Pegtibatinase as an Enzyme Therapy for Patients With Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (COMPOSE)
- Conditions
- Homocystinuria
- Registration Number
- NCT03406611
- Lead Sponsor
- Travere Therapeutics, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Active, not recruiting
- Sex
- All
- Target Recruitment
- 32
Inclusion Criteria:<br><br> - Confirmed diagnosis of homocystinuria based on genetic confirmation and plasma tHcy<br> =50 micromoles and documentation of previous tHcy level =80 micromoles<br><br> - Willing and able to provide written, signed informed consent and to comply with all<br> study related procedures.<br><br> - Subjects born biologically as female who are of child-bearing potential must have a<br> negative pregnancy test at screening and be willing to have additional pregnancy<br> tests during the study. Subjects born biologically as male who identify as female<br> and are not of childbearing potential are not required to undergo pregnancy tests<br><br> - Sexually active subjects who have childbearing potential or those who have partners<br> of childbearing potential must be willing to use acceptable methods of contraception<br> while on the study and for 4 weeks after the end of study<br><br> - Willing to maintain a stable diet with no significant modifications while on study<br> and complete a daily diet diary.<br><br>Exclusion Criteria:<br><br> - Previous exposure to pegtibatinase and/or previous participation in a clinical trial<br> that included administration of pegtibatinase<br><br> - Use of any investigational product or investigational medical device within 30 days<br> prior to Screening, or while on study<br><br> - Use or planned use of any injectable drugs containing PEG (other than pegtibatinase<br> or COVID-19 vaccines), including medroxyprogesterone (eg, Depo-Provera) injection,<br> within 3 months prior to Screening and during study participation<br><br> - Known hypersensitivity to PEG-containing product or any components of pegtibatinase<br><br> - A positive test for HIV antibody, hepatitis B surface antigen, or hepatitis C<br> antibody<br><br> - A history of organ transplantation, chronic immunosuppressive therapy, or substance<br> abuse<br><br> - Pregnant or breastfeeding at Screening or planning to become pregnant (self or<br> partner) or to breastfeed at any time during the study<br><br> - Concurrent disease or condition or planned major surgery that would interfere with<br> study participation or safety in the opinion of the investigator.<br><br> - Any condition that, in the view of the investigator, places the subject at high risk<br> of poor treatment compliance or of not completing the study<br><br> - Major surgery planned during the study period
Not provided
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Incidence of AEs;Anti-pegtibatinase antibodies;Anti-PEG antibodies
- Secondary Outcome Measures
Name Time Method Changes in pegtibatinase levels;Changes in Met cycle metabolites levels - tHcy;Changes in Met cycle metabolites levels - total Cys;Changes in Met cycle metabolites levels - Met;Changes in Met cycle metabolites levels - Cth;Changes in Met cycle metabolites levels - Phe;Descriptive ophthalmology examination findings;Bone densitometry using dual-energy X-ray absorptionmetry (DEXA) scans;Cognitive assessments using the National Institutes of Health Toolbox Cognition Battery score;Patient Reported Outcome (PRO): Quality of Life in Neurological Disorders [Neuro-QoL];Patient Reported Outcome (PRO): Quality of Life by 36-Item Short Form Survey [SF-36];Patient Reported Outcome (PRO): Quality of Life by EuroQol 5-Dimentional Instrument [EQ 5D]