CDKL5-deficiency disorder

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At a Glance

Live from database

1FDA treatments

5Recruiting trials

Regulatory Watchboard

8 events

Jun 2026Double-blind, Randomized, Placebo-controlled Trial of Ganaxolone in CDKL5 Deficiency Patients 6 Months to Less Than 2 Years Old

Immedica Pharma AB — PHASE3

TrialNOT YET RECRUITING

May 2026A Multicenter Open-label Phase II Study of Cemiplimab Plus Chemotherapy, Selected on the Basis of Baseline Cytidine Deaminase Activity, in Advanced Squamous Non-small Cell Lung Cancer

Gruppo Oncologico Italiano di Ricerca Clinica — PHASE2

TrialNOT YET RECRUITING

Apr 2024Targeting the Gut to Improve Seizure Control in CDKL5 Deficiency Disorder (CDD)

University of Milan — NA

TrialRECRUITING

Sep 2022Observational Study in Patients With Cyclin-dependent Kinase-like 5 Deficiency Disorder

Loulou Foundation

TrialACTIVE NOT RECRUITING

Mar 2022A Study to Investigate the Efficacy and Safety of ZX008 in Subjects With CDKL5 Deficiency Disorder

Zogenix, Inc. — PHASE3

TrialACTIVE NOT RECRUITING

Feb 2021International CDKL5 Clinical Research Network

University of Colorado, Denver

TrialRECRUITING

Apr 2019Dose-Escalation Study of E7727, an Oral Cytidine Deaminase Inhibitor With Oral Decitabine in Subjects With Solid Tumors

Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins — PHASE1

TrialACTIVE NOT RECRUITING

Dec 2018International CDKL5 Registry

University of Pennsylvania — NA

TrialRECRUITING

Data from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Financial Assistance

FINANCIAL LANDSCAPE SUMMARY

0

Total programs

No financial assistance programs currently listed for CDKL5-deficiency disorder.
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Approved Treatments

1 FDA-approved

Ztalmy

(ganaxolone)Orphan drug

Marinus Pharmaceuticals

Neuroactive Steroid Gamma-Aminobutyric Acid A Receptor Positive Modulator [EPC]

12.1 Mechanism of Action The precise mechanism by which ganaxolone exerts its therapeutic effects in the treatment of seizures associated with CDD is ...

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Clinical Trial Landscape

5 active trials

1Phase 3

2N/A

2Unknown

5Total recruiting

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Recent News & Research

No recent news articles indexed yet for CDKL5-deficiency disorder.
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Specialist Network

Top 6 by expertise

Timothy A Benke, MD PhD, MD

University of Colorado, Denver

📍 AURORA, CO

Score 281 trial

Alan K Percy, MD, M.D

University of Alabama at Birmingham

📍 BIRMINGHAM, AL

Score 202 trials1 pub

Aglaia Vignoli, MD

University of Milan

Score 191 trial1 pub

Orrin Devinsky, MD, M.D

NYU Langone Health

📍 NEW YORK, NY

Score 194 trials

Joseph Hulihan, MD, M.D

Marinus Pharmaceuticals, Inc.

📍 NEWTOWN, PA

Score 132 trials

Dennis J Dlugos, MD, M.D

Children's Hospital of Philadelphia

📍 PHILADELPHIA, PA

Score 62 trials

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