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A Study of ATL1102 or Placebo in Participants With Non-ambulatory Duchenne Muscular Dystrophy

Primary Purpose

Duchenne Muscular Dystrophy

Status
Recruiting
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
ATL1102 25mg
ATL1102 50mg
Placebo
Sponsored by
Antisense Therapeutics Limited
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Duchenne Muscular Dystrophy focused on measuring Non-ambulatory, DMD

Eligibility Criteria

10 Years - 17 Years (Child)MaleDoes not accept healthy volunteers

Key Inclusion Criteria: Has a clinical diagnosis of DMD confirmed by validated genetic testing Is considered to be non-ambulatory, defined as unable to walk 10 meters without assistance or help at Screening. Male aged 10 to less than 18 years, at the time of Screening. Body weight of at least 25 kg at Screening. If receiving corticosteroid therapy, therapy was initiated at least six months prior to the baseline visit and a stable daily dose for at least 3 months prior to baseline Participant has a Performance of Upper Limb Module for DMD 2.0 (PUL 2.0) Entry Item A score ≥2. Able to perform spirometry and has sufficient Respiratory function defined as reproducible percent predicted FVC ≥50%. Has adequate cardiac function defined as left ventricular ejection fraction (LVEF) ≥45% by echocardiogram and if receiving cardiac medication, must be currently on a stable regimen and doses of cardiac therapy (at least 3 months prior to baseline Day 1) Participant and their parent/guardian/carer are willing and able to comply with scheduled visits, study medication administration and study procedures. Key Exclusion Criteria: Participation in another clinical trial (non-interventional) or administration of any investigational product or experimental product within 12 weeks or 5 half-lives (whichever is longer) preceding Day 1. Exposure to more than 3 investigational products within the 12 months prior to Day 1. History of clinically significant bleeding or coagulation abnormalities or clinically significant abnormal coagulation parameters. Currently receiving antiplatelet or anticoagulant therapy or has taken medication with an antiplatelet or anticoagulant effect within 4 weeks prior Day 1 Any evidence of clinically significant structural or functional heart abnormality (cardiomyopathy that is managed by ACEi or beta blockers is acceptable provided the LVEF inclusion criterion is met). Known history of or a positive test for hepatitis B surface antigen (HBsAg), hepatitis C (HCV) antibodies, human immunodeficiency virus (HIV) antibodies at Screening. Evidence of renal impairment and/or cystatin C >1.4 mg/L. Received a live vaccine (including intranasal influenza vaccine) within 4 weeks prior to Day 1 or planned live vaccination during the study period. Asthma (if requiring regular medication), bronchitis/chronic obstructive pulmonary disease (COPD), bronchiectasis, emphysema, pneumonia or the presence of any non-DMD respiratory illness that affects PEF and FVC or other respiratory measures. Requires day-time assisted mechanical or non-invasive ventilation (NIV) (night time NIV is permitted). Chronic use (daily intake >14 days), within one month of Day 1, of beta-2 agonists or any use of other bronchodilating medication (e.g., inhaled steroids, sympathomimetics, anticholinergics). Used carnitine, creatine, glutamine, oxatomide, idebenone or other forms of coenzyme Q10 or vitamin E or any other nutritional or antioxidant supplements or herbal medicines or anabolic steroids other than standard corticosteroids or puberty testosterone supplementation within 4 weeks of Day 1. Has an increased risk for opportunistic infections or systemic medical conditions resulting in significantly compromised immune system function

Sites / Locations

  • The Children's Hospital at Westmead
  • Queensland Children's Hospital
  • Royal Childrens HospitalRecruiting
  • UMHAT Aleksandrovska Neurology clinicRecruiting
  • Marmara Universitesi Pendik Egitim ve Arastirma HastanesiRecruiting
  • Eskisehir Osmangazi Universitesi Tip FakultesiRecruiting
  • Yeditepe Üniversitesi Hastanesi [Yeditepe University Hospital]Recruiting
  • Birmingham Heartlands Hospital
  • The General Infirmary at Leeds, Leeds Teaching Hospital NHS Trust
  • University College London (UCL) - Great Ormond Street Institute of Child Health (ICH)Recruiting
  • Manchester Royal InfirmaryRecruiting
  • The Robert Jones and Agnes Hunt Orthopaedic Hospital
  • Greater Glasgow and Clyde Yorkhill Hospital
  • Alder Hey Children's Hospital

