Clenbuterol on Motor Function in Individuals With Amyotrophic Lateral Sclerosis
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|ClinicalTrials.gov Identifier: NCT04245709|
Recruitment Status : Completed
First Posted : January 29, 2020
Last Update Posted : March 23, 2021
|Condition or disease||Intervention/treatment||Phase|
|Amyotrophic Lateral Sclerosis||Drug: Clenbuterol||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||25 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Clinical Investigation of the Safety and Efficacy of Clenbuterol on Motor Function in Individuals With Amyotrophic Lateral Sclerosis|
|Actual Study Start Date :||February 10, 2020|
|Actual Primary Completion Date :||March 10, 2021|
|Actual Study Completion Date :||March 10, 2021|
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.
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 ERT (Koeberl et al. 2018).
- Number of adverse events as measured by patient reporting with dose increase [ Time Frame: Up to 24 weeks ]The primary endpoint is safety of clenbuterol at 80 mcg BID. Adverse and serious AEs will be systematically gathered as the dose is increased.
- Change in motor function measured by ALSFRS-R [ Time Frame: Baseline, week 4, week 12, week 16, week 20, and week 24 ]The ALS Functional Rating Scale- 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.
- Change in motor function measured by forced vital capacity (FVC) [ Time Frame: Baseline, week 4, week 12, and week 24 ]FVC is the total amount of air exhaled
- Change in motor function as measured by sniff nasal inspiratory [ Time Frame: Baseline, week 4, week 12, and week 24 ]Sniff nasal inspiratory pressure measures nasal pressure in an occluded nostril during a maximal sniff performed through the contralateral nostril
- Change in motor function as measured by isometric muscle strength [ Time Frame: Baseline, week 4, week 12, and week 24 ]This monitors muscle strength deterioration in ALS using the microFET 2 digital hand held dynamometer muscle tester.
- Change in motor function as measured by sub-maximum hand grip fatigue [ Time Frame: Baseline, week 4, week 12, and week 24 ]Hand strength deterioration is measured by squeezing a dynamometer for a least 5 seconds.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04245709
|United States, North Carolina|
|Duke University Medical center|
|Durham, North Carolina, United States, 27705|