Back to resultsAMX0035 in Adult Patients With Wolfram Syndrome
Primary Purpose
Wolfram Syndrome
Status
Recruiting
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
AMX0035
Sponsored by
About this trial
This is an interventional treatment trial for Wolfram Syndrome focused on measuring Wolfram, Wolfram syndrome
Eligibility Criteria
Key Inclusion Criteria: Definitive diagnosis of Wolfram syndrome Insulin dependent diabetes mellitus due to Wolfram syndrome At least 17 years of age Participant must be willing to wear a CGM device for the duration of the study Key Exclusion Criteria: Presence of pathologies that can alter the enterohepatic circulation of bile acids (e.g., ileal resection and stoma, regional ileitis) Any history of heart failure per New York Heart Association (NYHA) History of or family history of breast and/or ovarian cancer Participant under severe salt restriction where the added salt intake due to treatment would put the patient at risk, in the Investigator's judgment Received treatment with any investigational drug or device within the 30 days (or 5 half-lives, whichever is longer) prior to first dose at Day 1 Previous treatment with gene or cellular therapy
Sites / Locations
- Washington UniversityRecruiting
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
AMX0035
Arm Description
AMX0035 administered by mouth for 24 weeks: Once daily for first 3 weeks and then twice daily for the remainder of the study if tolerated by participant
Outcomes
Primary Outcome Measures
To evaluate the effect of AMX0035 during a 0-240 minutes mixed-meal tolerance test (MMTT)
C-peptide AUC response at Week 24
Change from Baseline in C-peptide at Week 24
To assess the safety and tolerability of AMX0035in adult participants with Wolfram syndrome
Incidence and severity of Adverse Events and Serious Adverse Events
Incidence of abnormalities in clinical laboratory assessments
Secondary Outcome Measures
To assess the effect size of AMX0035 on visual acuity
Change from Baseline on best-corrected visual acuity (Logmar) at Week 24
To evaluate the effect of AMX0035 on total daily insulin dose
Change from Baseline of exogenous insulin dose to Week 24
To evaluate the effect of AMX0035 on glucose range
Change from Baseline to Week 24
Time in good range
Time below range
Time above range
To evaluate the effect of AMX0035 on HbA1c levels
Change from Baseline to Week 24 in HbA1c level
Full Information
NCT ID
NCT05676034
First Posted
December 22, 2022
Last Updated
April 24, 2023
Sponsor
Amylyx Pharmaceuticals Inc.
1. Study Identification
Unique Protocol Identification Number
NCT05676034
Brief Title
AMX0035 in Adult Patients With Wolfram Syndrome
Official Title
A Phase II Study of Safety and Efficacy of AMX0035 in Adult Patients With Wolfram Syndrome
Study Type
Interventional
2. Study Status
Record Verification Date
April 2023
Overall Recruitment Status
Recruiting
Study Start Date
March 3, 2023 (Actual)
Primary Completion Date
February 2024 (Anticipated)
Study Completion Date
February 2024 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Amylyx Pharmaceuticals Inc.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
This study is an open label Phase II study to evaluate the safety and efficacy of AMX0035 in adults with Wolfram syndrome.
Detailed Description
AMX0035 is a combination therapy designed to reduce neuronal death through blockade of key cellular death pathways originating in the mitochondria and endoplasmic reticulum (ER). This clinical trial is designed to demonstrate that treatment is safe, tolerable, and to evaluate the effect of AMX0035 on residual beta cell functions by monitoring c-peptide levels during a 0-240 minute mixed-meal tolerance test. The trial will also assess the effects of AMX0035 on changes to diabetic measurements including daily insulin dose, time in good glucose range, and HbA1c levels. Effect on best-corrected visual acuity in both eyes will also be evaluated.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Wolfram Syndrome
Keywords
Wolfram, Wolfram syndrome
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Model Description
A 24 week open-label treatment period of AMX0035 administered twice daily
Masking
None (Open Label)
Allocation
N/A
Enrollment
12 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
AMX0035
Arm Type
Experimental
Arm Description
AMX0035 administered by mouth for 24 weeks: Once daily for first 3 weeks and then twice daily for the remainder of the study if tolerated by participant
Intervention Type
Drug
Intervention Name(s)
AMX0035
Other Intervention Name(s)
Proprietary formulation of taurursodiol and sodium phenylbutyrate
Intervention Description
AMX0035
Primary Outcome Measure Information:
Title
To evaluate the effect of AMX0035 during a 0-240 minutes mixed-meal tolerance test (MMTT)
Description
C-peptide AUC response at Week 24
Change from Baseline in C-peptide at Week 24
Time Frame
28 weeks
Title
To assess the safety and tolerability of AMX0035in adult participants with Wolfram syndrome
Description
Incidence and severity of Adverse Events and Serious Adverse Events
Incidence of abnormalities in clinical laboratory assessments
Time Frame
28 weeks
Secondary Outcome Measure Information:
Title
To assess the effect size of AMX0035 on visual acuity
Description
Change from Baseline on best-corrected visual acuity (Logmar) at Week 24
Time Frame
24 weeks
Title
To evaluate the effect of AMX0035 on total daily insulin dose
Description
Change from Baseline of exogenous insulin dose to Week 24
Time Frame
24 weeks
Title
To evaluate the effect of AMX0035 on glucose range
Description
Change from Baseline to Week 24
Time in good range
Time below range
Time above range
Time Frame
24 weeks
Title
To evaluate the effect of AMX0035 on HbA1c levels
Description
Change from Baseline to Week 24 in HbA1c level
Time Frame
24 weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
17 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Key Inclusion Criteria:
Definitive diagnosis of Wolfram syndrome
Insulin dependent diabetes mellitus due to Wolfram syndrome
At least 17 years of age
Participant must be willing to wear a CGM device for the duration of the study
Key Exclusion Criteria:
Presence of pathologies that can alter the enterohepatic circulation of bile acids (e.g., ileal resection and stoma, regional ileitis)
Any history of heart failure per New York Heart Association (NYHA)
History of or family history of breast and/or ovarian cancer
Participant under severe salt restriction where the added salt intake due to treatment would put the patient at risk, in the Investigator's judgment
Received treatment with any investigational drug or device within the 30 days (or 5 half-lives, whichever is longer) prior to first dose at Day 1
Previous treatment with gene or cellular therapy
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Amy Gauger
Phone
857-320-6200
Email
clinicaltrials@amylyx.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Mathias Leinders, Dr.
Organizational Affiliation
Amylyx Pharmaceuticals
Official's Role
Study Director
Facility Information:
Facility Name
Washington University
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Stacy Hurst, R, BSN, CDE
Phone
314-747-3294
Email
shurst@wustl.edu
First Name & Middle Initial & Last Name & Degree
Fumihiko Urano, MD, PhD
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
AMX0035 in Adult Patients With Wolfram Syndrome
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