Retrospective Observational Safety Effectiveness With Kuvan in hpA

See also
	Status	Clinical Trial	Phase
	Completed	`NCT04375592` - Acceptability and Tolerance of a Ready-to-use Protein Substitute in Tablet Form for the Dietary Management of Phenylketonuria
	Withdrawn	`NCT04014712` - O2 Transport and Utilization in Health and Lung Disease	Phase 1

See also

Status

Clinical Trial

Phase

Completed

NCT04375592 - Acceptability and Tolerance of a Ready-to-use Protein Substitute in Tablet Form for the Dietary Management of Phenylketonuria

Withdrawn

NCT04014712 - O2 Transport and Utilization in Health and Lung Disease

Phase 1

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	NCT03864029
Study type	Observational
Source	BioMarin Pharmaceutical
Contact
Status	Completed
Phase
Start date	October 10, 2017
Completion date	July 25, 2018

Retrospective Observational Safety Effectiveness With Kuvan in hpA — ROSEKA

Clinical Trial Summary

Clinical Trial Description

Study Design

Related Conditions & MeSH terms