Trial of Theracurmin for Patients With Amyotrophic Lateral Sclerosis (ALS)
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|ClinicalTrials.gov Identifier: NCT04499963|
Recruitment Status : Enrolling by invitation
First Posted : August 5, 2020
Last Update Posted : August 19, 2021
|Condition or disease||Intervention/treatment||Phase|
|Amyotrophic Lateral Sclerosis||Drug: Theracurmin HP||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||100 participants|
|Intervention Model:||Parallel Assignment|
|Intervention Model Description:||This will be a 6-month, widely inclusive, largely remote/virtual, single-center, open-label pilot trial utilizing a historical control group.|
|Masking:||None (Open Label)|
|Official Title:||An Open-label, Single-center, 6-month Trial of Theracurmin for Patients With Amyotrophic Lateral Sclerosis (ALS)|
|Actual Study Start Date :||August 28, 2020|
|Estimated Primary Completion Date :||August 15, 2022|
|Estimated Study Completion Date :||August 15, 2022|
Experimental: Open Label Arm
The intervention is twice daily dosage of Theracurmin 90 mg capsules. This same dose was used in a successful trial of patients with mild cognitive impairment. Theracurmin HP capsules containing 90 mg curcumin each that will be taken as one capsule twice daily for 6 months. Each capsule contains 300 mg Theracurmin enhanced bioavailable water-dispersible turmeric rhizome complex providing 30% curcumin (90 mg). The content of Theracurmin HP has been independently certified by NSF International under NSF/ANSI 173.
Drug: Theracurmin HP
The intervention is based on the twice daily dosage of Theracurmin 90 mg capsules used in a trial of patients with mild cognitive impairment
No Intervention: Healthy Control Arm
We will seek to enroll 50 healthy control participants. We will attempt to enroll one control subject from each enrolled primary participant's home, preferably a spouse or partner of similar age if possible. We plan to use this data to compare the microbiome of control participants to that of the ALS participants at baseline, week 4 and month 6. We will not conduct further follow-up or collect additional samples with the control subjects.
- Change in ALSFRS-R score [ Time Frame: Starting at week 4 and then once every 30 days for 6 months ]The ALSFRS-R (ALS Functional Rating Scale-Revised) will be determined at all video/telephone visits. ALSFRS-R is a quickly administered (five minute) ordinal rating scale (ratings 0-4) used to determine patients' assessments of their capability and independence in 12 functional activities. All 12 activities are relevant in ALS. Change in ALSFRS-R scores correlate with change in strength over time, and it is closely associated with quality of life measures and predicted survival. Each task is rated on a five-point scale from 0 = can't do, to 4 = normal ability. Individual item scores are summed to produce a reported score of between 0=worst and 48=best.
- ALS Reversals [ Time Frame: month 6 ]To look for an increase in the frequency of ALS reversals, we will count the number of participants who have an ALSFRS-R score that improves by 4 points or more over 6 months. The observed frequency of spontaneous ALS reversals defined in this way is 2-5% (30). We will look for an increase in this frequency to at least 10% (1 in 10).
- Saliva Microbiome Sequencing change [ Time Frame: Enrollment, week 4 and month 6 visits ]The microbiome of study participants will be analyzed in saliva samples at enrollment, week 4 and month 6 visits. We will compare the changes in the saliva microbiome over time in patients with ALS on Theracurmin to the changes observed in untreated healthy controls.Data will be compared within subjects with ALS to assess possible changes over the length of the study and identify microbial correlates of disease progression.
- Number of adverse events as measured by patient reporting [ Time Frame: Study duration, up to 6 months ]Adverse and serious adverse events will be recorded throughout the study. The PI will determine whether these are related to treatment. Patients will also measure and record their side effects at Month 0-6 Internet Visits on their PLM profile.
- Enrollment Rate [ Time Frame: one month ]Enrollment rate will be calculated as the number of participants enrolled per month.
- Retention [ Time Frame: month 6 ]Retention will be assessed by looking at the dropout rate at 6 months (the percentage of surviving enrolled patients who complete the Month 6 Visit).
- Stool Microbiome Sequencing change [ Time Frame: Enrollment, week 4 and month 6 visits ]The microbiome of study participants will be analyzed in stool samples at enrollment, week 4 and month 6 visits. Changes will be compared in the stool microbiome over time in patients with ALS on Theracurmin to the changes observed in untreated healthy controls. Metagenomic analysis of deidentified selected fecal sample will be done on patients that positively respond to Theracurmin to achieve strain level identification of microbes positively and negatively associated with improved outcomes. The selected anaerobes that have been linked to ALS severity will be cultured for further genetic sand phenotypic characterization.
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): NCT04499963
|United States, North Carolina|
|Duke University Medical Center|
|Durham, North Carolina, United States, 27705|
|Principal Investigator:||Richard Bedlack, MD, PhD||Duke University|