Amyotrophic Lateral SclerosisPhase 2/3NCT04297683
HEALEY ALS Platform Trial - Master Protocol
A Phase 2/3 study testing Zilucoplan, Verdiperstat and CNM-Au8 in people with Amyotrophic Lateral Sclerosis, run by Merit E. Cudkowicz, MD at 81 sites in United States.
Trial summary
The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- Zilucoplan, Verdiperstat and CNM-Au8
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1,500 participants
- Where
- 81 sitesUnited States
- Study dates
- 14 Jun 2020to Aug 2028
- Phase
- Phase 2/3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Sporadic or familial ALS diagnosed as clinically possible, probable, lab-supported probable, or definite ALS defined by revised El Escorial criteria.
- Age 18 years or older.
- Capable of providing informed consent and complying with study procedures, in the SI's opinion.
- Time since onset of weakness due to ALS ≤ 24 months at the time of the Master Protocol Screening Visit.
- Vital Capacity ≥ 50% of predicted capacity at the time of the Master Protocol Screening Visit measured by Slow Vital Capacity (SVC), or, if required due to pandemic-related restrictions, Forced Vital Capacity (FVC) measured in person.
- Participants must either not take riluzole or be on a stable dose of riluzole for ≥ 30 days prior to the Master Protocol Screening Visit.
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- Participants must either not take edaravone or have completed at least one cycle (typically 14 days) of edaravone prior to the Master Protocol Screening Visit.
- Participants must have the ability to swallow pills and liquids at the time of the Master Protocol Screening Visit and, in the SI's opinion, have the ability to swallow for the duration of the study.
- Geographically accessible to the site.
Who cannot join
- Clinically significant unstable medical condition (other than ALS) that would pose a risk to the participant, according to SI's judgment (e.g., cardiovascular instability, systemic infection), or clinically significant laboratory abnormality or EKG changes. Clinically significant abnormal liver or kidney function is exclusionary. The following values [alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 3 times the upper limit of normal (ULN) or estimated Glomerular Filtration Rate (eGFR) < 30 mL/min/1.73m2] are exclusionary regardless of clinical symptoms.
- 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.
- 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.
- Use of investigational treatments for ALS (off-label use or active participation in a clinical trial) within 5 half-lives (if known) or 30 days (whichever is longer) prior to the Master Protocol Screening Visit.
- Exposure at any time to any gene therapies under investigation for the treatment of ALS (off-label use or investigational).
- If female, breastfeeding, known to be pregnant, planning to become pregnant during the study, or of child-bearing potential and unwilling to use effective contraception, for the duration of the trial and for 3 months, or as specified in each RSA, after discontinuing study treatment.
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- If male of reproductive capacity, unwilling to use effective contraception for the duration of the trial and for 3 months, or as specified in each RSA, after discontinuing study treatment.
- Anything that would place the participant at increased risk or preclude the participant's full compliance with or completion of the study, in the SI's opinion.
- If a participant is being re-screened, the disqualifying condition has not been resolved, or the mandatory wash-out duration has not occurred.
Trial locations and contacts
81 sites in 1 country.
