A Phase I Dose-Escalation Clinical Study Evaluating Stiripentol Combined with Immunochemotherapy for the Treatment of HER2-Negative Unresectable Advanced Gastric Cancer

Start Date01 Jan 2026

Sponsor / Collaborator

The Seventh Affiliated Hospital of Sun Yat-sen University

CTR20251833

/ RecruitingNot Applicable

司替戊醇干混悬剂治疗Dravet综合征的真实世界临床研究

[Translation] A real-world clinical study of stiripentol dry suspension in the treatment of Dravet syndrome

评价在真实世界中使用司替戊醇干混悬剂治疗Dravet 综合征的安全性和有效性

[Translation]

To evaluate the safety and efficacy of stiripentol dry suspension in the treatment of Dravet syndrome in a real-world setting

Start Date26 Aug 2025

Sponsor / Collaborator

Shijiazhuang No. 4 Pharmaceutical Co., Ltd.

NCT07176832

/ CompletedPhase 1

Bioavailability Study of Stiripentol After Single Oral Administration of Two Different Formulations (Capsule and Oral Suspension) in 24 Healthy Subjects

This is a Phase I, open-label, randomized, single-center, two-way cross-over study evaluating the relative bioavailability, pharmacokinetics, safety, and palatability of two formulations of stiripentol (Diacomit®), indicated in Dravet syndrome. The investigational products are 500 mg capsules (reference) and a 50 mg/mL oral suspension (test).
The primary objective is to compare the relative bioavailability of the two formulations after a single 1,000 mg oral dose under fed conditions, based on Cmax, AUC0-t, and AUC0-∞. Secondary objectives include other PK parameters (tmax, tlag, ke, t1/2) and characterization of metabolites MIa and MIb. Palatability of the suspension will be assessed by questionnaire.
Safety evaluation will include adverse events, laboratory tests, ECGs, urinalysis, drug and alcohol screening, serology, and vital signs.
Twenty-four healthy volunteers (18-50 years) will be enrolled. Eligibility: BMI 18-30 kg/m², weight ≥50 kg, normal ECG and labs, and informed consent. Women of childbearing potential must use effective contraception and test negative for pregnancy. Exclusions: significant disease, recent surgery or blood donation, hypersensitivity, difficulty swallowing, use of CYP modulators (e.g., carbamazepine, grapefruit, herbal products), drug or alcohol abuse, smoking >5 cigarettes/day, or inability to follow dietary restrictions. Subjects testing positive for HIV, HBV, HCV, or drugs of abuse will also be excluded.
Each participant will attend a screening visit within 28 days before dosing, then two 3-day hospitalizations separated by a 7-15-day washout. On Day 1 of each period, they will receive either two capsules (1,000 mg) or 20 mL suspension (1,000 mg). Blood will be collected at 36 timepoints (≈180 mL total) for PK assessment.
The total study duration per subject is about seven weeks, including screening, hospitalization, dosing, washout, and follow-up. Treatment consists of one dosing day per period.
Sample size was based on prior data: 21 pairs provide 80% power for bioequivalence within 0.80-1.25 bounds; 24 subjects will be recruited to account for dropouts. Analyses will include the Safety Set, PK Concentrations Set, and PK Analysis Set.
This trial aims to establish whether the oral suspension provides a PK profile comparable to capsules, while generating safety, tolerability, and palatability data to support a more convenient formulation for Dravet syndrome patients.

Start Date01 Jun 2025

Sponsor / Collaborator

Biocodex SAS

100 Clinical Results associated with Stiripentol

100 Translational Medicine associated with Stiripentol

100 Patents (Medical) associated with Stiripentol

453

Literatures (Medical) associated with Stiripentol

01 Aug 2026·Advanced Science

Epigenetic Silencing of RFX7 Defines a Transcriptional Axis Linking Lactate Metabolism to Immune Checkpoint Therapy in Glioblastoma

