RegulationPriority Review (United States), Orphan Drug (United States), Orphan Drug (European Union), Priority Review (China), Rare Pediatric Disease (United States)

Structure/Sequence

Molecular FormulaC22H36O2

InChIKeyPGTVWKLGGCQMBR-FLBATMFCSA-N

CAS Registry38398-32-2

Clinical Trials associated with Ganaxolone

NCT05249556

/ Not yet recruitingPhase 3

Double-blind, Randomized, Placebo-controlled Trial of Adjunctive Ganaxolone in the Treatment of Seizures Associated With Genetically Confirmed Cyclin-dependent Kinase-like 5 (CDKL5) Deficiency Disorder (CDD) in Pediatric Patients From 6 Months to Less Than 2 Years of Age.

This study will assess the efficacy, safety, and tolerability of ganaxolone (GNX) compared with placebo (PBO) as adjunctive therapy to the participant's standard anti-epileptic medication for the treatment of seizures in pediatric patients from 6 months to less than 2 years old with genetically confirmed CDD during a 12-week, DB phase. Pharmacokinetic (PK) assessments and population PK analyses will also be performed during this time. The DB phase will be followed by an optional long-term OL phase at which time all participants will receive GNX as an adjunct to their standard anti-seizure medication. Efficacy, safety and tolerability, and PK assessments will continue to be performed.

Start Date01 Mar 2027

Sponsor / Collaborator

Immedica Pharma AB

NCT07635862

/ Not yet recruitingPhase 2IIT

A Randomized Controlled Trial of Ganaxolone for Children With Autism Spectrum Disorder

The goal of this study is to conduct a randomized, placebo-controlled trial (RCT) of ganaxolone, a neuroactive steroid (NAS), in autistic children and adolescents aged 5 to 17 years old. Ganaxolone is approved and effective for treating seizures in children as young as 2 years old who have CDKL5 deficiency disorder (CDD), a neurogenetic condition associated with developmental delays, seizure disorder, hypotonia, visual impairments, and autistic features. The primary outcome of interest for this trial is irritability on the Aberrant Behavior Checklist (ABC) because it is a common symptom of emotion dysregulation in ASD that impacts quality of life, including mental health, independence, educational opportunities, and integration into the community. The secondary domains of interest for this trial are restricted and repetitive behaviors (RRB), specifically insistence on sameness (IS), a subdomain of RRB characterized by inflexibility and a strong preference for predictable routines and familiar environments. Secondary outcome measures include the IS subscale from the Dimensional Assessment of Repetitive Behaviors (DARB) and subscales of the Clinical Global Impressions Scale for irritability (CGI-IR) and IS (CGI-IS). For participants living within 150 miles of Stanford University, we require participants to attend site visits and attempt EEG and MRI procedures before and after the trial, though we are recruiting nationally and the study can be completed without site vists.

Start Date01 Jan 2027

Sponsor / Collaborator

Stanford University

[+1]

CTR20250141

/ Active, not recruitingPhase 4

一项关于加那索龙口服混悬剂治疗2岁及以上细胞周期蛋白依赖性激酶5（CDKL5）缺乏症（CDD）中国患者癫痫发作的安全性和有效性的多中心、前瞻性、观察性上市后研究

[Translation] A multicenter, prospective, observational post-marketing study to investigate the safety and efficacy of ganaxolone oral suspension for the treatment of seizures in Chinese patients aged 2 years and older with cyclin-dependent kinase 5 (CDKL5) deficiency (CDD)

主要目的评价加那索龙口服混悬剂用于治疗中国CDD患者癫痫发作的安全性次要目的评价加那索龙口服混悬剂用于治疗中国CDD患者癫痫发作的有效性

[Translation]

Primary objective To evaluate the safety of ganaxolone oral suspension in the treatment of epileptic seizures in Chinese CDD patients Secondary objective To evaluate the effectiveness of ganaxolone oral suspension in the treatment of epileptic seizures in Chinese CDD patients

Start Date22 Apr 2025

Sponsor / Collaborator

Altasciences Cdmo Philadelphia LLC

[+2]

