A Clinical Investigation of the Safety and Efficacy of Clenbuterol on Motor Function in Individuals With Amyotrophic Lateral Sclerosis

Study Purpose:

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.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Enrolling

Phase:

Phase II

Study Chair(s)/Principal Investigator(s):

Dwight Koeberl, M.D., Ph.D., Duke University

Clinicaltrials.gov ID (11 digit #):

NCT04245709

Neals Affiliated?

No

Coordinating Center Contact Information

Duke University Medical Center
Lisa Harrison, RN / .(JavaScript must be enabled to view this email address) / 919-613-2681
.(JavaScript must be enabled to view this email address) Durham, North Carolina 27705 United States

Full Study Summary:

This is an open label pilot trial in which 25 people with ALS will take clenbuterol orally at 40-80 micrograms twice daily for 24 weeks. In person visits will occur at weeks 0, 4, 12 and 24. Telephone visits will occur at weeks 1, 6, 16 and 20. During these visits several safety and efficacy outcome measures will be performed for research purposes including safety labs, thyroid functions, pregnancy testing, ALSFRS-R, FVC, and muscle strength testing (myometry). The critical test of treatment efficacy will be the comparison of the ALSFRS-R slope during treatment to the estimated pre-treatment slope. All participants will continue to have standard follow up care for their ALS at Duke (for patients followed here) or their local ALS clinic

Study Sponsor:

Dwight Koeberl, M.D., Ph.D.

Participant Duration:

24 weeks

Estimated Enrollment:

25

Estimated Study Start Date:

01/31/2020

Estimated Study Completion Date:

02/10/2021

Posting Last Modified Date:

07/29/2020

Date Study Added to alsconsortium.org:

01/30/2020
  • Eligibility Criteria

    Gender:

    Female

    Minimum Age:

    18

    Maximum Age:

    N/A

    Min Vital Capacity (% predicted normal):

    50

    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:

    • Diagnosis of possible or more definite ALS according to the El Escorial criteria
    • SVC >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); MAO 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).

  • Site Contact Information

    Duke University Medical Center
    Lisa Harrison, RN / .(JavaScript must be enabled to view this email address) / 919-613-2681
    Durham, North Carolina 27705
    United States