A Study Exploring the PK/PD Relationship of QRL-101 in Adults With ALS

NCT06714396 | Completed Phase 1

• 1 other identifier: QRL-101-04
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief Summary

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

Outcome Measures

Primary Outcomes (2)

• QRL-101 plasma concentration

  Baseline through Follow up (Day 10)

• PK/PD Relationship: Strength-Duration Time Constant (SDTC)

  Baseline through Follow up (Day 10)

Secondary Outcomes (5)

• Safety and Tolerability: Adverse Events (AEs)

  Baseline through Follow up (Day 10)

• Safety and Tolerability: Serious Adverse Events (SAEs)

  Baseline through Follow up (Day 10)

• Plasma PK profile: Maximum concentration observed (Cmax)

  Baseline through Follow up (Day 10)

• Plasma PK profile: Time of maximum drug concentration (Tmax)

  Baseline through Follow up (Day 10)

• Plasma PK profile: Area under the concentration time curve from time 0 to 24 hours (AUC0-24hr)

  Baseline through Follow up (Day 10)

Study Arms (2)

QRL-101

EXPERIMENTAL

Single doses of QRL-101 will be orally administered. The dose levels may change subject to available nonclinical, clinical, safety, and PK data.

Drug: QRL-101

Placebo

EXPERIMENTAL

Single doses of comparator placebo will be orally administered. The dose levels may change subject to available nonclinical, clinical, safety, and PK data.

Other: Placebo

Interventions

QRL-101 DRUG

Single doses of QRL-101 will be orally administered. The dose levels may change subject to available nonclinical, clinical, safety, and PK data.

QRL-101

Placebo OTHER

Single doses of compartor placebo will be orally administered. The dose levels may change subject to available nonclinical, clinical, safety, and PK data.

Placebo

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

• 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.
• 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.
• Off-label use of drugs or devices that are being used or investigated as disease-modifying therapies in ALS
• 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.
• 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

Sponsors & Collaborators

• QurAlis Corporation: lead

Study Sites (1)

Universitair Medisch Centrum Utrecht

Utrecht, Netherlands, 9728 NZ, Netherlands

Location

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 27, 2024
• First Posted: December 3, 2024
• Study Start: November 11, 2024
• Primary Completion: August 22, 2025
• Study Completion: August 22, 2025
• Last Updated: September 2, 2025

Record last verified: 2025-08

Locations

NL (1)