100 Clinical Results associated with Ganaxolone

100 Translational Medicine associated with Ganaxolone

100 Patents (Medical) associated with Ganaxolone

227

Literatures (Medical) associated with Ganaxolone

01 Dec 2026·MOLECULAR BIOLOGY REPORTS

Molecular mechanisms of neurosteroids in temporal lobe epilepsy: current insights and therapeutic perspectives

Review

Author: Al-Qahtani, Zainah ; Al-Gareeb, Ali I ; Hussain, Nawar R ; Waheed, Huda J ; Al-Kuraishy, Hayder M ; Mustafa, Aya M ; El-Saber Batiha, Gaber ; Papadakis, Marios ; Albuhadily, Ali K ; Alexiu, Athanasios ; Alruwaili, Mubarak

Neurosteroids (NSs) are endogenous progesterone derivatives that act as natural allosteric modulators of GABA_A receptors, exerting distinct effects from conventional receptor agonists. They play critical roles in modulating neuronal excitability, cognitive function, and stress responses, and have shown potential in mitigating seizure severity and frequency, particularly in temporal lobe epilepsy (TLE). TLE is a common drug-resistant focal epilepsy characterized by complex pathophysiology involving hippocampal sclerosis, neuroinflammation, synaptic dysfunction, and alterations in GABA_A receptor subunits. Accumulating evidence indicates that NSs, including Allopregnanolone, brexanolone, and ganaxolone, may exert neuroprotective and anti-epileptogenic effects through both direct GABAergic modulation and indirect regulation of mTORC1, AMPK, and BDNF signaling pathways, as well as autophagy activation. Experimental studies demonstrate that NSs can prolong the latent period before epilepsy onset, reduce seizure recurrence, and potentially improve cognitive outcomes. However, clinical findings remain inconsistent, and prolonged NS exposure may lead to reduced receptor sensitivity. This review synthesizes current knowledge on the mechanistic roles of NSs in TLE, highlighting their potential as adjuvant therapies alongside antiepileptic drugs. Further translational studies are warranted to clarify optimal dosing strategies, identify patient subgroups most likely to benefit, and develop targeted NS analogues for refractory TLE management.

01 Nov 2026·CNS DRUGS

Cognitive Side Effects of Antiseizure Medications in Adults with Epilepsy: An Update with a Focus on New Therapeutic Agents

Review

Author: Mula, Marco

Antiseizure medications (ASMs) are the mainstay of epilepsy treatment but may adversely affect cognitive functions, deepening the cognitive and psychosocial burden intrinsic to epilepsy. Cognitive side effects vary widely across drug classes, doses, treatment regimens, and individual susceptibility, typically affecting attention, processing speed, memory, language, and executive function. This narrative review provides an updated synthesis of the clinical evidence on the cognitive effects of ASMs, mostly in adults, building on a prior 2009 review and focusing on agents introduced into clinical practice since then. A literature search of PubMed identified studies published between January 2009 and December 2025, yielding data from randomised controlled trials, observational studies, meta-analyses, and systematic reviews. Overall, newer-generation ASMs, including rufinamide, lacosamide, brivaracetam, cannabidiol, fenfluramine, and ganaxolone, demonstrate generally favourable cognitive profiles when used at recommended doses, particularly in monotherapy or rational polytherapy. Eslicarbazepine and cenobamate may be associated with mild, dose-dependent cognitive effects, occurring only at the upper end of the recommended dose range. In contrast, old ASMs and certain second-generation agents, notably topiramate and zonisamide, remain consistently associated with higher cognitive risks. Special populations, including older adults and individuals with intellectual disabilities, are particularly vulnerable to cognitive adverse effects and benefit from agents with low interaction potential and benign neuropsychological profiles. Cognitive dysfunction in epilepsy is multifactorial, reflecting the interaction between disease-related neurobiological mechanisms and treatment effects. Optimal management requires balancing seizure control with cognitive preservation through individualised drug selection, cautious titration, and minimisation of polytherapy to achieve the best functional and quality-of-life outcomes.

