Ganaxolone

Generic Name: Ganaxolone

Brand Names: Ztalmy

Drug Type: Small Molecule

Chemical Formula: C_₂₂H_₃₆O_₂

CAS Number: 38398-32-2

Unique Ingredient Identifier: 98WI44OHIQ

Background: Ganaxolone is the 3β-methylated synthetic analog of allopregnanolone, a metabolite of progesterone. Ganaxolone belongs to a class of compounds referred to as neurosteroids. Endogenous neurosteroids, which comprise certain metabolites of progesterone and deoxycorticosterone, bind potently and specifically to GABA receptors to enhance their inhibitory effects, and are thus known to have anxiolytic, analgesic, anticonvulsant, sedative, hypnotic, and anesthetic properties.

Ganaxolone, similar to its endogenous counterparts, is a positive allosteric modulator of GABA receptors. It was approved under the brand name ZTALMY by the US FDA in March 2022 for the treatment of seizures associated with CDKL5 deficiency disorder (CDD), becoming the first FDA-approved treatment indicated specifically for CDD. In July 2023, ganaxolone was also approved under the same brand name and for the same indication by the EMA.

Indication: Ganaxolone is indicated for the treatment of seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients ≥2 years old by the FDA. It is also approved as an adjuvant treatment for the same condition in patients aged 2 to 17, although it may be continued in patients 18 years old or older, by the EMA.

Associated Conditions: Epileptic seizure, Seizures

Associated Therapies: -

Clinical Trials

Augmentation of Treatment-Resistant Depression With An Analog of the Neuroactive Steroid Allopregnanolone

Not Applicable

Completed

Conditions: Major Depressive Disorder

Interventions: Drug: Ganaxolone

First Posted Date: 2016-09-14

Last Posted Date: 2019-02-18

Lead Sponsor: Massachusetts General Hospital

Target Recruit Count: 10

Registration Number: NCT02900092

Locations: 🇺🇸
Massachusetts General Hospital, Boston, Massachusetts, United States

A Two-year Open-label Extension Study of Ganaxolone in Patients With Drug-resistant Partial-onset Seizures

Phase 3

Terminated

Conditions: Drug Resistant Partial Onset Seizure

Interventions: Drug: ganaxolone

First Posted Date: 2015-08-11

Last Posted Date: 2023-06-28

Lead Sponsor: Marinus Pharmaceuticals

Target Recruit Count: 26

Registration Number: NCT02519439

Locations: 🇺🇸
Bluegrass Epilepsy Research, LLC, Lexington, Kentucky, United States
🇺🇸
Minneapolis Clinic of Neurology, Golden Valley, Minnesota, United States
🇺🇸
Neurological Research Institute, Santa Monica, California, United States

and more 2 locations

Open-Label PoC Trial of Ganaxolone in Children With PCDH19 Female Pediatric Epilepsy and Other Rare Genetic Epilepsies

Phase 2

Completed

Conditions: Epilepsy

Interventions: Drug: Ganaxolone

First Posted Date: 2015-02-09

Last Posted Date: 2023-03-21

Lead Sponsor: Marinus Pharmaceuticals

Target Recruit Count: 30

Registration Number: NCT02358538

Locations: 🇺🇸
Center for Rare Neurological Diseases, Norcross, Georgia, United States
🇮🇹
Bambino Gesu Children's Hospital, IRCCS, Rome, Italy
🇺🇸
Phoenix Children's Hospital, Phoenix, Arizona, United States

and more 7 locations

Phase 3 Study of Adjunctive Ganaxolone in Adults With Drug-resistant Partial Onset Seizures and Open-label Extension

Phase 3

Completed

Conditions: Drug Resistant Partial Onset Seizure

Interventions: Drug: ganaxolone
Drug: Placebo

First Posted Date: 2013-10-16

Last Posted Date: 2023-02-14

Lead Sponsor: Marinus Pharmaceuticals

Target Recruit Count: 405

Registration Number: NCT01963208

Locations: 🇺🇸
The MORE Foundation, Sun City, Arizona, United States
🇺🇸
Clinical Trials Inc., Little Rock, Arkansas, United States
🇦🇺
The Royal Melbourne Hospital, Parkville, Victoria, Australia

and more 57 locations

Proof-Of-Concept Investigation With a Neurosteroid Analog (Ganaxolone) as a Smoking Cessation Candidate

