Amyotrophic Lateral SclerosisPhase 2/3NCT04436497
HEALEY ALS Platform Trial - Regimen A Zilucoplan
A Phase 2/3 study testing Zilucoplan in people with Amyotrophic Lateral Sclerosis, run by Merit E. Cudkowicz, MD at 1 site 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. Regimen A will evaluate the safety and efficacy of a single study drug, zilucoplan, in participants with 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
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 162 participants
- Where
- 1 siteUnited States
- Study dates
- 29 Jul 2020to 4 May 2022
- Phase
- Phase 2/3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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
- The following inclusion criterion is in addition to the inclusion criteria specified in the Master Protocol (NCT NCT04297683).
- Vaccination with a quadrivalent meningococcal vaccine and meningococcal serotype B vaccine at least 14 days prior to the first dose of study drug at the Baseline (Day 0) visit. Meningococcal vaccines (including boosters) should be administered in accordance with the study's vaccination worksheet.
Who cannot join
- The following exclusion criteria are in addition to the exclusion criteria specified in the Master Protocol (NCT NCT04297683).
- History of meningococcal disease.
- Prior treatment with a complement inhibitor.
Trial locations and contacts
1 site in 1 country.
United States1 site
Healey Center for ALS at Mass General
Boston, Massachusetts, 02114
About this study
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.
Regimen A will evaluate the safety and efficacy of a single study drug, zilucoplan, in participants with 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. The HEALEY ALS Platform Trial Master Protocol is registered as NCT04297683.
Once a participant enrolls into the Master Protocol and meets all eligibility criteria, the participant will be eligible to be randomized into any currently enrolling regimen. All participants will have an equal chance of being randomized to any currently enrolling regimen.
If a participant is randomized to Regimen A - Zilucoplan, the participant will complete a screening visit to assess additional Regimen A eligibility criteria. Once Regimen A eligibility criteria are confirmed, participants will complete a baseline assessment and be randomized in a 3:1 ratio to either active zilucoplan or matching placebo.
Regimen A will enroll by invitation, as participants may not choose to enroll in Regimen A. Participants must first enroll into the Master Protocol and be eligible to participate in the Master Protocol before being able to be randomly assigned to Regimen A.
For a list of enrolling sites, please see the HEALEY ALS Platform Trial Master Protocol under NCT04297683.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- Zilucoplan, Matching Placebo
- Keywords
- ALS, Placebo-Controlled, Double-Blind, Regimen-Specific Appendix, Lou Gehrig's Disease, Zilucoplan, Ra Pharmaceuticals
- 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
Dosage: 0.3mg/kg administered daily
Matching PlaceboDrug
Drug: Matching Placebo
Administration: Subcutaneous injection
Dosage: Daily subcutaneous injection
| Study group | Role | What they receive |
|---|---|---|
| Zilucoplan | Experimental | Drug: Zilucoplan Administration: Subcutaneous injection
Dosage: 0.3mg/kg administered daily Receives: Drug: Zilucoplan |
| Matching Placebo | Placebo comparator | Administration: Subcutaneous injection
Dosage: Daily subcutaneous injection Receives: Drug: Matching Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Disease Progression as Assessed by the ALSFRS-R Total Score
Change in disease severity as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) total score using a Bayesian repeated measures model that accounts for loss to follow-up due to mortality. Each of 12 questions assessing distinct functional ability 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.
Measured: Baseline through 24 Weeks
Mortality Event Rate
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.
Measured: Baseline through 24 Weeks
Secondary outcomes (3)
Respiratory Function
Change in respiratory function as assessed by slow vital capacity (SVC).
Measured: Baseline and 24 Weeks
Muscle Strength
Change in muscle strength as measured isometrically using hand-held dynamometry (HHD).
Measured: Baseline and 24 Weeks
Number of Participants That Experienced Death or Death Equivalent
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 use of permanent assisted ventilation (PAV) for more than 22 hours per day for more than 7 days in a row.