Article

Author: Zhang, Yue ; Chen, Fan ; Zhang, Ziwen ; Cui, Wenxing ; Wang, Liang ; Ma, Shuoyao ; Wang, Qiang ; Feng, Tian ; Wang, Liangbo ; Zhu, Gang ; Sun, Fang ; Wang, Jiahui ; Han, Liying ; Wang, Kai ; Liu, Haixiao ; Yuan, Chenyi ; Guo, Chengxuan ; Qu, Yan ; Zhou, Jinpeng ; Li, Leiyang ; Li, Wencong

ABSTRACT:

Glioblastoma (GBM) is a highly aggressive brain tumor characterized by rapid proliferation, diffuse invasion, and robust immunosuppression. Although excessive aerobic glycolysis and lactate accumulation are known to contribute to an immunosuppressive microenvironment, the upstream transcriptional mechanisms connecting metabolic reprogramming and immunotherapy resistance in GBM remain unclear.By integrating transcriptomic profiling, chromatin immunoprecipitation sequencing, and metabolic analysis with gene perturbation experiments, we identified a regulatory axis comprising RFX7 and its downstream target PIK3IP1. In GBM tissues, RFX7 expression was reduced due to promoter hypermethylation. Restoration of RFX7 enhanced PIK3IP1 expression, suppressed PI3K/AKT activation, and inhibited malignant progression in GBM. Loss of PIK3IP1 increased lactate production and histone H4K12 lactylation (H4K12la), coinciding with upregulation of PD‐L1 and CSF1 and enhanced tumor immunosuppressive features. Pharmacological inhibition of lactate production with Stiripentol reduced H4K12la level, intracranial tumor growth, and immunosuppressive cell infiltration, while improving survival and response to immune checkpoint based therapy in experimental models.These findings identify an upstream transcriptional pathway linking lactate metabolism, histone lactylation, and immune suppression in GBM. Targeting the RFX7‐PIK3IP1 axis provides a mechanistic rationale for metabolic‐immune modulation in therapy, addressing an aspect that has remained insufficiently understood in GBM immune resistance.

01 Jun 2026·ANNALS OF PHARMACOTHERAPY

Relieving the Weight: Fenfluramine’s Re-emergence as Antiseizure Medication for Lennox-Gastaut and Dravet Syndrome

Review

Author: McCay, Simone A. ; Shiue, Harn J. ; Hoerth, Matthew T.

Objective::

To review the pharmacology, usage, efficacy, and safety of fenfluramine in treating seizures in pediatric patients with Lennox-Gastaut syndrome or Dravet syndrome.

Data sources::

The PubMed database was searched for studies using the search terms “Lennox-Gastaut Syndrome,” “Dravet Syndrome,” “fenfluramine,” and “Fintepla©.”

Study selection and data extraction::

Articles were selected based on the following criteria: published in English, published between January 1950 and September 2025, and included clinical relevancy for pharmacology, efficacy, and adverse events.

Data synthesis::

In a phase 3 trial, patients with Lennox-Gastaut syndrome experienced a median 26.5% decrease in frequency of drop seizures when taking the 0.7 mg/kg/d dose. In a 2-part phase 2 trial, 54% of patients with Dravet syndrome concurrently taking stiripentol experienced a >50% decrease in monthly seizures at a dosage of 0.4 mg/kg/d; 72.9% of patients with Dravet syndrome not concurrently taking stiripentol experienced a >50% decrease in monthly seizures at a dosage of 0.7 mg/kg/d. Most common adverse effects in both trials included lethargy, decreased weight, and decreased appetite.

Relevance to patient care and clinical practice::

Fenfluramine’s dual mechanism of action makes it uniquely suited to reduce seizure activity after previous failures in treatment. Some providers are hesitant to prescribe fenfluramine due to its historical association with severe cardiovascular risks. However, proper dosage and titration, as well as adherence to the Risk Evaluation and Mitigation Strategy program can sufficiently mitigate risk for the outcome of seizure reduction and increased quality of life.