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Experimental

Placebo Comparator

Arm Label

ATL1102 25mg

ATL1102 50mg

Placebo

Arm Description

ATL1102 25mg administered subcutaneously once weekly

ATL1102 50mg administered subcutaneously once weekly

Placebo is administered subcutaneously once weekly

Outcomes

Primary Outcome Measures

Change in the Performance of Upper Limb (PUL) 2.0 score from baseline to Week 25 (blinded treatment period).
The PUL is an assessment used to evaluate the upper limb strength for individuals with DMD where a higher score indicates a better outcome with a minimum of 0 and a maximum score of 42
Change in the Performance of Upper Limb (PUL) 2.0 score from Week 25 to Week 49 (open label treatment period).
The PUL is an assessment used to evaluate the upper limb strength for individuals with DMD where a higher score indicates a better outcome with a minimum of 0 and a maximum score of 42
Change in the Performance of Upper Limb (PUL) 2.0 score from baseline to Week 49 (combined treatment period).
The PUL is an assessment used to evaluate the upper limb strength for individuals with DMD where a higher score indicates a better outcome with a minimum of 0 and a maximum score of 42
Safety measured by the incidence and frequency of adverse events, serious adverse events and suspected unexpected adverse events from baseline to Week 65
An Adverse Event is any untoward medical occurrence in a participant and does not necessarily have to have a causal relationship with the intervention.