81 sites in 1 country
United States81 sites
Lehigh Valley Health Network
Allentown, Pennsylvania, 18103
Dent Neurologic Institute
Amherst, New York, 14226
University of Michigan
Ann Arbor, Michigan, 48109
Augusta University
Augusta, Georgia, 30912
University of Colorado
Aurora, Colorado, 80045
Austin Neuromuscular Clinic
Austin, Texas, 78759
Johns Hopkins University
Baltimore, Maryland, 21205
Saint Alphonsus Regional Medical Center
Boise, Idaho, 83704
Massachusetts General Hospital
Boston, Massachusetts, 02114
Beth Israel Deaconess Medical Center
Boston, Massachusetts, 02215
Lahey Hospital and Medical Center
Burlington, Massachusetts, 01805
University of North Carolina
Chapel Hill, North Carolina, 27599
Atrium Health
Charlotte, North Carolina, 28207
University of Virginia
Charlottesville, Virginia, 22908
Northwestern University
Chicago, Illinois, 60611
University of Chicago
Chicago, Illinois, 60637
University of Cincinnati
Cincinnati, Ohio, 45219
Cleveland Clinic
Cleveland, Ohio, 44195
University of Missouri Health Care
Columbia, Missouri, 65212
The Ohio State University
Columbus, Ohio, 43221
Texas Neurology
Dallas, Texas, 75214
Nova Southeastern University
Davie, Florida, 33024
Henry Ford Health System
Detroit, Michigan, 48202
Essentia Health
Duluth, Minnesota, 55805
Duke University
Durham, North Carolina, 27702
University of Kansas Medical Center
Fairway, Kansas, 66205
University of Florida
Gainesville, Florida, 32610
Spectrum Health/Corewell Health
Grand Rapids, Michigan, 49525
Hackensack University Medical Center
Hackensack, New Jersey, 07601
Virginia Commonwealth University
Henrico, Virginia, 23233
Penn State Hershey
Hershey, Pennsylvania, 17033
Baylor College of Medicine
Houston, Texas, 77030
Houston Methodist
Houston, Texas, 77030
Indiana University Health
Indianapolis, Indiana, 46202
University of Iowa Hospitals and Clinics
Iowa City, Iowa, 52242
Mayo Clinic Florida
Jacksonville, Florida, 32224
UC San Diego
La Jolla, California, 92037
Dartmouth-Hitchcock Medical Center
Lebanon, New Hampshire, 03756
University of Kentucky
Lexington, Kentucky, 40536
Neurology Associates, P.C./Somnos Clinical Research
Lincoln, Nebraska, 68506
University of Arkansas for Medical Sciences
Little Rock, Arkansas, 72205
Loma Linda University Health
Loma Linda, California, 92354
Kaiser Permanente Los Angeles Medical Center
Los Angeles, California, 90027
University of Southern California
Los Angeles, California, 90033
Cedars-Sinai Medical Center
Los Angeles, California, 90048
University of Miami
Miami, Florida, 33136
Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
University of Minnesota Medical School
Minneapolis, Minnesota, 55455
Vanderbilt University Medical Center
Nashville, Tennessee, 37232
Hospital for Special Care
New Britain, Connecticut, 06053
Yale University
New Haven, Connecticut, 06519
Ochsner Health System
New Orleans, Louisiana, 70115
Mount Sinai
New York, New York, 10003
Columbia University
New York, New York, 10032
University of Massachusetts Medical School
North Worcester, Massachusetts, 01655
University of Nebraska Medical Center
Omaha, Nebraska, 68198
University of California, Irvine
Orange, California, 92868
Jefferson Weinberg ALS Center, Thomas Jefferson University
Philadelphia, Pennsylvania, 19107
University of Pennsylvania
Philadelphia, Pennsylvania, 19107
Lewis Katz School of Medicine at Temple University
Philadelphia, Pennsylvania, 19140
Barrow Neurological Institute
Phoenix, Arizona, 85013
University of Pittsburg Medical Center
Pittsburgh, Pennsylvania, 15232
Providence Brain and Spine Institute ALS Center
Portland, Oregon, 97213
Mayo Clinic - Rochester
Rochester, Minnesota, 55902
University of Rochester
Rochester, New York, 14642
University of Utah
Salt Lake City, Utah, 84132
UTHSCSA
San Antonio, Texas, 78229
Forbes Norris MDA/ALS Research Center, California Pacific Medical Center
San Francisco, California, 94115
University of California, San Francisco
San Francisco, California, 94143
Mayo Clinic Scottsdale
Scottsdale, Arizona, 85259
Swedish Medical Center
Seattle, Washington, 98122
University of Washington
Seattle, Washington, 98195
Saint Louis University
St Louis, Missouri, 63104
Washington University School of Medicine
St Louis, Missouri, 63110
Stony Brook University Hospital
Stony Brook, New York, 11794
SUNY Upstate
Syracuse, New York, 13202
University of South Florida
Tampa, Florida, 33612
Georgetown University
Washington D.C., District of Columbia, 20007
George Washington University
Washington D.C., District of Columbia, 20037
OhioHealth Research Institute
Westerville, Ohio, 43082
Wake Forest Health Science
Winston-Salem, North Carolina, 27157
About this study
Official title: HEALEY ALS Platform Trial
The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS.
Read the detailed description
The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS. This trial is designed as a perpetual platform trial. This means that there is a single Master Protocol dictating the conduct of the trial.
In this trial, multiple investigational products for ALS will be tested simultaneously or sequentially. Each investigational product will be tested in a regimen. Each regimen consists of a placebo-controlled trial, meaning that the active investigational product and matching placebo will be tested in each regimen.