Conclusions::

As seen through the results of multiple clinical trials, fenfluramine can provide significant seizure reduction for Lennox-Gastaut and Dravet patients with treatment-resistant seizures. Risks can be mitigated through adherence to proper programs and schedules, and can provide enough benefits to outweigh risks in patients with particularly resistant epilepsies.

01 Jun 2026·Epilepsia Open

Practical consensus recommendations for polytherapy involving stiripentol in Dravet syndrome: A nominal group approach

Article

Author: Joseph Sullivan ; J. Helen Cross ; Barry Gidal ; Elaine Wirrell ; Rima Nabbout

Abstract:

Objectives:

Dravet syndrome (DS) is a drug‐resistant developmental and epileptic encephalopathy, often requiring three or more antiseizure medications (ASMs). Consequently, the therapeutic landscape is highly complex, combining DS‐specific agents (stiripentol, STP; cannabidiol, CBD; fenfluramine, FFA), non‐specific ASMs, and non‐pharmacologic options such as dietary therapy or neuromodulation. In addition, recognition of patients' changing needs across the lifespan, together with the essential contribution of caregiver education to effective treatment oversight—including awareness of potential drug–drug interactions and adverse events—emphasizes the value of an integrated and forward‐looking approach. A consensus initiative was developed to guide clinicians in the management of patients with DS, with a particular focus on STP‐containing regimens.

Methods:

Based on a literature review and clinical vignette discussions, a nominal group of five international DS experts developed 38 statements across six themes: (i) general principles, (ii) guiding physicians to empower families, (iii) STP as add‐on to specific and non‐specific Dravet ASMs, (iv) FFA in regimens containing STP, (v) CBD in regimens containing STP, and (vi) patient follow‐up during treatment initiation. Statements were scored on a 9‐point Likert scale (1 = strongly disagree, 9 = strongly agree) first by the nominal group and then by an international panel of 59 DS clinicians. Consensus was defined as ≥75% rating ≥7 and/or a median ≥8.

Results:

Thirty‐four of the 38 statements (89%) reached consensus among both the nominal group and the international panel; one did not reach consensus in either, and three reached consensus only among the nominal group. Both panels agreed on the importance of involving families in treatment decisions and follow‐up, as well as on key aspects of polytherapy management, including ASM initiation, treatment adjustments, and adverse event management.

Significance:

This international consensus integrates evidence and clinical expertise to establish shared principles that address a long‐standing gap in the polytherapeutic management of Dravet syndrome.

Plain Language Summary:

Dravet syndrome is a rare and severe form of epilepsy that begins in early childhood, with seizures that often persist throughout life. Because the disease is highly drug resistant, most patients are treated with polytherapy, making clinical management complex for clinicians. This consensus brings together published evidence and expert experience to provide practical guidance on the management of polytherapy in Dravet syndrome, supporting clinicians in optimizing patient care.

News (Medical) associated with Stiripentol

04 Sep 2026

·www.pharmaceutical-technology.com

Médunik and Biocodex sign exclusive agreement for Diacomit in Canada

Dravet syndrome is described as a rare lifelong neurological disorder marked by frequent, often treatment-resistant seizures from infancy. Credit: one photo / Shutterstock.com. Médunik Canada has entered an exclusive promotional services agreement with Biocodex for Diacomit, a medication approved in Canada as an add-on therapy for seizures associated with Dravet syndrome. The agreement gives Médunik Canada immediate responsibility for Diacomit’s sales, marketing, and medical affairs activities throughout the country. It aims to leverage Médunik Canada’s network and rare disease expertise to strengthen engagement with Canadian paediatric neurologists, epileptologists, and comprehensive epilepsy centres. When used together with clobazam and valproate, Diacomit is indicated by Health Canada for managing seizures associated with Dravet syndrome, a severe and rare developmental and epileptic encephalopathy. As part of the agreement, Médunik Canada will handle all commercial and medical engagement for the therapy while working to maintain consistency and reliability across the product’s supply chain and patient access. The goal is to expand educational programmes and support for Canada’s epilepsy community while ensuring ongoing access to the medication for eligible patients. Biocodex rare diseases international business head Hubert Chapellier said: “This alliance between our two companies marks a real turning point in our approach to Dravet syndrome in the Canadian market. “We are confident that our efforts will help patients and families better understand this complex condition. I am extremely excited about the partnership between Biocodex and Médunik Canada.” Dravet syndrome is described as a rare and lifelong neurological disorder, marked by frequent and often treatment-resistant seizures beginning in infancy. The condition can have a considerable impact on patients and their caregivers and typically needs the attention of specialised epilepsy care teams. Médunik Canada, part of Duchesnay Pharmaceutical Group (DPG), focuses on providing access to orphan drugs in Canada. DPG is headquartered in Blainville, Quebec, and operates several companies in both Canada and the US, offering a range of speciality and generic medicines.