Secondary Outcome Measures

Change in the grip strength of the hand from baseline to Week 25 using a handheld dynamometer tool (MyoGrip) (blinded treatment period).
The handheld dynamometer tool (MyoGrip) use strain gauge technology to measure the hand strength in Kilograms exerted by the participants with higher recordings indicating greater hand strength.
Change in the pinch strength of the fingers from baseline to Week 25 using handheld dynamometer tool (MyoPinch) (blinded treatment period).
The handheld dynamometer tool (MyoPinch) use strain gauge technology to measure the pinch strength of the fingers in Kilograms exerted by the participants with higher recordings indicating greater hand strength.
Change in the respiratory function assessed by Forced Vital Capacity (FVC) from baseline to Week 25 (blinded treatment period).
The percent predicted for Forced Vital Capacity (FVC%p) will be calculated at multiple timepoints after respiratory function is assessed using spirometry tests
Change in the respiratory function assessed by peak expiratory flow (PEF) from baseline to Week 25 (blinded treatment period).
The percent predicted for peak expiratory flow (PEF%p) will be calculated at multiple timepoints after respiratory function is assessed using spirometry tests
Change in the Paediatric Quality of Life (PedsQL™) questionnaire Duchenne Muscular Dystrophy (DMD) Module from baseline to Week 25 (blinded treatment period).
Health related quality of life is assessed by percentage of change in the score collected in the Paediatric Quality of Life (PedsQL™) Duchenne Muscular Dystrophy (DMD) Module for participants and parents at multiple timepoints. A higher score indicates a better health related quality of life with a minimum of 0 and a maximum score of 100.
Safety measured by the incidence and frequency of adverse events, serious adverse events and suspected unexpected adverse events from baseline to Week 25 (blinded treatment period).
An Adverse Event is any untoward medical occurrence in a participant and does not necessarily have to have a causal relationship with the intervention.
Maximum and minimum plasma concentration (Cmax and Cmin) for ATL1102 over multiple timepoints
Pharmacokinetic evaluation to evaluate dose response
Area under the plasma concentration time curve (AUC) for ATL1102 over multiple timepoints
Pharmacokinetic evaluation to evaluate dose concentration over time
Time to Cmax and Cmin for ATL1102 over multiple timepoints
Pharmacokinetic evaluation to evaluate concentration of ATL1102
The terminal half life for ATL1102
Pharmacokinetic evaluation to evaluate the time for the ATL1102 concentration to reduce by half
Change in the grip strength of the hand from Week 25 to Week 49 using a handheld dynamometer tool (MyoGrip) (open label treatment period).
The handheld dynamometer tool (MyoGrip) use strain gauge technology to measure the hand strength in Kilograms exerted by the participants with higher recordings indicating greater hand strength.
Change in the pinch strength of the fingers from Week 25 to Week 49 using handheld dynamometer tool (MyoPinch) (open label treatment period).
The handheld dynamometer tool (MyoPinch) use strain gauge technology to measure the pinch strength of the fingers in Kilograms exerted by the participants with higher recordings indicating greater hand strength.
Change in the respiratory function assessed by Forced Vital Capacity (FVC) from Week 25 to Week 49 (open label treatment period).
The percent predicted for Forced Vital Capacity (FVC%p) will be calculated at multiple timepoints after respiratory function is assessed using spirometry tests
Change in the respiratory function assessed by peak expiratory flow (PEF) from Week 25 to Week 49 (open label treatment period).
The percent predicted for peak expiratory flow (PEF%p) will be calculated at multiple timepoints after respiratory function is assessed using spirometry tests
Change in the Paediatric Quality of Life (PedsQL) questionnaire Duchenne Muscular Dystrophy (DMD) Module from Week 25 to Week 49 (open label treatment period).
Health related quality of life is assessed by percentage of change in the score collected in the Paediatric Quality of Life (PedsQL™) Duchenne Muscular Dystrophy (DMD) Module for participants and parents at multiple timepoints. A higher score indicates a better health related quality of life with a minimum of 0 and a maximum score of 100.
Change in the grip strength of the hand from baseline to Week 49 using a handheld dynamometer tool (MyoGrip) (combined treatment period).
The handheld dynamometer tool (MyoGrip) use strain gauge technology to measure the hand strength in Kilograms exerted by the participants with higher recordings indicating greater hand strength.
Change in the pinch strength of the fingers from Baseline to Week 49 using handheld dynamometer tool (MyoPinch) (combined treatment period).
The handheld dynamometer tool (MyoPinch) use strain gauge technology to measure the pinch strength of the fingers in Kilograms exerted by the participants with higher recordings indicating greater hand strength.
Change in the respiratory function assessed by Forced Vital Capacity (FVC) from Baseline to Week 49 (combined treatment period).
The percent predicted for Forced Vital Capacity (FVC%p) will be calculated at multiple timepoints after respiratory function is assessed using spirometry tests
Change in the respiratory function assessed by peak expiratory flow (PEF) from baseline to Week 49 (combined treatment period).
The percent predicted for peak expiratory flow (PEF%p) will be calculated at multiple timepoints after respiratory function is assessed using spirometry tests
Change in the Paediatric Quality of Life (PedsQL) questionnaire Duchenne Muscular Dystrophy (DMD) Module from Baseline to Week 49 (combined treatment period).
Health related quality of life is assessed by percentage of change in the score collected in the Paediatric Quality of Life (PedsQL™) Duchenne Muscular Dystrophy (DMD) Module for participants and parents at multiple timepoints. A higher score indicates a better health related quality of life with a minimum of 0 and a maximum score of 100.

Full Information

First Posted
June 5, 2023
Last Updated
October 18, 2023
Sponsor
Antisense Therapeutics Limited
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1. Study Identification

Unique Protocol Identification Number
NCT05938023
Brief Title
A Study of ATL1102 or Placebo in Participants With Non-ambulatory Duchenne Muscular Dystrophy
Official Title
A Multicentre, Randomised, Double-blind, Placebo-controlled and Open Label Extension Study to Assess the Efficacy, Safety, and Pharmacokinetic Profile of of ATL1102 in Non-ambulatory Participants With Duchenne Muscular Dystrophy
Study Type
Interventional

2. Study Status

Record Verification Date
August 2023
Overall Recruitment Status
Recruiting
Study Start Date
May 18, 2023 (Actual)
Primary Completion Date
November 13, 2024 (Anticipated)
Study Completion Date
March 5, 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Antisense Therapeutics Limited

