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Fenfluramine in CDD

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03861871
Recruitment Status : Not yet recruiting
First Posted : March 4, 2019
Last Update Posted : March 4, 2019
Information provided by (Responsible Party):
NYU Langone Health

Brief 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).

Condition or disease Intervention/treatment Phase
CDD Drug: Fenfluramine Hydrochloride Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 10 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Fenfluramine in CKDL5 Deficiency Disorder (CDD)
Estimated Study Start Date : April 2019
Estimated Primary Completion Date : April 2021
Estimated Study Completion Date : April 2021

Arm Intervention/treatment
Experimental: Fenfluramine Hydrochloride Drug: Fenfluramine Hydrochloride
Oral Solution 2.5mg/mL
Other Name: ZX008

Primary Outcome Measures :
  1. Median monthly convulsive seizure frequency [ Time Frame: 14 Weeks ]

Secondary Outcome Measures :
  1. Caregiver Global Impression of Change (CGIC). [ Time Frame: 14 Weeks ]
    The parent/caregiver and investigator rated change on a 7-point scale: 1-very much improved; 2-much improved; 3-minimally improved; 4-no change; 5-minimally worse; 6-much worse; and 7-very much worse

  2. Quality of Life in Childhood Epilepsy Scale [ Time Frame: 14 Weeks ]
    A low-burden parent/caregiver completed assessment that looks at how epilepsy affects day-to-day functioning of their child in various life areas, including physical activities, well-being, cognition, social activities, behavior and general health.

  3. Pediatric Quality Of Life Inventory (PEDS-QL) [ Time Frame: 14 Weeks ]
    A parent/caregiver completed assessment that looks at how epilepsy affects day-to-day functioning of their child in various life areas, including physical activities, well-being, cognition, social activities, behavior and general health during a specific time frame

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   2 Years to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Confirmed clinical/genetic diagnosis of CDKL5 Deficiency Disorder CDD
  • Ages 2-18 years old. Subject is male or non-pregnant, non-lactating female. Female subjects of childbearing potential must not be pregnant or breast-feeding. Female subjects of childbearing potential must have a negative urine pregnancy test. Subjects of childbearing or child-fathering potential must be willing to use medically acceptable forms of birth control, which includes abstinence, while being treated on this study and for 30 days after the last dose of study drug.
  • Subject has been informed of the nature of the study and informed consent has been obtained from the legally responsible parent/guardian.
  • Subject has provided assent in accordance with Investigational Review Board/Independent Ethics Committee (IRB/IEC) requirements, if capable.
  • Subject's caregiver is willing and able to be compliant with diary completion, visit schedule and study drug accountability.
  • Subjects must be receiving a therapeutically relevant and stable dose of anti-seizure medications, dietary therapies for epilepsy or vagus nerve stimulation settings for at least 4 weeks prior to screening and are expected to remain stable throughout the study.
  • ≥4 convulsive seizures (tonic-clonic, tonic, atonic, clonic, focal motor) per 4-week period; each convulsive seizure must last ≥3 seconds.

Exclusion Criteria:

  • Subject has a known hypersensitivity to fenfluramine or any of the excipients in the study medication.
  • Subject has current or past history of cardiovascular or cerebrovascular disease, myocardial infarction or stroke.
  • Subject has current or past history of cardiovascular or cerebrovascular disease, such as cardiac valvulopathy, myocardial infarction or stroke, or clinically significant structural cardiac abnormality, including but not limited to mitral valve prolapse, atrial or ventricular septal defects, patent ductus arteriosis, and patent foramen ovale with reversal of shunt. (note: Patent foramen ovale or a bicuspid valve are is not considered exclusionary, but may be associated with the following diseases, which are exclusionary: coarctation of the aorta, Turner syndrome, supravalvular aortic stenosis, subvalvular aortic stenosis, patent ductus arteriosus, Sinus of Valsalva aneurysm, ventricular septal defect, Shone's complex, ascending aortic aneurysm, Loeys-Dietz syndrome, ACTA2 mutation familial thoracic aortic aneurysm syndrome, and MAT2A mutation familial thoracic aortic aneurysm syndrome).
  • Subject has current or recent history of Anorexia Nervosa, bulimia, or depression within the prior year that required medical treatment or psychological treatment for a duration greater than 1 month.
  • Subjects who are currently on CBD/THC or any MMJ or those who have tested positive urine tetrahydrocannabinol (THC) Panel or whole blood cannabidiol (CBD).
  • Subject has participated in another clinical trial within the past 30 days (calculated from that study's last scheduled visit).
  • Subject is at imminent risk of self-harm or harm to others, in the investigator's opinion, based on clinical interview.
  • Subject has a current or past history of glaucoma.
  • Subject is receiving concomitant therapy with: centrally-acting anorectic agents; monoamine-oxidase inhibitors; any centrally-acting compound with clinically appreciable amount of serotonin agonist or antagonist properties, including serotonin reuptake inhibition; atomoxetine, or other centrally-acting noradrenergic agonist; or cyproheptadine. (see appendix 1)
  • Subject has moderate or severe hepatic impairment. Asymptomatic subjects with mild hepatic impairment (elevated liver enzymes <3x upper limited of normal [ULN] and/or elevated bilirubin <2x ULN) may be entered into the study after review and approval by the Medical Monitor in conjunction with the sponsor, in consideration of comorbidities and concomitant medications.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03861871

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Contact: Latoya King 646 558 0838

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United States, New York
New York Langone Health Not yet recruiting
New York, New York, United States, 10016
Contact: Latoya King    646-558-0838   
Principal Investigator: Latoya King, MD         
Sponsors and Collaborators
NYU Langone Health
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Principal Investigator: Orrin Devinsky, MD NYU Langone Health

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Responsible Party: NYU Langone Health Identifier: NCT03861871     History of Changes
Other Study ID Numbers: 18-01530
First Posted: March 4, 2019    Key Record Dates
Last Update Posted: March 4, 2019
Last Verified: March 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No

Keywords provided by NYU Langone Health:
CKDL5 Deficiency Disorder

Additional relevant MeSH terms:
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Serotonin Uptake Inhibitors
Neurotransmitter Uptake Inhibitors
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action
Neurotransmitter Agents
Serotonin Agents
Physiological Effects of Drugs