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A study to investigate efficacy and safety with pegtibatinase compared to placebo in participants ≥12 to ≤65 years of age with classical homocystinuria (HCU) due to cystathionine beta synthase deficiency receiving standard of care treatment
EU CTIS ID: 2023-504135-40-00
What this study is testing
Efficacy: To determine the effect of pegtibatinase administered subcutaneously on plasma tHcy and methionine (Met) as compared with placebo in participants with HCU receiving standard of care Safety: To determine the safety and tolerability of pegtibatinase administered subcutaneously in participants with HCU
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Individuals eligible to be enrolled into this study must have a diagnosis of HCU based on clinical, biochemical, and/or molecular genetic testing. Approximately 25% of participants may be enrolled with a Screening plasma tHcy ≥50 to <80 µM, and the remaining majority (approximately 75%) of participants will be enrolled with a Screening plasma tHcy ≥80 µM.
- Individuals must also be ≥12 to ≤65 years of age at Screening, and be willing to maintain a stable diet with consistent levels of DIPI, and HCU-related therapies such as betaine, pyridoxine, and medical food (eg, metabolic formula), if applicable, as part of pre-study treatment
You likely can't join if
- Diagnosis of Marfan syndrome, methylenetetrahydrofolate reductase (MTHFR) deficiency, or disorders of cobalamin metabolism; body weight ≥160 kg (measured from screening through the PND)
- Any prior exposure to pegtibatinase or pegtarviliase
- Use or planned use of any injectable drug containing PEG (other than pegtibatinase or PEG containing vaccines)
- History of a severe immune reaction to a PEG containing product; organ transplant; or intensive chronic immunosuppressive treatment within the 6 months prior to screening
The study team makes the final eligibility decision.
Where it's taking place
- Turkey
- Australia
- United States
- Qatar
- United Kingdom
- Saudi Arabia
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18-64 years, 0-17 years, 65+ years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Turkey; Australia; United States; Qatar; United Kingdom; Saudi Arabia. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.