OT-58 as an Enzyme Therapy for Patients With Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency
Study Phase: Phase 1/Phase 2
Recruitment Status: Recruiting
Start Date: January 22, 2019
End Date: December 01, 2021
Inclusion Criteria:
- Confirmed diagnosis of homocystinuria based on genetic confirmation and plasma tHcy ≥80 micromoles
- Willing and able to provide written, signed informed consent and to comply with all study related procedures
- Females of child-bearing potential must have a negative pregnancy test at screening and be willing to have additional pregnancy tests during the study
- Sexually active patients must be willing to use acceptable method 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
- In generally good health, except for possible complications of homocystinuria
Exclusion Criteria:
- 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 including medroxyprogesterone (e.g. Depo-Provera) injection within 3 months prior to Screening and while on study
- Known hypersensitivity to PEG-containing product or any components of OT-58
- 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.
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Conditions:
- Homocystinuria