A Trial to Evaluate the Efficacy and Safety of Reldesemtiv in Patients with Amyotrophic Lateral Sclerosis (ALS)

Overview

About this study

The purpose of this study is to assess the effect of Reldesemtiv versus placebo on functional outcomes in Amyotrophic Lateral Sclerosis (ALS).

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

Key Inclusion Criteria:

- Males or Females between the ages of 18 and 80 years of age, inclusive

- Diagnosis of familial or sporadic ALS (defined as meeting the laboratory-supported
probable, probable, or definite criteria for ALS according to the World Federation of
Neurology El Escorial criteria). Patients who meet the possible criteria are eligible
if they have lower motor neuron findings; those who have purely upper motor neuron
findings are ineligible.

- First symptom of ALS ≤ 24 months prior to screening. The qualifying first symptoms of
ALS are limited to manifestations of weakness in extremity, bulbar, or respiratory
muscles.

- ALSFRS-R total score ≤ 44 at screening. Patients with a total score of 45 or higher
may be rescreened 60±7 days following the original screening date.

- Upright FVC ≥ 65.0% of predicted for age, height, sex and ethnicity at screening
according to Global Lung Initiative equation

- Must be either on riluzole for ≥ 30 days prior to screening or have not taken it for
at least 30 days prior to screening

- Must have completed at least 2 cycles of edaravone at the time of screening or have
not received it for at least 30 days prior to screening

- Able to swallow whole tablets

Exclusion Criteria:

- eGFRCysC < 45.0 mL/min/1.73 m2 at screening

- Urine protein/creatinine ratio > 1 mg/mg (113 mg/mmol) at screening

- Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) ≥ 3-times the upper
limit of normal (ULN)

- Total bilirubin (TBL), direct or indirect bilirubin above the ULN.

- Cognitive impairment, related to ALS or otherwise that impairs the patient's ability
to understand and/or comply with study procedures and provide informed consent

- Other medically significant neurological conditions that could interfere with the
assessment of ALS symptoms, signs or progression.

- Has a tracheostomy

Note: Other protocol defined Inclusion/Exclusion criteria may apply.

Eligibility last updated 7/27/22. Questions regarding updates should be directed to the study team contact

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status Contact

Jacksonville, Fla.

Mayo Clinic principal investigator

Bjorn Oskarsson, M.D.

Open for enrollment

Contact information:

Clinical Studies Unit

(904) 953-2255

More information

Publications

Publications are currently not available
.
CLS-20516787

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