Phase 3: testing Pegtibatinase for Homocystinuria
Researchers are looking for a better way to treat people living with classical homocystinuria (HCU), a rare inherited condition. It is caused by changes in the cystathionine beta-synthase (or "CBS") gene and prevents an enzyme from working correctly in the body.
Official registry: https://clinicaltrials.gov/study/NCT06247085 · Travere Therapeutics, Inc. · enrollment listed as 70
What this phase means
Phase 3: this treatment already looked useful in smaller studies. Now it is tested in a large group, often against standard care or a dummy treatment, to see if it should be approved.
Phase labels are FDA/ICH categories applied to the registry Phase field. They are not a finding that this study succeeded.
What the registry says happens
If you join, the listed intervention(s) are Pegtibatinase, or Placebo. The registry lists allocation as randomized. Researchers are looking for a better way to treat people living with classical homocystinuria (HCU), a rare inherited condition.
They plan to enroll about 70 people at 52 sites. Taking part typically means taking a study drug (or dummy pill) plus check-ins.
Official title (unrewritten)
A Phase 3, Parallel-Group Treatment, Blinded, Randomized, Placebo-Controlled Study To Assess The Efficacy And Safety Of Pegtibatinase Administered Subcutaneously In Addition To Standard Of Care In Participants With Classical Homocystinuria Due To Cystathionine Beta Synthase Deficiency (HARMONY)
Interventions: Pegtibatinase, Placebo
Primary outcome (registry wording): Change from baseline in plasma tHcy levels - Weeks 6 to 12
Who it is for (from inclusion criteria)
- Ages 12 Years to 65 Years
- Must have a diagnosis of classical HCU based on clinical, biochemical, and/or molecular genetic testing
- Plasma tHcy ≥80 µM at Screening visit, with allowance for up to 18 participants who may be enrolled with a Screening plasma tHcy ≥50 to \<80 µM
- Participants who can become pregnant must have a negative pregnancy test before starting the study and must use a highly effective form of birth control (less than 1% risk of pregnancy per year) during the study and for at least 4 weeks after the last dose
- Willing to maintain a generally stable diet for the duration of the study (unless changes are required based on medical/safety reasons)
- Willing to maintain generally stable intake and doses of betaine, pyridoxine, and medical food for the duration of the study (unless changes are required based on medical/safety reasons)
Who the registry excludes
- Diagnosis of Marfan syndrome, methylenetetrahydrofolate reductase (MTHFR) deficiency, or disorder of cobalamin metabolism
- History of major thrombotic event (eg, cerebrovascular accident, myocardial infarction, pulmonary embolism) in the previous 6 months
- Body weight ≥160 kg
- Use or planned use of any injectable drugs containing PEG (excluding PEG-containing vaccines)
- Any previous exposure to pegtibatinase and/or previous participation in a clinical study that included administration of pegtibatinase or pegtarviliase
- Prior severe immune reaction to a PEG-containing product
Public study contact (from the registry)
Travere Call Center
Published on ClinicalTrials.gov. Contact them as a possible participant. They decide eligibility.
Sites listed on ClinicalTrials.gov
52 listed- Phoenix Children's Hospital — Phoenix, Arizona (recruiting)
- Yale University School of Medicine — New Haven, Connecticut (recruiting)
- Emory University — Atlanta, Georgia (recruiting)
- Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois (recruiting)
- Maine Health - Maine Medical Center — Portland, Maine (recruiting)
- Uncommon Cures — Chevy Chase, Maryland (recruiting)
- The Mount Sinai Hospital — New York, New York (recruiting)
- Science 37 - Virtual Site — Morrisville, North Carolina (recruiting)
- Oregon Health and Science University — Portland, Oregon (not_yet_recruiting)
- Children's Hospital of Philadelphia — Philadelphia, Pennsylvania (recruiting)
- University of Pittsburgh Medical Center - Children's Hospital of Pittsburgh — Pittsburgh, Pennsylvania (recruiting)
- University of Texas Southwestern Medical Center — Dallas, Texas (recruiting)
+ 39 sites outside the US on the same record
FAQ about this NCT record
Is NCT06247085 still recruiting?
The sponsor posted status “Recruiting” on ClinicalTrials.gov at last ingest. Sites can pause independently. Confirm with the official record and the listed contact.
What phase is NCT06247085?
Phase 3. Phase labels are FDA/ICH categories applied to the registry Phase field. They are not a finding that this study succeeded.
Can PlainTrials tell me if I qualify for NCT06247085?
No. The inclusion and exclusion lists on this page are readable extracts of the public eligibility block. Only the study team can screen you.
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