CDD Clinical Trial - Fenfluramine in CDKL5 Deficiency Disorder (CDD)

Clinical Trial Summary

This study will be enrolling 10 patients, ages 2-18 years old, with a confirmed genetic/clinical diagnosis of CDKL5 Deficiency Disorder (CDD) in an open label trial of fenfluramine for seizure control. Patients will be titrated over 14 days to a dose of ZX008 0.8 mg/kg/day (maximum dose 30 mg/d).

See also
	Status	Clinical Trial	Phase
	Recruiting	`NCT05558371` - International CDKL5 Clinical Research Network

See also

Status

Clinical Trial

Phase

Recruiting

NCT05558371 - International CDKL5 Clinical Research Network

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	NCT03861871
Study type	Interventional
Source	NYU Langone Health
Contact
Status	Completed
Phase	Phase 2
Start date	October 29, 2019
Completion date	October 24, 2022

Fenfluramine in CDKL5 Deficiency Disorder (CDD)

Clinical Trial Summary

Clinical Trial Description

Study Design

Related Conditions & MeSH terms