01 Oct 2026·Orthodontics & Craniofacial Research

Machine Learning‐Based Model for Predicting Short‐ and Long‐Term Growth in Untreated Class III Malocclusion

Article

Author: Nieri, Michele ; Rutili, Valentina ; Chicchi, Lorenzo ; Auconi, Pietro ; McNamara, James A. ; Giuntini, Veronica ; Buffoni, Lorenzo ; Franchi, Lorenzo ; Statie, Maria Denisa

ABSTRACT:

Objective:

To develop a Machine Learning‐based model to predict growth of Caucasian subjects with untreated Class III malocclusion in the short‐ and long‐term.

Materials and Methods:

A longitudinal sample of 144 Caucasian subjects with untreated Class III malocclusion was selected (80% training data; 20% test data). Cephalograms of the subjects of the test group were divided into short‐ and long‐term observations. Sixteen cephalometric landmarks were digitised in an X‐Y Cartesian coordinate system. The trained model was a Graph Neural Network. A one‐sample t ‐test and the Euclidean distances between predicted and observed values were calculated.

Results:

In the short‐term prediction, 16 subjects were examined. On the X‐Y axis, the following cephalometric points were statistically significant: SX, PgY, BY, PNSY, NY and SY. Mean Euclidean distance between predicted and actual values revealed high values for the mandibular points Go (2.6 mm), Me (1.9 mm), Gn (1.9 mm), Pg (2.0 mm), and B (2.0 mm). In the long‐term prediction, 13 subjects were examined. On the X‐Y axis, the following cephalometric points were statistically significant: MeX, GnX, PgX, BX, BY and PtY. Mean Euclidean distance between predicted and actual values revealed high values for the mandibular points Go (3.1 mm), Me (4.3 mm), Gn (4.1 mm), Pg (4.5 mm), and B Point (4.1 mm).

Conclusions:

The ML‐based prediction model was accurate for the majority of the landmarks. Cephalometric mandibular landmarks (Go, Me, Gn, Pg, and B Points) showed the highest mean Euclidean distances between predicted and observed values, indicating lower prediction accuracy.

149

News (Medical) associated with Ganaxolone

26 Oct 2026

·www.immedica.com

Immedica to advance IV ganaxolone into a new phase 3 study in refractory status epilepticus

