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NCT04679389 · ClinicalTrials.gov registry record · Phase 2
Acetazolamide Efficacy in Ataxia in PMM2-CDG
A Phase 2 study, sponsored by Mayo Clinic.
- Terminated
- Registry status
- Phase 2
- Development phase
- 25
- Enrollment target
NCT04679389: Clinical Trial Phase 2 study, sponsored by Mayo Clinic.
NCT04679389 is a Phase 2 study that was terminated before completion, run by Mayo Clinic. The registered enrollment target is 25 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (81% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04679389, a Phase 2 study, was terminated before completion, sponsored by Mayo Clinic.
- TERMINATED
- Registry status
- Phase 2
- Development phase
- 25 participants
- Enrollment target
Study Summary
Objective 1 (Primary): To determine the efficacy of acetazolamide in improving ataxia in patients with PMM2-CDG. Objective 2 (Secondary): To evaluate for any adverse events related to longer term acetazolamide administration. Objective 3 (Secondary): To examine the effect of acetazolamide on PMM2 biomarkers including carbohydrate deficient transferrin results, electrolytes (Na, K, Cl, CO2), VBG (pH, pCO2, PO2, CO2, Base excess), liver function tests (AST, ALT, GGT, indirect and direct bilirubin, total protein, albumin, alkaline phosphatase), kidney function tests (BUN, Creatinine, Urinalysis, urine calcium/creatinine ratio, urine protein/creatinine ratio), growth (height, weight, head circumference), vital signs (blood pressure, respiratory rate, heart rate), PROMIS scores, dysarthria using the PATA score, and NPCRS score. Objective 4 (Secondary): To explore characteristics of individuals with PMM2-CDG who do not respond to acetazolamide.
Primary Outcome
To achieve this goal, we compared the change of Mini-ICARS score from baseline to after six months of treatment between the placebo and active treatment groups. Minimal score is 0, maximum score is 100, higher score indicates greater impairment. Each subscale has an ordinal scale with a 0 indicating normal and the higher score indicating greater impairment or that the patient was unable to complete the task.
Interventions
- DRUG Placebo
- DRUG Acetazolamide
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 25 participants |
| Start Date | 2021-03-17 |
| Est. Completion | 2024-01-24 |
| Phase | Phase 2 |
Why NCT04679389 stopped before completion
NCT04679389 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 25 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (81% lower).
The record links to 0 conditions, and to 2 interventions - of which Placebo is the first listed.
NCT04679389 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT04679389 about?
NCT04679389 is a clinical study titled "Acetazolamide Efficacy in Ataxia in PMM2-CDG". Objective 1 (Primary): To determine the efficacy of acetazolamide in improving ataxia in patients with PMM2-CDG. Objective 2 (Secondary): To evaluate for any adverse events related to longer term acetazolamide administration. Objective 3 (Secondary): To examine the effect of acetazolamide on PMM2 ...
What is the current status of trial NCT04679389?
This trial is currently terminated. It is a Phase 2 study. The enrollment target is 25 participants. The study started on 2021-03-17. Estimated completion is 2024-01-24.
What interventions are being tested in trial NCT04679389?
The interventions under investigation include: Placebo (DRUG), Acetazolamide (DRUG).
Who is sponsoring clinical trial NCT04679389?
This trial is sponsored by Mayo Clinic, which has 2,844 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT04679389's enrollment target sits among peer trials
25 1719th of 2000 higher than 245 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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