Doxecitine and doxribtimine
(Brand name KYGEVVI)
Approved in the U.S. for children and adults with TK2d whose symptoms started at age 12 or younger. It is not approved in Australia.
Study details
A Phase 2 Open-Label Study of Continuation Treatment With Combination Pyrimidine Nucleosides in Patients With Thymidine Kinase 2 Deficiency (NCT03845712).
Which people with mito might this therapy help?
People with genetically confirmed Thymidine kinase 2 deficiency (TK2d) whose symptoms started at age 12 or younger.
KYGEVVI is a powder mixed with water and taken by mouth. It is usually taken three times a day with food.
KYGEVVI is designed to support mitochondrial DNA production in muscles. This is important because TK2d affects the body’s ability to maintain mitochondrial DNA.
What research has been done on this therapy?
KYGEVVI has been studied in people with TK2d. The U.S. Food and Drug Administration (FDA) approval was supported by:
- one Phase 2 clinical study
- two medical record review studies
- an expanded access program.
These included 82 people treated with KYGEVVI or related pyrimidine nucleosides whose TK2d symptoms started at age 12 or younger.
UCB reported that treatment reduced the risk of death compared with an untreated group. Because TK2d is very rare, the research used a mix of clinical study data, medical record data and expanded access data.
Possible side effects include diarrhoea, nausea, vomiting, stomach pain and reduced appetite. KYGEVVI may also affect liver blood test results, so liver tests are recommended before treatment starts.
Clinical trial information
Study name
A Phase 2 Open-Label Study of Continuation Treatment With Combination Pyrimidine Nucleosides in Patients With Thymidine Kinase 2 Deficiency (NCT03845712).
Location
Multiple sites overseas, including USA, Israel and Spain. There are no Australian sites.
Who is funding this study?
UCB Biosciences.
This study is looking at the safety and efficacy of doxecitine and doxribtimine in people with TK2d.
The study includes people who had already taken part in an earlier TK2d study, or who were already receiving the therapy and were approved by the study sponsor.
Important information
Find out more
Emerging therapies for mito
Learn about developing therapies and opportunities to take part in research.
Clinical trials explained
A guide to help understand participation and make informed decisions.
