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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: ClinicalTrials.gov
Last refreshed on: 12 December 2020
Main ID:  NCT03843710
Date of registration: 14/02/2019
Prospective Registration: Yes
Primary sponsor: Clene Nanomedicine
Public title: 31P-MRS Imaging to Assess the Effects of CNM-Au8 on Impaired Neuronal Redox State in Amyotrophic Lateral Sclerosis (REPAIR-ALS) REPAIR-ALS
Scientific title: A Phase 2, Pilot Open Label, Sequential Group, Investigator Blinded Study of Magnetic Resonance Spectroscopy (31P-MRS) to Assess the Effects of CNM-Au8 for the Bioenergetic Improvement of Impaired Neuronal Redox State in Amyotrophic Lateral Sclerosis
Date of first enrolment: April 30, 2021
Target sample size: 24
Recruitment status: Not yet recruiting
URL:  https://clinicaltrials.gov/show/NCT03843710
Study type:  Interventional
Study design:  Allocation: Non-Randomized. Intervention model: Sequential Assignment. Primary purpose: Treatment. Masking: None (Open Label).  
Phase:  Phase 2
Countries of recruitment
United States
Contacts
Name:     Jeffery Elliott, MD
Address: 
Telephone: 214-648-5437
Email: Jeffery.Elliott@UTSouthwestern.edu
Affiliation: 
Name:     Robert Glanzman, MD
Address: 
Telephone: (801) 676-9695
Email: info@clene.com
Affiliation: 
Name:     Jeffery Elliott, MD
Address: 
Telephone:
Email:
Affiliation:  UT Southwestern
Key inclusion & exclusion criteria

Inclusion Criteria:

1. Able to understand and give written informed consent.

2. Male or female patients aged 35 years or greater (inclusive) and less than 75 years of
age at the time of ALS diagnosis.

3. Patients with a confirmed ALS diagnosis: "definite ALS" or "probable ALS" or
"possible" diagnostic criteria per the revised El Escorial Criteria as determined by a
neurologist subspecializing in ALS (e.g., the Principal Investigator by study site).

4. Stable background therapy (e.g., stable dosing of riluzole within the prior 6-weeks)
per Investigator discretion.

5. At the time of Screening disease duration less than or equal to 24-months from symptom
onset OR within 12-moths of a confirmed ALS diagnosis.

6. Forced vital capacity (FVC) >/= 60% of predicted value as adjusted for gender, height,
and age at the Screening Visit.

7. Patients who are ambulatory (e.g., normal ambulation, early ambulation difficulties,
or walks with assistance) on the ALSFRS-R scale.

Exclusion Criteria:

1. At Screening patients who utilize, or in the Investigator's judgment will be
imminently dependent upon during the course of this study:

1. Non-invasive ventilation

2. Gastrostomy (e.g., use of percutaneous endoscopic gastrostomy tube)

3. Use of wheel chair

2. Patient who have previously undergone tracheostomy.

3. Patient with a history of significant other major medical condition based on the
Investigator's judgment.

4. Based on the investigator's judgment, patients who may have difficulty complying with
the protocol and/or study procedures.

5. Patient with clinically significant abnormalities in hematology, blood chemistry, ECG,
or physical examination not resolved by the Baseline visit which according to
Investigator can interfere with study participation.

6. Patient participating in any other investigational drug trial or using investigational
drug (within 12 weeks prior to screening and thereafter)

7. Females who are pregnant or nursing or who plan to get pregnant during the course of
this clinical trial or within 6 months of the end of this trial.

8. Positive screen for drugs of abuse or known alcohol abuse.

9. Women of child-bearing potential, or men, who are unwilling or unable to use accepted
methods of birth control during the study or for 6 months following completion of
study participation.

10. Women with a positive pregnancy test, are lactating, or are planning to become
pregnant during the study.

11. Patients with implanted metal objects in their body that may be affected by an MRI
procedure.

12. Patients who are claustrophobic or otherwise unlikely to be able to complete the MRI
scanning procedures.

13. Patients with a history of gold allergy.



Age minimum: 35 Years
Age maximum: 75 Years
Gender: All
Health Condition(s) or Problem(s) studied
Amyotrophic Lateral Sclerosis
Intervention(s)
Drug: Gold Nanocrystals
Primary Outcome(s)
Change in 31P-MRS Redox Ratio (NAD+/NADH) [Time Frame: At 12 Weeks]
Secondary Outcome(s)
Secondary ID(s)
CNMAu8.203
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
University of Texas Southwestern Medical Center
Ethics review
Results
Results available:
Date Posted:
Date Completed:
URL:
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