ALS dementia
Conditions
Interventions
All patients will receive intrathecal (IT) doses of the study drug starting at
Week 0.
The initial dose level is planned to be 10 mg of the study drug administered no
more often than every 12 weeks
Sponsors
Wave Life Sciences UK Limited
Eligibility
Age
18 Years to 99 Years
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. 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.
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Primary Endpoint: - Incidence of patients with adverse events (AEs), severe AEs, serious adverse events (SAEs), withdrawals due to AEs | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary Endpoints: Clinical Assessments: - Clinical Dementia Rating plus National Alzheimer*s Coordinating Center Frontotemporal Lobar Degeneration (CDR® plus NACC FTLD) - ALS Functional Rating Scale-Revised (ALSFRS-R) - Handheld dynamometry (HHD) - Pulmonary function testing (forced vital capacity [FVC]) - Amyotrophic Lateral Sclerosis Assessment Questionnaire (ALSAQ)-5 Pharmacodynamic Effects: - Change from baseline in concentration of poly-glycine-proline (poly-GP) levels in the cerebrospinal fluid (CSF) | — |
Countries
Netherlands
Outcome results
None listed