The additional details that govern the testing of each investigational product will be summarized in separate regimen-specific appendices (RSAs). Each regimen will have a separate ClinicalTrials.gov posting, which will include specific information about the regimen. All regimen-specific outcome measures will be detailed in each regimen posting.
Participants will have an equal chance to be randomized to all regimens that are active at the time of screening. Once randomized to a regimen, participants will be randomized in a 3:1 ratio to either study drug or placebo.
The following regimens are active in the trial:
Regimen I - NUZ-001
New regimens will be continuously added as new investigational products become available. The HEALEY ALS Platform Trial will enroll additional participants as each new regimen is available.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- Zilucoplan, Verdiperstat, CNM-Au8, Pridopidine, SLS-005 Trehalose
- Keywords
- ALS, Placebo-Controlled, Double-Blind, Master Protocol, Lou Gehrig's Disease
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ZilucoplanDrug
Drug: Zilucoplan Administration: Subcutaneous injection Dose: Minimum of .0.22 mg/kg daily to a maximum dose of 0.42 mg/kg daily, dependent on weight
VerdiperstatDrug
Drug: Verdiperstat Administration: Oral Dose: 600mg twice daily
CNM-Au8Drug
Drug: CNM-Au8 Administration: Oral Dose: 30 mg or 60 mg daily
PridopidineDrug
Drug: Pridopidine Administration: Oral Dose: 45mg twice daily
SLS-005 TrehaloseDrug
Drug: SLS-005 Trehalose Administration: Infusion Dose: 0.75 g/kg weekly
ABBV-CLS-7262Drug
Drug: ABBV-CLS-7162 Administration: Oral Dose: Dose 1 or Dose 2
DNL343Drug
Drug: DNL343 Administration: Oral Dose: Once per day
NUZ-001Drug
Drug: NUZ-001. Administration: Oral. Dose: Once per day.
| Study group | Role | What they receive |
|---|---|---|
| Regimen A - Zilucoplan | Experimental | Participants are randomized to receive either active zilucoplan or matching placebo.
NO LONGER RECRUITING; RESULTS REPORTED. Receives: Drug: Zilucoplan |
| Regimen B - Verdiperstat | Experimental | Participants are randomized to receive either active verdiperstat or matching placebo.
NO LONGER RECRUITING; RESULTS REPORTED. Receives: Drug: Verdiperstat |
| Regimen C - CNM-Au8 | Experimental | Participants are randomized to receive either active CNM-Au8 or matching placebo.
NO LONGER RECRUITING; RESULTS REPORTED. Receives: Drug: CNM-Au8 |
| Regimen D - Pridopidine | Experimental | Participants are randomized to receive either active Pridopidine or matching placebo.
NO LONGER RECRUITING; RESULTS REPORTED. Receives: Drug: Pridopidine |
| Regimen E - SLS-005 Trehalose | Experimental | Participants are randomized to receive either active SLS-005 Trehalose or matching placebo.
NO LONGER RECRUITING; RESULTS REPORTED. Receives: Drug: SLS-005 Trehalose |
| Regimen F- ABBV-CLS-7262 | Experimental | Participants are randomized to receive either active ABBV-CLS-7262 or matching placebo.
NO LONGER RECRUITING; RESULTS REPORTED. Receives: Drug: ABBV-CLS-7262 |
| Regimen G - DNL343 | Experimental | Participants are randomized to receive either active DNL343 or matching placebo.
NO LONGER RECRUITING; RESULTS REPORTED. Receives: Drug: DNL343 |
| Regimen I - NUZ-001 | Experimental | Participants are randomized to receive either active NUZ-001 or matching placebo. ACTIVE, NOT RECRUITING Receives: Drug: NUZ-001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Disease Progression
Change in disease severity as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) total score and survival.
Measured: 36 Weeks
Secondary outcomes (2)
Respiratory Function
Change in respiratory function over time as measured by Slow Vital Capacity (SVC).
Measured: 36 Weeks
Survival
Comparison of rate of occurrence between groups.
Measured: 36 Weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Mar 2020
- Study start14 Jun 2020
- Primary completionJul 2027 (estimated)
- Study completionAug 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- As new investigational products become available, additional regimens will be added to the HEALEY ALS Platform Trial.