Phase 2

Completed

Conditions: Smoking Cessation

Interventions: Drug: Ganaxolone
Drug: Nicotine Patch

First Posted Date: 2013-05-20

Last Posted Date: 2014-09-08

Lead Sponsor: Jed E. Rose

Target Recruit Count: 36

Registration Number: NCT01857531

Locations: 🇺🇸
Duke Center for Smoking Cessation, Winston-Salem, North Carolina, United States

Ganaxolone Treatment in Children With Fragile X Syndrome

Phase 2

Completed

Conditions: Fragile x Syndrome

Interventions: Drug: Ganaxolone
Drug: Placebo

First Posted Date: 2012-11-12

Last Posted Date: 2023-04-10

Lead Sponsor: Marinus Pharmaceuticals

Target Recruit Count: 59

Registration Number: NCT01725152

Locations: 🇧🇪
Antwerp University Hospital, Edegem, Belgium
🇺🇸
M.I.N.D. Institute at University of California at Davis Medical Center, Sacramento, California, United States

Ganaxolone in Posttraumatic Stress Disorder (PTSD)

Phase 2

Completed

Conditions: PTSD

Interventions: Drug: Placebo
Drug: Ganaxolone

First Posted Date: 2011-04-21

Last Posted Date: 2022-12-29

Lead Sponsor: Marinus Pharmaceuticals

Target Recruit Count: 112

Registration Number: NCT01339689

Locations: 🇺🇸
White River Junction VA Medical Center/ Dartmouth College, White River Junction, Vermont, United States
🇺🇸
Washington DC VA Medical Center/ Uniformed Services University of the Health Services, Washington, District of Columbia, United States
🇺🇸
Durham VA Medical Center /Duke University Medical Center, Durham, North Carolina, United States

and more 5 locations

A Treatment Use Protocol for Subjects Continuing on From the Open-label Extension 0601

Phase 2

Completed

Conditions: Epilepsy, Complex Partial

Interventions: Drug: ganaxolone

First Posted Date: 2009-10-27

Last Posted Date: 2022-09-07

Lead Sponsor: Marinus Pharmaceuticals

Target Recruit Count: 11

Registration Number: NCT01002820

Locations: 🇺🇸
Ohio State University, Columbus, Ohio, United States
🇺🇸
Riddle Health Care Center II, Philadelphia, Pennsylvania, United States
🇺🇸
Thomas Jefferson University, Comprehensive Epilepsy Center, Philadelphia, Pennsylvania, United States

and more 4 locations

Open-label Extension to Protocol 1042-0600

Phase 2

Completed

Conditions: Epilepsies, Partial

Interventions: Drug: ganaxolone

First Posted Date: 2007-08-07

Last Posted Date: 2023-01-17

Lead Sponsor: Marinus Pharmaceuticals

Target Recruit Count: 123

Registration Number: NCT00512317

Locations: 🇺🇸
University of California-Davis, Sacramento, California, United States
🇺🇸
Southern Illinois University Medical Center, Springfield, Illinois, United States
🇺🇸
Arkansas Epilepsy Program, Little Rock, Arkansas, United States

and more 22 locations

A Randomized, Controlled Trial of Ganaxolone in Adult Uncontrolled Partial-Onset Seizures

Phase 2

Completed

Conditions: Partial Epilepsy
Catamenial Epilepsy

Interventions: Other: Placebo
Drug: Ganaxolone

First Posted Date: 2007-04-25

Last Posted Date: 2023-03-15

Lead Sponsor: Marinus Pharmaceuticals

Target Recruit Count: 147

Registration Number: NCT00465517

Locations: 🇺🇸
SUNY Upstate Medical University, Syracuse, New York, United States
🇺🇸
University of Southern California Adult Comprehensive Epilepsy Center, Los Angeles, California, United States
🇺🇸
Neurosciences Institute at Albany Medical Center, Albany, New York, United States

and more 24 locations

Drug Development Updates

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