4. Oversight

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

5. Study Description

Brief Summary
This Phase IIb study is a two part, multicenter study to evaluate the efficacy, safety, pharmacokinetics and pharmacodynamics of ATL1102 in non-ambulant boys with Duchenne Muscular Dystrophy aged 10 to <18 years old. The study includes a randomised, double-blind, placebo-controlled treatment period (Part A), followed by an open labelled treatment period (Part B).
Detailed Description
This Phase IIb study is a two part, multicenter study to evaluate the efficacy, safety, pharmacokinetics and pharmacodynamics of ATL1102 and will enroll 45 non-ambulant boys with Duchenne Muscular Dystrophy (DMD) aged 10 to <18 years old. During the 24 week randomised, double-blind, placebo-controlled treatment period (Part A) participants will be enrolled and randomised to receive either ATL1102 25mg, ATL1102 50mg or matched placebo in a 1:1:1 ratio given as a weekly subcutaneous injection. Participants will then continue to the 24 week Open Labelled Treatment Period (Part B) and continue to receive ATL1102 25mg or ATL1102 50mg for a further 24 weeks. Participants on placebo in Part A will transition to ATL1102. The study will consist of a 4 week screening period, 24 week randomised, double-blind, placebo-controlled treatment period (Part A), 24 week open label treatment period (Part B) and 16 week follow up period.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Duchenne Muscular Dystrophy
Keywords
Non-ambulatory, DMD

