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

Primary Purpose

Amyotrophic Lateral Sclerosis

Status
Recruiting
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
QRL-201 - Dose 1
QRL-201 - Dose 2
QRL-201 - Dose 3
QRL-201 - Dose 4
QRL-201 - Dose 5
QRL-201 - Dose 6
QRL-201 - Dose 7
QRL-201- Dose 8
Sponsored by
QurAlis Corporation
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Amyotrophic Lateral Sclerosis focused on measuring ALS, Stathmin-2, STMN2, ASO, Antisense Oligonucleotide

Eligibility Criteria

18 Years - 80 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Universitaire Ziekenhuizen Leuven (UZ Leuven)
  • University of CalgaryRecruiting
  • University of AlbertaRecruiting
  • CHUM - Hopital Notre-DameRecruiting
  • Montreal Neurological Institute-HospitalRecruiting
  • University Hospital Schleswig-Holstein (UKSH) Campus Lübeck, Department for Neurology/ Precision NeurologyRecruiting
  • Universitätsklinikum Ulm
  • St James's HospitalRecruiting
  • Universitair Medisch Centrum UtrechtRecruiting
  • The University of Sheffield, Royal Hallamshire Hospital

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm 5

Arm 6

Arm 7

Arm 8

Arm Type

Experimental

Experimental

Experimental

Experimental

Experimental

Experimental

Experimental

Experimental

Arm Label

QRL-201 - Arm 1

QRL-201 - Arm 2

QRL-201 - Arm 3

QRL-201 - Arm 4

QRL-201 - Arm 5

QRL-201 - Arm 6

QRL-201 - Arm 7

QRL-201 - Arm 8

Arm Description

Placebo Comparator: Placebo consists of the same components as the formulation buffer for QRL-201

Placebo Comparator: Placebo consists of the same components as the formulation buffer for QRL-201

Placebo Comparator: Placebo consists of the same components as the formulation buffer for QRL-201

Placebo Comparator: Placebo consists of the same components as the formulation buffer for QRL-201

Placebo Comparator: Placebo consists of the same components as the formulation buffer for QRL-201

Placebo Comparator: Placebo consists of the same components as the formulation buffer for QRL-201

Placebo Comparator: Placebo consists of the same components as the formulation buffer for QRL-201

Placebo Comparator: Placebo consists of the same components as the formulation buffer for QRL-201

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

Pharmacokinetics (plasma): Maximum observed concentration of QRL-201 (Cmax)
Endpoints: PK: Cmax of QRL-201
Pharmacokinetics (plasma): Area under the concentration time curve from zero to infinity (AUCinf) of QRL-201
Endpoints: PK: AUC (0-inf) of QRL-201
Pharmacokinetics (plasma): Time of maximum concentration (Tmax) of QRL-201
Endpoints: PK: Tmax of QRL-201

Full Information

First Posted
November 21, 2022
Last Updated
October 19, 2023
Sponsor
QurAlis Corporation
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1. Study Identification

Unique Protocol Identification Number
NCT05633459
Brief Title
A Study Evaluating the Safety and Tolerability of QRL-201 in ALS
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
Study Type
Interventional

2. Study Status

Record Verification Date
October 2023
Overall Recruitment Status
Recruiting
Study Start Date
December 16, 2022 (Actual)
Primary Completion Date
May 6, 2025 (Anticipated)
Study Completion Date
May 6, 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
QurAlis Corporation

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Amyotrophic Lateral Sclerosis
Keywords
ALS, Stathmin-2, STMN2, ASO, Antisense Oligonucleotide