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 1,500 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Merit E. Cudkowicz, MD
- Sponsor type
- University, hospital or other
- Collaborators
- Massachusetts General Hospital
- Responsible party
- Sponsor-investigator, Merit E. Cudkowicz, MD, Massachusetts General Hospital
- Investigators
- Merit Cudkowicz, MD, Principal investigator, Massachusetts General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04297683
- Sponsor's protocol ID
- 2019P003518
- Status last verified
- Jul 2026
- First submitted
- 3 Mar 2020
- Last updated
- 21 Jul 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Writing Committee for the HEALEY ALS Platform Trial; Berry JD, Maragakis NJ, Macklin EA, Chibnik LB, Quintana M, Saville BR, Detry MA, Vestrucci M, Marion J, McGlothlin A, Stommel EW, Chase M, Pothier L, Harkey BA, Yu H, Sherman A, Shefner J, Hall M, Kittle G, Babu S, Andrews J, D'Agostino D, Tustison E, Scirocco E, Giacomelli E, Alameda G, Locatelli E, Ho D, Quick A, Ajroud-Driss S, Katz J, Heitzman D, Appel SH, Shroff S, Felice KJ, Simmons Z, Miller T, Olney N, Weiss MD, Goutman SA, Fernandes JA Jr, Jawdat O, Owegi MA, Foster L, Vu T, Ilieva H, Newman DS, Arcila-Londono X, Jackson C, Ladha S, Heiman-Patterson T, Caress J, Swenson A, Peltier A, Lewis R, Fee D, Elliott M, Bedlack R, Kasarskis EJ, Elman L, Rosenfeld J, Walk D, McIlduff CE, Twydell P, Young E, Johnson K, Rezania K, Goyal NA, Cohen JA, Benatar M, Jones V, Glass J, Shah J, Beydoun SR, Wymer JP, Zilliox L, Nayar S, Pattee GL, Martinez-Thompson J, Rynders A, Evan J, Evan J, Hartford A, Sepassi M, Ho KS, Glanzman R, Greenberg B, Hotchkin MT, Paganoni S, Cudkowicz ME; HEALEY ALS Platform Trial Study Group. CNM-Au8 in Amyotrophic Lateral Sclerosis: The HEALEY ALS Platform Trial. JAMA. 2025 Feb 17;333(13):1138-49. doi: 10.1001/jama.2024.27643. Online ahead of print.
PubMed 40067821Writing Committee for the HEALEY ALS Platform Trial; Shefner JM, Oskarsson B, Macklin EA, Chibnik LB, Quintana M, Saville BR, Detry MA, Vestrucci M, Marion J, McGlothlin A, Heiman-Patterson T, Chase M, Pothier L, Harkey BA, Yu H, Sherman AV, Hall M, Kittle G, Berry JD, Babu S, Andrews J, D'Agostino D, Tustison E, Scirocco E, Giacomelli E, Alameda G, Locatelli E, Ho D, Quick A, Ajroud-Driss S, Katz J, Heitzman D, Appel SH, Shroff S, Felice K, Maragakis NJ, Simmons Z, Miller TM, Olney N, Weiss MD, Goutman SA, Fernandes JA, Jawdat O, Owegi MA, Foster LA, Vu T, Ilieva H, Newman DS, Arcila-Londono X, Jackson CE, Ladha S, Caress JB, Swenson A, Peltier A, Lewis RA, Fee D, Elliott M, Bedlack R, Kasarskis EJ, Elman L, Rosenfeld J, Walk D, McIlduff C, Twydell P, Young E, Johnson K, Rezania K, Goyal NA, Cohen JA, Benatar M, Jones V, Shah J, Beydoun SR, Wymer JP, Zilliox L, Nayar S, Pattee GL, Martinez-Thompson J, Leitner ML, Chen K, Goldberg YP, Cohen Y, Geva M, Hayden MR, Paganoni S, Cudkowicz ME; HEALEY ALS Platform Trial Study Group. Pridopidine in Amyotrophic Lateral Sclerosis: The HEALEY ALS Platform Trial. JAMA. 2025 Feb 17;333(13):1128-37. doi: 10.1001/jama.2024.26429. Online ahead of print.
