← Back to Search

Other

Deoxynucleoside Therapy for Mitochondrial Disease (dC-dT-MDS Trial)

Phase 2

Recruiting

Led By Kenneth Alexis MD Myers, MD PhD FRCPC

Research Sponsored by McGill University Health Centre/Research Institute of the McGill University Health Centre

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Clinical Diagnosis of a Mitochondrial Depletion Disorder

Clinical Diagnosis of a Mitochondrial Depletion Disorder.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 104 weeks

Awards & highlights

dC-dT-MDS Trial Summary

This trial is to study if a mix of Deoxynucleosides Pyrimidine is effective and safe as treatment of MDS.

Who is the study for?

This trial is for children and adults (0-60 years old) with a confirmed diagnosis of Mitochondrial Depletion Disorder. Participants must have specific genetic mutations (POLG, C10orf2, RRM2B, MPV17, SUCLA2, SUCLG1, FBXL4). Women who can bear children must test negative for pregnancy and agree to use contraception.Check my eligibility

What is being tested?

The trial tests a combination of deoxycytidine and deoxythymidine as an early treatment for Mitochondrial Depletion Syndrome. This phase II trial aims to confirm the safety and effectiveness of these compounds in improving mitochondrial function.See study design

What are the potential side effects?

While the description does not specify side effects, previous trials suggest that the mix of deoxynucleosides being tested is generally well-tolerated. However, potential side effects are not detailed here.

dC-dT-MDS Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I have been diagnosed with a mitochondrial depletion disorder.

Select...

I have been diagnosed with a mitochondrial depletion disorder.

Select...

My genetic test shows mutations in specific energy production genes.

Select...

I am 18 years old or younger.

dC-dT-MDS Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 104 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~104 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and 104 weeks for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Rate of Responder versus Non-Responder Status with investigational product

Secondary outcome measures

Number of participants experiencing dose-limiting toxicities, adverse events (AEs), serious adverse events (SAEs)

dC-dT-MDS Trial Design

1Treatment groups

Experimental Treatment

Group I: dC/dT100-400 ArmExperimental Treatment1 Intervention

Children & Adult (0-60 Y), who takes the investigational product deoxynucleosides pyrimidine (mix of deoxycytidine and deoxythymidine), following the protocol.

0 / 4Eligibility criteria met

Find a Location

Who is running the clinical trial?

McGill University Health Centre/Research Institute of the McGill University Health CentreLead Sponsor

443 Previous Clinical Trials

159,448 Total Patients Enrolled

Kenneth Alexis MD Myers, MD PhD FRCPCPrincipal InvestigatorRI-MUHC, Children Hospital of Montreal (MUHC), McGill University

Media Library

Deoxycytidine and Deoxythymidine (Other) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04802707 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are enrollment opportunities still available for this trial?

"Clinicaltrials.gov has verified that this medical trial is accruing new patients, having been initially posted on October 18th 2021 and recently edited on July 4th 2022."

Answered by AI

How many individuals can participate in this experiment?

"Yes, according to clinicaltrials.gov these medical research is at present accepting applicants. This trial was first posted on October 18th 2021 and last updated on July 4th 2022; it requires 15 individuals from one centre for the experiment."

Answered by AI

Is this medical experiment offering a unique treatment approach?

"Presently, there are nine active experiments concerning deoxycytidine and deoxythymidine across 10 cities in five countries. The first exploration of these drugs began back in 2014 thanks to GenVivo, Inc.'s sponsorship which included 122 participants and culminated with Phase 1 approval. Subsequently, four other trials have been successfully finished."

Answered by AI

Has research been conducted with similar compounds such as deoxycytidine and deoxythymidine?

"Currently, 9 clinical trials centred around deoxycytidine and deoxythymidine are underway with none in Phase 3. Buffalo is the primary hub for these studies but other cities across the US have 26 locations conducting related research."

Answered by AI

Are there any specific characteristics of participants that would make them ideal candidates for this experiment?

"This medical trial is seeking 15 minors aged 1 month to 18 with a mitochondrial encephalomyopathy diagnosis. Furthermore, those accepted must possess written parental consent, have no pathogenic variants in POLG, C10orf2, RRM2B, MPV17 SUCLA2 ,SUCLG1 or FBXL4 genes and pass a urine pregnancy test at screening time. Additionally all participants are expected to use reliable contraception during the entirety of the study period."

Answered by AI

Is this trial open to persons aged sixty or older?

"This research project is seeking young participants between the ages of 1 month and 18 years."

Answered by AI

What potential harm could be caused to patients by the administration of deoxycytidine and deoxythymidine?

"Though no prior data exists on the efficacy of deoxycytidine and deoxythymidine, there is some evidence for their safety. As such, we have assigned these substances a rating of 2."

Answered by AI