Intermediate-Sized Expanded Access Study

An Expanded Access Protocol of Intravenous Trehalose Injection 90.5 mg/mL Treatment of Patients With Amyotrophic Lateral Sclerosis

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.

Study Overview

• Status: No longer available
• Conditions: Amyotrophic Lateral Sclerosis
• Intervention / Treatment: Drug: Trehalose

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.

• Study Type: Expanded Access
• Expanded Access Type: Intermediate-size Population

Contacts and Locations

Study Locations

• United States
  • Arizona
    • Phoenix, Arizona, United States, 85013
      • Barrow Neurological Institute
  • California
    • Orange, California, United States, 92697
      • University of California, Irvine
    • San Francisco, California, United States, 94115
      • University of California, San Francisco
  • Colorado
    • Aurora, Colorado, United States, 80045
      • University of Colorado
  • Connecticut
    • New Britain, Connecticut, United States, 06053
      • Hospital for Special Care
  • District of Columbia
    • Washington D.C., District of Columbia, United States, 20052
      • George Washington University
  • Florida
    • Fort Lauderdale, Florida, United States, 33328
      • Nova Southeastern University
    • Fort Lauderdale, Florida, United States, 33308
      • Holy Cross Hospital - Phil Smith Neuroscience Institute
  • Idaho
    • Boise, Idaho, United States, 83706
      • Saint Alphonsus Regional Medical Center
  • Iowa
    • Iowa City, Iowa, United States, 52242
      • University of Iowa Hospitals and Clinics
  • Maryland
    • Baltimore, Maryland, United States, 21201
      • University of Maryland School of Medicine
  • Massachusetts
    • Boston, Massachusetts, United States, 02215
      • Beth Israel Deaconess Medical Center
    • Boston, Massachusetts, United States, 02114
      • Sean M. Healey & AMG Center for ALS Neurological Clinical Research Institute, Massachusetts General Hospital
  • Michigan
    • Grand Rapids, Michigan, United States, 49503
      • Spectrum Health/Corewell Health
  • Minnesota
    • Minneapolis, Minnesota, United States, 55455
      • University of Minnesota
  • Missouri
    • St Louis, Missouri, United States, 63110
      • Washington University School of Medicine
  • New York
    • Syracuse, New York, United States, 13210
      • SUNY Upstate Medical University
  • Pennsylvania
    • Allentown, Pennsylvania, United States, 18105
      • Lehigh Valley Hospital
    • Philadelphia, Pennsylvania, United States, 19107
      • Thomas Jefferson University
  • Texas
    • Dallas, Texas, United States, 75206
      • Texas Neurology, P.A.-Neal Site:769
  • Virginia
    • Richmond, Virginia, United States, 23298
      • Virginia Commonwealth University Clinical Research Unit

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: N/A

Description

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.

Study Plan

How is the study designed?

Collaborators and Investigators

• Sponsor: Seelos Therapeutics, Inc.
• Collaborators: Massachusetts General Hospital; National Institute of Neurological Disorders and Stroke (NINDS); National Institutes of Health (NIH)
• Investigators: Principal Investigator: Suma Babu, MD,MPH,MBBS, Massachusetts General Hospital; Principal Investigator: James D Berry, MD,MPH, Massachusetts General Hospital; Principal Investigator: Sabrina Paganoni, MD,PhD, Massachusetts General Hospital

Publications and helpful links

General Publications

• Krivickas B, Scirocco E, Giacomelli E, Sharma S, Benson M, Keegan M, Kulesa-Kelley J, Chibnik LB, Casagrande G, Heyd L, Chase M, Drake K, Mohapatra S, Hagar JL, Hasenoehrl MG, Dagostino D, Sherman AV, Leite A, Yu H, Rosenthal J, Miller T, McCaffrey A, Gwathmey K, Locatelli E, Bayat E, Heitzman D, Young E, Goyal NA, Whitesell J, Felice K, Ilieva H, Swenson A, Walk D, Alameda G, Foster L, McIlduff CE, Walsh A, Zilliox L, Ajroud-Driss S, Bodkin C, Katz J, Ladha S, Rivner M, Rosow L, Twydell P, Wasiewski W, Babu S, Berry JD, Paganoni S. Multicenter Expanded Access Protocol for Research Through Access to Trehalose in People With Amyotrophic Lateral Sclerosis. Muscle Nerve. 2025 Aug 26. doi: 10.1002/mus.70011. Online ahead of print.

Study record dates

Study Registration Dates

• First Submitted: October 24, 2022
• First Submitted That Met QC Criteria: October 24, 2022
• First Posted (Actual): October 28, 2022

Study Record Updates

• Last Update Posted (Estimated): September 2, 2025
• Last Update Submitted That Met QC Criteria: August 29, 2025
• Last Verified: August 1, 2025

More Information

Terms related to this study

• Keywords: ALS; Lou Gehrig's Disease; Trehalose; Seelos Therapeutics; SLS-005
• Additional Relevant MeSH Terms: Central Nervous System Diseases; Nervous System Diseases; Neuromuscular Diseases; Metabolic Diseases; Neurodegenerative Diseases; Spinal Cord Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Motor Neuron Disease; Nutritional and Metabolic Diseases; Amyotrophic Lateral Sclerosis; Carbohydrates; Polysaccharides; Disaccharides; Oligosaccharides; Sugars; Glucans; Trehalose
• Other Study ID Numbers: SLS-005-EAP; UF1NS131791 (U.S. NIH Grant/Contract)