Trials / Completed
CompletedNCT05017818
A Retrospective Study of Subjects With Thymidine Kinase 2 Deficiency
A Retrospective Study of Subjects With Thymidine Kinase 2 Deficiency Treated With the Combination of Pyrimidine Nucleos(t)Ides as Well as Untreated Subjects to Collect Vital Status Data and Supporting Information
- Status
- Completed
- Phase
- —
- Study type
- Observational
- Enrollment
- 61 (actual)
- Sponsor
- Zogenix MDS, Inc. · Industry
- Sex
- All
- Age
- —
- Healthy volunteers
- Not accepted
Summary
This is a multicenter, multinational, retrospective chart-review study to evaluate survival in patients with Thymidine Kinase 2 deficiency (TK2d).
Detailed description
This is a retrospective, chart-review study. The primary goal is to use data on survival and other related information to support a comprehensive evaluation of patients with Thymidine Kinase 2 deficiency (TK2d) who have not been treated with pyrimidine nucleos(t)ides including deoxycytidine monophosphate/deoxythymidine monophosphase (dCMP/dTMP), and deoxycytidine/deoxythymidine (dC/dT), and/or MT1621(dC/dT), and those that have received one or more of these treatments. The secondary goals of this study will focus on describing the participant's clinical course and treatment experience.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| OTHER | Observational retrospective data | Non-intervention |
Timeline
- Start date
- 2021-07-23
- Primary completion
- 2022-01-21
- Completion
- 2022-01-21
- First posted
- 2021-08-24
- Last updated
- 2023-09-01
Locations
12 sites across 4 countries: United States, Russia, Spain, Turkey (Türkiye)
Source: ClinicalTrials.gov record NCT05017818. Inclusion in this directory is not an endorsement.