Clinical trial NCT05597436: Intermediate-Sized Expanded Access Study
Official title: An Expanded Access Protocol of Intravenous Trehalose Injection 90.5 mg/mL Treatment of Patients With Amyotrophic Lateral Sclerosis
Study type: EXPANDED_ACCESS
Overall status: NO_LONGER_AVAILABLE
Conditions: Amyotrophic Lateral Sclerosis
Keywords: ALS
Lou Gehrig's Disease
Trehalose
Seelos Therapeutics
SLS-005
Lead sponsor: Seelos Therapeutics, Inc.
Sponsor class: INDUSTRY
Collaborators: name: Massachusetts General Hospital
name: National Institute of Neurological Disorders and Stroke (NINDS)
name: National Institutes of Health (NIH)
First posted: 2022-10-28
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT

Brief summary: This expanded access protocol is to provide access to the investigational product, SLS-005, to participants with ALS who are not eligible to participate in clinical trials.

Detailed description: Approximately 70 participants receive a weekly infusion of SLS-005 for up to 24 weeks. There are 25 infusions over 24 weeks (first infusion occurs at the Screening/Baseline Visit). In addition to infusion visits, participants will have three scheduled in-clinic visits at Screening/Baseline, Week 3, and Week 24, and 2 phone call or telemedicine visits at Week 12 and approximately 28 days following the last dose of the investigational medical product.

Eligibility criteria: Inclusion Criteria:

1. Sporadic or familial ALS.
2. Age 18 years or older.
3. Cohort 1: Patients who do not qualify for any reasonably accessible ongoing clinical trial.
4. Cohort 2: Patients who have completed the open label extension (OLE) period of Regimen E of the HEALEY ALS Platform Trial and are not eligible for enrollment in another treatment regimen of the platform study.
5. Capable of providing informed consent and complying with study procedures, in the Site Investigator's (SI's) opinion.
6. Participants have established care with a physician at the specialized ALS center involved in the study and will maintain this clinical care throughout the duration of the EAP.
7. Participants must have a life expectancy of at least 6 months in SI's opinion.

Exclusion Criteria:

1. Current diagnosis or healthcare professional-recommended treatment (medication, exercise or diet) of diabetes mellitus.
2. Screening glucose \>=140 mg/dl.
3. Known hypersensitivity to trehalose.
4. Current use of oral trehalose.
5. Inability for participant to return to site for weekly drug administration, until approved for home infusions.
6. Screening body weight \>144 kilograms.
7. Participant with a history of any clinically significant or unstable medical condition or lab abnormality based on the SI's judgment that may interfere with assessment of the study objectives, with safety or full participation.
8. Females who are pregnant or nursing or who plan to get pregnant during the course of the EAP.
9. Females of child-bearing potential, or males, who are unwilling or unable to use highly effective methods of birth control.
10. Use of investigational treatments for ALS (as part of participation in a clinical trial or another EAP) within 5 half-lives (if known) or 30 days (whichever is longer) prior to the Screening Visit.
11. Permanent assisted ventilation (PAV), defined as more than 22 hours per day of noninvasive or invasive mechanical ventilation for more than seven consecutive days. The date of onset of PAV is the first day of the seven days.
12. Active cancer or history of cancer, except for the following: basal cell carcinoma or successfully treated squamous cell carcinoma of the skin, cervical carcinoma in situ, prostatic carcinoma in situ, or other malignancies curatively treated and with no evidence of disease recurrence for at least 3 years.
13. Presence of unstable psychiatric disease, cognitive impairment, dementia, or substance abuse that would impair ability of the participant to provide informed consent, in the SI's opinion.
14. Patients who chose to take experimental medications and/or supplements, and that is the only reason they are not eligible for trials, won't be eligible for the EAP.

Primary outcomes:
Secondary outcomes:
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Barrow Neurological Institute
state: Arizona
city: San Francisco
country: United States
facility: University of California, San Francisco
state: California
city: Aurora
country: United States
facility: University of Colorado
state: Colorado
city: New Britain
country: United States
facility: Hospital for Special Care
state: Connecticut
city: Fort Lauderdale
country: United States
facility: Holy Cross Hospital - Phil Smith Neuroscience Institute
state: Florida
city: Fort Lauderdale
country: United States
facility: Nova Southeastern University
state: Florida
city: Boise
country: United States
facility: Saint Alphonsus Regional Medical Center
state: Idaho
city: Bloomington
country: United States
facility: Indiana University
state: Indiana
city: Iowa City
country: United States
facility: University of Iowa Hospitals and Clinics
state: Iowa
city: Baltimore
country: United States
facility: University of Maryland School of Medicine
state: Maryland
city: Boston
country: United States
facility: Sean M. Healey & AMG Center for ALS Neurological Clinical Research Institute, Massachusetts General Hospital
state: Massachusetts
city: Minneapolis
country: United States
facility: University of Minnesota
state: Minnesota
city: Saint Louis
country: United States
facility: Washington University School of Medicine
state: Missouri
city: Syracuse
country: United States
facility: SUNY Upstate Medical University
state: New York
city: Philadelphia
country: United States
facility: Thomas Jefferson University
state: Pennsylvania
city: Dallas
country: United States
facility: Texas Neurology, P.A.-Neal Site:769
state: Texas
city: Richmond
country: United States
facility: Virginia Commonwealth University Clinical Research Unit
state: Virginia
Study officials:
affiliation: Massachusetts General Hospital
name: Suma Babu, MD,MPH,MBBS
role: PRINCIPAL_INVESTIGATOR
affiliation: Massachusetts General Hospital
name: James D Berry, MD,MPH
role: PRINCIPAL_INVESTIGATOR
affiliation: Massachusetts General Hospital
name: Sabrina Paganoni, MD,PhD
role: PRINCIPAL_INVESTIGATOR
References: