Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)NCT03768817
Clinical Outcome of Triheptanoin Treatment in Patients With Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) Treated Under Expanded Access Program
An observational study of Long-chain Fatty Acid Oxidation Disorders (LC-FAOD), run by Ultragenyx Pharmaceutical Inc at 15 sites in United States.
Trial summary
The primary objective of the study is to evaluate the clinical outcomes for the trigger event of patients who receive triheptanoin in the emergency Investigational New Drug (eIND) program. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
- How many people
- 20 participants
- Where
- 15 sitesUnited States
- Study dates
- 30 Jan 2019to 8 Jun 2020
- Phase
- Observational
- 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 Any age
- All sexes
- Child, Adult, Older adult
Study population: Medical records of patients in the USA with LC-FAOD who received triheptanoin via eIND request from their metabolic or treating physician before 01 September 2018.
Who may be able to join
- Male or female patients of any age with confirmed diagnosis of one of the LC-FAOD disorders including: mitochondrial trifunctional protein (TFP) deficiency, carnitine palmitoyltransferase deficiencies (CPT I and CPT II), very long chain acyl-CoA dehydrogenase (VLCAD) deficiency, long-chain 3-hydroxy-acyl-CoA dehydrogenase (LCHAD) deficiency, and carnitine acylcarnitine translocase (CATR) deficiency
- Treatment initiation with triheptanoin was via eIND before 01 September 2018
- Treated with triheptanoin in the USA
- Willing and able to provide written, signed informed consent, or where appropriate for subjects under the age of 18, or provide written assent and written informed consent by a legally authorized representative after the nature of the study has been explained and prior to any research-related procedures. To obtain and review medical records of deceased individuals, informed consent from next of kin or appropriate legal entity will be obtained, as applicable.
Who cannot join
- Unwilling to sign informed consent or assent to release of medical records
Trial locations and contacts
15 sites in 1 country.
15 sites in 1 country
United States15 sites
Emory University
Atlanta, Georgia, 30322
Boston Children's Hospital
Boston, Massachusetts, 02115
Duke University
Durham, North Carolina, 27710
Children's Hospital of Los Angeles
Los Angeles, California, 90027
Kaiser Permanente
Los Angeles, California, 90027
University of Minnesota Masonic Children's Hospital
Minneapolis, Minnesota, 55455
Yale University
New Haven, Connecticut, 06511
Children's Hospital of Orange County
Orange, California, 92868
Stanford University
Palo Alto, California, 94304
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224
Oregon Health and Science University
Portland, Oregon, 97239
Seattle Children's Hospital
Seattle, Washington, 98105
University of South Florida
Tampa, Florida, 33606
Children's National Hospital
Washington D.C., District of Columbia, 20010
About this study
Official title: A Retrospective Chart Review Study to Assess the Clinical Outcome of Triheptanoin Treatment in Patients With Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) Treated Under Expanded Access Program
The primary objective of the study is to evaluate the clinical outcomes for the trigger event of patients who receive triheptanoin in the emergency Investigational New Drug (eIND) program.
Read the detailed description
The study is a retrospective chart review of patients in the USA with LC-FAOD who received triheptanoin via eIND request from their metabolic or treating physician before 01 September 2018. Participating study sites will provide the medical records of subjects who meet eligibility criteria, and relevant data regarding the trigger event (the reason the patient was placed on triheptanoin) and key details about hospitalizations during the study period will be extracted.
- Being tested
- No Intervention
- Keywords
- Long-chain Fatty Acid Oxidation Disorders (LC-FAOD), Triheptanoin
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
No InterventionOther
This is a non-interventional retrospective medical record review study. No study investigational product will be administered in this protocol.
| Study group | Role | What they receive |
|---|---|---|
| Patients treated with triheptanoin | Patients with LC-FAOD treated with triheptanoin before 01 September 2018 under eIND Receives: Other: No Intervention |
What is being measured
The primary outcome is the main question the study is designed to answer.
Duration of Hospitalization for Trigger Event
Measured: Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment
Disposition on Discharge of Trigger Event
Measured: Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment
Duration of Important Interventions for Trigger Event
Measured: Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment
Number of Critical Medical Assessments Related to LC-FAOD for Trigger Event
Measured: Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment
Secondary outcomes (4)
Duration of Hospitalization for Other Major Clinical Events
Measured: Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment
Disposition on Discharge of Other Major Clinical Events
Measured: Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment
Number of Important Medical Interventions for Other Major Clinical Events
Measured: Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment
Number of Critical Medical Assessments Related to LC-FAOD for Other Major Clinical Events
Measured: Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Dec 2018
- Study start30 Jan 2019
- Primary completion8 Jun 2020
- Study completion8 Jun 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)Studies of the same condition, open and under way.
