Status:
ACTIVE_NOT_RECRUITING
Pegtibatinase As an Enzyme Therapy for Patients with Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (COMPOSE)
Lead Sponsor:
Travere Therapeutics, Inc.
Conditions:
Homocystinuria
Eligibility:
All Genders
12-65 years
Phase:
PHASE1
PHASE2
Brief Summary
Homocystinuria caused by Cystathionine Beta-Synthase (CBS) Deficiency is a rare autosomal-recessive metabolic condition characterized by an excess of homocysteine (Hcy) in the plasma, tissues and urin...
Eligibility Criteria
Inclusion
- Confirmed diagnosis of homocystinuria based on genetic confirmation and plasma tHcy ≥50 micromoles and documentation of previous tHcy level ≥80 micromoles
- Willing and able to provide written, signed informed consent and to comply with all study related procedures.
- Subjects born biologically as female who are of child-bearing potential must have a negative pregnancy test at screening and be willing to have additional pregnancy tests during the study. Subjects born biologically as male who identify as female and are not of childbearing potential are not required to undergo pregnancy tests
- Sexually active subjects who have childbearing potential or those who have partners of childbearing potential must be willing to use acceptable methods of contraception while on the study and for 4 weeks after the end of study
- Willing to maintain a stable diet with no significant modifications while on study and complete a daily diet diary.
Exclusion
- Previous exposure to pegtibatinase and/or previous participation in a clinical trial that included administration of pegtibatinase
- Use of any investigational product or investigational medical device within 30 days prior to Screening, or while on study
- Use or planned use of any injectable drugs containing PEG (other than pegtibatinase or COVID-19 vaccines), including medroxyprogesterone (eg, Depo-Provera) injection, within 3 months prior to Screening and during study participation
- Known hypersensitivity to PEG-containing product or any components of pegtibatinase
- A positive test for HIV antibody, hepatitis B surface antigen, or hepatitis C antibody
- A history of organ transplantation, chronic immunosuppressive therapy, or substance abuse
- Pregnant or breastfeeding at Screening or planning to become pregnant (self or partner) or to breastfeed at any time during the study
- Concurrent disease or condition or planned major surgery that would interfere with study participation or safety in the opinion of the investigator.
- Any condition that, in the view of the investigator, places the subject at high risk of poor treatment compliance or of not completing the study
- Major surgery planned during the study period
Key Trial Info
Start Date :
January 22 2019
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
December 1 2026
Estimated Enrollment :
32 Patients enrolled
Trial Details
Trial ID
NCT03406611
Start Date
January 22 2019
End Date
December 1 2026
Last Update
November 22 2024
Active Locations (8)
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1
Travere Investigational Site - Virtual Site
Culver City, California, United States, 90230
2
Travere Investigational Site
Aurora, Colorado, United States, 80045
3
Travere Investigational Site
Miami, Florida, United States, 33136
4
Travere Investigational Site
Indianapolis, Indiana, United States, 46202