CLINICAL TRIALS PROFILE FOR STIRIPENTOL

All Clinical Trials for stiripentol

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT02205931 ↗	Ketogenic Diet in Infants With Epilepsy (KIWE)	Unknown status	Alder Hey Children's NHS Foundation Trust	Phase 4	2015-01-01	Epilepsy, a condition where individuals are prone to recurrent epileptic seizures, is the most common chronic neurological disorder in children. Epilepsy onset is most common in the first two years of life and is associated with poor prognosis for seizure control and neurodevelopmental outcome. The ketogenic diet (KD) is a medically supervised diet that is high in fat and restricted in carbohydrates and protein. KD therapy has shown to be an effective treatment for seizures in children with epilepsy older than two. Associated benefits include: a reduced requirement for routine and emergency antiepileptic drugs (AED) and fewer seizure related hospital admissions. Although reports suggest that KD therapy improves seizures in younger children there is no high quality trial data that demonstrates effectiveness and safety in this age group. The KD is resource intensive, requiring dietetic and physician time; data is required to justify expansion of services to cater for the apparent need. The investigators therefore propose a prospective multicentre randomised trial to investigate the effectiveness and safety of the KD in children with epilepsy under the age of 2, who have failed to respond to two or more AEDs. Children will be randomly assigned to either receive the KD or further AEDs. The allocated treatment will be started after a 2week baseline period, and it's effectiveness assessed after 8 weeks. Seizure diaries will be used to record seizures and related events, a questionnaire will be used to assess diet tolerance; also growth and blood biochemistry will be monitored. The information obtained from this study is necessary to optimise choices in epilepsy treatment, aiming to improve outcomes and thus determine whether and when the KD should should be used.
NCT02205931 ↗	Ketogenic Diet in Infants With Epilepsy (KIWE)	Unknown status	Birmingham Children's Hospital NHS Foundation Trust	Phase 4	2015-01-01	Epilepsy, a condition where individuals are prone to recurrent epileptic seizures, is the most common chronic neurological disorder in children. Epilepsy onset is most common in the first two years of life and is associated with poor prognosis for seizure control and neurodevelopmental outcome. The ketogenic diet (KD) is a medically supervised diet that is high in fat and restricted in carbohydrates and protein. KD therapy has shown to be an effective treatment for seizures in children with epilepsy older than two. Associated benefits include: a reduced requirement for routine and emergency antiepileptic drugs (AED) and fewer seizure related hospital admissions. Although reports suggest that KD therapy improves seizures in younger children there is no high quality trial data that demonstrates effectiveness and safety in this age group. The KD is resource intensive, requiring dietetic and physician time; data is required to justify expansion of services to cater for the apparent need. The investigators therefore propose a prospective multicentre randomised trial to investigate the effectiveness and safety of the KD in children with epilepsy under the age of 2, who have failed to respond to two or more AEDs. Children will be randomly assigned to either receive the KD or further AEDs. The allocated treatment will be started after a 2week baseline period, and it's effectiveness assessed after 8 weeks. Seizure diaries will be used to record seizures and related events, a questionnaire will be used to assess diet tolerance; also growth and blood biochemistry will be monitored. The information obtained from this study is necessary to optimise choices in epilepsy treatment, aiming to improve outcomes and thus determine whether and when the KD should should be used.
NCT02205931 ↗	Ketogenic Diet in Infants With Epilepsy (KIWE)	Unknown status	Birmingham Women's and Children's NHS Foundation Trust	Phase 4	2015-01-01	Epilepsy, a condition where individuals are prone to recurrent epileptic seizures, is the most common chronic neurological disorder in children. Epilepsy onset is most common in the first two years of life and is associated with poor prognosis for seizure control and neurodevelopmental outcome. The ketogenic diet (KD) is a medically supervised diet that is high in fat and restricted in carbohydrates and protein. KD therapy has shown to be an effective treatment for seizures in children with epilepsy older than two. Associated benefits include: a reduced requirement for routine and emergency antiepileptic drugs (AED) and fewer seizure related hospital admissions. Although reports suggest that KD therapy improves seizures in younger children there is no high quality trial data that demonstrates effectiveness and safety in this age group. The KD is resource intensive, requiring dietetic and physician time; data is required to justify expansion of services to cater for the apparent need. The investigators therefore propose a prospective multicentre randomised trial to investigate the effectiveness and safety of the KD in children with epilepsy under the age of 2, who have failed to respond to two or more AEDs. Children will be randomly assigned to either receive the KD or further AEDs. The allocated treatment will be started after a 2week baseline period, and it's effectiveness assessed after 8 weeks. Seizure diaries will be used to record seizures and related events, a questionnaire will be used to assess diet tolerance; also growth and blood biochemistry will be monitored. The information obtained from this study is necessary to optimise choices in epilepsy treatment, aiming to improve outcomes and thus determine whether and when the KD should should be used.
NCT02205931 ↗	Ketogenic Diet in Infants With Epilepsy (KIWE)	Unknown status	Bristol Royal Hospital for Children	Phase 4	2015-01-01	Epilepsy, a condition where individuals are prone to recurrent epileptic seizures, is the most common chronic neurological disorder in children. Epilepsy onset is most common in the first two years of life and is associated with poor prognosis for seizure control and neurodevelopmental outcome. The ketogenic diet (KD) is a medically supervised diet that is high in fat and restricted in carbohydrates and protein. KD therapy has shown to be an effective treatment for seizures in children with epilepsy older than two. Associated benefits include: a reduced requirement for routine and emergency antiepileptic drugs (AED) and fewer seizure related hospital admissions. Although reports suggest that KD therapy improves seizures in younger children there is no high quality trial data that demonstrates effectiveness and safety in this age group. The KD is resource intensive, requiring dietetic and physician time; data is required to justify expansion of services to cater for the apparent need. The investigators therefore propose a prospective multicentre randomised trial to investigate the effectiveness and safety of the KD in children with epilepsy under the age of 2, who have failed to respond to two or more AEDs. Children will be randomly assigned to either receive the KD or further AEDs. The allocated treatment will be started after a 2week baseline period, and it's effectiveness assessed after 8 weeks. Seizure diaries will be used to record seizures and related events, a questionnaire will be used to assess diet tolerance; also growth and blood biochemistry will be monitored. The information obtained from this study is necessary to optimise choices in epilepsy treatment, aiming to improve outcomes and thus determine whether and when the KD should should be used.
NCT01533506 ↗	Stiripentol in Dravet Syndrome	No longer available	Mayo Clinic		2012-02-01	The patient has failed all other available agents and has intractable epilepsy due to Dravet Syndrome. Stiripentol is highly efficacious in Dravet Syndrome. The overall goals of therapy with Stiripentol are primarily to significantly reduce the frequency and severity of seizures.
NCT01835314 ↗	Compassionate Use of Stiripentol in Dravet Syndrome	No longer available	University of Colorado, Denver		1969-12-31	Compassionate use of Stiripentol in Dravet Syndrome. This is a treatment protocol, not a research study, therefore children will only be monitored on a clinical basis for seizure improvement predominantly by parent and caregiver report.
NCT01983722 ↗	Treatment Plan to Provide Expanded Access to Stiripentol for Patients With Dravet Syndrome	Approved for marketing	Children's Hospital Medical Center, Cincinnati		1969-12-31	Expanded access to Stiripentol for patients with Dravet Syndrome.

