Other Clinical Trial - Efficacy & Safety Study of Fosmetpantotenate

See also
Status	Clinical Trial	Phase
Completed	`NCT02174848` - Long-term Deferiprone Treatment in Patients With Pantothenate Kinase-Associated Neurodegeneration	Phase 3
Active, not recruiting	`NCT04182763` - CoA-Z in Pantothenate Kinase-associated Neurodegeneration (PKAN)	N/A
Completed	`NCT01741532` - Efficacy and Safety Study of Deferiprone in Patients With Pantothenate Kinase-associated Neurodegeneration (PKAN)	Phase 3
No longer available	`NCT02635841` - Compassionate Use of Deferiprone in Patients With PKAN

See also

Status

Clinical Trial

Phase

Completed

NCT02174848 - Long-term Deferiprone Treatment in Patients With Pantothenate Kinase-Associated Neurodegeneration

Phase 3

Active, not recruiting

NCT04182763 - CoA-Z in Pantothenate Kinase-associated Neurodegeneration (PKAN)

N/A

Completed

NCT01741532 - Efficacy and Safety Study of Deferiprone in Patients With Pantothenate Kinase-associated Neurodegeneration (PKAN)

Phase 3

No longer available

NCT02635841 - Compassionate Use of Deferiprone in Patients With PKAN

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	NCT03041116
Study type	Interventional
Source	Travere Therapeutics, Inc.
Contact
Status	Terminated
Phase	Phase 3
Start date	July 17, 2017
Completion date	December 30, 2019

Efficacy and Safety Study of Fosmetpantotenate (RE-024) in PKAN Participants — PKAN

Clinical Trial Summary

Clinical Trial Description

Study Design

Related Conditions & MeSH terms