Either you have JavaScript disabled or your browser does not support Javascript . To work properly, this page requires JavaScript to be enabled.
How to enable JavaScript in your browser?

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).

Trial Profile

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).

Status: Active, no longer recruiting
Phase of Trial: Phase III

Latest Information Update: 11 Apr 2025

Price :
$35 *
Note:
  • Adis is an information provider.
  • Final gross price and currency may vary according to local VAT and billing address.
  • Your purchase entitles you to full access to the information contained in our trial profile at the time of purchase.
  • A link to download a PDF version of the trial profile will be included in your email receipt.

At a glance

  • Drugs Pegtibatinase (Primary)
  • Indications Homocystinuria
  • Focus Registrational; Therapeutic Use
  • Acronyms HARMONY
  • Sponsors Travere Therapeutics

Most Recent Events

  • 20 Feb 2025 According to a Travere Therapeutics media release, company is on track to restart enrollment in the Phase 3 HARMONY Study in 2026.
  • 26 Sep 2024 event According to a Travere Therapeutics media release, status changed from recruiting to active, no longer recruiting.
  • 26 Sep 2024 According to a Travere Therapeutics media release, company announced a voluntary pause of enrollment in the trial, this pause enables the Company to work to address necessary process improvements in manufacturing scale-up to support commercial scale manufacturing as well as full enrollment in the HARMONY Study. And expects restart enrollment in 2026.

You need to be a logged in or subscribed to view this content

Request demo (opens in a new window)
If your organization or you do not have a subscription, try one of the following: If your organization has a subscription, there are several access options, even while working remotely:
  • Working within your organization’s network
  • with username/password or try to via your institution
  • Persisted access using your organization’s identifier stored in your user browser for 90 days
Back to top