HEALEY ALS Platform Trial - Master Protocol

  • STATUS
    Recruiting
  • End date
    Nov 4, 2022
  • participants needed
    480
  • sponsor
    Merit E. Cudkowicz, MD
Updated on 4 November 2020
weakness
riluzole
edaravone

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.

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 A - Zilucoplan Regimen B - Verdiperstat Regimen C - CNM-Au8

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.

Details
Treatment CNM-Au8, Verdiperstat, zilucoplan
Clinical Study IdentifierNCT04297683
SponsorMerit E. Cudkowicz, MD
Last Modified on4 November 2020

Adding a note
adding personal notes guide

Select a piece of text and start making personal notes.

Eligibility

Yes No Not Sure

Inclusion Criteria

Is your age greater than or equal to 18 yrs?
Gender: Male or Female
Do you have any of these conditions: Amyotrophic Lateral Sclerosis or Myelopathy or Amyotrophic Lateral Sclerosis (ALS) or Spinal Cord Disorders?
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 36 months at the time of the Master Protocol Screening Visit
Vital Capacity 50% of predicted capacity for age, height, and sex 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)
Participants must either not take riluzole or be on a stable dose of riluzole for 30 days prior to the Master Protocol Screening Visit. Riluzole-nave participants are permitted in the study
Participants must either not take edaravone or have completed at least one cycle of edaravone prior to the Master Protocol Screening Visit. Edaravone-nave participants are permitted in the study
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

Exclusion Criteria

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, untreated thyroid dysfunction, or clinically significant laboratory abnormality or EKG changes)
Lab abnormalities include, but are not limited to: Hemoglobin < 10 g/dL, White
Blood Cells < 3.0 x 103/mm3, Neutrophils, Absolute 1000/mm3, Eosinophilia
(absolute eosinophil count of 500 eosinophils per microliter), low platelet
counts (< 150 x 109 per liter), alanine aminotransferase (ALT) or aspartate
aminotransferase (AST) greater than 3 times the upper limit of normal (ULN)
eGFR < 30 mL/min/1.73m2, thyroid-stimulating hormone (TSH) levels >10 mIU/L or
<0.01 mIU/L
\. 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
longer as specified in each RSA, after discontinuing study treatment
\. If male of reproductive capacity, unwilling to use effective contraception
for the duration of the trial and for 3 months, or longer 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
\. For those participating in the optional CSF collection, contraindication
to undergoing a lumbar puncture (LP) in the SI's opinion. Participants
undergoing the LP must not be currently taking anticoagulation medications
such as warfarin that would be a contraindication to LP; aspirin and non-
steroidal anti-inflammatories are allowed
Clear my responses

How to participate?

Step 1 Connect with a site
What happens next?
  • You can expect the study team to contact you via email or phone in the next few days.
  • Sign up as volunteer to help accelerate the development of new treatments and to get notified about similar trials.

You are contacting

Investigator Avatar
Name

Primary Contact

site
Name

Phone Email

0/250
Please verify that you are not a bot.
Step 2 Get screened

Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

Learn more
Step 3 Enroll in the clinical study

If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

Learn more
Step 4 Get your study results

Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

Learn more

Similar trials to consider

Loading...

Browse trials for

Not finding what you're looking for?

Every year hundreds of thousands of volunteers step forward to participate in research. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.

Sign up as volunteer

Study

user name

Annotated by • 

 • 

Private

Reply by • Private
Loading...

Lorem ipsum dolor sit amet consectetur, adipisicing elit. Ipsa vel nobis alias. Quae eveniet velit voluptate quo doloribus maxime et dicta in sequi, corporis quod. Ea, dolor eius? Dolore, vel!

  The passcode will expire in None.
Loading...

No made yet