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A Study Evaluating the Safety and Tolerability of QRL-201 in ALS

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05633459
Recruitment Status : Recruiting
First Posted : December 1, 2022
Last Update Posted : December 20, 2023
Sponsor:
Information provided by (Responsible Party):
QurAlis Corporation

Study Description
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Brief 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

Condition or disease Intervention/treatment Phase
Amyotrophic Lateral Sclerosis Drug: QRL-201 - Dose 1 Drug: QRL-201 - Dose 2 Drug: QRL-201 - Dose 3 Drug: QRL-201 - Dose 4 Drug: QRL-201 - Dose 5 Drug: QRL-201 - Dose 6 Drug: QRL-201 - Dose 7 Drug: QRL-201- Dose 8 Phase 1

Detailed 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.
Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 64 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description: Multiple-ascending doses of QRL-201 or placebo will be administered. The dose levels may change subject to available nonclinical, clinical, safety, and PK data.
Primary Purpose: Treatment
Official Title: A Multi-Center, Randomized, Double-Blind Placebo Controlled Multiple-Ascending Dose Study to Evaluate the Safety and Tolerability of QRL-201 in Amyotrophic Lateral Sclerosis
Actual Study Start Date : December 16, 2022
Estimated Primary Completion Date : May 6, 2025
Estimated Study Completion Date : May 6, 2025

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: QRL-201 - Arm 1
Placebo Comparator: Placebo consists of the same components as the formulation buffer for QRL-201
 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)
Experimental: QRL-201 - Arm 2
Placebo Comparator: Placebo consists of the same components as the formulation buffer for QRL-201
 Drug: 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)
Experimental: QRL-201 - Arm 3
Placebo Comparator: Placebo consists of the same components as the formulation buffer for QRL-201
 Drug: 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)
Experimental: QRL-201 - Arm 4
Placebo Comparator: Placebo consists of the same components as the formulation buffer for QRL-201
 Drug: 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)
Experimental: QRL-201 - Arm 5
Placebo Comparator: Placebo consists of the same components as the formulation buffer for QRL-201
 Drug: 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)
Experimental: QRL-201 - Arm 6
Placebo Comparator: Placebo consists of the same components as the formulation buffer for QRL-201
 Drug: 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)
Experimental: QRL-201 - Arm 7
Placebo Comparator: Placebo consists of the same components as the formulation buffer for QRL-201
 Drug: 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)
Experimental: QRL-201 - Arm 8
Placebo Comparator: Placebo consists of the same components as the formulation buffer for QRL-201
 Drug: 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)


Outcome Measures
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Primary Outcome Measures :
  1. Number of participants with one or more treatment emergent adverse events and serious adverse events [ Time Frame: Baseline through 253 Days ]
    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.


Secondary Outcome Measures :
  1. Pharmacokinetics (plasma): Maximum observed concentration of QRL-201 (Cmax) [ Time Frame: Predose up to 24 hours postdose ]
    Endpoints: PK: Cmax of QRL-201

  2. Pharmacokinetics (plasma): Area under the concentration time curve from zero to infinity (AUCinf) of QRL-201 [ Time Frame: Predose up to 24 hours postdose ]
    Endpoints: PK: AUC (0-inf) of QRL-201

  3. Pharmacokinetics (plasma): Time of maximum concentration (Tmax) of QRL-201 [ Time Frame: Predose up to 24 hours postdose ]
    Endpoints: PK: Tmax of QRL-201


Eligibility Criteria
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Ages Eligible for Study:   18 Years to 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05633459


Contacts
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Contact: QurAlis Corporation 617-720-9566 clinicaltrials@quralis.com

Locations
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Belgium
Universitaire Ziekenhuizen Leuven (UZ Leuven) Recruiting
Leuven, Belgium, B-3000
Canada, Alberta
University of Calgary Recruiting
Calgary, Alberta, Canada, T2N 1N4
Contact    403-210-7009    wongb@ucalgary.ca   
Principal Investigator: Thomas Mobach, MD         
University of Alberta Recruiting
Edmonton, Alberta, Canada, T6G 2G3
Contact: Kelsey Tymkow    780-492-7690    tymkow@ualberta.ca   
Principal Investigator: Wendy Johnston         
Canada, Quebec
CHUM - Hopital Notre-Dame Recruiting
Montréal, Quebec, Canada, H2L 4M1
Contact    514-890-8000 ext 30737    uit.eligibilite.chum@ssss.gouv.qc.ca   
Principal Investigator: Genevieve Matte, MD         
Montreal Neurological Institute-Hospital Recruiting
Montréal, Quebec, Canada, H3A 2B4
Contact    514-398-6183    als-cru.neuro@mcgill.ca   
Principal Investigator: Rami Massie, MD         
Germany
Charité Research Organisation Recruiting
Berlin, Germany, 10117
Contact: André S Maier, MD    +49 30 450 539 200      
Principal Investigator: André S Maier         
University Hospital Schleswig-Holstein (UKSH) Campus Lübeck, Department for Neurology/ Precision Neurology Recruiting
Lübeck, Germany
Contact: Julian Grosskreutz, MD    +49 451-500-43468    pnl@neuro.uni-luebeck.de   
Principal Investigator: Julian Grosskreutz         
Universitätsklinikum Ulm Recruiting
Ulm, Germany, 89081
Contact: Bettina Hepp       Bettina.hepp@uniklinik-ulm.de   
Contact: Albert C Ludolph, MD    +49 731 177 1201    albert.ludolph@rku.de   
Ireland
St James's Hospital Recruiting
Dublin, Ireland, D08 A978
Contact       HARDIMAO@tcd.ie   
Principal Investigator: Orla Hardiman         
Netherlands
Universitair Medisch Centrum Utrecht Recruiting
Utrecht, Netherlands
Principal Investigator: Leonard Van Den Berg         
United Kingdom
The University of Sheffield, Royal Hallamshire Hospital Recruiting
Sheffield, United Kingdom, S10 2JF
Contact: Christopher McDermott, MD    +44-114-2222264      
Contact: Madalina Roman, NP       madalina.roman@nhs.net   
Sponsors and Collaborators
QurAlis Corporation
Investigators
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Study Director: Angela Genge, MD QurAlis Corporation
More Information
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Responsible Party: QurAlis Corporation
ClinicalTrials.gov Identifier: NCT05633459    
Other Study ID Numbers: QRL-201-01
First Posted: December 1, 2022    Key Record Dates
Last Update Posted: December 20, 2023
Last Verified: December 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by QurAlis Corporation:
ALS, Stathmin-2, STMN2, ASO, Antisense Oligonucleotide
Additional relevant MeSH terms:
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Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Sclerosis
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
 Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases