Mitochondrial DiseasesPhase 1NCT00060515
RG2133 (2',3',5'-Tri-O-Acetyluridine) in Mitochondrial Disease
A Phase 1 study testing RG2133 (2',3',5'-tri-O-acetyluridine) in people with Mitochondrial Diseases, run by Repligen Corporation.
Trial summary
The objective of the study is to determine the safety and tolerability of RG2133 in patients with Mitochondrial Disease. Read more about this study
- Who is it for?
- Ages 3 years and older
- Conditions the study is for
- Mitochondrial Diseases
- What is being tested?
- RG2133 (2',3',5'-tri-O-acetyluridine)
- How many people
- 12 participants
- Where
- No sites listed yet
- Study dates
- Start not givento not given
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Mitochondrial Disease
About this study
Official title: An Open-Label Dose-Escalation Phase I Study to Asses the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of RG2133 (2',3',5'-Tri-O-Acetyluridine) in the Treatment of Inherited Mitochondrial Diseases
The objective of the study is to determine the safety and tolerability of RG2133 in patients with Mitochondrial Disease.
- Conditions
- Mitochondrial Diseases
- Being tested
- RG2133 (2',3',5'-tri-O-acetyluridine)
- Medical subject headings
- Mitochondrial Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RG2133 (2',3',5'-tri-O-acetyluridine)Drug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 May 2003 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Mitochondrial DiseasesStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Enrolment
- 12 participants
Sponsor and investigators
- Lead sponsor
- Repligen Corporation
- Sponsor type
- Industry (pharma, biotech, devices)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00060515
- Sponsor's protocol ID
- RG2133-01
- Status last verified
- Aug 2005
- First submitted
- 7 May 2003
- Last updated
- 5 Aug 2005
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Aug 2005. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Dose-Escalation Phase I Study to Asses the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of RG2133 (2',3',5'-Tri-O-Acetyluridine) in the Treatment of Inherited Mitochondrial Diseases
- ClinicalTrials.gov ID
- NCT00060515
- Acronym / sponsor ID
- RG2133-01
- Conditions
- Mitochondrial Diseases
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Terminated
- Enrolment
- 12 participants
- Lead sponsor
- Repligen CorporationIndustry (pharma, biotech, devices)
- First posted
- 8 May 2003 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The objective of the study is to determine the safety and tolerability of RG2133 in patients with Mitochondrial Disease.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
Who may qualify
- Ages
- 3 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RG2133 (2',3',5'-tri-O-acetyluridine) | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
The study was stopped early. Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Aug 2005.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing RG2133 (2',3',5'-tri-O-acetyluridine) in people with Mitochondrial Diseases, run by Repligen Corporation.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 5 Aug 2005. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early. Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Aug 2005.