Stockholm, Sweden, October 26, 2026 - Immedica Pharma AB ("Immedica") today announced that it will advance the development of intravenous (IV) ganaxolone for the treatment of refractory status epilepticus (RSE) and plans to initiate a new phase 3 clinical study in 2027. The decision follows an extensive review of the available clinical data and productive interactions with the U.S. Food and Drug Administration (FDA), which have provided a clear regulatory path forward for the program. Immedica is now progressing the activities required to prepare and operationalize the new study, including finalization of the study design, manufacturing of clinical trial material and clinical site planning. RSE is a life-threatening neurological emergency in which ongoing seizure activity does not respond to at least two different antiseizure medications. Patients who continue to experience seizures after first- and second-line treatment may require intravenous anesthetics and medically induced coma, involving invasive treatment and intensive care. Despite the severity of the condition, treatment options remain limited, and there is currently no approved therapy specifically for the condition. There is a significant need for therapies that can rapidly control seizures and reduce the need for escalation to intravenous anesthesia. "Advancing IV ganaxolone into a new phase 3 study is an important step for Immedica and reflects our commitment to developing treatments for people affected by severe conditions with high unmet medical need," said Anders Edvell, CEO of Immedica. "The program also marks an important evolution for our company as we broaden our capabilities beyond commercialization and strengthen our focus on clinical development. Following a comprehensive evaluation of the clinical evidence and constructive discussions with the FDA, we believe there is a well-defined path forward, and we are now focused on preparing the study and bringing IV ganaxolone into clinical development." "Refractory status epilepticus is a serious medical emergency for which additional treatment options are urgently needed. IV ganaxolone works through a mechanism distinct from currently used therapies. A new phase 3 study is expected to elucidate the role of IV ganaxolone for patients whose seizures continue despite standard treatment", commented Dr Aatif M. Husain, Professor of Neurology and Division Chief, Epilepsy, Sleep, and Clinical Neurophysiology. Duke University School of Medicine, Durham, NC, USA Immedica acquired the global rights to ganaxolone through its acquisition of Marinus Pharmaceuticals in 2025. The company is building on the existing clinical and scientific evidence for the molecule and expanding its clinical development capabilities within rare neurology. About refractory status epilepticus (RSE) Status epilepticus (SE) is a life-threatening, neurological emergency characterized by prolonged, self-sustaining or repeated uncontrolled seizure activity without sufficient recovery between seizures. Management of SE centers around rapid and sustained seizure control. Refractory SE (RSE) is defined as SE resistant to treatment with one first-line ASM (e.g. benzodiazepines) and one second-line ASM. The condition is associated with a risk of neurological injury, including alterations of neuronal networks and neuronal death, complications from prolonged intensive care, and high morbidity and mortality. Current treatment may involve intravenous anesthetics and medically induced coma. These interventions have been associated with additional morbidity, highlighting the need for additional treatment options that can rapidly and durably control seizures. About IV ganaxolone Internalization of GABAA receptors occurs within minutes after SE onset. This results in functional loss of GABAergic inhibition and contributes to SE becoming treatment resistant. While extrasynaptic GABAA receptors are not internalized, they are insensitive to standard GABAergic agents. Ganaxolone, a neuroactive steroid and a synthetic analogue of allopregnanolone, positively modulates both synaptic and extrasynaptic GABAA receptors, thus enhancing inhibitory signaling in the central nervous system. The intravenous formulation of ganaxolone is being developed as an investigational treatment for refractory status epilepticus. IV ganaxolone is not approved for the treatment of refractory status epilepticus. About Immedica Immedica is a pharmaceutical company, headquartered in Stockholm, Sweden, focused on the commercialization of medicines for rare diseases and specialty care products. Immedica's capabilities cover marketing and sales, market access, compliance, pharmacovigilance, quality assurance, regulatory, medical affairs and clinical development, as well as a global distribution network serving patients in more than 50 countries. Immedica is fully dedicated to helping those living with diseases which have a large unmet medical need. Immedica's therapeutic areas are within RARE metabolic, RARE hematology & oncology, RARE neurology, RARE endocrinology and specialty care. Immedica was founded in 2018 and employs today around 200 people across Europe, the Middle East and the United States. Immedica is backed by the investment firms KKR and Impilo. For more information visit www.immedica.com. Immedica contact: Linda Holmström Head of HR & Communication linda.holmstrom@immedica.com We believe we have an important role to fill in society – bringing new, innovative treatments to patients with rare diseases. Focus areas are within RARE metabolic, RARE neurology, RARE hematology & oncology and specialty care. We operate products from phase 2 and all the way to mature products. ESG is an integrated part of our business model. Stay up to date with our latest news. Telephone: +46 (0) 8 533 39 500 E-mail: info@immedica.com

