Top
Late-Onset OTC Deficiency - Healing Genes
1325
post-template-default,single,single-post,postid-1325,single-format-standard,mkdf-bmi-calculator-1.0,mkd-core-1.1.1,tribe-no-js,wellspring-ver-1.8,mkdf-smooth-page-transitions,mkdf-ajax,mkdf-grid-1300,mkdf-blog-installed,mkdf-header-standard,mkdf-no-behavior,mkdf-default-mobile-header,mkdf-sticky-up-mobile-header,mkdf-dropdown-slide-from-bottom,mkdf-full-width-wide-menu,mkdf-search-dropdown,elementor-default,elementor-template-full-width,elementor-kit-3486,elementor-page-2540

Late-Onset OTC Deficiency

Share This Post

Share on facebook
Share on linkedin
Share on twitter
Share on email

Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset OTC Deficiency (CAPtivate)

A Phase 1/2, Open-Label Safety and Dose-Finding Study of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Adults With Late-Onset OTC Deficiency


Phase 1/2

DESCRIPTION:

Doctors at university medical centers in CA, CO, MA, NY, OH, and OR are recruiting adult patients with late-onset Ornithine transcarbamylase (OTC) deficiency to trial an investigative gene therapy, DTX301. The therapy involves a single infusion of a genetically engineered virus designed to transfer a functional version of the OTC gene. This replacement gene may produce OTC and achieve a meaningful increase the urea cycle in the liver.

A single infusion of the drug at one of 3 dosages is required, with follow up for 1 year. Subjects will be followed for 52 weeks and then asked to enroll in a 4-year extension study to evaluate the long term (a total of 5 years) safety and efficacy of DTX301.


PATIENT MUST:

  • Be 18 years of age or older
  • Diagnosis of late-onset OTC with confirmation by molecular testing
  • Be on stable dose of ammonia scavenger therapy for ≥4 weeks

THE STUDY INVOLVES:

  1. Prescreening tests to confirm eligibility of the patient to participate.
  2. A single intravenous administration of the trial drug.
  3. Follow up over the year after treatment.

LOCATIONS AND CONTACTS:
The following study sites are coordinating:
Ronald Reagan UCLA Medical Center in Los Angeles, CA, Map.
University of California San Francisco in San Francisco, CA, Map.
The Children’s Hospital Colorado in Aurora, CO, Map.
Boston Children’s Hospital in Boston, MA, Map.
Icahn School of Medicine at Mount Sinai in New York, NY, Map.
University Hospital Cleveland Medical Center/Case Western Reserve University in Cleveland, OH, Map.
Oregon Health and Science University in Portland, OR, Map.

Contacts:
Patient Advocacy  |  1-415-483-8800  |  [email protected]
HCPs Contact  |  1-888-756-8657  |  [email protected]

SPONSOR INFORMATION:
Ultragenyx Pharmaceutical Inc

Or go online:
https://clinicaltrials.gov/ct2/show/NCT02991144

Subscribe To Our Newsletter

Get updates and learn from the best

More To Explore

Vanquishing the Virus: 160+ COVID-19 Drug and Vaccine Candidates in Development

While President Trump continues to promote antimalarial drugs such as hydroxychloroquine sulfate and azithromycin to treat COVID-19—“What do you have to lose?” he exclaimed during the April 4 Coronavirus Task Force Briefing—the global community of drug discovery researchers and biopharmas is developing a much broader