Phase 2 safety and tolerability study using ANX005 for ALS
- Conditions
- Amyotrophic Lateral Scelrosis (ALS)MedDRA version: 21.1Level: PTClassification code 10002026Term: Amyotrophic lateral sclerosisSystem Organ Class: 10029205 - Nervous system disordersTherapeutic area: Diseases [C] - Nervous System Diseases [C10]
- Registration Number
- EUCTR2021-000325-26-FR
- Lead Sponsor
- Annexon Biosciences
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- All
- Target Recruitment
- 25
1. Males or females aged 18 and above at the time of informed consent.
2. Diagnosis of ALS (probable, possible, laboratory-supported probable or definite) according to the World Federation of Neurology revised E1 Escorial criteria
3. Onset of weakness within 3 years prior to enrollment
4. Slow Vital Capacity = 60% of predicted normal adjusted for sex, age, and height (from the sitting position).
5. Able to perform reproducible pulmonary function tests
6. ALS concomitant medications (e.g., riluzole, edaravone) stable for at least 2 months prior to Screening and expected to remain at that dose until Week 24, the end of study visit.
7. ALSFRS-R = 30 at the Screening Visit
8. If female, must be postmenopausal (for at least 2 years), surgically sterilized (bilateral tubal ligation, bilateral oophorectomy, or hysterectomy), or agree to use highly effective methods of
contraception from screening through Week 24
9. Males with a woman of childbearing potential partner must agree to use highly effective methods of contraception from Screening through Week 24 (section 6.3).
10. Documented evidence of previous vaccinations within 5 years prior to screening visit against encapsulated bacterial pathogens (Neisseria meningitidis including serogroup B meningococcus, Haemophilus influenzae, and Streptococcus pneumoniae) or willing to undergo vaccination.
11. Able to comply with the requirements of the study and complete the full sequence of protocol related procedures and evaluations, including lumbar punctures for collection of cerebrospinal fluid.
12. If a caregiver/study partner is required, per Investigator discretion, must have a competent caregiver/study partner who is at least 18 years of age and is willing to accompany the subject to select trial visits and to be available by phone if needed, and who is and will remain sufficiently knowledgeable of subject’s ongoing condition to respond to Study Center inquiries about the subject.
13. Able to understand and provide written, informed consent
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 19
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 6
1. Be at risk of suicide or self-harm within the preceding 12 months, in the opinion of the investigator.
2. Subjects with body weight > 150 kg.
3. Clinically significant findings on screening laboratory testing, physical examination or vital signs that are not specific to ALS that could interfere with the conduct of the study, the interpretation of the data or increase subject risk.
4. Signs and symptoms of, or a diagnosis consistent with a chronic
autoimmune disorder and/or an ANA titer = 1:160.
5. History of previous infusion reactions, sensitivities, allergic, or anaphylactic reactions to previous medications, environmental stimuli or other substances
6. Receipt of an experimental agent within 60 days or five half-lives, whichever is longer, prior to Screening or through Week 24.
7. Prior treatment with any monoclonal antibody within 6 months of screening.
8. Hypersensitivity to any of the excipients in the ANX005 drug product
9. Clinically significant intercurrent illness, medical condition, or medical history (including neurological or mental illness, HIV, any active infection, including Hepatitis B or C) that would jeopardize the safety of the subject, limit participation, or compromise the
interpretation of the data derived from the subject.
10. Any known genetic deficiencies of the complement-cascade system.
11. History of chronic systemic steroid use or immunosuppressant medication ending less than 1 month prior to screening.
12. Active alcohol, drug abuse or substance abuse, or any other reason that makes it unlikely that the subject will comply with study procedures per Investigator discretion.
13. Females who are pregnant (positive pregnancy test at screening or prior to infusion of ANX005), breast feeding, or unable or unwilling to use highly effective methods of contraception throughout the study.
14. Contraindication to undergoing an LP including, but not limited to: inability to tolerate an appropriately flexed position for the time necessary to perform an LP; international normalized ratio (INR) >
1.4 or other coagulopathy; platelet count of < 120,000/µL; infection at the desired lumbar puncture site; taking anti-platelet or coagulant medication within 60 days of screening (Note: low dose aspirin is permitted); history of severe degenerative arthritis of the lumbar spine; suspected non-communicating hydrocephalus or intracranial mass; prior history of spinal mass or trauma
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method