Phase 3Drug Approval

10 Sep 2026

·www.immedica.com

Abstract highlighting three-year ganaxolone data in CDD presented at EEC 2026

Stockholm, Sweden, Sept 10, 2026 - Immedica Pharma AB announced that data evaluating the long-term efficacy of ganaxolone in patients with CDKL5 deficiency disorder (CDD) was presented at the 16th European Epilepsy Congress (EEC), taking place in Athens, Greece, September 5-9, 2026. The poster presentation will feature results from a post-hoc analysis titled "Sustained clinical efficacy of ganaxolone in CDKL5 deficiency disorder: three-year follow-up." The analysis evaluated long-term seizure outcomes in patients who participated in the open-label extension phase following the pivotal, Phase III Marigold study (NCT03572933). 88 patients (aged 2 to 19) who completed the 17-week double-blind phase continued into the open-label extension study, where all participants received ganaxolone treatment. The study was completed after 233 weeks (~4.25 years; inclusive of the 17-week double blind phase) after FDA approval was granted in the United States in March of 2022. Results demonstrated sustained reductions in major motor seizure frequency (MMSF) throughout the open-label extension period, with the proportion of participants with ≥50% reduction from baseline in 28-day MMSF consistently having met or exceeded that achieved in the ganaxolone group during the double-blind phase (24.5%) at all 3-month intervals for the first 3 years of treatment in the open-label extension. After 163 weeks (~3 years) of ganaxolone treatment in the Marigold open label extension, the median change from baseline in the 28-day MMSF was 35.6%, and 42.1% of participants demonstrated ≥50% reduction in the 28-day MMSF. No new safety findings were identified during the open label extension. Poster Details Title: Sustained clinical efficacy of ganaxolone in CDKL5 deficiency disorder: three-year follow-up Presenter: Sam Amin, Department of Paediatric Neurology, Bristol Royal Hospital for Children, United Kingdom Authors: Sam Amin, Department of Paediatric Neurology, Bristol Royal Hospital for Children, Bristol, United Kingdom, Michelle L. Shuffett (submitting author): Immedica Pharma US Inc., Chicago, IL, Jenny Samuelsson: Immedica Pharma AB, Stockholm, Sweden, Kristina Lindsten: Immedica Pharma AB, Stockholm, Sweden, Kenneth Johnson: Immedica Pharma US Inc., Chicago, IL, Christine Ochoa Escamilla: Immedica Pharma US Inc., Chicago, IL, Scott T. Demarest: Children's Hospital Colorado, Department of Pediatrics, University of Colorado, School of Medicine, University of Colorado, Aurora, CO, USA. About ZTALMY® (ganaxolone) ZTALMY® is indicated specifically for seizures associated with cyclin-dependent kinase-like 5 deficiency disorder (CDD) in patients two years of age and older. ZTALMY® is approved in the United States, the European Union, United Kingdom, China, Kuwait and UAE. ZTALMY is a neuroactive steroid anticonvulsant, that acts as a positive allosteric modulator of GABAA receptors in the central nervous system. About CDKL5 Deficiency Disorder CDKL5 deficiency disorder (CDD) is a serious and rare genetic disorder that is caused by a pathogenic variant in the cyclin-dependent kinase-like 5 (CDKL5) gene, located on the X chromosome. CDD is characterized by early-onset, difficult-to-control seizures and severe neuro-developmental impairment. About the Marigold Trial ZTALMY was approved by the U.S. Food and Drug Administration in March 2022 based on data from the Phase 3 Marigold double-blind placebo-controlled trial of 101 patients with CDD, published in The Lancet Neurology, in which ZTALMY significantly reduced the frequency of monthly major motor seizures by a median of 30.7% compared with 6.9% for placebo (p=0.0036). In this trial, ZTALMY demonstrated efficacy, safety and tolerability with the most common adverse reactions (incidence ≥5% and at least twice the rate of placebo) in the ZTALMY group being somnolence, pyrexia, salivary hypersecretion and seasonal allergy. About Immedica Immedica is a pharmaceutical company, headquartered in Stockholm, Sweden, focused on the commercialization of medicines for rare diseases and specialty care products. Immedica's capabilities cover marketing and sales, compliance, pharmacovigilance, quality assurance, regulatory, medical affairs and market access, as well as a global distribution network serving patients in more than 50 countries. Immedica is fully dedicated to helping those living with diseases which have a large unmet medical need. Immedica's therapeutic areas are within RARE metabolic, RARE hematology & oncology, RARE neurology, RARE Endocrinology and specialty care. Immedica was founded in 2018 and employs today around 180 people across Europe, the Middle East and the United States. Immedica is backed by the investment firms KKR and Impilo. For more information visit www.immedica.com. Immedica contact: Linda Holmström Head of HR & Communication linda.holmstrom@immedica.comFormulärets överkant Formulärets nederkant We believe we have an important role to fill in society – bringing new, innovative treatments to patients with rare diseases. Focus areas are within RARE metabolic, RARE neurology, RARE hematology & oncology and specialty care. We operate products from phase 2 and all the way to mature products. ESG is an integrated part of our business model. Stay up to date with our latest news. Telephone: +46 (0) 8 533 39 500 E-mail: info@immedica.com

Clinical ResultDrug ApprovalPhase 3

25 Jun 2026

·www.immedica.com

Immedica announces marketing authorization approval in the UAE for Ztalmy® (ganaxolone) for CDKL5 deficiency disorder

