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Treatment of TK2 Deficiency With Thymidine and Deoxycytidine
NCT03639701 · View on ClinicalTrials.gov ↗
Study Summary
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 synthesis. This results in mitochondrial DNA depletion syndrome (i.e less number of functional mitochondrial DNA). Patients with confirmed TK2 deficiency will be treated with open label deoxythymidine (dThd) and deoxycytidine (dCyt), which are nucleotide precursors, with the expectation that the cells could make additional mitochondrial DNA. This in turn may help reduce the clinical symptoms.
Conditions Studied
Interventions
- DRUG Thymidine
Study Locations (1)
New York
- Columbia University Irving Medical Center — New York
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 23 participants |
| Start Date | 2017-05-16 |
| Est. Completion | 2026-12-31 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT03639701
The ClinicalTrials.gov registry entry for NCT03639701 describes a study currently listed as active not recruiting. It is categorized as Phase 1, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 23 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Columbia University, which has 875 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 2 conditions, with Thymidine Kinase 2 Deficiency appearing as the primary indexed condition, and to 1 intervention — of which Thymidine is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT03639701 reports 1 study location spanning 1 distinct geographic area — top geographies include New York. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT03639701 about?
NCT03639701 is a clinical study titled "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 synthesis. This results in mitochondrial DNA depletion syndrome (i.e less num...
What is the current status of trial NCT03639701?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 23 participants. The study started on 2017-05-16. Estimated completion is 2026-12-31.
What conditions does trial NCT03639701 study?
This clinical trial studies the following conditions: Thymidine Kinase 2 Deficiency, Mitochondrial DNA Depletion Syndrome 2 Myopathic Type. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT03639701?
The interventions under investigation include: Thymidine (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT03639701?
This trial is sponsored by Columbia University, which has 875 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT03639701 being conducted?
This trial has 1 study location across New York. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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