PubMed 40067755Writing Committee for the HEALEY ALS Platform Trial; Andrews J, Paganoni S, Macklin EA, Chibnik LB, Quintana M, Saville BR, Detry MA, Vestrucci M, Marion J, McGlothlin A, Young E, Chase M, Pothier L, Harkey B, Yu H, Sherman A, Shefner J, Hall M, Kittle G, Connolly MR, Berry JD, D'Agostino D, Tustison E, Giacomelli E, Scirocco E, Alameda G, Locatelli E, Ho D, Quick A, Heitzman D, Ajroud-Driss S, Appel SH, Shroff S, Katz J, Felice K, Maragakis NJ, Simmons Z, Goutman SA, Olney N, Miller T, Fernandes JA, Ilieva H, Jawdat O, Weiss MD, Foster L, Vu T, Ladha S, Owegi MA, Newman DS, Arcila-Londono X, Jackson CE, Swenson A, Heiman-Patterson T, Caress J, Fee D, Peltier A, Lewis R, Rosenfeld J, Walk D, Johnson K, Elliott M, Kasarskis EJ, Rutkove S, McIlduff CE, Bedlack R, Elman L, Goyal NA, Rezania K, Twydell P, Benatar M, Glass J, Cohen JA, Jones V, Zilliox L, Wymer JP, Beydoun SR, Shah J, Pattee GL, Martinez-Thompson J, Nayar S, Granit V, Donohue M, Grossman K, Campbell DJ, Qureshi IA, Cudkowicz ME, Babu S. Verdiperstat in Amyotrophic Lateral Sclerosis: Results From the Randomized HEALEY ALS Platform Trial. JAMA Neurol. 2025 Apr;82(4):333-343. doi: 10.1001/jamaneurol.2024.5249. Epub 2025 Feb 17.
PubMed 40067754Paganoni S, Fournier CN, Macklin EA, Chibnik LB, Quintana M, Saville BR, Detry MA, Vestrucci M, Marion J, McGlothlin A, Ajroud-Driss S, Chase M, Pothier L, Harkey BA, Yu H, Sherman AV, Shefner JM, Hall M, Kittle G, Berry JD, Babu S, Andrews J, Dagostino D, Tustison E, Giacomelli E, Scirocco E, Alameda G, Locatelli E, Ho D, Quick A, Katz J, Heitzman D, Appel SH, Shroff S, Felice K, Maragakis NJ, Simmons Z, Miller TM, Olney N, Weiss MD, Goutman SA, Fernandes JA, Jawdat O, Owegi MA, Foster LA, Vu T, Ilieva H, Newman DS, Arcila-Londono X, Jackson CE, Ladha S, Heiman-Patterson T, Caress JB, Swenson A, Peltier A, Lewis R, Fee D, Elliott M, Bedlack R, Kasarskis EJ, Elman L, Rosenfeld J, Walk D, McIlduff C, Twydell P, Young E, Johnson K, Rezania K, Goyal NA, Cohen JA, Benatar M, Jones V, Glass J, Shah J, Beydoun SR, Wymer JP, Zilliox L, Nayar S, Pattee GL, Martinez-Thompson J, Harvey B, Patel S, Mahoney P, Duda PW, Cudkowicz ME; HEALEY ALS Platform Trial Study Group. Efficacy and Safety of Zilucoplan in Amyotrophic Lateral Sclerosis: A Randomized Clinical Trial. JAMA Netw Open. 2025 Feb 3;8(2):e2459058. doi: 10.1001/jamanetworkopen.2024.59058.
PubMed 39960672
Study at a glance
From the registry record, last updated by the sponsor on 21 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- HEALEY ALS Platform Trial
- ClinicalTrials.gov ID
- NCT04297683
- Acronym / sponsor ID
- 2019P003518
- Conditions
- Amyotrophic Lateral Sclerosis
- Keywords
- ALS, Placebo-Controlled, Double-Blind, Master Protocol, Lou Gehrig's Disease
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 1,500 participants (estimated)
- Lead sponsor
- Merit E. Cudkowicz, MDUniversity, hospital or other
- Collaborators
- Massachusetts General Hospital
- Study start
- 14 Jun 2020 (actual)
- Primary completion
- Jul 2027 (estimated)
- Study completion
- Aug 2028 (estimated)
- First posted
- 5 Mar 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS.
Detailed description
The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS. This trial is designed as a perpetual platform trial. This means that there is a single Master Protocol dictating the conduct of the trial.
In this trial, multiple investigational products for ALS will be tested simultaneously or sequentially. Each investigational product will be tested in a regimen. Each regimen consists of a placebo-controlled trial, meaning that the active investigational product and matching placebo will be tested in each regimen.
The additional details that govern the testing of each investigational product will be summarized in separate regimen-specific appendices (RSAs). Each regimen will have a separate ClinicalTrials.gov posting, which will include specific information about the regimen. All regimen-specific outcome measures will be detailed in each regimen posting.
Participants will have an equal chance to be randomized to all regimens that are active at the time of screening. Once randomized to a regimen, participants will be randomized in a 3:1 ratio to either study drug or placebo.
The following regimens are active in the trial:
Regimen I - NUZ-001
New regimens will be continuously added as new investigational products become available. The HEALEY ALS Platform Trial will enroll additional participants as each new regimen is available.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 8
- Model description
- As new investigational products become available, additional regimens will be added to the HEALEY ALS Platform Trial.
| Arm | Type | Receives |
|---|---|---|
| Regimen A - Zilucoplan | Experimental | Drug: ZilucoplanParticipants are randomized to receive either active zilucoplan or matching placebo. NO LONGER RECRUITING; RESULTS REPORTED. |
| Regimen B - Verdiperstat | Experimental | Drug: VerdiperstatParticipants are randomized to receive either active verdiperstat or matching placebo. NO LONGER RECRUITING; RESULTS REPORTED. |
| Regimen C - CNM-Au8 | Experimental | Drug: CNM-Au8Participants are randomized to receive either active CNM-Au8 or matching placebo. NO LONGER RECRUITING; RESULTS REPORTED. |
| Regimen D - Pridopidine | Experimental | Drug: PridopidineParticipants are randomized to receive either active Pridopidine or matching placebo. NO LONGER RECRUITING; RESULTS REPORTED. |
| Regimen E - SLS-005 Trehalose | Experimental | Drug: SLS-005 TrehaloseParticipants are randomized to receive either active SLS-005 Trehalose or matching placebo. NO LONGER RECRUITING; RESULTS REPORTED. |
| Regimen F- ABBV-CLS-7262 | Experimental | Drug: ABBV-CLS-7262Participants are randomized to receive either active ABBV-CLS-7262 or matching placebo. NO LONGER RECRUITING; RESULTS REPORTED. |
| Regimen G - DNL343 | Experimental | Drug: DNL343Participants are randomized to receive either active DNL343 or matching placebo. NO LONGER RECRUITING; RESULTS REPORTED. |
| Regimen I - NUZ-001 | Experimental | Drug: NUZ-001Participants are randomized to receive either active NUZ-001 or matching placebo. ACTIVE, NOT RECRUITING |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Sporadic or familial ALS diagnosed as clinically possible, probable, lab-supported probable, or definite ALS defined by revised El Escorial criteria.
- Age 18 years or older.
- Capable of providing informed consent and complying with study procedures, in the SI's opinion.
- Time since onset of weakness due to ALS ≤ 24 months at the time of the Master Protocol Screening Visit.
- Vital Capacity ≥ 50% of predicted capacity at the time of the Master Protocol Screening Visit measured by Slow Vital Capacity (SVC), or, if required due to pandemic-related restrictions, Forced Vital Capacity (FVC) measured in person.
Key exclusion criteria (9 listed)
- Clinically significant unstable medical condition (other than ALS) that would pose a risk to the participant, according to SI's judgment (e.g., cardiovascular instability, systemic infection), or clinically significant laboratory abnormality or EKG changes. Clinically significant abnormal liver or kidney function is exclusionary. The following values [alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 3 times the upper limit of normal (ULN) or estimated Glomerular Filtration Rate (eGFR) < 30 mL/min/1.73m2] are exclusionary regardless of clinical symptoms.
- 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.
- 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.
- Use of investigational treatments for ALS (off-label use or active participation in a clinical trial) within 5 half-lives (if known) or 30 days (whichever is longer) prior to the Master Protocol Screening Visit.
- Exposure at any time to any gene therapies under investigation for the treatment of ALS (off-label use or investigational).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ZilucoplanDrug: Zilucoplan Administration: Subcutaneous injection Dose: Minimum of .0.22 mg/kg daily to a maximum dose of 0.42 mg/kg daily, dependent on weight | Drug | Regimen A - Zilucoplan |
| VerdiperstatDrug: Verdiperstat Administration: Oral Dose: 600mg twice daily | Drug | Regimen B - Verdiperstat |
| CNM-Au8Drug: CNM-Au8 Administration: Oral Dose: 30 mg or 60 mg daily | Drug | Regimen C - CNM-Au8 |
| PridopidineDrug: Pridopidine Administration: Oral Dose: 45mg twice daily | Drug | Regimen D - Pridopidine |
| SLS-005 TrehaloseDrug: SLS-005 Trehalose Administration: Infusion Dose: 0.75 g/kg weekly | Drug | Regimen E - SLS-005 Trehalose |
| ABBV-CLS-7262Drug: ABBV-CLS-7162 Administration: Oral Dose: Dose 1 or Dose 2 | Drug | Regimen F- ABBV-CLS-7262 |
| DNL343Drug: DNL343 Administration: Oral Dose: Once per day | Drug | Regimen G - DNL343 |
| NUZ-001Drug: NUZ-001. Administration: Oral. Dose: Once per day. | Drug | Regimen I - NUZ-001 |
Primary outcome
Disease Progression
Time frame: 36 Weeks
Change in disease severity as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) total score and survival.
2 secondary outcomes
Respiratory Function
Time frame: 36 Weeks
Survival
Time frame: 36 Weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Jul 2026.
- Derived from this study: PubMed 40067821Writing Committee for the HEALEY ALS Platform Trial; Berry JD, Maragakis NJ, Macklin EA, Chibnik LB, Quintana M, Saville BR, Detry MA, Vestrucci M, Marion J, McGlothlin A, Stommel EW, Chase M, Pothier L, Harkey BA, Yu H, Sherman A, Shefner J, Hall M, Kittle G, Babu S, Andrews J, D'Agostino D, Tustison E, Scirocco E, Giacomelli E, Alameda G, Locatelli E, Ho D, Quick A, Ajroud-Driss S, Katz J, Heitzman D, Appel SH, Shroff S, Felice KJ, Simmons Z, Miller T, Olney N, Weiss MD, Goutman SA, Fernandes JA Jr, Jawdat O, Owegi MA, Foster L, Vu T, Ilieva H, Newman DS, Arcila-Londono X, Jackson C, Ladha S, Heiman-Patterson T, Caress J, Swenson A, Peltier A, Lewis R, Fee D, Elliott M, Bedlack R, Kasarskis EJ, Elman L, Rosenfeld J, Walk D, McIlduff CE, Twydell P, Young E, Johnson K, Rezania K, Goyal NA, Cohen JA, Benatar M, Jones V, Glass J, Shah J, Beydoun SR, Wymer JP, Zilliox L, Nayar S, Pattee GL, Martinez-Thompson J, Rynders A, Evan J, Evan J, Hartford A, Sepassi M, Ho KS, Glanzman R, Greenberg B, Hotchkin MT, Paganoni S, Cudkowicz ME; HEALEY ALS Platform Trial Study Group. CNM-Au8 in Amyotrophic Lateral Sclerosis: The HEALEY ALS Platform Trial. JAMA. 2025 Feb 17;333(13):1138-49. doi: 10.1001/jama.2024.27643. Online ahead of print.
- Derived from this study: PubMed 40067755Writing Committee for the HEALEY ALS Platform Trial; Shefner JM, Oskarsson B, Macklin EA, Chibnik LB, Quintana M, Saville BR, Detry MA, Vestrucci M, Marion J, McGlothlin A, Heiman-Patterson T, Chase M, Pothier L, Harkey BA, Yu H, Sherman AV, Hall M, Kittle G, Berry JD, Babu S, Andrews J, D'Agostino D, Tustison E, Scirocco E, Giacomelli E, Alameda G, Locatelli E, Ho D, Quick A, Ajroud-Driss S, Katz J, Heitzman D, Appel SH, Shroff S, Felice K, Maragakis NJ, Simmons Z, Miller TM, Olney N, Weiss MD, Goutman SA, Fernandes JA, Jawdat O, Owegi MA, Foster LA, Vu T, Ilieva H, Newman DS, Arcila-Londono X, Jackson CE, Ladha S, Caress JB, Swenson A, Peltier A, Lewis RA, Fee D, Elliott M, Bedlack R, Kasarskis EJ, Elman L, Rosenfeld J, Walk D, McIlduff C, Twydell P, Young E, Johnson K, Rezania K, Goyal NA, Cohen JA, Benatar M, Jones V, Shah J, Beydoun SR, Wymer JP, Zilliox L, Nayar S, Pattee GL, Martinez-Thompson J, Leitner ML, Chen K, Goldberg YP, Cohen Y, Geva M, Hayden MR, Paganoni S, Cudkowicz ME; HEALEY ALS Platform Trial Study Group. Pridopidine in Amyotrophic Lateral Sclerosis: The HEALEY ALS Platform Trial. JAMA. 2025 Feb 17;333(13):1128-37. doi: 10.1001/jama.2024.26429. Online ahead of print.