License out/inDrug Approval

03 Sep 2026

·www.prnewswire.com

Médunik Canada enters into exclusive promotional services agreement with Biocodex for Diacomit® in Canada

Agreement expands support for Canadian healthcare professionals and families impacted by Dravet syndrome BLAINVILLE, QC, Sept. 3, 2026 /PRNewswire/ -- Médunik Canada, a Canadian owned and operated pharmaceutical company dedicated to improving the health and quality of life of people living with rare and debilitating diseases, part of Duchesnay Pharmaceutical Group, is pleased to announce that it has entered into an exclusive promotional services agreement with Biocodex for Diacomit®. Diacomit® is approved by Health Canada in conjunction with clobazam and valproate as adjunctive therapy for the treatment of seizures associated with Dravet syndrome, a rare and severe form of developmental and epileptic encephalopathy. Continue Reading Promotional Services Agreement between Médunik Canada and Biocodex Under the terms of the agreement, Médunik Canada assumes immediate and exclusive responsibility for all Canadian sales, marketing, and medical affairs activities related to Diacomit®. The partnership will leverage Médunik Canada's specialized rare disease infrastructure and expertise to strengthen engagement with Canadian pediatric neurologists, epileptologists, and specialized comprehensive epilepsy centers across the country. "Dravet syndrome requires highly specialized care, and we are excited to deploy our field teams to support Dravet specialists across the country. Our focus is to maximize the therapeutic reach of this medicine while ensuring that clinical support and medical education are closer to the specialists who need them," said Michael Gallo, Vice President, Regulatory & Medical Affairs, DPG. The agreement is designed to enhance commercial and medical engagement while preserving continuity and reliability across the product's supply chain and access infrastructure. "This alliance between our two companies marks a real turning point in our approach to Dravet syndrome in the Canadian market. We are confident that our efforts will help patients and families better understand this complex condition. I am extremely excited about the partnership between Biocodex and Médunik Canada," said Hubert Chapellier, Head of International Business, Biocodex Rare Diseases. Dravet syndrome is a rare, lifelong neurological disorder characterized by frequent and often treatment-resistant seizures that typically begin in infancy. The condition can significantly impact quality of life for patients and caregivers and generally requires management by specialized epilepsy care teams. Through this agreement, Médunik Canada and Biocodex aim to strengthen educational outreach and support for the Canadian epilepsy community while ensuring uninterrupted access to Diacomit® for eligible patients across Canada. ABOUT MÉDUNIK CANADA Médunik Canada's vision is to improve the health and quality of life of Canadians living with a rare and debilitating disease. Founded in December 2009, and part of Duchesnay Pharmaceutical Group, the company provides access to orphan drugs through strategic global partnerships as well as domestic and international research and development projects. For more information about Médunik Canada, please visit . Follow us on LinkedIn. ABOUT DUCHESNAY PHARMACEUTICAL GROUP Duchesnay Pharmaceutical Group (DPG), with its affiliated companies, is headquartered in Blainville, Quebec. The Group consists of six pharmaceutical companies to meet the needs of patients in Canada, the U.S. and abroad. The companies are Duchesnay (Canada) and Duchesnay USA, both dedicated to women's health; Médunik Canada and Medunik USA, which provide treatments for rare and debilitating diseases; and Analog Pharma Canada and Analog Pharma, specializing in orphan generic medications. From its state-of-the-art manufacturing plant, DPG exports its innovative treatments to more than 50 countries. DPG is one of the eight companies across the country chosen to participate in the Government of Canada's Global Hypergrowth Project. This appointment offers exclusive and personalized support for at least two years, in order to accelerate its growth to become an anchor firm in the Canadian economy. DPG is the winner of the 2024 Life Sciences Innovation Award by ADRIQ, the Association for the Development of Research and Innovation of Quebec, which recognizes DPG's healthy workplace culture and commitment to pharmaceutical innovation, while DPG president Éric Gervais is the recipient of the 2024 Bernard-Landry Award by ADRIQ which acknowledges his impactful leadership on Quebec's research and innovation ecosystem. DPG, through its proprietary research and development, and through exclusive partnerships, offers innovative treatments for a variety of medical conditions in women's health, urology, oncology and for rare diseases, plus lower-cost generic medications. DPG recognizes the dedication and professionalism of its employees and promotes a positive culture and flexible work environment. It is deeply committed to environmental responsibility and to giving back to the community through the support of various charitable organizations. For more information, please visit . Follow us on LinkedIn. SOURCE Médunik Canada 21% more press release views with Request a Demo