Clinical Trial Conditions for stiripentol

Condition Name

Condition Name for stiripentol
Intervention	Trials
Dravet Syndrome	6
Epilepsy	3
Chronic Renal Insufficiency	1
Dravet Syndrome, Intractable	1

Condition MeSH

Condition MeSH for stiripentol
Intervention	Trials
Syndrome	6
Epilepsies, Myoclonic	6
Epilepsy	5
Hyperoxaluria, Primary	1

Clinical Trial Locations for stiripentol

Trials by Country

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Trials by Country for stiripentol
Location	Trials
United States	11
France	4
Spain	3
Canada	3
Netherlands	3

Trials by US State

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Trials by US State for stiripentol
Location	Trials
Ohio	2
Colorado	2
Minnesota	2
Pennsylvania	1
Michigan	1

Clinical Trial Progress for stiripentol

Clinical Trial Phase

Clinical Trial Phase for stiripentol
Clinical Trial Phase	Trials
Phase 4	2
Phase 3	2
Phase 2	3

Clinical Trial Status

Clinical Trial Status for stiripentol
Clinical Trial Phase	Trials
Completed	5
No longer available	3
Not yet recruiting	3

Clinical Trial Sponsors for stiripentol

Sponsor Name

Sponsor Name for stiripentol
Sponsor	Trials
Biocodex	4
GW Research Ltd	2
Zogenix, Inc.	1

Sponsor Type

Sponsor Type for stiripentol
Sponsor	Trials
Other	23
Industry	8

Stiripentol: Clinical Trials, Market Analysis, and Projections

Introduction to Stiripentol

Stiripentol, marketed under the brand name Diacomit, is a medication primarily used to treat seizures associated with Dravet syndrome, a severe form of epilepsy. Here, we will delve into the clinical trials, market analysis, and future projections for this drug.

Clinical Trials and Efficacy

Key Clinical Trials

Stiripentol has been evaluated in several pivotal clinical trials to assess its efficacy and safety in patients with Dravet syndrome. Two notable trials, STICLO France (BC.299) and STICLO Italy (BC.385), demonstrated the effectiveness of stiripentol when used in conjunction with other antiepileptic drugs like clobazam (CLB) and valproate (VPA)[3].

