Phase 2 N=25 Treatment

Clenbuterol on Motor Function in Individuals With Amyotrophic Lateral Sclerosis

Amyotrophic Lateral Sclerosis

Bottom Line

View on ClinicalTrials.gov: NCT04245709 ↗

Enrolled (actual)

Serious AEs

8.0%

Results posted

Sep 2022

Primary outcome: Primary: Number of Participants With Serious Adverse Events as Measured by Patient Reporting — 2 Participants

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: Clenbuterol (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: Dwight Koeberl, M.D., Ph.D.
Primary completion: Mar 2021

Outcome Measures

Outcome	Result	p-value
PRIMARY Number of Participants With Serious Adverse Events as Measured by Patient Reporting	2	—
SECONDARY Change in Motor Function Measured by ALSFRS-R	-0.17	—
SECONDARY FVC Decline, Per-protocol Comparison	-0.25	—

Summary

The purpose of this study is to assess the safety and tolerability of clenbuterol (taken by mouth) in subjects with ALS (amyotrophic lateral sclerosis) and to assess the effectiveness of clenbuterol with regard to motor function in subjects with ALS. Subjects will be in this study approximately 24 weeks. The study drug, clenbuterol, is taken twice a day. As part of this study subjects will have the following tests and procedures: medical history, vital signs, physical examination, blood tests, heart and lung function tests, muscle function test, ALSFRS-R (ALS Functional Rating Scale Revised), thyroid function and for women who can become pregnant, pregnancy tests.

Eligibility Criteria

Inclusion Criteria

Diagnosis of possible or more definite ALS according to the El Escorial criteria
FVC >50% of predicted for age, height and gender.
At least four of 12 ALSFRS-R questions scored as 2 or 3 at screening.
Diminished but measurable grip strength (1) in at least one hand (females:10-50 pounds; males, 10-70 pounds).
Taking riluzole at a stable dose or not taking riluzole at screening.
On Radicava at a stable dose for at least 30d or not taking this
Life expectancy at least 6 months
Able to swallow tablets without crushing.
Age: 18+ years at enrollment.
Subjects are capable of giving written consent.
If sexually active, must agree to use contraceptive or abstinence for duration of treatment
Females of child bearing age must have negative pregnancy test at screening

Exclusion Criteria

Concurrent illness or laboratory abnormalities that could confound the measurement of ALS progression or interfere with the ability to complete the study.
Taking any investigational study drug within 30 days of screening or five half-lives of the prior agent.
No previous exposure to clenbuterol.
Pregnancy
Clinically relevant EKG abnormality (arrhythmia, cardiomyopathy)
Tachycardia (resting heart rate greater than 100 beats per minute)
History of seizure disorder
Hyperthyroidism
Pheochromocytoma
Pregnancy
Have any other co-morbid conditions that in the opinion of the study investigator, places the participant at increased risk of complications, interferes with study participation or compliance, or confounds study objectives
History of hypersensitivity to 2-agonist drugs such as albuterol, levalbuterol (Xopenex), bitolterol (Tornalate), pirbuterol (Maxair), terbutaline, salmeterol (Serevent).
The use of the following concomitant meds is prohibited during the study:

diuretics (furosemide, Lasix), digoxin (digitalis, Lanoxin);blockers such as atenolol (Tenormin), metoprolol (Lopressor), and propranolol (Inderal); tricyclic antidepressants such as amitriptyline (Elavil, Etrafon), doxepin (Sinequan), imipramine (Janimine, Tofranil), and nortriptyline (Pamelor); monoamine oxidase inhibitors such as isocarboxazid (Marplan), phenelzine (Nardil), rasagiline (Azilect), selegiline (Eldepryl, Emsam), or tranylcypromine (Parnate); or other bronchodilators such as albuterol (Ventolin), levalbuterol (Xopenex), bitolterol (Tornalate), pirbuterol (Maxair), terbutaline (Brethine, Bricanyl), salmeterol (Serevent), isoetherine (Bronkometer), metaproterenol (Alupent, Metaprel), or isoproterenol (Isuprel Mistometer).

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT04245709). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.