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
Trial ID: NCT06714396
Interventional - Phase 1 - Completed
This Phase 1 randomized, placebo-controlled, double-blind study will evaluate the PK/PD relationship of single doses of QRL-101 in 12 ALS participants. The study will also assess safety and tolerability in participants receiving either QRL-101 or placebo.
Conditions
Amyotrophic Lateral Sclerosis
Interventions
QRL-101
Placebo
Eligibility
Inclusion criteria
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
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