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A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adult Male Subjects With Cerebral Adrenoleukodystrophy (CALYX)

Primary Purpose

Cerebral Adrenoleukodystrophy (cALD)

Status
Recruiting
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Leriglitazone
Placebo
Sponsored by
Minoryx Therapeutics, S.L.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Cerebral Adrenoleukodystrophy (cALD)

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)MaleDoes not accept healthy volunteers

Key Inclusion Criteria: Subject is male and aged ≥18 years. Subject has progressive cALD, defined as GdE+ brain lesions. Subjects for whom HSCT is not recommended by the investigator or subject is not willing to undergo HSCT. Subject has a Loes score ≥0.5 and ≤12 at Screening. Subject does not have major functional disability in the Major Functional Disabilities-Neurological Function Score (MFD-NFS), except for "wheelchair bound" or "total incontinence", which will be allowed as these are considered expected symptoms of AMN in the time course of the disease Subject does not have major cognitive impairment which would impair his ability to take part in the study as determined by the investigator at screening. Key Exclusion Criteria: Subject who had previous bone marrow transplantation (HSCT) or treatment with ex-vivo gene therapy (eli-Cel). Subject has known type 1 or type 2 diabetes. Subject has known hypersensitivity or intolerance to pioglitazone or any other thiazolidinedione. Subject is taking or has taken honokiol, pioglitazone, or other thiazolidinediones within 3 months prior to Screening. Subject with current participation in another interventional clinical study or within 1 month prior to Screening. Subject with other medical, neuropsychiatric or social conditions that, in the opinion of the investigator, are likely to adversely affect the risk-benefit of study participation, interfere with study compliance, or confound the study results.

Sites / Locations

  • Stanford University Medical CenterRecruiting
  • Neuro Medicine Hospital / UF Health
  • Kennedy Krieger InstituteRecruiting
  • Massachusetts General HospitalRecruiting
  • University of MinnesotaRecruiting
  • Health University of UtahRecruiting
  • Hospital Austral
  • Federal University of Sao Paulo

Arms of the Study

Arm 1

Arm 2

Arm Type

Active Comparator

Placebo Comparator

Arm Label

Leriglitazone

Placebo

Arm Description

Leriglitazone Treatment

Placebo

Outcomes

Primary Outcome Measures

The primary endpoint will be the time to death or the subject becoming bedridden with a requirement for permanent ventilatory support, wichever comes earlier, in subjects treated with leriglitazone compared to placebo.
The primary endpoint will be the time to death or the subject becoming bedridden with a requirement for permanent ventilatory support, wichever comes earlier, in subjects treated with leriglitazone compared to placebo.
The primary endpoint will be the time to death or the subject becoming bedridden with a requirement for permanent ventilatory support, wichever comes earlier, in subjects treated with leriglitazone compared to placebo.
The primary endpoint will be the time to death or the subject becoming bedridden with a requirement for permanent ventilatory support, wichever comes earlier, in subjects treated with leriglitazone compared to placebo.
The primary endpoint will be the time to death or the subject becoming bedridden with a requirement for permanent ventilatory support, wichever comes earlier, in subjects treated with leriglitazone compared to placebo.
The primary endpoint will be the time to death or the subject becoming bedridden with a requirement for permanent ventilatory support, wichever comes earlier, in subjects treated with leriglitazone compared to placebo.

Secondary Outcome Measures

Change from Baseline in Loes Score.
Change from Baseline in Loes Score. Loes Score Minimum = 0 Maximum =34 0 better outcome (healthy) 34 worst osutcome
Change from Baseline in Loes Score
Change from Baseline in Loes Score. Loes Score Minimum = 0 Maximum =34 0 better outcome (healthy) 34 worst osutcome
Change from Baseline in Loes Score
Change from Baseline in Loes Score. Loes Score Minimum = 0 Maximum =34 0 better outcome (healthy) 34 worst osutcome

Full Information

First Posted
March 23, 2023
Last Updated
October 23, 2023
Sponsor
Minoryx Therapeutics, S.L.
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1. Study Identification

Unique Protocol Identification Number
NCT05819866
Brief Title
A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adult Male Subjects With Cerebral Adrenoleukodystrophy
Acronym
CALYX
Official Title
A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adult Male Subjects With Cerebral Adrenoleukodystrophy
Study Type
Interventional

2. Study Status

Record Verification Date
October 2023
Overall Recruitment Status
Recruiting
Study Start Date
July 12, 2023 (Actual)
Primary Completion Date
May 2027 (Anticipated)
Study Completion Date
May 2027 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Minoryx Therapeutics, S.L.

