Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program

Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) Clinical Trial

Official title:

Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program (LC-FAOD DMP)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04632953
• Other study ID #: UX007-CL401
• Status: Recruiting
• Start date: November 30, 2021
• Est. completion date: December 2035

Study information

• Verified date: May 2024
• Source: Ultragenyx Pharmaceutical Inc
• Contact: Patients Contact: Trial Recruitment
• Phone: 1-888-756-8657
• Email: trialrecruitment[@]ultragenyx.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

The primary objective of this study is to assess the long-term safety, including pregnancy, infant, and lactation outcomes, of patients with LC-FAOD who are enrolled in the DMP.

Description:

The LC-FAOD DMP is a global observational long-term prospective outcomes study aiming to collect information for up to 10 years from adult and pediatric patients with LC-FAOD, regardless of disease management, including treatment with triheptanoin, and those who have previously participated in triheptanoin clinical trials or Expanded Access Program (EAP).

Patients enrolling in the LC-FAOD DMP will be managed at the discretion of their physicians and may or may not be treated with triheptanoin during the course of the study. Patients will only have access to triheptanoin through authorized commercial use (if approved in their country) or available EAP but not from the LC-FAOD DMP itself.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 200
• Est. completion date: December 2035
• Est. primary completion date: December 2035
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

Inclusion Criteria:

• Confirmed diagnosis of any LC-FAOD sub-type. Diagnosis must be confirmed by results of acylcarnitine profiles, and/or genetic testing results obtained from medical records or equivalent documentation.

• Willing and able to comply with all study procedures.

• Willing and able to provide consent or, if a minor, provide assent and informed consent by their legally authorized representative.

• Female of child-bearing potential who become pregnant during the study will be invited to remain in the study. Pregnant females with LC-FAOD will be informed of the study and invited to enroll.

• Pregnant females not affected by LC-FAOD carrying a fetus affected by LC-FAOD with confirmed pre-natal diagnosis of LC-FAOD.

Exclusion Criteria:

• Presence of a concurrent disease or condition that would interfere with study participation or affect patient's safety in the opinion of the Investigator.

• Presence or history of any condition that, in the view of the Investigator, places the patient at high risk of not completing the study or would affect the interpretation of study results.

Related Conditions & MeSH terms

• Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)

Intervention

Other:
• No Intervention
No Intervention

Locations

Country	Name	City	State
Canada	University of Alberta	Edmonton	Alberta
Canada	CHEO (Children's Hospital Eastern Ontario)	Ottawa	Ontario
Canada	SickKids (The Hospital for Sick Children)	Toronto	Ontario
United States	The Emory Clinic	Atlanta	Georgia
United States	Children's Hospital of Colorado	Aurora	Colorado
United States	Boston Children's Hospital	Boston	Massachusetts
United States	Ann & Robert H. Lurie Children's Hospital	Chicago	Illinois
United States	Nationwide Children's Hospital	Columbus	Ohio
United States	University of Minnesota	Minneapolis	Minnesota
United States	Columbia University	New York	New York
United States	Phoenix Children's Hospital	Phoenix	Arizona
United States	Children's Hospital of Pittsburgh of UPMC	Pittsburgh	Pennsylvania
United States	University of Utah	Salt Lake City	Utah
United States	University of California San Francisco	San Francisco	California
United States	Seattle Children's Hospital	Seattle	Washington
United States	University of South Florida	Tampa	Florida

Sponsors (1)

Lead Sponsor	Collaborator
Ultragenyx Pharmaceutical Inc

Countries where clinical trial is conducted

United States,  Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Long-Term Safety of Patients with LC-FAOD as Assessed by Incidence of Serious Adverse Events (SAEs) and Adverse Events (AEs) During Pregnancy for all Patients with LC-FAOD		10 years
Primary	Long-Term Safety of Patients with LC-FAOD as Assessed by Neonate and Infant Outcomes from Pregnancy Throughout the First Year of Life		10 years
Primary	Long-Term Safety of Patients with LC-FAOD as Assessed by Incidence of SAEs Assessed as Related to Triheptanoin Treatment by Study Investigator		10 years
Primary	Long-Term Safety of Patients with LC-FAOD as Assessed by Incidence of all Colon Cancer or Gastrointestinal (GI) Cancer, GI Dysplasia, and GI Neoplasia, SAE's and AEs Reported for all Patients with LC-FAOD		10 years
Primary	Long-Term Safety of Patients with LC-FAOD as Assessed by Incidence of all SAEs and AEs in Nursing Mothers and Their Breast-Milk-Fed Infants		10 years
Secondary	Long-term Effectiveness of Triheptanoin in Patients with LC-FAOD as Assessed by Major Clinical Events (MCEs)	Number and duration of MCEs, including rhabdomyolysis, cardiomyopathy, liver damage, and hypoglycemia	10 years
Secondary	Long-term Effectiveness of Triheptanoin in Patients with LC-FAOD as Assessed by Hospitalizations		10 years
Secondary	Long-term Effectiveness of Triheptanoin in Patients with LC-FAOD as Assessed by Mortality		10 years
Secondary	Long-term Effectiveness of Triheptanoin in Patients with LC-FAOD as Assessed by 12-Minute Walk Test (12MWT)		10 years
Secondary	Long-term Effectiveness of Triheptanoin in Patients with LC-FAOD as Assessed by Time to First MCE in Newborns and Pediatrics		10 Years
Secondary	Long-term Effectiveness of Triheptanoin in Patients with LC-FAOD as Assessed by Height/Recumbent Length		10 years
Secondary	Long-term Effectiveness of Triheptanoin in Patients with LC-FAOD as Assessed by Weight		10 years
Secondary	Long-term Effectiveness of Triheptanoin in Patients with LC-FAOD as Assessed by Head Circumference		10 years
Secondary	Natural History, Progression, and Burden of LC-FAOD from the Patient/Caregiver's Perspective as Assessed by the Infant and Toddler Quality of Life (ITQOL)		10 years
Secondary	Natural History, Progression, and Burden of LC-FAOD from the Patient/Caregiver's Perspective as Assessed by the Medical Outcomes Study 10-Item Short Form (SF-10)		10 years
Secondary	Natural History, Progression, and Burden of LC-FAOD from the Patient/Caregiver's Perspective as Assessed by the Medical Outcomes Study 12-Item Short Form (SF-12 v2) assessments		10 years
Secondary	Natural History, Progression, and Burden of LC-FAOD from the Patient/Caregiver's Perspective as Assessed by the Clinical Global Impression of Severity (CGI-S) assessment		10 years
Secondary	Natural History, Progression, and Burden of LC-FAOD from the Patient/Caregiver's Perspective as Assessed by Nutrition Management and Dosing		10 years
Secondary	Natural History, Progression, and Burden of LC-FAOD from the Patient/Caregiver's Perspective as Assessed by Medical Resource Utilization		10 years
Secondary	Natural History, Progression, and Burden of LC-FAOD from the Patient/Caregiver's Perspective as Assessed by LC-FAOD Symptoms and Impact		10 years
Secondary	Natural History, Progression, and Burden of LC-FAOD from the Patient/Caregiver's Perspective as Assessed by the Work Productivity Questionnaire		10 years
Secondary	Natural History, Progression, and Burden of LC-FAOD from the Patient/Caregiver's Perspective as Assessed by the School Impact Questionnaire		10 years

External Links

•   Ultragenyx Patient Advocacy/LC-FAOD Disease Information