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Model Description
randomized, double-blind, placebo controlled treatment periods followed by open labelled treatment period
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
45 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
ATL1102 25mg
Arm Type
Experimental
Arm Description
ATL1102 25mg administered subcutaneously once weekly
Arm Title
ATL1102 50mg
Arm Type
Experimental
Arm Description
ATL1102 50mg administered subcutaneously once weekly
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Placebo is administered subcutaneously once weekly
Intervention Type
Drug
Intervention Name(s)
ATL1102 25mg
Intervention Description
Dose and scheduled as specified in the Arm description
Intervention Type
Drug
Intervention Name(s)
ATL1102 50mg
Intervention Description
Dose and scheduled as specified in the Arm description
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Dose and scheduled as specified in the Arm description
Primary Outcome Measure Information:
Title
Change in the Performance of Upper Limb (PUL) 2.0 score from baseline to Week 25 (blinded treatment period).
Description
The PUL is an assessment used to evaluate the upper limb strength for individuals with DMD where a higher score indicates a better outcome with a minimum of 0 and a maximum score of 42
Time Frame
25 weeks
Title
Change in the Performance of Upper Limb (PUL) 2.0 score from Week 25 to Week 49 (open label treatment period).
Description
The PUL is an assessment used to evaluate the upper limb strength for individuals with DMD where a higher score indicates a better outcome with a minimum of 0 and a maximum score of 42
Time Frame
49 weeks
Title
Change in the Performance of Upper Limb (PUL) 2.0 score from baseline to Week 49 (combined treatment period).
Description
The PUL is an assessment used to evaluate the upper limb strength for individuals with DMD where a higher score indicates a better outcome with a minimum of 0 and a maximum score of 42
Time Frame
49 weeks
Title
Safety measured by the incidence and frequency of adverse events, serious adverse events and suspected unexpected adverse events from baseline to Week 65
Description
An Adverse Event is any untoward medical occurrence in a participant and does not necessarily have to have a causal relationship with the intervention.
Time Frame
65 weeks
Secondary Outcome Measure Information:
Title
Change in the grip strength of the hand from baseline to Week 25 using a handheld dynamometer tool (MyoGrip) (blinded treatment period).
Description
The handheld dynamometer tool (MyoGrip) use strain gauge technology to measure the hand strength in Kilograms exerted by the participants with higher recordings indicating greater hand strength.
Time Frame
25 weeks
Title
Change in the pinch strength of the fingers from baseline to Week 25 using handheld dynamometer tool (MyoPinch) (blinded treatment period).
Description
The handheld dynamometer tool (MyoPinch) use strain gauge technology to measure the pinch strength of the fingers in Kilograms exerted by the participants with higher recordings indicating greater hand strength.
Time Frame
25 weeks
Title
Change in the respiratory function assessed by Forced Vital Capacity (FVC) from baseline to Week 25 (blinded treatment period).
Description
The percent predicted for Forced Vital Capacity (FVC%p) will be calculated at multiple timepoints after respiratory function is assessed using spirometry tests
Time Frame
25 weeks
Title
Change in the respiratory function assessed by peak expiratory flow (PEF) from baseline to Week 25 (blinded treatment period).
Description
The percent predicted for peak expiratory flow (PEF%p) will be calculated at multiple timepoints after respiratory function is assessed using spirometry tests
Time Frame
25 weeks
Title
Change in the Paediatric Quality of Life (PedsQL™) questionnaire Duchenne Muscular Dystrophy (DMD) Module from baseline to Week 25 (blinded treatment period).
Description
Health related quality of life is assessed by percentage of change in the score collected in the Paediatric Quality of Life (PedsQL™) Duchenne Muscular Dystrophy (DMD) Module for participants and parents at multiple timepoints. A higher score indicates a better health related quality of life with a minimum of 0 and a maximum score of 100.
Time Frame
25 weeks
Title
Safety measured by the incidence and frequency of adverse events, serious adverse events and suspected unexpected adverse events from baseline to Week 25 (blinded treatment period).
Description
An Adverse Event is any untoward medical occurrence in a participant and does not necessarily have to have a causal relationship with the intervention.
Time Frame
25 weeks
Title
Maximum and minimum plasma concentration (Cmax and Cmin) for ATL1102 over multiple timepoints
Description
