Your session is about to expire
← Back to Search
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Subjects with rapidly progressing disease ("fast" progressors), defined as average ALS Functional Rating Scale - Revised decline ≥1.0 per month calculated from score at onset of symptoms compared to score at Screening ALSFRS-R.
Subjects diagnosed with mutant SOD1-mediated ALS experiencing signs and/or symptoms of lower motor neuron dysfunction, with or without upper motor neuron symptoms.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 5 years
Awards & highlights
Summary
"This trial is testing a new gene therapy called AMT-162 in people with SOD1-ALS to see if it is safe, well-tolerated, and potentially effective. It involves giving
Who is the study for?
This trial is for adults with SOD1-ALS, a form of motor neuron disease. Participants should be in the early to mid-stages of the disease, have normal kidney and blood clotting functions, and not have severe respiratory issues. They must be able to consent and follow trial procedures. People with very high levels of certain antibodies or those who are 'fast' progressors may not qualify.
What is being tested?
The study tests AMT-162, an intrathecal gene therapy for safety, tolerability, and preliminary effectiveness in treating SOD1-ALS. It's a Phase 1/2 trial where participants receive one dose of the treatment at increasing levels to find out what's safe.
What are the potential side effects?
Potential side effects aren't specified but could include typical gene therapy reactions such as headache, fever, nausea or pain at injection site. Since it's a safety study, part of its purpose is to identify any side effects.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
My ALS is worsening quickly, losing more than 1 point per month on the ALSFRS-R scale.
Select...
I have ALS with signs of lower motor neuron issues, possibly with upper motor neuron symptoms.
Select...
My liver disease is not in its most severe stage.
Select...
My blood clotting ability is normal.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 5 years
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~5 years
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
To evaluate the safety and tolerability of ascending doses of intrathecally administered AMT-162 in Participants with SOD1-ALS
Secondary outcome measures
Characterization of Immune Response to AMT-162 and Shedding of intrathecally administered AMT-162.
Characterization of the Effect of intrathecally administered AMT-162
Other outcome measures
Efficacy of AMT-162
Trial Design
2Treatment groups
Experimental Treatment
Group I: EXPANSION COHORTExperimental Treatment1 Intervention
Expansion cohort: To further test selected dose from the SAD part in approximately 6 to 8 participants
The study will be open-label. Each Participant will receive a single dose of AMT-162 delivered via an intrathecal (IT) infusion and will be followed for up to 5 years after AMT-162 administration.
Group II: 3 single Ascending Dose LevelsExperimental Treatment1 Intervention
Experimental: 3 single Ascending Dose Levels
The study will be open-label with an initial plan to explore 3 dose levels of AMT-162 in approximately 6 to 12 Participants in total. Each Participant will receive a single dose of AMT-162 delivered via an intrathecal (IT) infusion and will be followed for up to 5 years after AMT-162 administration.
Find a Location
Who is running the clinical trial?
UniQure Biopharma B.V.Lead Sponsor
10 Previous Clinical Trials
232 Total Patients Enrolled
Executive Director, Clinical DevelopmentStudy DirectorUniQure Biopharma B.V.
Other Trials to Consider
Popular Categories
Share this study with friends
Copy Link
Messenger