- More trials run by Ultragenyx Pharmaceutical IncThe sponsor's other registered studies.
- 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
- Observational
- Observational model
- Case only
- Time perspective
- Retrospective
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Ultragenyx Pharmaceutical Inc
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03768817
- Sponsor's protocol ID
- UX007-CL003
- Status last verified
- Aug 2020
- First submitted
- 5 Dec 2018
- Last updated
- 28 Aug 2020
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Aug 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Retrospective Chart Review Study to Assess the Clinical Outcome of Triheptanoin Treatment in Patients With Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) Treated Under Expanded Access Program
- ClinicalTrials.gov ID
- NCT03768817
- Acronym / sponsor ID
- UX007-CL003
- Keywords
- Long-chain Fatty Acid Oxidation Disorders (LC-FAOD), Triheptanoin
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- Ultragenyx Pharmaceutical IncIndustry (pharma, biotech, devices)
- Study start
- 30 Jan 2019 (actual)
- Primary completion
- 8 Jun 2020 (actual)
- Study completion
- 8 Jun 2020 (actual)
- First posted
- 7 Dec 2018 (actual)
- 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 primary objective of the study is to evaluate the clinical outcomes for the trigger event of patients who receive triheptanoin in the emergency Investigational New Drug (eIND) program.
Detailed description
The study is a retrospective chart review of patients in the USA with LC-FAOD who received triheptanoin via eIND request from their metabolic or treating physician before 01 September 2018. Participating study sites will provide the medical records of subjects who meet eligibility criteria, and relevant data regarding the trigger event (the reason the patient was placed on triheptanoin) and key details about hospitalizations during the study period will be extracted.
Study design
- Observational model
- Case only
- Time perspective
- Retrospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Patients treated with triheptanoin | – | Other: No InterventionPatients with LC-FAOD treated with triheptanoin before 01 September 2018 under eIND |
Who may qualify
- Sex
- All
- Age groups
- Child, Adult, Older adult
- Study population
- Medical records of patients in the USA with LC-FAOD who received triheptanoin via eIND request from their metabolic or treating physician before 01 September 2018.
Key inclusion criteria (4 listed)
- Male or female patients of any age with confirmed diagnosis of one of the LC-FAOD disorders including: mitochondrial trifunctional protein (TFP) deficiency, carnitine palmitoyltransferase deficiencies (CPT I and CPT II), very long chain acyl-CoA dehydrogenase (VLCAD) deficiency, long-chain 3-hydroxy-acyl-CoA dehydrogenase (LCHAD) deficiency, and carnitine acylcarnitine translocase (CATR) deficiency
- Treatment initiation with triheptanoin was via eIND before 01 September 2018
- Treated with triheptanoin in the USA
- Willing and able to provide written, signed informed consent, or where appropriate for subjects under the age of 18, or provide written assent and written informed consent by a legally authorized representative after the nature of the study has been explained and prior to any research-related procedures. To obtain and review medical records of deceased individuals, informed consent from next of kin or appropriate legal entity will be obtained, as applicable.
Key exclusion criteria (1 listed)
- Unwilling to sign informed consent or assent to release of medical records
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 |
|---|---|---|
| No InterventionThis is a non-interventional retrospective medical record review study. No study investigational product will be administered in this protocol. | Other | Patients treated with triheptanoin |
Primary outcome
Duration of Hospitalization for Trigger Event
Time frame: Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment
Disposition on Discharge of Trigger Event
Time frame: Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment
Duration of Important Interventions for Trigger Event
Time frame: Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment
Number of Critical Medical Assessments Related to LC-FAOD for Trigger Event
Time frame: Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment
4 secondary outcomes
Duration of Hospitalization for Other Major Clinical Events
Time frame: Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment
Disposition on Discharge of Other Major Clinical Events
Time frame: Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment
Number of Important Medical Interventions for Other Major Clinical Events
Time frame: Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment
Number of Critical Medical Assessments Related to LC-FAOD for Other Major Clinical Events
Time frame: Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Aug 2020.
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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.
Medical Director study director
Ultragenyx Pharmaceutical Inc
15 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Long-chain Fatty Acid Oxidation Disorders (LC-FAOD), run by Ultragenyx Pharmaceutical Inc at 15 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 1 exclusion criteria. 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: finished, as of the sponsor's last update on 28 Aug 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
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.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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.
What does the primary outcome measure?
Duration of Hospitalization for Trigger Event (time frame: Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
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 28 Aug 2020.