Pharmacokinetic evaluation to evaluate dose response
Time Frame
65 weeks
Title
Area under the plasma concentration time curve (AUC) for ATL1102 over multiple timepoints
Description
Pharmacokinetic evaluation to evaluate dose concentration over time
Time Frame
65 weeks
Title
Time to Cmax and Cmin for ATL1102 over multiple timepoints
Description
Pharmacokinetic evaluation to evaluate concentration of ATL1102
Time Frame
65 weeks
Title
The terminal half life for ATL1102
Description
Pharmacokinetic evaluation to evaluate the time for the ATL1102 concentration to reduce by half
Time Frame
65 weeks
Title
Change in the grip strength of the hand from Week 25 to Week 49 using a handheld dynamometer tool (MyoGrip) (open label treatment period).
Description
The handheld dynamometer tool (MyoGrip) use strain gauge technology to measure the hand strength in Kilograms exerted by the participants with higher recordings indicating greater hand strength.
Time Frame
49 weeks
Title
Change in the pinch strength of the fingers from Week 25 to Week 49 using handheld dynamometer tool (MyoPinch) (open label treatment period).
Description
The handheld dynamometer tool (MyoPinch) use strain gauge technology to measure the pinch strength of the fingers in Kilograms exerted by the participants with higher recordings indicating greater hand strength.
Time Frame
49 weeks
Title
Change in the respiratory function assessed by Forced Vital Capacity (FVC) from Week 25 to Week 49 (open label treatment period).
Description
The percent predicted for Forced Vital Capacity (FVC%p) will be calculated at multiple timepoints after respiratory function is assessed using spirometry tests
Time Frame
49 weeks
Title
Change in the respiratory function assessed by peak expiratory flow (PEF) from Week 25 to Week 49 (open label treatment period).
Description
The percent predicted for peak expiratory flow (PEF%p) will be calculated at multiple timepoints after respiratory function is assessed using spirometry tests
Time Frame
49 weeks
Title
Change in the Paediatric Quality of Life (PedsQL) questionnaire Duchenne Muscular Dystrophy (DMD) Module from Week 25 to Week 49 (open label treatment period).
Description
Health related quality of life is assessed by percentage of change in the score collected in the Paediatric Quality of Life (PedsQL™) Duchenne Muscular Dystrophy (DMD) Module for participants and parents at multiple timepoints. A higher score indicates a better health related quality of life with a minimum of 0 and a maximum score of 100.
Time Frame
49 weeks
Title
Change in the grip strength of the hand from baseline to Week 49 using a handheld dynamometer tool (MyoGrip) (combined treatment period).
Description
The handheld dynamometer tool (MyoGrip) use strain gauge technology to measure the hand strength in Kilograms exerted by the participants with higher recordings indicating greater hand strength.
Time Frame
49 weeks
Title
Change in the pinch strength of the fingers from Baseline to Week 49 using handheld dynamometer tool (MyoPinch) (combined treatment period).
Description
The handheld dynamometer tool (MyoPinch) use strain gauge technology to measure the pinch strength of the fingers in Kilograms exerted by the participants with higher recordings indicating greater hand strength.
Time Frame
49 weeks
Title
Change in the respiratory function assessed by Forced Vital Capacity (FVC) from Baseline to Week 49 (combined treatment period).
Description
The percent predicted for Forced Vital Capacity (FVC%p) will be calculated at multiple timepoints after respiratory function is assessed using spirometry tests
Time Frame
49 weeks
Title
Change in the respiratory function assessed by peak expiratory flow (PEF) from baseline to Week 49 (combined treatment period).
Description
The percent predicted for peak expiratory flow (PEF%p) will be calculated at multiple timepoints after respiratory function is assessed using spirometry tests
Time Frame
49 weeks
Title
Change in the Paediatric Quality of Life (PedsQL) questionnaire Duchenne Muscular Dystrophy (DMD) Module from Baseline to Week 49 (combined treatment period).
Description
Health related quality of life is assessed by percentage of change in the score collected in the Paediatric Quality of Life (PedsQL™) Duchenne Muscular Dystrophy (DMD) Module for participants and parents at multiple timepoints. A higher score indicates a better health related quality of life with a minimum of 0 and a maximum score of 100.
Time Frame
49 weeks
Other Pre-specified Outcome Measures:
Title
Changes in lymphocyte populations to assess pharmacodynamic effects of ATL1102 from baseline to Week 57
Description
Lymphocyte population (cells/L) including cells expressing CD49d will be evaluated at multiple timepoints during the study utilizing chip cytometry.
Time Frame
57 weeks

