Safety and tolerability of VTx-002 given into the cisterna in people with ALS

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

PIONEER-ALS is a first-in-human, open-label Phase 1/2 study using ascending doses of VTx-002 administered as a single intracisternal injection. Two dose cohorts (low and high) will be enrolled and all participants will be followed for up to 5 years and 5 weeks with scheduled in-person and remote visits. The first year includes 12 site visits including one overnight stay after dosing, then biannual follow-up visits in years 2–5, totaling up to 20 follow-up visits. Clinical outcomes and biomarker measures will be collected throughout to monitor safety, tolerability, and any effects on disease-related measures.

Why it matters

Who can join: the full rules

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 of breath)
6. Erect (seated) SVC % predicted ≥ 80% at Screening
7. Treatment Research Initiative to Cure ALS (TRICALS) risk score between -2 and -6 at Screening
8. Has a reliable caregiver/partner/legal representative willing and able to support the participant in participation in the study and to give informed consent on behalf of the participant in the case that disease progression prevents the participant of giving consent (local legal rules will apply).
9. Treatment with riluzole and/or edaravone is allowed if treatment was started and has remained at a stable dose for at least 2 weeks (riluzole) or one treatment cycle (edaravone) before the Screening visit
10. Women of childbearing potential (WOCBP) and male participants with female partners who are WOCBP must agree to use highly effective contraception during and after the study. WOCBP cannot be pregnant or breastfeeding
11. Women of nonchildbearing potential must be post-menopausal or surgically sterile (e.g. hysterectomy, bilateral tubal ligation, ovaries removed)

Key Exclusion Criteria:

1. Diagnosis of a significant CNS or peripheral nervous system disease other than ALS that may be a cause for the participant's ALS symptoms or may confound study objectives
2. Spinal, cervical, or brain MRI/MRA indicating clinically significant abnormality
3. Presence of tracheostomy and feeding tube at Screening
4. Contraindications to corticosteroid use (e.g. due to osteoporosis, uncontrolled blood pressure, diabetes or cholesterol).

5\. Significant concomitant disease or condition within 6 months of Screening that could pose an unacceptable safety risk to the participant or interfere with the participant's ability to comply with study procedures, e.g. heart disease, uncontrolled diabetes, liver disease, autoimmune diseases needing strong immune-suppressing drugs, cancer, etc or a current psychiatric diagnosis.

6\. Clinically significant abnormalities in laboratory test results at Screening for example poor liver or kidney function, abnormal clotting or infections such as Hepatitis or HIV

7\. Use of blood thinners (e.g., warfarin, heparin, and novel oral anticoagulants) and being unable to safely stop them before certain study procedures.

8\. Contraindications to imaging methods MRI, MRA, CT due to claustrophobia and/or intolerance to contrast agents.

9\. Contraindications to general anaesthesia (GA) or deep sedation

10 Positive test for illegal drugs (except prescribed medications or permitted medicinal/recreational marijuana if used responsibly)

11\. Generally frail or if the Investigator deems participation in the study would not be in the best interest of the participant or is likely to prohibit further participation during the study

Other protocol-defined inclusion/exclusion criteria may apply

\-

Where it's running

• University of California San Diego Medical Center
  San Diego, California, United States Recruiting
• Mayo Clinic in Florida
  Jacksonville, Florida, United States Recruiting
• Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital
  Boston, Massachusetts, United States Recruiting
• University of Pennsylvania
  Philadelphia, Pennsylvania, United States Recruiting
• UZ Leuven
  Leuven, Belgium Recruiting
• UMC Utrecht
  Utrecht, Netherlands Recruiting

Study contacts

• Study coordinator: Dr Olga Uspenskaya Chief medical Officer, VectorY Therapeutics, M.D; PhD
• Email: patients@vectorytx.com
• Phone: patients@vectorytx.com

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Implanted Brain-Computer Interface to Help People with Severe Arm Impairment

  We’re testing the N1 System, a fully implanted brain interface in the motor cortex with a robot, to see if it can help adults with spinal cord injury, ALS, or chronic stroke use a computer or keyboard more easily.

  Recruiting Neuralink Corp · Miami, Florida · NCT07929792

• Brain-computer interface chat tool for people with ALS

  This test compares a wearable brain-computer interface communication device to eye-tracking devices that people with ALS currently use to help them communicate.

  Recruiting Cognixion · Santa Barbara, California · NCT07871071

• BIIB147 safety and how it behaves in adults with ALS

  This study tests BIIB147 in adults with ALS to see if it is safe and how the body handles it, including early and long-term effects.

  Recruiting at 2 sites Phase 1/2 Biogen · St Louis, Missouri and 1 other locations · NCT07855211

• Aims to restore UNC13A function in people with ALS

  This trial tests TRCN-1023 in adults with ALS to see if it’s safe, tolerated, and shows early signs of benefit.

  Recruiting at 6 sites Phase 1/2 Trace Neuroscience, Inc. · Lübeck, Schleswig-Holstein and 5 other locations · NCT07674667

• LTX-002 in adults with ALS

  A study will test whether the experimental drug LTX-002 is safe and tolerable in adults with ALS and see how the body handles the drug and how it affects ALS indicators.

  Recruiting at 4 sites Phase 1/2 Leal Therapeutics, Inc · Lübeck and 4 other locations · NCT07660614

• Repeated ultrasound-assisted blood-brain barrier opening in ALS

  This trial tests whether repeatedly opening the blood-brain barrier with ultrasound is safe and may help people with ALS.

  Recruiting Phase 1/2 Assistance Publique - Hôpitaux de Paris · Paris · NCT07571486

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.