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HEALEY ALS Platform Trial - Master Protocol

ConditionAmyotrophic Lateral Sclerosis
Trial statusRecruiting
Trial phasePhase 2, Phase 3
Trial typeInterventional
Biological sexAll
Age18+
SponsorMerit E. Cudkowicz, MD

About this 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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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.

Disqualifiers

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.

Trial design

Design model

Parallel

Treatments tested in this trial

  • Zilucoplan

    Drug

    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

  • Verdiperstat

    Drug

    Drug: Verdiperstat Administration: Oral Dose: 600mg twice daily

  • CNM-Au8

    Drug

    Drug: CNM-Au8 Administration: Oral Dose: 30 mg or 60 mg daily

  • Pridopidine

    Drug

    Drug: Pridopidine Administration: Oral Dose: 45mg twice daily

  • SLS-005 Trehalose

    Drug

    Drug: SLS-005 Trehalose Administration: Infusion Dose: 0.75 g/kg weekly

  • ABBV-CLS-7262

    Drug

    Drug: ABBV-CLS-7162 Administration: Oral Dose: Dose 1 or Dose 2

  • DNL343

    Drug

    Drug: DNL343 Administration: Oral Dose: Once per day

  • NUZ-001

    Drug

    Drug: NUZ-001. Administration: Oral. Dose: Once per day.

Treatment groups

1,500 Participants
are divided into 8 treatment groups

8

Treatment groups

See each treatment group below.

Group A: Regimen A - ZilucoplanExperimental treatment 1 intervention
Group B: Regimen B - VerdiperstatExperimental treatment 1 intervention
Group C: Regimen C - CNM-Au8Experimental treatment 1 intervention
Group D: Regimen D - PridopidineExperimental treatment 1 intervention
Group E: Regimen E - SLS-005 TrehaloseExperimental treatment 1 intervention
Group F: Regimen F- ABBV-CLS-7262Experimental treatment 1 intervention
Group G: Regimen G - DNL343Experimental treatment 1 intervention
Group H: Regimen I - NUZ-001Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Disease Progression

Change in disease severity as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) total score and survival.

Time frame
36 Weeks

Secondary outcomes

1

Respiratory Function

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

Time frame
36 Weeks
2

Survival

Comparison of rate of occurrence between groups.

Time frame
36 Weeks

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Merit E. Cudkowicz, MD

Lead sponsor

Massachusetts General Hospital

Sponsor institution

Massachusetts General Hospital

Collaborator

Source ClinicalTrials.gov
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