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Parallel Assignment
Model Description
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes 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.
Allocation
Randomized
Enrollment
64 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
QRL-201 - Arm 1
Arm Type
Experimental
Arm Description
Placebo Comparator: Placebo consists of the same components as the formulation buffer for QRL-201
Arm Title
QRL-201 - Arm 2
Arm Type
Experimental
Arm Description
Placebo Comparator: Placebo consists of the same components as the formulation buffer for QRL-201
Arm Title
QRL-201 - Arm 3
Arm Type
Experimental
Arm Description
Placebo Comparator: Placebo consists of the same components as the formulation buffer for QRL-201
Arm Title
QRL-201 - Arm 4
Arm Type
Experimental
Arm Description
Placebo Comparator: Placebo consists of the same components as the formulation buffer for QRL-201
Arm Title
QRL-201 - Arm 5
Arm Type
Experimental
Arm Description
Placebo Comparator: Placebo consists of the same components as the formulation buffer for QRL-201
Arm Title
QRL-201 - Arm 6
Arm Type
Experimental
Arm Description
Placebo Comparator: Placebo consists of the same components as the formulation buffer for QRL-201
Arm Title
QRL-201 - Arm 7
Arm Type
Experimental
Arm Description
Placebo Comparator: Placebo consists of the same components as the formulation buffer for QRL-201
Arm Title
QRL-201 - Arm 8
Arm Type
Experimental
Arm Description
Placebo Comparator: Placebo consists of the same components as the formulation buffer for QRL-201
Intervention Type
Drug
Intervention Name(s)
QRL-201 - Dose 1
Intervention Description
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)
Intervention Type
Drug
Intervention Name(s)
QRL-201 - Dose 2
Intervention Description
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)
Intervention Type
Drug
Intervention Name(s)
QRL-201 - Dose 3
Intervention Description
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)
Intervention Type
Drug
Intervention Name(s)
QRL-201 - Dose 4
Intervention Description
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)
Intervention Type
Drug
Intervention Name(s)
QRL-201 - Dose 5
Intervention Description
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)
Intervention Type
Drug
Intervention Name(s)
QRL-201 - Dose 6
Intervention Description
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)
Intervention Type
Drug
Intervention Name(s)
QRL-201 - Dose 7
Intervention Description
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)
Intervention Type
Drug
Intervention Name(s)
QRL-201- Dose 8
Intervention Description
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)
Primary Outcome Measure Information:
Title
Number of participants with one or more treatment emergent adverse events and serious adverse events
Description
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.
Time Frame
Baseline through 253 Days
Secondary Outcome Measure Information:
Title
Pharmacokinetics (plasma): Maximum observed concentration of QRL-201 (Cmax)
Description
Endpoints: PK: Cmax of QRL-201
Time Frame
Predose up to 24 hours postdose
Title
Pharmacokinetics (plasma): Area under the concentration time curve from zero to infinity (AUCinf) of QRL-201
Description
Endpoints: PK: AUC (0-inf) of QRL-201
Time Frame
Predose up to 24 hours postdose
Title
Pharmacokinetics (plasma): Time of maximum concentration (Tmax) of QRL-201
Description
Endpoints: PK: Tmax of QRL-201
Time Frame
Predose up to 24 hours postdose

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
80 Years
Accepts Healthy Volunteers
No
Eligibility 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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
QurAlis Corporation
Phone
617-720-9566
Email
clinicaltrials@quralis.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Angela Genge, MD
Organizational Affiliation
QurAlis Corporation
Official's Role
Study Director
Facility Information:
Facility Name
Universitaire Ziekenhuizen Leuven (UZ Leuven)
City
Leuven
ZIP/Postal Code
B-3000
Country
Belgium
Individual Site Status
Not yet recruiting
Facility Name
University of Calgary
City
Calgary
State/Province
Alberta
ZIP/Postal Code
T2N 1N4
Country
Canada
Individual Site Status
Recruiting
Facility Contact:
Phone
403-210-7009
Email
wongb@ucalgary.ca
First Name & Middle Initial & Last Name & Degree
Thomas Mobach, MD
Facility Name
University of Alberta
City
Edmonton
State/Province
Alberta
ZIP/Postal Code
T6G 2G3
Country
Canada
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Kelsey Tymkow
Phone
780-492-7690
Email
tymkow@ualberta.ca
First Name & Middle Initial & Last Name & Degree
Wendy Johnston
Facility Name
CHUM - Hopital Notre-Dame
City
Montréal
State/Province
Quebec
ZIP/Postal Code
H2L 4M1
Country
Canada
Individual Site Status
Recruiting
Facility Contact:
Phone
514-890-8000
Ext
30737
Email
uit.eligibilite.chum@ssss.gouv.qc.ca
First Name & Middle Initial & Last Name & Degree
Genevieve Matte, MD
Facility Name
Montreal Neurological Institute-Hospital
City
Montréal
State/Province
Quebec
ZIP/Postal Code
H3A 2B4
Country
Canada
Individual Site Status
Recruiting
Facility Contact:
Phone
514-398-6183
Email
als-cru.neuro@mcgill.ca
First Name & Middle Initial & Last Name & Degree
Rami Massie, MD
Facility Name
University Hospital Schleswig-Holstein (UKSH) Campus Lübeck, Department for Neurology/ Precision Neurology
City
Lübeck
Country
Germany
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Julian Grosskreutz, MD
Phone
+49 451-500-43468
Email
pnl@neuro.uni-luebeck.de
First Name & Middle Initial & Last Name & Degree
Julian Grosskreutz
Facility Name
Universitätsklinikum Ulm
City
Ulm
ZIP/Postal Code
89081
Country
Germany
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Bettina Hepp
Email
Bettina.hepp@uniklinik-ulm.de
First Name & Middle Initial & Last Name & Degree
Albert C Ludolph, MD
Phone
+49 731 177 1201
Email
albert.ludolph@rku.de
Facility Name
St James's Hospital
City
Dublin
ZIP/Postal Code
D08 A978
Country
Ireland
Individual Site Status
Recruiting
Facility Contact:
Email
HARDIMAO@tcd.ie
First Name & Middle Initial & Last Name & Degree
Orla Hardiman
Facility Name
Universitair Medisch Centrum Utrecht
City
Utrecht
Country
Netherlands
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Leonard Van Den Berg
Facility Name
The University of Sheffield, Royal Hallamshire Hospital
City
Sheffield
ZIP/Postal Code
S10 2JF
Country
United Kingdom
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Christopher McDermott, MD
Phone
+44-114-2222264
First Name & Middle Initial & Last Name & Degree
Madalina Roman, NP
Email
madalina.roman@nhs.net

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

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