Expanded Access Protocol of BHV-0223 for Patients With Amyotrophic Lateral Sclerosis (ALS)

• ClinicalTrials.gov Identifier: NCT03537807
• Expanded Access Status: No longer available
• First Posted: May 25, 2018
• Last Update Posted: July 26, 2019
• Sponsor: Biohaven Pharmaceuticals, Inc.
• Information provided by (Responsible Party): Biohaven Pharmaceuticals, Inc.

Study Description

Brief Summary:

This is an open label expanded access protocol for the treatment of up to approximately 250 adult patients with amyotrophic lateral sclerosis (ALS) who have difficulty swallowing oral riluzole tablets and may be able to derive benefit from treatment with an alternative oral formulation of riluzole.

Condition or disease	Intervention/treatment
Amyotrophic Lateral Sclerosis; ALS; Lou Gehrig Disease; Lou Gehrig's Disease; Lou-Gehrigs Disease; Motor Neuron Disease, Amyotrophic Lateral Sclerosis	Drug: Riluzole

Detailed Description:

Riluzole is indicated in the U.S. for the treatment of patients with amyotrophic lateral sclerosis (ALS). The commercially available dosage form is a 50mg oral tablet. This expanded access protocol (EAP) is designed to provide access to a dissolving tablet formulation of riluzole designed for sublingual (SL) administration, in patients with ALS who, in the opinion and clinical judgement of the treating physician, would benefit from treatment with BHV-0223.

Study Design

• Study Type: Expanded Access
• Expanded Access Type: Treatment IND/Protocol
• Official Title: BHV-0223 Expanded Access Protocol in Patients With Amyotrophic Lateral Sclerosis

Interventions

Intervention Details:

• Drug: Riluzole
  For adult patients, one 40mg of BHV-0223, administered sublingually, on a twice daily basis (approximately every 12 hours).
  Other Name: Riluzole Zydis® sublingual

Eligibility Criteria

• Ages Eligible for Study: 18 Years and older (Adult, Older Adult)
• Sexes Eligible for Study: All

Criteria

Inclusion Criteria:

• Patients with diagnosed ALS of any type or duration
• Current or previous treatment with oral riluzole tablets, or patients who have never taken riluzole oral tablets, or patients who have successfully completed a clinical trial with BHV-0223 and were not withdrawn prematurely due to adverse events
• Swallowing difficulties, or patient or caregiver report choking one or more times per week, or investigator deems appropriate to treat with sublingual BHV-0223 because (s)he deems the patient cannot be satisfactorily treated with Rilutek®
• Adequate hepatic function

Exclusion Criteria:

• Patient with history of severe hypersensitivity reaction to riluzole oral tablets or BHV-0223
• Patient is known to have any other acute or chronic liver disease

Contacts and Locations

No Contacts or Locations Provided

More Information

• Responsible Party: Biohaven Pharmaceuticals, Inc.
• ClinicalTrials.gov Identifier: NCT03537807
• Other Study ID Numbers: BHV0223-401
• First Posted: May 25, 2018
• Last Update Posted: July 26, 2019
• Last Verified: July 2019

Keywords provided by Biohaven Pharmaceuticals, Inc.:

Amyotrophic Lateral Sclerosis; ALS; Lou Gehrig Disease; Lou Gehrig's Disease; Lou-Gehrigs Disease; Motor Neuron Disease, Amyotrophic Lateral Sclerosis; Riluzole; Rilutek®; Sublingual; Oral formulation; Neuroprotective Agents; Administration, Sublingual; Administration, Oral; Biological Availability

Additional relevant MeSH terms:

Motor Neuron Disease; Amyotrophic Lateral Sclerosis; Sclerosis; Pathologic Processes; Neurodegenerative Diseases; Nervous System Diseases; Neuromuscular Diseases; Spinal Cord Diseases; Central Nervous System Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Metabolic Diseases; Riluzole; Anticonvulsants; Excitatory Amino Acid Antagonists; Excitatory Amino Acid Agents; Neurotransmitter Agents; Molecular Mechanisms of Pharmacological Action; Physiological Effects of Drugs; Neuroprotective Agents; Protective Agents