03 Sep 2026

·www.biospace.com

Médunik Canada enters into exclusive promotional services agreement with Biocodex for Diacomit® in Canada

Agreement expands support for Canadian healthcare professionals and families impacted by Dravet syndrome BLAINVILLE, QC , Sept. 3, 2026 /CNW/ -- Médunik Canada, a Canadian owned and operated pharmaceutical company dedicated to improving the health and quality of life of people living with rare and debilitating diseases, part of Duchesnay Pharmaceutical Group, is pleased to announce that it has entered into an exclusive promotional services agreement with Biocodex for Diacomit®. Diacomit® is approved by Health Canada in conjunction with clobazam and valproate as adjunctive therapy for the treatment of seizures associated with Dravet syndrome, a rare and severe form of developmental and epileptic encephalopathy. Under the terms of the agreement, Médunik Canada assumes immediate and exclusive responsibility for all Canadian sales, marketing, and medical affairs activities related to Diacomit®. The partnership will leverage Médunik Canada's specialized rare disease infrastructure and expertise to strengthen engagement with Canadian pediatric neurologists, epileptologists, and specialized comprehensive epilepsy centers across the country. "Dravet syndrome requires highly specialized care, and we are excited to deploy our field teams to support Dravet specialists across the country. Our focus is to maximize the therapeutic reach of this medicine while ensuring that clinical support and medical education are closer to the specialists who need them," said Michael Gallo, Vice President, Regulatory & Medical Affairs, DPG. ezstandalone.cmd.push(function () { ezstandalone.showAds(131); }); The agreement is designed to enhance commercial and medical engagement while preserving continuity and reliability across the product's supply chain and access infrastructure. "This alliance between our two companies marks a real turning point in our approach to Dravet syndrome in the Canadian market. We are confident that our efforts will help patients and families better understand this complex condition. I am extremely excited about the partnership between Biocodex and Médunik Canada," said Hubert Chapellier, Head of International Business, Biocodex Rare Diseases. Dravet syndrome is a rare, lifelong neurological disorder characterized by frequent and often treatment-resistant seizures that typically begin in infancy. The condition can significantly impact quality of life for patients and caregivers and generally requires management by specialized epilepsy care teams. Through this agreement, Médunik Canada and Biocodex aim to strengthen educational outreach and support for the Canadian epilepsy community while ensuring uninterrupted access to Diacomit® for eligible patients across Canada. ezstandalone.cmd.push(function () { ezstandalone.showAds(132); }); ABOUT MÉDUNIK CANADA Médunik Canada's vision is to improve the health and quality of life of Canadians living with a rare and debilitating disease. Founded in December 2009, and part of Duchesnay Pharmaceutical Group, the company provides access to orphan drugs through strategic global partnerships as well as domestic and international research and development projects. For more information about Médunik Canada, please visit . Follow us on LinkedIn . ezstandalone.cmd.push(function () { ezstandalone.showAds(133); }); ABOUT DUCHESNAY PHARMACEUTICAL GROUP Duchesnay Pharmaceutical Group (DPG), with its affiliated companies, is headquartered in Blainville, Quebec. The Group consists of six pharmaceutical companies to meet the needs of patients in Canada, the U.S. and abroad. The companies are Duchesnay (Canada) and Duchesnay USA, both dedicated to women's health; Médunik Canada and Medunik USA, which provide treatments for rare and debilitating diseases; and Analog Pharma Canada and Analog Pharma, specializing in orphan generic medications. From its state-of-the-art manufacturing plant, DPG exports its innovative treatments to more than 50 countries. DPG is one of the eight companies across the country chosen to participate in the Government of Canada's Global Hypergrowth Project. This appointment offers exclusive and personalized support for at least two years, in order to accelerate its growth to become an anchor firm in the Canadian economy. DPG is the winner of the 2024 Life Sciences Innovation Award by ADRIQ, the Association for the Development of Research and Innovation of Quebec, which recognizes DPG's healthy workplace culture and commitment to pharmaceutical innovation, while DPG president Éric Gervais is the recipient of the 2024 Bernard-Landry Award by ADRIQ which acknowledges his impactful leadership on Quebec's research and innovation ecosystem. ezstandalone.cmd.push(function () { ezstandalone.showAds(134); }); DPG, through its proprietary research and development, and through exclusive partnerships, offers innovative treatments for a variety of medical conditions in women's health, urology, oncology and for rare diseases, plus lower-cost generic medications. DPG recognizes the dedication and professionalism of its employees and promotes a positive culture and flexible work environment. It is deeply committed to environmental responsibility and to giving back to the community through the support of various charitable organizations. For more information, please visit . Follow us on LinkedIn . View original content to download multimedia: ezstandalone.cmd.push(function () { ezstandalone.showAds(135); }); SOURCE Médunik Canada

