Drugs / deoxythymidine
Trials 3
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 11 trial | ||||||
| Phase 1 | NCT06817590 | Sep 2025 → Jun 2028 expected | Hoyeraal-Hreidarsson syndrome, Revesz syndrome, pulmonary fibrosis and/or bone marrow failure, telomere-related | Suneet Agarwal | Recruiting | No outcome recorded |
| Phase 21 trial | ||||||
| Phase 2 | NCT04802707 | Oct 2021 → Jun 2029 expected | mitochondrial DNA depletion syndrome, mitochondrial encephalomyopathy | Kenneth Myers, MD | Recruiting | No outcome recorded |
| Phase 31 trial | ||||||
| Phase 3 | NCT04581733 | Sep 2022 → Mar 2025 | mitochondrial DNA depletion syndrome, myopathic form | Zogenix MDS, Inc. | Withdrawn | No outcome recorded Stop: Business |
News releases announcing trial results or a regulatory action · 2
| Date | Issuer | Release |
|---|---|---|
| 2025-10-13 | UCB Pharma | Results Results published in Neurology highlight a 95% reduction in risk of death with pyrimidine nucleos(t)ide therapy in patients with thymidine kinase 2 deficiency (TK2d) ucb.com ↗
The results indicated that pyrimidine nucleoside and/or nucleotide therapy may reduce risk of death and can impact functional outcomes in patients with TK2d. |
| 2025-03-19 | UCB Pharma | Results New data on investigational therapy for thymidine kinase 2 deficiency presented at Muscular Dystrophy Association (MDA) 2025 Conference ucb.com ↗
The data show that in individuals with TK2d who were aged 12 years or less when their symptoms first appeared, treatment with pyrimidine nucleoside and/or nucleotide therapy significantly decreased mortality and increased survival. |
All press releases naming this drug 2 releases
Evidence & citations 2 cited values
Every value below carries the sentence it was read from. 3 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | deoxythymidine | “Deoxycytidine (dC) and deoxythymidine (dT)” NCT04581733 ↗ |
| Route | Oral | “will be administered orally every day (QD)” NCT04802707 ↗ |