Study of Protein Translocation in Patients With Beta-Oxidation Disorders

Clinical Trial Description

PROTOCOL OUTLINE: Patients undergo clinical and molecular analysis of beta-oxidation enzyme metabolism. The evaluation includes a urinary metabolite profile, and DNA and familial studies. ;

Study Design

See also
Status	Clinical Trial	Phase
Recruiting	`NCT02171104` - MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis	Phase 2
Completed	`NCT00007020` - Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid	Phase 3
Completed	`NCT01043640` - Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders	Phase 2
Recruiting	`NCT03163771` - Evaluation of a New Strategy for the Diagnosis of Peroxisomal Diseases

See also

Status

Clinical Trial

Phase

Recruiting

NCT02171104 - MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis

Phase 2

Completed

NCT00007020 - Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid

Phase 3

Completed

NCT01043640 - Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders

Phase 2

Recruiting

NCT03163771 - Evaluation of a New Strategy for the Diagnosis of Peroxisomal Diseases

Interventional studies are often prospective and are specifically tailored to evaluate direct impacts of treatment or preventive measures on disease.

Observational studies are often retrospective and are used to assess potential causation in exposure-outcome relationships and therefore influence preventive methods.

Expanded access is a means by which manufacturers make investigational new drugs available, under certain circumstances, to treat a patient(s) with a serious disease or condition who cannot participate in a controlled clinical trial.

Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.

Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.

NCT number	NCT00004348
Study type	Observational
Source	National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Contact
Status	Completed
Phase
Start date	September 1995
Completion date	March 31, 1998

Study of Protein Translocation in Patients With Beta-Oxidation Disorders

Clinical Trial Summary

Clinical Trial Description

Study Design

Related Conditions & MeSH terms