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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

Tracking Information
First Submitted Date  ICMJE November 21, 2022
First Posted Date  ICMJE December 1, 2022
Last Update Posted Date December 20, 2023
Actual Study Start Date  ICMJE December 16, 2022
Estimated Primary Completion Date May 6, 2025   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: November 21, 2022)		 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.
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: November 21, 2022)
  • Pharmacokinetics (plasma): Maximum observed concentration of QRL-201 (Cmax) [ Time Frame: Predose up to 24 hours postdose ]
    Endpoints: PK: Cmax of QRL-201
  • 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
  • Pharmacokinetics (plasma): Time of maximum concentration (Tmax) of QRL-201 [ Time Frame: Predose up to 24 hours postdose ]
    Endpoints: PK: Tmax of QRL-201
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study Evaluating the Safety and Tolerability of QRL-201 in ALS
Official Title  ICMJE 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
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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE 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
Condition  ICMJE Amyotrophic Lateral Sclerosis
Intervention  ICMJE
  • 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)

  • 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)

  • 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)

  • 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)

  • 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)

  • 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)

  • 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)

  • 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)
Study Arms  ICMJE
  • Experimental: QRL-201 - Arm 1
    Placebo Comparator: Placebo consists of the same components as the formulation buffer for QRL-201
    Intervention: Drug: QRL-201 - Dose 1
  • Experimental: QRL-201 - Arm 2
    Placebo Comparator: Placebo consists of the same components as the formulation buffer for QRL-201
    Intervention: Drug: QRL-201 - Dose 2
  • Experimental: QRL-201 - Arm 3
    Placebo Comparator: Placebo consists of the same components as the formulation buffer for QRL-201
    Intervention: Drug: QRL-201 - Dose 3
  • Experimental: QRL-201 - Arm 4
    Placebo Comparator: Placebo consists of the same components as the formulation buffer for QRL-201
    Intervention: Drug: QRL-201 - Dose 4
  • Experimental: QRL-201 - Arm 5
    Placebo Comparator: Placebo consists of the same components as the formulation buffer for QRL-201
    Intervention: Drug: QRL-201 - Dose 5
  • Experimental: QRL-201 - Arm 6
    Placebo Comparator: Placebo consists of the same components as the formulation buffer for QRL-201
    Intervention: Drug: QRL-201 - Dose 6
  • Experimental: QRL-201 - Arm 7
    Placebo Comparator: Placebo consists of the same components as the formulation buffer for QRL-201
    Intervention: Drug: QRL-201 - Dose 7
  • Experimental: QRL-201 - Arm 8
    Placebo Comparator: Placebo consists of the same components as the formulation buffer for QRL-201
    Intervention: Drug: QRL-201- Dose 8
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: November 21, 2022)		 64
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE May 6, 2025
Estimated Primary Completion Date May 6, 2025   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 80 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: QurAlis Corporation 617-720-9566 clinicaltrials@quralis.com
Listed Location Countries  ICMJE Belgium,   Canada,   Germany,   Ireland,   Netherlands,   United Kingdom
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT05633459
Other Study ID Numbers  ICMJE QRL-201-01
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Current Responsible Party QurAlis Corporation
Original Responsible Party Same as current
Current Study Sponsor  ICMJE QurAlis Corporation
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Angela Genge, MD QurAlis Corporation
PRS Account QurAlis Corporation
Verification Date December 2023

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP