Trial Outcomes & Findings for HEALEY ALS Platform Trial - Regimen E SLS-005 - Trehalose (NCT NCT05136885)

Last Updated: 2025-05-14

Results Overview

Change in disease severity over time as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R). Each type of function is scored from 4 (normal) to 0 (no ability), with a maximum total score of 48 and a minimum total score of 0. Patients with higher scores have more physical function.

Recruitment status

COMPLETED

Study phase

PHASE2/PHASE3

Target enrollment

161 participants

Primary outcome timeframe

24 Weeks

Results posted on

2025-05-14

Participant Flow

Participant milestones

Participant milestones
Measure	SLS-005 SLS-005 is administered via infusion once weekly for 24 weeks. SLS-005: Administration: Infusion Dose: 0.75 g/kg weekly	Matching Placebo Matching placebo is administered via infusion once weekly for 24 weeks. Matching Placebo: Administration: Infusion Dose: equivalent weight-based volume as described for trehalose
Overall Study STARTED	120	41
Overall Study COMPLETED	92	37
Overall Study NOT COMPLETED	28	4

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Number analyzed in row differs from overall number due to missing data.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	SLS-005 n=120 Participants SLS-005 is administered via infusion once weekly for 24 weeks. SLS-005: Administration: Infusion Dose: 0.75 g/kg weekly	Matching Placebo n=41 Participants Matching placebo is administered via infusion once weekly for 24 weeks. Matching Placebo: Administration: Infusion Dose: equivalent weight-based volume as described for trehalose	Total n=161 Participants Total of all reporting groups
Age, Continuous	59.3 years STANDARD_DEVIATION 10.73 • n=120 Participants	61.3 years STANDARD_DEVIATION 10.19 • n=41 Participants	59.8 years STANDARD_DEVIATION 10.60 • n=161 Participants
Sex: Female, Male Female	55 Participants n=120 Participants	24 Participants n=41 Participants	79 Participants n=161 Participants
Sex: Female, Male Male	65 Participants n=120 Participants	17 Participants n=41 Participants	82 Participants n=161 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	2 Participants n=120 Participants	1 Participants n=41 Participants	3 Participants n=161 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	115 Participants n=120 Participants	40 Participants n=41 Participants	155 Participants n=161 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	3 Participants n=120 Participants	0 Participants n=41 Participants	3 Participants n=161 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=120 Participants	0 Participants n=41 Participants	0 Participants n=161 Participants
Race (NIH/OMB) Asian	5 Participants n=120 Participants	1 Participants n=41 Participants	6 Participants n=161 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=120 Participants	0 Participants n=41 Participants	0 Participants n=161 Participants
Race (NIH/OMB) Black or African American	2 Participants n=120 Participants	1 Participants n=41 Participants	3 Participants n=161 Participants
Race (NIH/OMB) White	109 Participants n=120 Participants	39 Participants n=41 Participants	148 Participants n=161 Participants
Race (NIH/OMB) More than one race	1 Participants n=120 Participants	0 Participants n=41 Participants	1 Participants n=161 Participants
Race (NIH/OMB) Unknown or Not Reported	3 Participants n=120 Participants	0 Participants n=41 Participants	3 Participants n=161 Participants
ALS Diagnosis from R El Escorial Criteria Clinically Definite ALS	44 Participants n=120 Participants	17 Participants n=41 Participants	61 Participants n=161 Participants
ALS Diagnosis from R El Escorial Criteria Clinically Possible ALS	8 Participants n=120 Participants	3 Participants n=41 Participants	11 Participants n=161 Participants
ALS Diagnosis from R El Escorial Criteria Clinically Probable ALS	40 Participants n=120 Participants	13 Participants n=41 Participants	53 Participants n=161 Participants
ALS Diagnosis from R El Escorial Criteria Clinically Probable ALS - Laboratory Supported	28 Participants n=120 Participants	8 Participants n=41 Participants	36 Participants n=161 Participants
ALS Onset Location Bulbar	25 Participants n=120 Participants	11 Participants n=41 Participants	36 Participants n=161 Participants
ALS Onset Location Limb	93 Participants n=120 Participants	27 Participants n=41 Participants	120 Participants n=161 Participants
ALS Onset Location Multiple	2 Participants n=120 Participants	1 Participants n=41 Participants	3 Participants n=161 Participants
ALS Onset Location Respiratory	0 Participants n=120 Participants	2 Participants n=41 Participants	2 Participants n=161 Participants
Time Since Symptom Onset at Baseline	19.6 Months STANDARD_DEVIATION 8.61 • n=120 Participants	19.9 Months STANDARD_DEVIATION 8.20 • n=41 Participants	19.7 Months STANDARD_DEVIATION 8.48 • n=161 Participants
Delay in ALS Symptom Onset and Diagnosis	10.7 Months STANDARD_DEVIATION 6.86 • n=120 Participants	10.1 Months STANDARD_DEVIATION 4.82 • n=41 Participants	10.5 Months STANDARD_DEVIATION 6.39 • n=161 Participants
Baseline Edaravone Use No	84 Participants n=120 Participants	28 Participants n=41 Participants	112 Participants n=161 Participants
Baseline Edaravone Use Yes	36 Participants n=120 Participants	13 Participants n=41 Participants	49 Participants n=161 Participants
Baseline Riluzole Use No	33 Participants n=120 Participants	11 Participants n=41 Participants	44 Participants n=161 Participants
Baseline Riluzole Use Yes	87 Participants n=120 Participants	30 Participants n=41 Participants	117 Participants n=161 Participants
ALSFRS-R Total Score	35.8 Points STANDARD_DEVIATION 6.43 • n=120 Participants	35.0 Points STANDARD_DEVIATION 6.71 • n=41 Participants	35.6 Points STANDARD_DEVIATION 6.49 • n=161 Participants
Baseline Decline in ALSFRS-R	0.76 points per month STANDARD_DEVIATION 0.608 • n=120 Participants	0.76 points per month STANDARD_DEVIATION 0.504 • n=41 Participants	0.76 points per month STANDARD_DEVIATION 0.582 • n=161 Participants
SVC	74.5 percent predicted STANDARD_DEVIATION 18.81 • n=119 Participants • Number analyzed in row differs from overall number due to missing data.	77.9 percent predicted STANDARD_DEVIATION 16.54 • n=41 Participants • Number analyzed in row differs from overall number due to missing data.	75.4 percent predicted STANDARD_DEVIATION 18.26 • n=160 Participants • Number analyzed in row differs from overall number due to missing data.
King Stage 1 Region with Neuromuscular Dysfunction	20 Participants n=120 Participants	8 Participants n=41 Participants	28 Participants n=161 Participants
King Stage 2 Region with Neuromuscular Dysfunction	38 Participants n=120 Participants	13 Participants n=41 Participants	51 Participants n=161 Participants
King Stage 3 Region with Neuromuscular Dysfunction	36 Participants n=120 Participants	5 Participants n=41 Participants	41 Participants n=161 Participants
King Stage 4a/b Nutritional/Respiratory Failure	26 Participants n=120 Participants	15 Participants n=41 Participants	41 Participants n=161 Participants
Weight	79.3 kg STANDARD_DEVIATION 21.44 • n=120 Participants	76.8 kg STANDARD_DEVIATION 17.65 • n=41 Participants	78.6 kg STANDARD_DEVIATION 20.52 • n=161 Participants
Body Mass Index	26.7 kg/cm^2 STANDARD_DEVIATION 6.47 • n=120 Participants	26.8 kg/cm^2 STANDARD_DEVIATION 5.15 • n=41 Participants	26.7 kg/cm^2 STANDARD_DEVIATION 6.14 • n=161 Participants
Serum Creatinine Concentration	0.7 mg/dL STANDARD_DEVIATION 0.19 • n=120 Participants	0.7 mg/dL STANDARD_DEVIATION 0.16 • n=41 Participants	0.7 mg/dL STANDARD_DEVIATION 0.18 • n=161 Participants
Serum NfL Concentration	76.9 ng/L STANDARD_DEVIATION 51.08 • n=116 Participants • Number analyzed in row differs from overall number due to missing data.	76.5 ng/L STANDARD_DEVIATION 38.45 • n=39 Participants • Number analyzed in row differs from overall number due to missing data.	76.8 ng/L STANDARD_DEVIATION 48.09 • n=155 Participants • Number analyzed in row differs from overall number due to missing data.

PRIMARY outcome

Timeframe: 24 Weeks

Population: Outcome measure data was analyzed using the Full Analysis Set, which included shared placebo from other regimens, as pre-specified in the Regimen Statistical Analysis Plan. Regimen A (NCT04436497), Regimen B (NCT04436510), Regimen C (NCT04414345) and Regimen D (NCT04615923) contributed placebo participants into the shared placebo cohort used for analysis.

