- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04245709
Clenbuterol on Motor Function in Individuals With Amyotrophic Lateral Sclerosis
A Clinical Investigation of the Safety and Efficacy of Clenbuterol on Motor Function in Individuals With Amyotrophic Lateral Sclerosis
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
Study Type
Enrollment (Actual)
Phase
- Phase 2
Contacts and Locations
Study Locations
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North Carolina
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Durham, North Carolina, United States, 27705
- Duke University Medical Center
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
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).
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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Experimental: Open label Arm
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.
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The intervention is treatment with oral clenbuterol at 40-80 micrograms twice daily for 24 weeks.
Dosage will initially be 40 mcg daily for one week, then 40 mcg BID per oral daily for the next 5 weeks.
If the 40 mcg BID per oral is well tolerated in the opinion of Dr. Bedlack, the dose will be increased to 80 mcg each morning/40 mcg each evening for one week, followed by 80 mcg BID per oral for the remainder of the study.
The selected target dose (80 mcg BID) is based upon the experience with the long-term administration of clenbuterol, specifically the beneficial muscle effects in a Phase I/II clinical trial that enrolled patients with late-onset Pompe disease who were previously treated with enzyme replacement therapy (Koeberl et al. 2018).
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Number of Participants With Serious Adverse Events as Measured by Patient Reporting
Time Frame: Up to 24 weeks
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The primary endpoint is safety of clenbuterol at 80 mcg BID.
Adverse events and serious adverse events will be systematically gathered as the dose is increased.
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Up to 24 weeks
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Change in Motor Function Measured by ALSFRS-R
Time Frame: Baseline, week 4, week 12, week 16, week 20, and week 24
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The ALS Functional Rating Scale (ALSFRS-R) - 12 questions rated on a five-point scale, where 0= can't do, to 5= normal ability.
It is utilized for monitoring the progression of disability in patients with ALS.
The critical test for efficacy was comparison of the mean slope of the ALSFRS-R during treatment compared to pre-treatment.
Pre-treatment slope for each participant was estimated as follows: (48-enrollment ALSFRS-R)/months since symptom onset.
A statistically significant treatment effect was determined by a two-tailed, t-test, with a critical p value < .05.
Other analyses included a repeated measures ANOVA design (between and within subjects) of ALSFRS-R slopes before and during treatment.
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Baseline, week 4, week 12, week 16, week 20, and week 24
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FVC Decline, Per-protocol Comparison
Time Frame: Baseline, week 4, week 12, and week 24
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Comparison of the mean slope of percent predicted FVC during treatment versus pre-treatment.
Pre-treatment slope for each participant was estimated as follows: (100%-enrollment percent predicted FVC)/months since symptom onset.
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Baseline, week 4, week 12, and week 24
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Collaborators and Investigators
Sponsor
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Pathologic Processes
- Metabolic Diseases
- Central Nervous System Diseases
- Nervous System Diseases
- Neuromuscular Diseases
- Neurodegenerative Diseases
- Spinal Cord Diseases
- TDP-43 Proteinopathies
- Proteostasis Deficiencies
- Sclerosis
- Motor Neuron Disease
- Amyotrophic Lateral Sclerosis
- Physiological Effects of Drugs
- Adrenergic Agents
- Neurotransmitter Agents
- Molecular Mechanisms of Pharmacological Action
- Autonomic Agents
- Peripheral Nervous System Agents
- Adrenergic Agonists
- Bronchodilator Agents
- Anti-Asthmatic Agents
- Respiratory System Agents
- Adrenergic beta-Agonists
- Sympathomimetics
- Clenbuterol
Other Study ID Numbers
- Pro00103668
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
IPD Plan Description
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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