Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy

NCT01484678 | Recruiting

• 4 other identifiers: IRB201700056-NR01AR056973176-2010OCR16243
• Study Type: observational
• Target: 550
• Locations: 1 country
• Sites: 3

Brief Summary

The purpose of this research study is to determine the potential of magnetic resonance imaging, spectroscopy, and whole body imaging to monitor disease progression and to serve as an objective outcome measure for clinical trials in Muscular Dystrophy (MD). The investigators will compare the muscles of ambulatory or non-ambulatory boys/men with DMD with muscles of healthy individuals of the same age and monitor disease progression in those with DMD over a 5-10 year period. The amount of muscle damage and fat that the investigators measure will also be related to performance in daily activities, such as walking and the loss of muscle strength. In a small group of subjects the investigators will also assess the effect of corticosteroid drugs on the muscle measurements. Additionally, the investigators will map the progression of Becker MD following adults with this rare disease. The primary objective is to conduct a multi-centered study to validate the potential of non-invasive magnetic resonance imaging and magnetic resonance spectroscopy to monitor disease progression and to serve as a noninvasive surrogate outcome measure for clinical trials in DMD and BMD. The secondary objective is to characterize the progressive involvement of the lower extremity, upper extremity, trunk/respiratory muscles in boys/men with DMD and BMD guiding clinical trials.

Conditions

Duchenne Muscular Dystrophy; Becker Muscular Dystrophy

Keywords

Duchenne Muscular Dystrophy; Becker Muscular Dystrophy; Magnetic Resonance Spectroscopy; Magnetic Resonance Imaging; Muscle

Outcome Measures

Primary Outcomes (3)

• Change from baseline in intramuscular lipid up to 3-10 years

  In BMD and DMD, the from baseline in intramuscular lipid of upper/ lower extremity and trunk/respiratory muscles, as well as composite measures. MR measures of intramuscular lipid will be measured in yearly intervals for a period up to 3-10 years.

  Change in baseline up to 3-10 years

• Change from baseline in muscle T2 up to 3 months in DMD

  In a subgroup of subjects the effect of corticosteroids on muscle T2 will be measured at 3 and 6 months. Muscle T2 is a noninvasive marker of muscle damage/inflammation and will be measured using MR. This substudy requires its own Primary and Secondary Outcome measures.

  Change in baseline up to 3 months

• Correlation between MR measures of intramuscular lipid, functional endpoints and histological markers.

  In both BMD and DMD, the correlation between MR measures and functional endpoints will be determined, as well as the ability of MR measures to predict future change and loss in function.

  Through study completion, an average of 1 year

Secondary Outcomes (3)

• Change from baseline in muscle T2 up to 5-10 years

  Change in baseline up to 5-10 years

• Change from baseline in muscle contractile area up to 5-10 years

  change in baseline up to 5-10 years

• Change from baseline in muscle T2 at 6 months

  Change in baseline up to 6 months

Study Arms (3)

Age Matched Controls

Age matched non-affected (non-DMD) boys \* This arm is full Age matched non-affected men, matched for men with Becker MD \*Recruiting

Boys/Men with DMD

This group will include ambulatory and non-ambulatory boys/men with Duchenne Muscular Dystrophy ranging from 5-30 years old. \*Recruiting

Adults with Becker MD

This group will include ambulatory and non-ambulatory men with Becker Muscular Dystrophy ranging from 18-62 years old. \* Recruiting

Eligibility Criteria

• Age: 5 Years - 62 Years
• Sex: male
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

Subjects are recruited from across the country. Investigators have a website (www.imagingNMD.org) and advertise the study nationally through list serves. The study will be advertised at the website of non-profit MD organizations. General information will be emailed to faculty and colleagues around the country. Fliers and brochures will be distributed in participating local clinics, MDA clinics, non-profit organizations, local neuromuscular clinics, and in strategic locations in associated hospitals. Age-matched healthy men will be recruited to match subjects with Becker MD from local and university communities. Individuals interested in this study are asked to contact the site clinical coordinator, who will complete a telephone screening interview to assess eligibility.

You may qualify if:

• \. Ambulatory and non-ambulatory males (ages 5-30 at baseline testing) previously diagnosed with DMD based on:
• clinical features with onset of symptoms before age five
• elevated serum creatine kinase level or
• absence of dystrophin expression, as determined by immunostain or western blot (\<2%) and/or DNA confirmation of a dystrophin mutation \*Subjects will not be excluded based on corticosteroid treatment or other clinical trials
• Ambulatory males (ages 18-62) without disease or injury to the lower extremities
• Specific recruitment of a subset of individuals with deletion mutations in the dystrophin gene involving either exon 51 or exon 45.
• \. Ambulatory males (ages 18-62) without disease or injury to the lower and/or upper extremities will be eligible to participate in this study

You may not qualify if:

• Males with a contraindication to an MR examination
• Males with unstable medical problems
• Males who are not able to cooperate during testing
• Males with a secondary condition that may impact muscle metabolism, muscle function or functional ability (i.e. cerebral palsy, endocrine disorders, mitochondrial disease)
• Daytime ventilation
• Implantable Cardioverter Defibrillator- (ICD) or pace maker
• Healthy boys/men who participate in competitive sports specific training in excess of 8 hours per week

