Phase 1/2 Investigation of Novel Experimental Regimen in Amyotrophic Lateral Sclerosis (Pioneer-ALS): An Open-Label, Uncontrolled, Multicenter Study to Assess the Safety and Tolerability of Two Doses of VTx-002
Vector Y Therapeutics
Summary
PIONEER-ALS is a Phase 1/2, multicenter, open-label, ascending dose, uncontrolled, first-in-human study that will evaluate the safety, tolerability and effects on clinical and biomarker endpoints of intracisternal administration of Vtx-002 in participants with Amyotrophic Lateral Sclerosis (ALS). Two escalating dose (low dose and high dose) cohorts are planned. The duration of the study will be a maximum of 5 years and 5 weeks (265 weeks) for each participant. The screening period may last up to 5 weeks to complete screening procedures.
Description
All participants will receive a single injection of the study drug. During the first year of the study there will be 12 visits to the study center, including an overnight stay after dosing of at least 1 night. There will be a further 4 remote visits (telephone or video call). From Year 2-5 there will be 8 further visits. These will be every 6 months and will be either in-person at the study site or remote (telephone or video call) if needed or preferred. Throughout the 5-year observation period, there will be up to 20 study visits to complete follow-up tests and assessments and monitor the o…
Eligibility
- Age range
- 18+ years
- Sex
- All
- Healthy volunteers
- No
Key Inclusion Criteria: 1. Capable of, and willing to, provide written informed consent and comply with study procedures, including visits to the study site and visit requirements 2. Male or female ≥ 18 years of age 3. Has a diagnosis of ALS according to the El Escorial criteria (Brooks, et al., 2000) (probable, laboratory results supported; clinically probable, clinically definite) 4. Confirmed absence of ALS caused by FUS and SOD1 gene mutations confirmed by laboratory tests. 5. A maximum of 18 months since first appearance of weakness (e.g., limb weakness, dysarthria, dysphagia, shortness…
Interventions
- GeneticVTx-002
An investigational gene therapy targeting a specific protein.
- DrugPreventative (Prophylactic) Medication - Corticosteroids: Methylprednisolone
To reduce the risk of reactions caused by the study treatment, steroid medicines will be given in advance.
Locations (11)
- St Joseph's Hospital and medical Center - Barrow Neurological InstitutePhoenix, Arizona
- University of California San Diego Medical CenterSan Diego, California
- Mayo Clinic in FloridaJacksonville, Florida
- University of Miami School of ScienceMiami, Florida
- Sean M. Healey & AMG Center for ALS at Massachusetts General HospitalBoston, Massachusetts
- Herbert Irving Comprehensive Cancer CenterNew York, New York