4. Oversight

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

5. Study Description

Brief Summary
A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adults Male Subjects with Cerebral Adrenoleukodystrophy.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cerebral Adrenoleukodystrophy (cALD)

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigator
Allocation
Randomized
Enrollment
40 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Leriglitazone
Arm Type
Active Comparator
Arm Description
Leriglitazone Treatment
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Placebo
Intervention Type
Drug
Intervention Name(s)
Leriglitazone
Intervention Description
Leriglitazone at a strength of 15 mg/ml. Once-daily dosing at an initial volume of 10 ml
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Placebo will match the study drug visually and by taste
Primary Outcome Measure Information:
Title
The primary endpoint will be the time to death or the subject becoming bedridden with a requirement for permanent ventilatory support, wichever comes earlier, in subjects treated with leriglitazone compared to placebo.
Description
The primary endpoint will be the time to death or the subject becoming bedridden with a requirement for permanent ventilatory support, wichever comes earlier, in subjects treated with leriglitazone compared to placebo.
Time Frame
Interim analysis 1 (at 18 months of treatment)
Title
The primary endpoint will be the time to death or the subject becoming bedridden with a requirement for permanent ventilatory support, wichever comes earlier, in subjects treated with leriglitazone compared to placebo.
Description
The primary endpoint will be the time to death or the subject becoming bedridden with a requirement for permanent ventilatory support, wichever comes earlier, in subjects treated with leriglitazone compared to placebo.
Time Frame
Interim analysis 2 (at 27 months of treatment)
Title
The primary endpoint will be the time to death or the subject becoming bedridden with a requirement for permanent ventilatory support, wichever comes earlier, in subjects treated with leriglitazone compared to placebo.
Description
The primary endpoint will be the time to death or the subject becoming bedridden with a requirement for permanent ventilatory support, wichever comes earlier, in subjects treated with leriglitazone compared to placebo.
Time Frame
Final analysis (at 36 months of treatment)
Secondary Outcome Measure Information:
Title
Change from Baseline in Loes Score.
Description
Change from Baseline in Loes Score. Loes Score Minimum = 0 Maximum =34 0 better outcome (healthy) 34 worst osutcome
Time Frame
Interim analysis 1 (at 18 months of treatment)
Title
Change from Baseline in Loes Score
Description
Change from Baseline in Loes Score. Loes Score Minimum = 0 Maximum =34 0 better outcome (healthy) 34 worst osutcome
Time Frame
Interim analysis 2 (at 27 months of treatment)
Title
Change from Baseline in Loes Score
Description
Change from Baseline in Loes Score. Loes Score Minimum = 0 Maximum =34 0 better outcome (healthy) 34 worst osutcome
Time Frame
Final analysis (at 36 months of treatment)

10. Eligibility

Sex
Male
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Key Inclusion Criteria: Subject is male and aged ≥18 years. Subject has progressive cALD, defined as GdE+ brain lesions. Subjects for whom HSCT is not recommended by the investigator or subject is not willing to undergo HSCT. Subject has a Loes score ≥0.5 and ≤12 at Screening. Subject does not have major functional disability in the Major Functional Disabilities-Neurological Function Score (MFD-NFS), except for "wheelchair bound" or "total incontinence", which will be allowed as these are considered expected symptoms of AMN in the time course of the disease Subject does not have major cognitive impairment which would impair his ability to take part in the study as determined by the investigator at screening. Key Exclusion Criteria: Subject who had previous bone marrow transplantation (HSCT) or treatment with ex-vivo gene therapy (eli-Cel). Subject has known type 1 or type 2 diabetes. Subject has known hypersensitivity or intolerance to pioglitazone or any other thiazolidinedione. Subject is taking or has taken honokiol, pioglitazone, or other thiazolidinediones within 3 months prior to Screening. Subject with current participation in another interventional clinical study or within 1 month prior to Screening. Subject with other medical, neuropsychiatric or social conditions that, in the opinion of the investigator, are likely to adversely affect the risk-benefit of study participation, interfere with study compliance, or confound the study results.
Facility Information:
Facility Name
Stanford University Medical Center
City
Palo Alto
State/Province
California
ZIP/Postal Code
94304-5978
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Jacinda Sampson
Phone
650-723-0993
Email
jacindas@stanford.edu
Facility Name
Neuro Medicine Hospital / UF Health
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32608
Country
United States
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Hans Shuhaiber
Phone
312-286-1059
Email
hshuhaiber@ufl.edu
Facility Name
Kennedy Krieger Institute
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21205
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Ali Fatemi
Phone
443-923-2769
Email
fatemi@kennedykrieger.org
Facility Name
Massachusetts General Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Reza Seyedsadjadi
Phone
919-280-5582
Email
rseyedsadjadi@mgh.harvard.edu
Facility Name
University of Minnesota
City
Minnesota
State/Province
Minnesota
ZIP/Postal Code
55455
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Troy Lund
Phone
612-625-6687
Email
lundx072@umn.edu
Facility Name
Health University of Utah
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84112
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Joshua Bonksowsky
Phone
801-213-3599
Email
joshua.bonkowsky@hsc.utah.edu
Facility Name
Hospital Austral
City
Buenos Aires
Country
Argentina
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Hernan Amartino
Facility Name
Federal University of Sao Paulo
City
São Paulo
Country
Brazil
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Paulo Sgobbi

12. IPD Sharing Statement

Learn more about this trial

A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adult Male Subjects With Cerebral Adrenoleukodystrophy

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