containing contraceptives is willing to switch to a non-estrogen-containing contraceptive at least 1 week before dosing and for the duration of the study) and for 30 days after the last dose
• History of hypersensitivity to polyethylene glycol (PEG) that, in the judgment of the investigator, puts the subject at unacceptable risk for adverse events (AEs)
• Serum creatinine level >1.5× the upper limit of normal (ULN)
• Alanine aminotransferase (ALT), aspartate aminotransferase (AST), or total bilirubin level > 2× the ULN

Where it is running

• UT Southwestern Medical Center — Dallas, Texas, United States
• Westmead Hospital — Westmead, New South Wales, Australia
• Royal Children's Hospital — Parkville, Victoria, Australia
• Royal Melbourne Hospital — Parkville, Victoria, Australia
• University Hospitals Birmingham NHS — Birmingham, United Kingdom
• Great Ormond Street Hospital — London, United Kingdom
• Guy's and St Thomas' Hospital NHS Foundation Trust — London, United Kingdom
• University College London — London, United Kingdom
• Salford Royal NHS Foundation Trust — Salford, United Kingdom

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.