Outcome measures

Outcome measures
Measure	SLS-005 n=120 Participants SLS-005 is administered via infusion once weekly for 24 weeks. SLS-005: Administration: Infusion Dose: 0.75 g/kg weekly	Matching Placebo n=204 Participants Matching placebo is administered via infusion once weekly for 24 weeks. Matching Placebo: Administration: Infusion Dose: equivalent weight-based volume as described for trehalose
Disease Progression as Assessed by the ALSFRS-R Slope	-0.91 points per month Standard Deviation 0.090	-1.06 points per month Standard Deviation 0.091

PRIMARY outcome

Timeframe: Baseline to 24 Weeks

Mortality is defined as death or death equivalent. A participant is determined to meet the criteria of death equivalent if permanent assisted ventilation (PAV) is used for more than 22 hours per day for more than seven days in a row. The rate of mortality was estimated from a Bayesian shared-parametric model that assumed exponentially distributed survival times.

Outcome measures

Outcome measures
Measure	SLS-005 n=120 Participants SLS-005 is administered via infusion once weekly for 24 weeks. SLS-005: Administration: Infusion Dose: 0.75 g/kg weekly	Matching Placebo n=204 Participants Matching placebo is administered via infusion once weekly for 24 weeks. Matching Placebo: Administration: Infusion Dose: equivalent weight-based volume as described for trehalose
Mortality Event Rate	0.010 events per month Standard Deviation 0.0025	0.012 events per month Standard Deviation 0.0027

SECONDARY outcome

Timeframe: Baseline to 24 Weeks

Change in respiratory function over time as measured by Slow Vital Capacity (SVC).

Outcome measures

Outcome measures
Measure	SLS-005 n=119 Participants SLS-005 is administered via infusion once weekly for 24 weeks. SLS-005: Administration: Infusion Dose: 0.75 g/kg weekly	Matching Placebo n=201 Participants Matching placebo is administered via infusion once weekly for 24 weeks. Matching Placebo: Administration: Infusion Dose: equivalent weight-based volume as described for trehalose
Respiratory Function	-11.40 percent change Standard Error 1.431	-10.85 percent change Standard Error 1.089

SECONDARY outcome

Timeframe: Baseline to 24 Weeks

Change in muscle strength over time as measured isometrically using hand-held dynamometry (HHD).

Outcome measures

Outcome measures
Measure	SLS-005 n=120 Participants SLS-005 is administered via infusion once weekly for 24 weeks. SLS-005: Administration: Infusion Dose: 0.75 g/kg weekly	Matching Placebo n=203 Participants Matching placebo is administered via infusion once weekly for 24 weeks. Matching Placebo: Administration: Infusion Dose: equivalent weight-based volume as described for trehalose
Muscle Strength	-26.19 percent change Standard Error 2.568	-27.11 percent change Standard Error 1.876

SECONDARY outcome

Timeframe: Baseline to 24 Weeks

The number of participants who died or met the criterion for a death equivalent from the date of their baseline visit to the end of the Week 24 visit window (generally 175 days after baseline). The death equivalent criterion is the use of permanent assisted ventilation (PAV) for more than 22 hours per day for more than 7 days in a row.

Outcome measures

Outcome measures
Measure	SLS-005 n=120 Participants SLS-005 is administered via infusion once weekly for 24 weeks. SLS-005: Administration: Infusion Dose: 0.75 g/kg weekly	Matching Placebo n=203 Participants Matching placebo is administered via infusion once weekly for 24 weeks. Matching Placebo: Administration: Infusion Dose: equivalent weight-based volume as described for trehalose
Number of Participants That Experienced Death or Death Equivalent	10 Participants	10 Participants