10. Eligibility

Sex
Male
Gender Based
Yes
Gender Eligibility Description
DMD is a disease that predominantly affects males
Minimum Age & Unit of Time
10 Years
Maximum Age & Unit of Time
17 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Key Inclusion Criteria: Has a clinical diagnosis of DMD confirmed by validated genetic testing Is considered to be non-ambulatory, defined as unable to walk 10 meters without assistance or help at Screening. Male aged 10 to less than 18 years, at the time of Screening. Body weight of at least 25 kg at Screening. If receiving corticosteroid therapy, therapy was initiated at least six months prior to the baseline visit and a stable daily dose for at least 3 months prior to baseline Participant has a Performance of Upper Limb Module for DMD 2.0 (PUL 2.0) Entry Item A score ≥2. Able to perform spirometry and has sufficient Respiratory function defined as reproducible percent predicted FVC ≥50%. Has adequate cardiac function defined as left ventricular ejection fraction (LVEF) ≥45% by echocardiogram and if receiving cardiac medication, must be currently on a stable regimen and doses of cardiac therapy (at least 3 months prior to baseline Day 1) Participant and their parent/guardian/carer are willing and able to comply with scheduled visits, study medication administration and study procedures. Key Exclusion Criteria: Participation in another clinical trial (non-interventional) or administration of any investigational product or experimental product within 12 weeks or 5 half-lives (whichever is longer) preceding Day 1. Exposure to more than 3 investigational products within the 12 months prior to Day 1. History of clinically significant bleeding or coagulation abnormalities or clinically significant abnormal coagulation parameters. Currently receiving antiplatelet or anticoagulant therapy or has taken medication with an antiplatelet or anticoagulant effect within 4 weeks prior Day 1 Any evidence of clinically significant structural or functional heart abnormality (cardiomyopathy that is managed by ACEi or beta blockers is acceptable provided the LVEF inclusion criterion is met). Known history of or a positive test for hepatitis B surface antigen (HBsAg), hepatitis C (HCV) antibodies, human immunodeficiency virus (HIV) antibodies at Screening. Evidence of renal impairment and/or cystatin C >1.4 mg/L. Received a live vaccine (including intranasal influenza vaccine) within 4 weeks prior to Day 1 or planned live vaccination during the study period. Asthma (if requiring regular medication), bronchitis/chronic obstructive pulmonary disease (COPD), bronchiectasis, emphysema, pneumonia or the presence of any non-DMD respiratory illness that affects PEF and FVC or other respiratory measures. Requires day-time assisted mechanical or non-invasive ventilation (NIV) (night time NIV is permitted). Chronic use (daily intake >14 days), within one month of Day 1, of beta-2 agonists or any use of other bronchodilating medication (e.g., inhaled steroids, sympathomimetics, anticholinergics). Used carnitine, creatine, glutamine, oxatomide, idebenone or other forms of coenzyme Q10 or vitamin E or any other nutritional or antioxidant supplements or herbal medicines or anabolic steroids other than standard corticosteroids or puberty testosterone supplementation within 4 weeks of Day 1. Has an increased risk for opportunistic infections or systemic medical conditions resulting in significantly compromised immune system function
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Antisense Therapeutics
Phone
+61 3 9827 8999
Email
clinicaltrials@antisense.com.au
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Thomas Voit
Organizational Affiliation
UCL Great Ormond Street Institute of Child Health
Official's Role
Principal Investigator
Facility Information:
Facility Name
The Children's Hospital at Westmead
City
Westmead
State/Province
New South Wales
ZIP/Postal Code
2145
Country
Australia
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Michelle Lorentzos
Facility Name
Queensland Children's Hospital
City
South Brisbane
State/Province
Queensland
ZIP/Postal Code
4101
Country
Australia
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Anita Cairns
Facility Name
Royal Childrens Hospital
City
Melbourne
State/Province
Victoria
ZIP/Postal Code
3052
Country
Australia
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Ian Woodcock
Facility Name
UMHAT Aleksandrovska Neurology clinic
City
Sofia
ZIP/Postal Code
1432
Country
Bulgaria
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Ivaylov L Tarnev
Facility Name
Marmara Universitesi Pendik Egitim ve Arastirma Hastanesi
City
Pendik
State/Province
Istanbul
ZIP/Postal Code
34899
Country
Turkey
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Elif Acar Arslan Arslan
Facility Name
Eskisehir Osmangazi Universitesi Tip Fakultesi
City
Eskisehir
ZIP/Postal Code
26040
Country
Turkey
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Coskun Yarar
Facility Name
Yeditepe Üniversitesi Hastanesi [Yeditepe University Hospital]
City
Istanbul
Country
Turkey
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Haluk Topalogu
Facility Name
Birmingham Heartlands Hospital
City
Birmingham
State/Province
England
ZIP/Postal Code
B9 5SS
Country
United Kingdom
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Zoya Alhaswani
Facility Name
The General Infirmary at Leeds, Leeds Teaching Hospital NHS Trust
City
Leeds
State/Province
England
ZIP/Postal Code
LS2 9NS
Country
United Kingdom
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Anne-Marie Childs
Facility Name
University College London (UCL) - Great Ormond Street Institute of Child Health (ICH)
City
London
State/Province
England
ZIP/Postal Code
WC1N 3JH
Country
United Kingdom
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Giovanni Baranello
Facility Name
Manchester Royal Infirmary
City
Manchester
State/Province
England
ZIP/Postal Code
M13 9WL
Country
United Kingdom
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Gary McCullagh
Facility Name
The Robert Jones and Agnes Hunt Orthopaedic Hospital
City
Gobowen
State/Province
Shropshire
ZIP/Postal Code
SY10 7AG
Country
United Kingdom
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Tracey Willis
Facility Name
Greater Glasgow and Clyde Yorkhill Hospital
City
Glasgow
ZIP/Postal Code
G3 8SJ
Country
United Kingdom
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Iain Horrocks
Facility Name
Alder Hey Children's Hospital
City
Liverpool
ZIP/Postal Code
L12 2AP
Country
United Kingdom
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Rajesh Madhu

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

A Study of ATL1102 or Placebo in Participants With Non-ambulatory Duchenne Muscular Dystrophy

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