License out/inOrphan Drug

100 Deals associated with Stiripentol

External Link

KEGG	Wiki	ATC	Drug Bank
D05928	Stiripentol	N03AX17	DB09118

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
CDKL5 Deficiency Disorder	United States	Biocodex SAS	20 Aug 2018
Seizures	United States	Biocodex SAS	20 Aug 2018
Epilepsies, Myoclonic	European Union	Biocodex SAS	03 Jan 2007
Epilepsies, Myoclonic	Iceland	Biocodex SAS	03 Jan 2007
Epilepsies, Myoclonic	Liechtenstein	Biocodex SAS	03 Jan 2007
Epilepsies, Myoclonic	Norway	Biocodex SAS	03 Jan 2007
Epilepsy, Tonic-Clonic	European Union	Biocodex SAS	03 Jan 2007
Epilepsy, Tonic-Clonic	Iceland	Biocodex SAS	03 Jan 2007
Epilepsy, Tonic-Clonic	Liechtenstein	Biocodex SAS	03 Jan 2007
Epilepsy, Tonic-Clonic	Norway	Biocodex SAS	03 Jan 2007

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Primary Hyperoxaluria Type 1	Phase 3	-	Biocodex SAS	01 Aug 2024
Primary Hyperoxaluria Type 2	Phase 3	-	Biocodex SAS	01 Aug 2024
Primary hyperoxaluria type III	Phase 3	-	Biocodex SAS	01 Aug 2024
Hyperoxaluria, Primary	Phase 2	France	Biocodex SAS	21 May 2019
Glioblastoma Multiforme	Preclinical	United States	St. John's University	29 Apr 2025

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT06540378 (Pubmed \| Neurol Res Pract)	Not Applicable	Status Epilepticus	25	Stiripentol	pwlowguven(mssswalrlt) = sloziamita osdmcmtxgq (vaffmzedia ) View more	Positive	21 Oct 2024

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