Trial · NCT04176523
Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods Approach
NCT04176523, Recruiting, RECORDATI GROUP
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods Approach. Trials, record NCT04176523. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04176523
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods Approach
- NCT number
- NCT04176523
- Status
- Recruiting
- Enrollment
- 95 participants, estimated
- Lead sponsor
- RECORDATI GROUP
- Conditions
- Methylmalonic Acidemia, Propionic Acidemia
- Interventions
- Carglumic Acid
- First posted
- 2019-11-25
- Last updated
- 2024-02-05
Source ClinicalTrials.gov
Registered outcomes5
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change in Number and Duration of Decompensation Events | 54 months | Change in the number of decompensation events before and after treatment with carglumic acid |
| Secondary | Frequency and Duration of Healthcare Resource Utilization | 18 months | Estimation of the health care resource utilization associated with a decompensation event with number of inpatient hospitalization vs. outpatient vs. home. Treatment used to control ammonia levels during decompensation episodes. Number of emergency visits. number of visits to specialist or to general practitioner. Number of hospital stays. Number of bed days in hospital. |
| Secondary | Caregiver Burden Exposure Frequency and Duration | 18 months | description of the patient and caregiver burden associated with MMA and PA |
| Secondary | Caregiver Satisfaction Level | 18 months | Describe the MMA and PA patient and caregiver satisfaction with MMA or PA treatment |
| Secondary | Maintenance Dosage of NCG | 54 months | Document the dosages of NCG used in maintenance treatment |
Source ClinicalTrials.gov
Outcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
Papers8
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04176523
GET https://conductscience.org/api/trials/v1/NCT04176523/registered