• Trial Outcomes: These trials showed that stiripentol significantly reduced the frequency of convulsive seizures in patients with Dravet syndrome. The proposed dosing regimen of 50 mg/kg daily, administered in 2 or 3 divided doses, was found to be effective and safe[3].

Safety and Pharmacokinetics

• Pharmacokinetics: Stiripentol is metabolized through multiple CYP enzymes and glucuronidation. The drug's pharmacokinetics are slightly non-linear, with median half-lives varying depending on the dose. It is not recommended for patients with moderate to severe hepatic or renal impairment[3].

• Adverse Events: Common adverse events include somnolence, decreased appetite, and pyrexia. However, the overall safety profile of stiripentol has been deemed acceptable in clinical trials[3].

Market Analysis

Current Market Scenario

The Dravet syndrome treatment market has seen significant growth in recent years, driven by the approval of new drugs like stiripentol and cannabidiol (Epidiolex).

• Market Value: The Dravet syndrome treatment market was valued at $288.2 million in 2021 and is projected to grow at a CAGR of 11.7% from 2022 to 2030[2][5].

• Regional Dominance: North America currently dominates the global market due to rising awareness of neurological disorders, increasing demand for target-specific drugs, and advanced diagnostic technologies. The Asia Pacific region is expected to grow significantly during the forecast period due to increasing awareness and developing diagnostic technologies[2][5].

Stiripentol's Market Share

Stiripentol, approved by the FDA in August 2020, has seen a significant increase in market share due to its higher efficiency, safety, and prolonged relief from seizures.

• Market Growth: Stiripentol's market share is growing rapidly post-2020, contributing substantially to the overall growth of the Dravet syndrome treatment market. The drug's approval has attracted major pharmaceutical companies to the market[2][5].

Future Projections

Market Growth Drivers

Several factors are driving the growth of the Dravet syndrome treatment market, including:

• Recent Drug Approvals: The approval of stiripentol and cannabidiol has expanded the treatment options available for Dravet syndrome, attracting more patients and pharmaceutical companies to the market[2][5].

• Increasing Awareness: Rising awareness about neurological disorders and the increasing demand for target-specific drugs are key drivers of market growth[2][5].

• Developing Diagnostic Technologies: Advances in diagnostic technologies, particularly in regions like the Asia Pacific, are expected to further boost the market[2][5].

Emerging Trends

• Targeted Therapies: The market is shifting towards targeted therapies that provide symptomatic relief and potentially address the underlying causes of Dravet syndrome. Stiripentol and cannabidiol are at the forefront of this trend[2][5].

• Potential New Indications: Recent studies suggest that stiripentol may have potential in treating other conditions, such as gastric cancer, by inhibiting lactate production and reversing chemotherapy resistance. This could expand the drug's market beyond Dravet syndrome[4].

Key Takeaways

• Clinical Efficacy: Stiripentol has demonstrated significant efficacy in reducing convulsive seizures in patients with Dravet syndrome.
• Market Growth: The drug's market share is growing rapidly due to its safety, efficiency, and prolonged relief from seizures.
• Future Projections: The Dravet syndrome treatment market is expected to grow at a CAGR of 11.7% from 2022 to 2030, driven by recent drug approvals and increasing awareness.
• Emerging Trends: Stiripentol may have potential in new indications, such as gastric cancer treatment, which could further expand its market.

FAQs

What is stiripentol used for?

Stiripentol is primarily used to treat seizures associated with Dravet syndrome, a severe form of epilepsy.

When was stiripentol approved by the FDA?

Stiripentol was approved by the FDA in August 2020 under the brand name Diacomit.

What are the common adverse events associated with stiripentol?

Common adverse events include somnolence, decreased appetite, and pyrexia.

How does stiripentol affect the market for Dravet syndrome treatments?

Stiripentol's approval has significantly contributed to the growth of the Dravet syndrome treatment market due to its higher efficiency, safety, and prolonged relief from seizures.

Are there potential new indications for stiripentol?

Yes, recent studies suggest that stiripentol may have potential in treating gastric cancer by inhibiting lactate production and reversing chemotherapy resistance.

Sources

1. EU Clinical Trials Register: EudraCT Number 2016-000474-38 - Clinical trial results.
2. ResearchAndMarkets: Dravet Syndrome Treatment Market Size, Market Share, Application Analysis.
3. FDA: 206709Orig1s000 207223Orig1s000 - accessdata.fda.gov.
4. ASCO Post: Stiripentol Could Prolong Efficacy of Chemotherapy in Patients With Gastric Cancer.
5. Business Wire: Dravet Syndrome Treatment Market Report 2022-2029: Stiripentol and Cannabidiol to Contribute to Significant Growth of 11.7% - ResearchAndMarkets.com.