Measured: Baseline through 24 Weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Jun 2020
- Study start29 Jul 2020
- Primary completion13 Apr 2022
- Study completion4 May 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- 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
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 162 participants (actual)
Sponsor and investigators
- Lead sponsor
- Merit E. Cudkowicz, MD
- Sponsor type
- University, hospital or other
- Collaborators
- Ra Pharmaceuticals
- Responsible party
- Sponsor-investigator, Merit E. Cudkowicz, MD, Massachusetts General Hospital
- Investigators
- Merit Cudkowicz, Principal investigator, Massachusetts General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04436497
- Sponsor's protocol ID
- 2019P003518A
- Status last verified
- Jun 2023
- First submitted
- 1 Jun 2020
- Last updated
- 25 Jul 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Jul 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- HEALEY ALS Platform Trial - Regimen A Zilucoplan
- ClinicalTrials.gov ID
- NCT04436497
- Acronym / sponsor ID
- 2019P003518A
- Conditions
- Amyotrophic Lateral Sclerosis
- Keywords
- ALS, Placebo-Controlled, Double-Blind, Regimen-Specific Appendix, Lou Gehrig's Disease, Zilucoplan, Ra Pharmaceuticals
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 162 participants (actual)
- Lead sponsor
- Merit E. Cudkowicz, MDUniversity, hospital or other
- Collaborators
- Ra Pharmaceuticals
- Study start
- 29 Jul 2020 (actual)
- Primary completion
- 13 Apr 2022 (actual)
- Study completion
- 4 May 2022 (actual)
- First posted
- 18 Jun 2020 (actual)
- Results
- Posted 25 Jul 2023
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.
Regimen A will evaluate the safety and efficacy of a single study drug, zilucoplan, in participants with 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. The HEALEY ALS Platform Trial Master Protocol is registered as NCT04297683.
Once a participant enrolls into the Master Protocol and meets all eligibility criteria, the participant will be eligible to be randomized into any currently enrolling regimen. All participants will have an equal chance of being randomized to any currently enrolling regimen.
If a participant is randomized to Regimen A - Zilucoplan, the participant will complete a screening visit to assess additional Regimen A eligibility criteria. Once Regimen A eligibility criteria are confirmed, participants will complete a baseline assessment and be randomized in a 3:1 ratio to either active zilucoplan or matching placebo.
Regimen A will enroll by invitation, as participants may not choose to enroll in Regimen A. Participants must first enroll into the Master Protocol and be eligible to participate in the Master Protocol before being able to be randomly assigned to Regimen A.
For a list of enrolling sites, please see the HEALEY ALS Platform Trial Master Protocol under NCT04297683.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Zilucoplan | Experimental | Drug: ZilucoplanDrug: Zilucoplan Administration: Subcutaneous injection Dosage: 0.3mg/kg administered daily |
| Matching Placebo | Placebo comparator | Drug: Matching PlaceboAdministration: Subcutaneous injection Dosage: Daily subcutaneous injection |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- The following inclusion criterion is in addition to the inclusion criteria specified in the Master Protocol (NCT NCT04297683).
Key exclusion criteria (3 listed)
- The following exclusion criteria are in addition to the exclusion criteria specified in the Master Protocol (NCT NCT04297683).
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 Dosage: 0.3mg/kg administered daily | Drug | Zilucoplan |
| Matching PlaceboDrug: Matching Placebo Administration: Subcutaneous injection Dosage: Daily subcutaneous injection | Drug | Matching Placebo |
Primary outcome
Disease Progression as Assessed by the ALSFRS-R Total Score
Time frame: Baseline through 24 Weeks
Change in disease severity as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) total score using a Bayesian repeated measures model that accounts for loss to follow-up due to mortality. Each of 12 questions assessing distinct functional ability 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.
Mortality Event Rate
Time frame: Baseline through 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.
3 secondary outcomes
Respiratory Function
Time frame: Baseline and 24 Weeks
Muscle Strength
Time frame: Baseline and 24 Weeks
Number of Participants That Experienced Death or Death Equivalent
Time frame: Baseline through 24 Weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Jul 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- 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 principal investigator
Massachusetts General Hospital
1 site 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 in people with Amyotrophic Lateral Sclerosis, run by Merit E. Cudkowicz, MD at 1 site 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 2 inclusion and 3 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: finished, as of the sponsor's last update on 25 Jul 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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. Some participants in this study may receive a placebo. 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?
Yes. Results are posted in the registry (first posted 25 Jul 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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 as Assessed by the ALSFRS-R Total Score (time frame: Baseline through 24 Weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 25 Jul 2023.