Clinical trials: Thymidine Kinase 2 (TK2)
Thymidine Kinase 2 Deficiency
An Open-Label Study of Continuation Treatment With Combination Pyrimidine Nucleosides in Patients With TK2 Deficiency
This is a Phase 2 prospective open-label treatment study of the safety and efficacy of doxecitine and doxribtimine in study participants with thymidine kinase 2 (TK2) deficiency who participated in the retrospective study…
- Trial name
- Continuation (NCT03845712)
- Testing
- doxecitine and doxribtimine
- Where
- 8 sites in United States, Israel and 1 more
- Volunteers
- 47 people
- Sponsor
- UCB BIOSCIENCES, Inc.
Mitochondrial DNA Depletion Syndrome 2 Myopathic Type, Thymidine Kinase 2 Deficiency
Treatment of TK2 Deficiency With Thymidine and Deoxycytidine
Patients with confirmed mitochondrial DNA depletion syndrome 2 (thymidine kinase 2 [TK2] deficiency) have reduced levels of nucleotides (deoxythymidine monophosphate and deoxycytidine monophosphate) for mitochondrial DNA…
- Trial name
- NCT03639701
- Testing
- Thymidine
- Where
- United States
- Volunteers
- 23 people
- Sponsor
- Columbia University
Thymidine Kinase 2 (TK2)
A RETROspective Study of Patients With TK2d
This is a medical chart review study to collect information for patients who have been taking pyrimidine nucleosides for treatment of TK2 deficiency.
- Trial name
- RETRO (NCT03701568)
- Testing
- dC/dT
- Where
- 8 sites in United States, Israel and 1 more
- Volunteers
- 38 people
- Sponsor
- Zogenix MDS, Inc.
Thymidine Kinase 2 Deficiency
A Retrospective Study of Subjects With Thymidine Kinase 2 Deficiency
This is a multicenter, multinational, retrospective chart-review study to evaluate survival in patients with Thymidine Kinase 2 deficiency (TK2d).
- Trial name
- NCT05017818
- Testing
- Observational retrospective data
- Where
- 12 sites in United States, Russia and 2 more
- Volunteers
- 61 people
- Sponsor
- Zogenix MDS, Inc.
Thymidine Kinase 2 (TK2 ) Deficiency, Mitochondrial Myopathies
Doxecitin and Doxribthymine in Adult Subjects With Thymidine Kinase 2 (TK2) Deficiency
The purpose of this clinical trial is to evaluate the efficacy and safety of Doxecitin and Doxribtimine (dC+dT) in adult participants with thymidine kinase 2 (TK2) deficiency attended in the Neuromuscular Unit of '12 de Octubre'…
- Trial name
- NCT06754098
- Testing
- Doxecitine and Doxribtimine
- Where
- Spain
- Volunteers
- 15 people
- Sponsor
- Cristina Domínguez González
Thymine Kinase 2 Deficiency
Doxecitine and Doxribtimine-Expanded Access
This Expanded Access Record for doxecitine and doxribtimine includes the following managed access programs and status: TK0113: Available TK0115: No longer Available
- Trial name
- NCT06590493
- Testing
- doxecitine and doxribtimine
- Sponsor
- UCB BIOSCIENCES, Inc.
Thymidine Kinase 2 Deficiency
A Study of the Efficacy and Safety of MT1621 in Thymidine Kinase 2 (TK2) Deficiency (Treatment naïve)
This is a Phase 3b, prospective, single-arm, multicenter, open-label treatment study of the efficacy and safety of MT1621 in pediatric and adolescent patients with thymidine kinase 2 deficiency (TK2d).
- Trial name
- NCT04581733
- Testing
- MT1621
- Sponsor
- Zogenix MDS, Inc.
Primary Mitochondrial Myopathies
Natural History in Primary Mitochondrial Myopathies
This is a longitudinal study in a cohort of patients with a genetic diagnosis of Primary Mitochondrial Myopathy to describe the natural history of the disease and identify clinical, biochemical, molecular, and radiological…
- Trial name
- NHPMM (NCT05653544)
- Where
- Spain
- Volunteers
- 150 people
- Sponsor
- Cristina Domínguez González