Adverse Events

SLS-005

Serious events: 19 serious events

Other events: 107 other events

Deaths: 6 deaths

Matching Placebo

Serious events: 3 serious events

Other events: 33 other events

Deaths: 0 deaths

Serious adverse events

Other adverse events

Additional Information

Healey Center for ALS Project Management

Healey Center for ALS at Massachusetts General Hospital

Phone: 833-425-8257

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Measure	SLS-005 n=120 participants at risk SLS-005 is administered via infusion once weekly for 24 weeks. SLS-005: Administration: Infusion Dose: 0.75 g/kg weekly	Matching Placebo n=41 participants at risk Matching placebo is administered via infusion once weekly for 24 weeks. Matching Placebo: Administration: Infusion Dose: equivalent weight-based volume as described for trehalose
Respiratory, thoracic and mediastinal disorders Acute respiratory failure	2.5% 3/120 • Number of events 3 • Up to 35 weeks after participant signed Master Protocol consent.	0.00% 0/41 • Up to 35 weeks after participant signed Master Protocol consent.
Respiratory, thoracic and mediastinal disorders Pneumonia aspiration	0.83% 1/120 • Number of events 2 • Up to 35 weeks after participant signed Master Protocol consent.	2.4% 1/41 • Number of events 1 • Up to 35 weeks after participant signed Master Protocol consent.
Respiratory, thoracic and mediastinal disorders Respiratory failure	1.7% 2/120 • Number of events 2 • Up to 35 weeks after participant signed Master Protocol consent.	0.00% 0/41 • Up to 35 weeks after participant signed Master Protocol consent.
Respiratory, thoracic and mediastinal disorders Pulmonary embolism	0.83% 1/120 • Number of events 1 • Up to 35 weeks after participant signed Master Protocol consent.	0.00% 0/41 • Up to 35 weeks after participant signed Master Protocol consent.
Nervous system disorders Amyotrophic lateral sclerosis	2.5% 3/120 • Number of events 3 • Up to 35 weeks after participant signed Master Protocol consent.	0.00% 0/41 • Up to 35 weeks after participant signed Master Protocol consent.
Nervous system disorders Cerebrovascular accident	0.83% 1/120 • Number of events 1 • Up to 35 weeks after participant signed Master Protocol consent.	0.00% 0/41 • Up to 35 weeks after participant signed Master Protocol consent.
Nervous system disorders Syncope	0.83% 1/120 • Number of events 1 • Up to 35 weeks after participant signed Master Protocol consent.	0.00% 0/41 • Up to 35 weeks after participant signed Master Protocol consent.
Infections and infestations COVID-19	1.7% 2/120 • Number of events 2 • Up to 35 weeks after participant signed Master Protocol consent.	0.00% 0/41 • Up to 35 weeks after participant signed Master Protocol consent.
Infections and infestations Pneumonia	1.7% 2/120 • Number of events 2 • Up to 35 weeks after participant signed Master Protocol consent.	0.00% 0/41 • Up to 35 weeks after participant signed Master Protocol consent.
Injury, poisoning and procedural complications Hip fracture	0.83% 1/120 • Number of events 1 • Up to 35 weeks after participant signed Master Protocol consent.	2.4% 1/41 • Number of events 1 • Up to 35 weeks after participant signed Master Protocol consent.
Injury, poisoning and procedural complications Subdural haemorrhage	0.00% 0/120 • Up to 35 weeks after participant signed Master Protocol consent.	2.4% 1/41 • Number of events 1 • Up to 35 weeks after participant signed Master Protocol consent.
Gastrointestinal disorders Abdominal pain upper	0.83% 1/120 • Number of events 1 • Up to 35 weeks after participant signed Master Protocol consent.	0.00% 0/41 • Up to 35 weeks after participant signed Master Protocol consent.
Gastrointestinal disorders Colitis ischaemic	0.00% 0/120 • Up to 35 weeks after participant signed Master Protocol consent.	2.4% 1/41 • Number of events 1 • Up to 35 weeks after participant signed Master Protocol consent.
General disorders Complication associated with device	0.83% 1/120 • Number of events 1 • Up to 35 weeks after participant signed Master Protocol consent.	0.00% 0/41 • Up to 35 weeks after participant signed Master Protocol consent.
Immune system disorders Urticaria	0.83% 1/120 • Number of events 1 • Up to 35 weeks after participant signed Master Protocol consent.	0.00% 0/41 • Up to 35 weeks after participant signed Master Protocol consent.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Breast cancer	0.83% 1/120 • Number of events 1 • Up to 35 weeks after participant signed Master Protocol consent.	0.00% 0/41 • Up to 35 weeks after participant signed Master Protocol consent.
Vascular disorders Hypertension	0.83% 1/120 • Number of events 1 • Up to 35 weeks after participant signed Master Protocol consent.	0.00% 0/41 • Up to 35 weeks after participant signed Master Protocol consent.

