Recruiting

Observational Study

Sponsor:

Icahn School of Medicine at Mount Sinai

Code:

NCT07734090

Conditions

Multiple Acyl-CoA Dehydrogenase Deficiency

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Not accepted

Study Details

Brief summary:

The objective of this study is to conduct a longitudinal, observational investigation to determine the natural history of Multiple Acyl-CoA Dehydrogenase Deficiency (MADD), delineate the spectrum of its clinical features and their progression, identify biomarkers, and develop and validate patient reported outcomes.

Conditions

Multiple Acyl-CoA Dehydrogenase Deficiency

Study ID

NCT07734090

Start date

Oct, 2026

Status verified date

Sep, 2026

Completion date

Jul, 2031

Anticipated

Primary completion date

Jul, 2031

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Not accepted

Inclusion Criteria:

  • Have a biochemical and/or molecular diagnosis of Multiple Acyl-CoA Dehydrogenase Deficiency (MADD), as confirmed by a study investigator
  • Provision of signed and dated informed consent form (and assent when applicable) from subject or subject's legal representative

Exclusion Criteria:

\- Presence of a major unrelated condition

Study Design

Enrollment

50 participants

Anticipated

Interventions and Outcome Measures

Arms

Multiple Acyl-CoA Dehydrogenase Deficiency (MADD)

Patients with a molecular and/or biochemical diagnosis of Multiple Acyl-CoA Dehydrogenase Deficiency (MADD).

Primary outcome measure

  • Number of participants enrolled in the patient registry [ Time Frame: 5 years ]

Central Contacts and Locations

Central contacts

Mary Freeman, MS, CGC

(347) 668-9162mary.freeman@mssm.edu

Locations

Icahn School of Medicine at Mount Sinai

Recruiting

New York, New York, United States, 10029

More Information

Sponsor

Icahn School of Medicine at Mount Sinai

Last update posted

Sep 16, 2026

Last verified

Sep, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-18. This information was provided to ClinicalTrials.gov by Icahn School of Medicine at Mount Sinai on 2026-09-16.