Stockholm, Sweden, June 23, 2026 – Immedica today announced that the Emirates Drug Establishment (EDE) has granted Marketing Authorization (MA) approval for Ztalmy® (ganaxolone) for the treatment of seizures associated with CDKL5 deficiency disorder (CDD) in the United Arab Emirates (UAE). This marks an important milestone in making Ztalmy available to eligible patients in the UAE. “The approval of Ztalmy in the UAE is an important step in expanding access to treatment options for patients living with CDD,” said Ashraf Attia, General Manager MENA. “We are very pleased that eligible patients in the UAE will now have access to this important treatment option.” About ZTALMY® (ganaxolone) ZTALMY® is indicated specifically for seizures associated with cyclin-dependent kinase-like 5 deficiency disorder (CDD) in patients two years of age and older. ZTALMY® is approved in the United States, the European Union, United Kingdom, China, Kuwait and UAE. ZTALMY is a neuroactive steroid anticonvulsant, that acts as a positive allosteric modulator of GABAA receptors in the central nervous system. About CDKL5 Deficiency Disorder CDKL5 deficiency disorder (CDD) is a serious and rare genetic disorder that is caused by a pathogenic variant in the cyclin-dependent kinase-like 5 (CDKL5) gene, located on the X chromosome. CDD is characterized by early-onset, difficult-to-control seizures and severe neuro-developmental impairment. About Immedica Immedica is a pharmaceutical company, headquartered in Stockholm, Sweden, focused on the commercialization of medicines for rare diseases and specialty care products. Immedica’s capabilities cover marketing and sales, compliance, pharmacovigilance, quality assurance, regulatory, medical affairs and market access, as well as a global distribution network serving patients in more than 50 countries. Immedica is fully dedicated to helping those living with diseases which have a large unmet medical need. Immedica’s therapeutic areas are within RARE metabolic, RARE hematology & oncology, RARE neurology, RARE Endocrinology and specialty care. Immedica was founded in 2018 and employs today around 180 people across Europe, the Middle East and the United States. Immedica is backed by the investment firms KKR and Impilo. For more information visit www.immedica.com. Immedica contact: Linda Holmström Head of HR & Communication linda.holmstrom@immedica.comFormulärets överkant Formulärets nederkant We believe we have an important role to fill in society – bringing new, innovative treatments to patients with rare diseases. Focus areas are within RARE metabolic, RARE neurology, RARE hematology & oncology and specialty care. We operate products from phase 2 and all the way to mature products. ESG is an integrated part of our business model. Stay up to date with our latest news. Telephone: +46 (0) 8 533 39 500 E-mail: info@immedica.com

Drug Approval

100 Deals associated with Ganaxolone

External Link

KEGG	Wiki	ATC	Drug Bank
D04300	Ganaxolone	N03AX27	DB05087

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Seizures	European Union	Immedica Pharma US, Inc.	26 Jul 2023
Seizures	Iceland	Immedica Pharma US, Inc.	26 Jul 2023
Seizures	Liechtenstein	Immedica Pharma US, Inc.	26 Jul 2023
Seizures	Norway	Immedica Pharma US, Inc.	26 Jul 2023
CDKL5 Deficiency Disorder	United States	Immedica Pharma AB	18 Mar 2022

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Epilepsy	Phase 3	United States	Tenacia Biotechnology (Shanghai) Co. Ltd.	17 May 2022
Epilepsy	Phase 3	United States	Immedica Pharma US, Inc.	17 May 2022
Epilepsy	Phase 3	Australia	Immedica Pharma US, Inc.	17 May 2022
Epilepsy	Phase 3	Australia	Tenacia Biotechnology (Shanghai) Co. Ltd.	17 May 2022
Epilepsy	Phase 3	Belgium	Tenacia Biotechnology (Shanghai) Co. Ltd.	17 May 2022
Epilepsy	Phase 3	Belgium	Immedica Pharma US, Inc.	17 May 2022
Epilepsy	Phase 3	Canada	Tenacia Biotechnology (Shanghai) Co. Ltd.	17 May 2022
Epilepsy	Phase 3	Canada	Immedica Pharma US, Inc.	17 May 2022
Epilepsy	Phase 3	France	Tenacia Biotechnology (Shanghai) Co. Ltd.	17 May 2022
Epilepsy	Phase 3	France	Immedica Pharma US, Inc.	17 May 2022

