Exploring QRL-101 for treating ALS in adults

A Randomized, Placebo-Controlled, Double-Blind, Single-Dose, Phase 1 Study to Explore the PK/PD Relationship of QRL-101 in People Living With ALS

This study is testing a new drug called QRL-101 to see if it is safe and how it works in adults with ALS.

Quick facts

• Phase: Phase 1
• Study type: Interventional
• Enrollment: 12 (estimated)
• Ages: 18 Years to 80 Years
• Sex: All
• Sponsor: QurAlis Corporation (Industry-sponsored)
• Locations: 1 site (Utrecht)
• Trial ID: NCT06714396 on ClinicalTrials.gov

What this trial studies

This Phase 1 randomized, placebo-controlled, double-blind study aims to evaluate the pharmacokinetics (PK) and pharmacodynamics (PD) of single doses of QRL-101 in adults diagnosed with Amyotrophic Lateral Sclerosis (ALS). The study will involve approximately 12 participants who will receive either QRL-101 or a placebo in a controlled setting. The primary focus is to assess the safety, tolerability, and the relationship between the drug's pharmacokinetics and pharmacodynamics. Participants will be randomized into four groups to receive different doses of QRL-101 or placebo, allowing for a comprehensive analysis of the drug's effects.

Who should consider this trial

Good fit: Ideal candidates for this study are adults aged 18 to 80 with a confirmed diagnosis of ALS.

Not a fit: Patients currently enrolled in other clinical trials or with significant medical illnesses may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could lead to a new treatment option for patients with ALS, potentially improving their quality of life.

How similar studies have performed: While this approach is novel in the context of ALS, similar studies in other conditions have shown promise in evaluating PK/PD relationships.

Eligibility criteria

INCLUSION CRITERIA

1. Age 18 to 80 years of age inclusive at the time of signing the informed consent.
2. Diagnosis of ALS, by either Gold Coast Criteria or El Escorial Criteria (possible, probable laboratory supported, probable, or definite)
3. Clinical chemistry laboratory values within acceptable range for the population, as per investigator judgment.
4. Body mass index of 18 to 32 kg/m2 (inclusive).
5. Willing and able to practice effective contraception.

EXCLUSION CRITERIA

1. Currently enrolled in any other clinical trial involving a study drug, off-label drug or device use, or any other type of medical research judged not scientifically or medically compatible with this study.
2. History or presence of medical illness including, but not limited to, any cardiovascular, hepatic, respiratory, hematological, endocrine, psychiatric, or neurological disease, convulsions, or any clinically significant laboratory abnormality that, in the judgment of the investigator, indicate a medical problem that would preclude study participation.
3. Off-label use of drugs or devices that are being used or investigated as disease-modifying therapies in ALS
4. Participated in another clinical study within 30 days, or 5 half-lives of the previous investigational product, whichever is greater, prior to the planned randomization date.
5. Taking any prescription or nonprescription medicines that are cytochrome P450 3A4 inducers, inhibitors, or substrates within 30 days or 5 half-lives, whichever is greater, prior to the planned randomization date

Where this trial is running

Utrecht

• Universitair Medisch Centrum Utrecht — Utrecht, Netherlands (Recruiting)

Study contacts

• Study coordinator: QurAlis Corporation

Conditions Amyotrophic Lateral Sclerosis