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Classical Homocystinuria research hub

2 indexed studies · 0 currently recruiting

authorisedPhase I and Phase II (Integrated)- First administration to humans
A PHASE 1/2 STUDY TO ASSESS THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS, AND EFFECTS ON CLINICAL OUTCOMES OF PEGTIBATINASE (TVT-058) ADMINISTERED SUBCUTANEOUSLY IN SUBJECTS WITH CYSTATHIONINE BETASYNTHASE-DEFICIENT HOMOCYSTINURIA (COMPOSE)
This study tests a new medicine called pegtibatinase for classical homocystinuria. Homocystinuria is a rare condition that causes high levels of a chemical called homocysteine in the blood. Researchers want to find out if this injection is safe, well-tolerated, and able to lower homocysteine levels in the body.
France
authorisedTherapeutic confirmatory (Phase III)
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)
This study is looking into a new treatment called Pegtibatinase for people with a rare genetic condition called Classical Homocystinuria. Researchers want to see if this new medicine, given as a shot under the skin, can help lower harmful levels of a substance in the blood called tHcy.
Belgium · Ireland · Spain