Amyotrophic Lateral Sclerosis and Frontotemporal Dementia MedDRA version: 21.1 Level: PT Classification code 10068968 Term: Frontotemporal dementia System Organ Class: 10029205 - Nervous system disorders MedDRA version: 21.1 Level: PT Classification code 10002026 Term: Amyotrophic lateral sclerosis System Organ Class: 10029205 - Nervous system disorders
Conditions
Sponsors
Wave Life Sciences UK Limited
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Patient has the ability and is willing to provide informed consent prior to any study procedures. In instances where signed written informed consent is unable to be obtained, it is acceptable for the patient to provide consent with legally authorized representative signing on the patient's behalf. 2. Patient successfully completed the Phase 1b/2a study with WVE-004, WVE-004-001. 3. In the opinion of the Investigator, the patient is able to tolerate all study procedures, is willing to comply with all other protocol requirements, and tolerated study drug in the parent study. 4. Patient is willing to practice highly effective contraception for the duration of the study and for 5 months after the last dose of study drug if the patient or their partner are of childbearing potential. Non-childbearing potential and highly effective methods of contraception are defined in the protocol (Section 5.2.1). In addition, willingness to forego sperm or ova (egg) donation for the duration of the study and 5 months after completion of the study. 5. Patient has identified a study partner(s) for the duration of the study. 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 31 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 11
Exclusion criteria
Exclusion criteria: 1. Patient has a clinically significant medical finding on the physical examination other than C9orf72-associated ALS or FTD that, in the judgment of the Investigator or Sponsor, will make the patient unsuitable for participation in and/or completion of the trial procedures. a. Prior or ongoing medical conditions, including acute illness, within 28 days of Screening visit; b. Clinically significant abnormality on laboratory testing at Screening, including but not limited to renal insufficiency, which is defined as creatinine clearance <40 mL/min. 2. Patient has a positive hepatitis B surface antigen or hepatitis C antibody test. 3. Patients who are pregnant (as determined by a serum pregnancy test) or breast feeding at the Screening visit or plans to become pregnant during the trial. 4. Patients deemed to be at significant risk for suicidal behavior based on Investigator assessment and/or active suicidal ideation. 5. Patient has a bone, spine, bleeding (e.g., hemophilia, Von Willebrand disease, or liver disease), or other disorder that exposes the patient to a risk of injury or unsuccessful LP. 6. Patient received prior treatment with viral or cellular-based gene therapy. 7. Patient received any other investigational drug, biological agent, or device within 1 month or 5 half-lives of study agent, whichever is longer. Patient received an investigational oligonucleotide within the past 6 months or 5 half-lives of the drug, whichever is longer. 8. Patient anticipates using antiplatelet or anticoagulant therapy during the course of the study. Patients who received antiplatelet or anticoagulant therapy must complete one of the following washout periods before the Screening visit: a. A 7-day washout period for antiplatelet therapy, b. A 1-day washout period for anticoagulants (except warfarin), or c. A 5-day washout period for warfarin. 9. Patient was noncompliant in the opinion of the Investigator or Sponsor when participating in study WVE-004-001. 10. Patient is directly or indirectly involved in the conduct and administration of this trial as an Investigator, sub-investigator, trial coordinator, or other trial staff member, or the patient is a first-degree family member, significant other, or relative residing with one of the above persons involved directly or indirectly in the trial.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the safety and tolerability of long-term treatment with WVE-004 in patients with ALS or FTD with a documented mutation in the C9orf72 gene ;Secondary Objective: To evaluate the clinical and pharmacodynamic (PD) effects of WVE-004 in patients with ALS or FTD with a documented mutation in the C9orf72 gene ;Primary end point(s): Incidence of patients with adverse events (AEs), severe AEs, serious adverse events (SAEs), withdrawals due to AEs.;Timepoint(s) of evaluation of this end point: week 1-120 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Clinical Assessments: Change from baseline in: o Clinical Dementia Rating plus National Alzheimer’s Coordinating Center Frontotemporal Lobar Degeneration (CDR® plus NACC FTLD) o ALS Functional Rating Scale-Revised (ALSFRS-R) o Handheld dynamometry (HHD) o Pulmonary function testing (forced vital capacity [FVC]) o Amyotrophic Lateral Sclerosis Assessment Questionnaire (ALSAQ) 5 Pharmacodynamic Effects: • Change from baseline in concentration of poly-lycine-proline (poly-GP) levels in the cerebrospinal fluid (CSF) ;Timepoint(s) of evaluation of this end point: week 1-120 | — |
Countries
Australia, Belgium, Canada, Ireland, Netherlands, New Zealand, United Kingdom
Contacts
Public ContactRegulatory Submissions
Medpace, Inc.
Outcome results
None listed