- Derived from this study: PubMed 40067754Writing Committee for the HEALEY ALS Platform Trial; Andrews J, Paganoni S, Macklin EA, Chibnik LB, Quintana M, Saville BR, Detry MA, Vestrucci M, Marion J, McGlothlin A, Young E, Chase M, Pothier L, Harkey B, Yu H, Sherman A, Shefner J, Hall M, Kittle G, Connolly MR, Berry JD, D'Agostino D, Tustison E, Giacomelli E, Scirocco E, Alameda G, Locatelli E, Ho D, Quick A, Heitzman D, Ajroud-Driss S, Appel SH, Shroff S, Katz J, Felice K, Maragakis NJ, Simmons Z, Goutman SA, Olney N, Miller T, Fernandes JA, Ilieva H, Jawdat O, Weiss MD, Foster L, Vu T, Ladha S, Owegi MA, Newman DS, Arcila-Londono X, Jackson CE, Swenson A, Heiman-Patterson T, Caress J, Fee D, Peltier A, Lewis R, Rosenfeld J, Walk D, Johnson K, Elliott M, Kasarskis EJ, Rutkove S, McIlduff CE, Bedlack R, Elman L, Goyal NA, Rezania K, Twydell P, Benatar M, Glass J, Cohen JA, Jones V, Zilliox L, Wymer JP, Beydoun SR, Shah J, Pattee GL, Martinez-Thompson J, Nayar S, Granit V, Donohue M, Grossman K, Campbell DJ, Qureshi IA, Cudkowicz ME, Babu S. Verdiperstat in Amyotrophic Lateral Sclerosis: Results From the Randomized HEALEY ALS Platform Trial. JAMA Neurol. 2025 Apr;82(4):333-343. doi: 10.1001/jamaneurol.2024.5249. Epub 2025 Feb 17.
- Derived from this study: PubMed 39960672Paganoni S, Fournier CN, Macklin EA, Chibnik LB, Quintana M, Saville BR, Detry MA, Vestrucci M, Marion J, McGlothlin A, Ajroud-Driss S, Chase M, Pothier L, Harkey BA, Yu H, Sherman AV, Shefner JM, Hall M, Kittle G, Berry JD, Babu S, Andrews J, Dagostino D, Tustison E, Giacomelli E, Scirocco E, Alameda G, Locatelli E, Ho D, Quick A, Katz J, Heitzman D, Appel SH, Shroff S, Felice K, Maragakis NJ, Simmons Z, Miller TM, Olney N, Weiss MD, Goutman SA, Fernandes JA, Jawdat O, Owegi MA, Foster LA, Vu T, Ilieva H, Newman DS, Arcila-Londono X, Jackson CE, Ladha S, Heiman-Patterson T, Caress JB, Swenson A, Peltier A, Lewis R, Fee D, Elliott M, Bedlack R, Kasarskis EJ, Elman L, Rosenfeld J, Walk D, McIlduff C, Twydell P, Young E, Johnson K, Rezania K, Goyal NA, Cohen JA, Benatar M, Jones V, Glass J, Shah J, Beydoun SR, Wymer JP, Zilliox L, Nayar S, Pattee GL, Martinez-Thompson J, Harvey B, Patel S, Mahoney P, Duda PW, Cudkowicz ME; HEALEY ALS Platform Trial Study Group. Efficacy and Safety of Zilucoplan in Amyotrophic Lateral Sclerosis: A Randomized Clinical Trial. JAMA Netw Open. 2025 Feb 3;8(2):e2459058. doi: 10.1001/jamanetworkopen.2024.59058.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Merit Cudkowicz, MD principal investigator
Massachusetts General Hospital
81 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Zilucoplan, Verdiperstat and CNM-Au8 in people with Amyotrophic Lateral Sclerosis, run by Merit E. Cudkowicz, MD at 81 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 21 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 81 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Disease Progression (time frame: 36 Weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04297683, which also lists 4 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Jul 2026.