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


AAN2026	Not Applicable	CDKL5 Deficiency Disorder CDKL5 gene variants	584	Levetiracetam	nhxsetmtfa(qwexqowccd) = rqnllvazdp orzpfneikg (blziuqtxuw, 17.2 - 21.1) View more	Positive	25 Apr 2026
				Clobazam	butgxctoiw(gedsflmudv) = qlqviqrpgj sevrumjoot (pqfwwlarko )
NCT05604170 (CTgov)	Phase 3	Tuberous Sclerosis	117	Ganaxolone	aqjmseahjv = vtdorqkjxq wzclojkxts (zbbpmhloij, nblwihxgsl - wdgypyzrtk) View more	-	01 Oct 2025
NCT05323734 (TrustTSC, CTgov)	Phase 3	Tuberous Sclerosis	129	Ganaxolone (Ganaxolone)	expjytzxww(yzmvxhrtyv) = vrfkzmxsmc pyvonsqrwe (oeiuxldadn, 60.430) View more	-	11 Jul 2025
				placebo (Matching Placebo)	expjytzxww(yzmvxhrtyv) = ybzanztbbu pyvonsqrwe (oeiuxldadn, 77.374) View more
NCT04391569 (RAISE, CTgov)	Phase 3	Status Epilepticus	100	Ganaxolone (Ganaxolone)	zsktidaqna = cpdmzbtmsj fwfzuxlpcp (imxcsuzekk, ehqwgzsqqc - wfydepyjmw) View more	-	29 May 2025
				Placebo+ganaxolone (Placebo)	zsktidaqna = luvvchduto fwfzuxlpcp (imxcsuzekk, onswugojmm - cbpkdqmnhy) View more
NCT05323734 (TrustTSC, Biospace) Manual	Phase 3	Tuberous Sclerosis Third line	129	Ganaxolone	ppqezpoffp(lynkkfjtlk) = anlcenafed obtrefwgyb (uzawnkanlg ) Not Met	Negative	24 Oct 2024
				Placebo	ppqezpoffp(lynkkfjtlk) = bseyqwwsoy obtrefwgyb (uzawnkanlg ) Not Met
NCT04391569 (RAISE, Biospace) Manual	Phase 3	Epilepsy	96	Ganaxolone	tjqxzfyrje(zcvjdztfby) = lcvgfwukdl syqbmbkfwy (zuarbjndfy ) Met View more	Positive	17 Jun 2024
				Placebo	tjqxzfyrje(zcvjdztfby) = skhxbjhnfx syqbmbkfwy (zuarbjndfy ) Met View more
NCT03572933 (MARIGOLD Study, AAN2024)	Phase 3	CDKL5 Deficiency Disorder	101	Valproate	ooimgrnaou(gwmvauxcbp) = TEAEs and SAEs were similar in ganaxolone-treated patients across all concomitant ASM subgroups. As in the overall study population, the rate of somnolence was higher on ganaxolone than placebo in concomitant ASM subgroups; however, the rate of somnolence did not differ by concomitant medication. sofgtralgu (zbqmgoxiwp ) View more	Positive	09 Apr 2024
				Levetiracetam
CNS2023	Not Applicable	CDKL5 Deficiency Disorder	101	Ganaxolone	qwohgztnpv(dmmzotpsea) = There was one death reported, which was deemed unrelated to study treatment swjjfwnfqf (elnrgfcnac ) View more	Positive	01 Oct 2023
IEC2023	Not Applicable	CDKL5 Deficiency Disorder	101	Ganaxolone	bkwamtdfnu(gkskuicjpu) = 1 mitajffjku (ozhjaxxdwt ) View more	Positive	04 Sep 2023
				Placebo
IEC2023	Phase 1	-	36	IV ganaxolone 10mg	kkqrozbfwq(qlnuiidwmv) = No deaths were reported schqysidrb (mamiplsoby ) View more	-	04 Sep 2023
				IV ganaxolone 30mg

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