Measure	SLS-005 n=120 participants at risk SLS-005 is administered via infusion once weekly for 24 weeks. SLS-005: Administration: Infusion Dose: 0.75 g/kg weekly	Matching Placebo n=41 participants at risk Matching placebo is administered via infusion once weekly for 24 weeks. Matching Placebo: Administration: Infusion Dose: equivalent weight-based volume as described for trehalose
Nervous system disorders Muscular weakness	18.3% 22/120 • Number of events 30 • Up to 35 weeks after participant signed Master Protocol consent.	19.5% 8/41 • Number of events 14 • Up to 35 weeks after participant signed Master Protocol consent.
Nervous system disorders Neuromyopathy	15.0% 18/120 • Number of events 22 • Up to 35 weeks after participant signed Master Protocol consent.	9.8% 4/41 • Number of events 5 • Up to 35 weeks after participant signed Master Protocol consent.
Nervous system disorders Headache	9.2% 11/120 • Number of events 15 • Up to 35 weeks after participant signed Master Protocol consent.	0.00% 0/41 • Up to 35 weeks after participant signed Master Protocol consent.
Nervous system disorders Dysarthria	3.3% 4/120 • Number of events 4 • Up to 35 weeks after participant signed Master Protocol consent.	9.8% 4/41 • Number of events 4 • Up to 35 weeks after participant signed Master Protocol consent.
Injury, poisoning and procedural complications Fall	26.7% 32/120 • Number of events 63 • Up to 35 weeks after participant signed Master Protocol consent.	19.5% 8/41 • Number of events 17 • Up to 35 weeks after participant signed Master Protocol consent.
Injury, poisoning and procedural complications Infusion site bruising	5.8% 7/120 • Number of events 12 • Up to 35 weeks after participant signed Master Protocol consent.	4.9% 2/41 • Number of events 2 • Up to 35 weeks after participant signed Master Protocol consent.
Injury, poisoning and procedural complications Post lumbar puncture syndrome	6.7% 8/120 • Number of events 10 • Up to 35 weeks after participant signed Master Protocol consent.	0.00% 0/41 • Up to 35 weeks after participant signed Master Protocol consent.
General disorders Fatigue	15.8% 19/120 • Number of events 20 • Up to 35 weeks after participant signed Master Protocol consent.	14.6% 6/41 • Number of events 7 • Up to 35 weeks after participant signed Master Protocol consent.
General disorders Infusion related reaction	5.0% 6/120 • Number of events 33 • Up to 35 weeks after participant signed Master Protocol consent.	4.9% 2/41 • Number of events 30 • Up to 35 weeks after participant signed Master Protocol consent.
General disorders Oedema peripheral	5.0% 6/120 • Number of events 7 • Up to 35 weeks after participant signed Master Protocol consent.	4.9% 2/41 • Number of events 4 • Up to 35 weeks after participant signed Master Protocol consent.
Gastrointestinal disorders Constipation	12.5% 15/120 • Number of events 16 • Up to 35 weeks after participant signed Master Protocol consent.	4.9% 2/41 • Number of events 3 • Up to 35 weeks after participant signed Master Protocol consent.
Gastrointestinal disorders Diarrhoea	10.8% 13/120 • Number of events 15 • Up to 35 weeks after participant signed Master Protocol consent.	9.8% 4/41 • Number of events 4 • Up to 35 weeks after participant signed Master Protocol consent.
Gastrointestinal disorders Dysphagia	7.5% 9/120 • Number of events 10 • Up to 35 weeks after participant signed Master Protocol consent.	4.9% 2/41 • Number of events 2 • Up to 35 weeks after participant signed Master Protocol consent.
Gastrointestinal disorders Salivary hypersecretion	3.3% 4/120 • Number of events 4 • Up to 35 weeks after participant signed Master Protocol consent.	9.8% 4/41 • Number of events 5 • Up to 35 weeks after participant signed Master Protocol consent.
Gastrointestinal disorders Abdominal discomfort	5.0% 6/120 • Number of events 6 • Up to 35 weeks after participant signed Master Protocol consent.	0.00% 0/41 • Up to 35 weeks after participant signed Master Protocol consent.
Infections and infestations COVID-19	10.0% 12/120 • Number of events 12 • Up to 35 weeks after participant signed Master Protocol consent.	7.3% 3/41 • Number of events 3 • Up to 35 weeks after participant signed Master Protocol consent.
Infections and infestations Urinary tract infection	5.8% 7/120 • Number of events 10 • Up to 35 weeks after participant signed Master Protocol consent.	12.2% 5/41 • Number of events 7 • Up to 35 weeks after participant signed Master Protocol consent.
Respiratory, thoracic and mediastinal disorders Cough	6.7% 8/120 • Number of events 9 • Up to 35 weeks after participant signed Master Protocol consent.	4.9% 2/41 • Number of events 3 • Up to 35 weeks after participant signed Master Protocol consent.
Respiratory, thoracic and mediastinal disorders Dyspnoea	5.0% 6/120 • Number of events 6 • Up to 35 weeks after participant signed Master Protocol consent.	7.3% 3/41 • Number of events 3 • Up to 35 weeks after participant signed Master Protocol consent.
Musculoskeletal and connective tissue disorders Musculoskeletal pain	6.7% 8/120 • Number of events 8 • Up to 35 weeks after participant signed Master Protocol consent.	4.9% 2/41 • Number of events 2 • Up to 35 weeks after participant signed Master Protocol consent.