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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT05633459
Other study ID # QRL-201-01
Secondary ID
Status Recruiting
Phase Phase 1
First received
Last updated
Start date December 16, 2022
Est. completion date May 6, 2025

Study information

Verified date December 2023
Source QurAlis Corporation
Contact QurAlis Corporation
Phone 617-720-9566
Email clinicaltrials@quralis.com
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

The primary objective of this study is to determine the safety and tolerability of multiple doses of QRL-201 in people living with ALS


Description:

This first-in-human, Phase 1 study will evaluate safety, tolerability, and pharmacokinetics (PK) of QRL-201 administered intrathecal (IT) to participants with Amyotrophic Lateral Sclerosis. 8 cohorts of 8 participants each, in a 6:2 ratio of QRL-201 to placebo will be tested.


Recruitment information / eligibility

Status Recruiting
Enrollment 64
Est. completion date May 6, 2025
Est. primary completion date May 6, 2025
Accepts healthy volunteers No
Gender All
Age group 18 Years to 80 Years
Eligibility Inclusion Criteria: - Male or female participants aged 18 to 80 years diagnosed with ALS - ALS symptom onset within 24 months of Screening - Slow vital capacity >50% - Clinical evidence of lower motor neuron involvement - Not pregnant and not nursing - Willing and able to practice effective contraception - Able to tolerate lumbar puncture - If on approved therapies for the treatment of ALS during the course of the study, must be on a stable dose (at the Sponsor's discretion) Exclusion Criteria: - Pathogenic variant, likely pathogenic variant, or variant of uncertain significance in the superoxide dismutase 1 (SOD1) and/or fused in sarcoma (FUS) genes - Currently enrolled in any other clinical study involving either an investigational product (IP) or off-label use of a drug or device - Prior exposure to stem cell or gene therapy products - Any contraindication to intrathecal drug administration - Abnormal laboratory values deemed clinically significant by the Investigator - Significant infection, or known inflammatory process

Study Design


Intervention

Drug:
QRL-201 - Dose 1
Drug: Dose 1 of QRL-201 administered via intrathecal injection Diluent: artificial cerebrospinal fluid (aCSF) Placebo: consists of the same components as the formulation buffer for QRL-201 administered via intrathecal injection. Calculated volume to match active comparator Diluent: artificial cerebrospinal fluid (aCSF)
QRL-201 - Dose 2
Drug: Dose 2 of QRL-201 administered via intrathecal injection Diluent: artificial cerebrospinal fluid (aCSF) Placebo: consists of the same components as the formulation buffer for QRL-201 administered via intrathecal injection. Calculated volume to match active comparator Diluent: artificial cerebrospinal fluid (aCSF) Diluent: artificial cerebrospinal fluid (aCSF)
QRL-201 - Dose 3
Drug: Dose 3 of QRL-201 administered via intrathecal injection Diluent: artificial cerebrospinal fluid (aCSF) Placebo: consists of the same components as the formulation buffer for QRL-201 administered via intrathecal injection. Calculated volume to match active comparator Diluent: artificial cerebrospinal fluid (aCSF)
QRL-201 - Dose 4
Drug: Dose 4 of QRL-201 administered via intrathecal injection Diluent: artificial cerebrospinal fluid (aCSF) Placebo: consists of the same components as the formulation buffer for QRL-201 administered via intrathecal injection. Calculated volume to match active comparator Diluent: artificial cerebrospinal fluid (aCSF)
QRL-201 - Dose 5
Drug: Dose 5 of QRL-201 administered via intrathecal injection Diluent: artificial cerebrospinal fluid (aCSF) Placebo: consists of the same components as the formulation buffer for QRL-201 administered via intrathecal injection. Calculated volume to match active comparator Diluent: artificial cerebrospinal fluid (aCSF)
QRL-201 - Dose 6
Drug: Dose 6 of QRL-201 administered via intrathecal injection Diluent: artificial cerebrospinal fluid (aCSF) Placebo: consists of the same components as the formulation buffer for QRL-201 administered via intrathecal injection. Calculated volume to match active comparator Diluent: artificial cerebrospinal fluid (aCSF)
QRL-201 - Dose 7
Drug: Dose 7 of QRL-201 administered via intrathecal injection Diluent: artificial cerebrospinal fluid (aCSF) Placebo: consists of the same components as the formulation buffer for QRL-201 administered via intrathecal injection. Calculated volume to match active comparator Diluent: artificial cerebrospinal fluid (aCSF)
QRL-201- Dose 8
Drug: Dose 8 of QRL-201 administered via intrathecal injection Diluent: artificial cerebrospinal fluid (aCSF) Placebo: consists of the same components as the formulation buffer for QRL-201 administered via intrathecal injection. Calculated volume to match active comparator Diluent: artificial cerebrospinal fluid (aCSF)

Locations

Country Name City State
Belgium Universitaire Ziekenhuizen Leuven (UZ Leuven) Leuven
Canada University of Calgary Calgary Alberta
Canada University of Alberta Edmonton Alberta
Canada CHUM - Hopital Notre-Dame Montréal Quebec
Canada Montreal Neurological Institute-Hospital Montréal Quebec
Germany Charité Research Organisation Berlin
Germany University Hospital Schleswig-Holstein (UKSH) Campus Lübeck, Department for Neurology/ Precision Neurology Lübeck
Germany Universitätsklinikum Ulm Ulm
Ireland St James's Hospital Dublin
Netherlands Universitair Medisch Centrum Utrecht Utrecht
United Kingdom The University of Sheffield, Royal Hallamshire Hospital Sheffield

Sponsors (1)

Lead Sponsor Collaborator
QurAlis Corporation

Countries where clinical trial is conducted

Belgium,  Canada,  Germany,  Ireland,  Netherlands,  United Kingdom, 

Outcome

Type Measure Description Time frame Safety issue
Primary Number of participants with one or more treatment emergent adverse events and serious adverse events Endpoints: A summary of treatment emergent adverse events, serious adverse events, and other non-serious adverse events, regardless of causality, will be reported in the Reported Adverse Events module. Baseline through 253 Days
Secondary Pharmacokinetics (plasma): Maximum observed concentration of QRL-201 (Cmax) Endpoints: PK: Cmax of QRL-201 Predose up to 24 hours postdose
Secondary Pharmacokinetics (plasma): Area under the concentration time curve from zero to infinity (AUCinf) of QRL-201 Endpoints: PK: AUC (0-inf) of QRL-201 Predose up to 24 hours postdose
Secondary Pharmacokinetics (plasma): Time of maximum concentration (Tmax) of QRL-201 Endpoints: PK: Tmax of QRL-201 Predose up to 24 hours postdose
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