Sponsors & Collaborators

• University of Florida: lead
• Oregon Health and Science University: collaborator
• Children's Hospital of Philadelphia: collaborator
• Shriners Hospitals for Children: collaborator
• National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS): collaborator

Study Sites (3)

University of Florida

Gainesville, Florida, 32610, United States

RECRUITING

Oregon Health and Science University

Portland, Oregon, 97239, United States

RECRUITING

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

ACTIVE NOT RECRUITING

Related Publications (38)

• Willis AB, Zelikovich AS, Sufit R, Ajroud-Driss S, Vandenborne K, Demonbreun AR, Batra A, Walter GA, McNally EM. Serum protein and imaging biomarkers after intermittent steroid treatment in muscular dystrophy. medRxiv [Preprint]. 2024 Jun 16:2024.06.14.24308858. doi: 10.1101/2024.06.14.24308858.

  PMID: 38947030 BACKGROUND

• Muntoni F, Signorovitch J, Sajeev G, Done N, Yao Z, Goemans N, McDonald C, Mercuri E, Niks EH, Wong B, Vandenborne K, Straub V, de Groot IJM, Tian C, Manzur A, Dieye I, Lane H, Ward SJ, Servais L; PRO-DMD-01 study investigators; Association Francaise contre les Myopathies; UK NorthStar Clinical Network; ImagingDMD investigators; cTAP. Meaningful changes in motor function in Duchenne muscular dystrophy (DMD): A multi-center study. PLoS One. 2024 Jul 10;19(7):e0304984. doi: 10.1371/journal.pone.0304984. eCollection 2024.

  PMID: 38985784 BACKGROUND

• Ikelaar NA, Barnard AM, Eng SWM, Hosseini Vajargah S, Ha KCH, Kan HE, Vandenborne K, Niks EH, Walter GA, Spitali P. Large scale serum proteomics identifies proteins associated with performance decline and clinical milestones in Duchenne muscular dystrophy. medRxiv [Preprint]. 2024 Aug 7:2024.08.05.24311516. doi: 10.1101/2024.08.05.24311516.

  PMID: 39148831 BACKGROUND

• Kim J, Morales JF, Kang S, Klose M, Willcocks RJ, Daniels MJ, Belfiore-Oshan R, Walter GA, Rooney WD, Vandenborne K, Kim S. A model-informed clinical trial simulation tool with a graphical user interface for Duchenne muscular dystrophy. CPT Pharmacometrics Syst Pharmacol. 2025 Nov;14(11):1765-1774. doi: 10.1002/psp4.13246. Epub 2024 Oct 3.

  PMID: 39360574 BACKGROUND

• Jenkins BM, Dixon LD, Kokesh KJ, Zingariello CD, Vandenborne K, Walter GA, Barnard AM. Skeletal muscle symptoms and quantitative MRI in females with dystrophinopathy. Muscle Nerve. 2024 Nov;70(5):988-999. doi: 10.1002/mus.28235. Epub 2024 Sep 2.

  PMID: 39221574 BACKGROUND

• Akima H, Lott D, Senesac C, Deol J, Germain S, Arpan I, Bendixen R, Lee Sweeney H, Walter G, Vandenborne K. Relationships of thigh muscle contractile and non-contractile tissue with function, strength, and age in boys with Duchenne muscular dystrophy. Neuromuscul Disord. 2012 Jan;22(1):16-25. doi: 10.1016/j.nmd.2011.06.750. Epub 2011 Jul 31.

  PMID: 21807516 BACKGROUND

• Forbes SC, Walter GA, Rooney WD, Wang DJ, DeVos S, Pollaro J, Triplett W, Lott DJ, Willcocks RJ, Senesac C, Daniels MJ, Byrne BJ, Russman B, Finkel RS, Meyer JS, Sweeney HL, Vandenborne K. Skeletal muscles of ambulant children with Duchenne muscular dystrophy: validation of multicenter study of evaluation with MR imaging and MR spectroscopy. Radiology. 2013 Oct;269(1):198-207. doi: 10.1148/radiol.13121948. Epub 2013 May 21.

  PMID: 23696684 BACKGROUND

• Triplett WT, Baligand C, Forbes SC, Willcocks RJ, Lott DJ, DeVos S, Pollaro J, Rooney WD, Sweeney HL, Bonnemann CG, Wang DJ, Vandenborne K, Walter GA. Chemical shift-based MRI to measure fat fractions in dystrophic skeletal muscle. Magn Reson Med. 2014 Jul;72(1):8-19. doi: 10.1002/mrm.24917. Epub 2013 Sep 4.

  PMID: 24006208 BACKGROUND

• Willcocks RJ, Arpan IA, Forbes SC, Lott DJ, Senesac CR, Senesac E, Deol J, Triplett WT, Baligand C, Daniels MJ, Sweeney HL, Walter GA, Vandenborne K. Longitudinal measurements of MRI-T2 in boys with Duchenne muscular dystrophy: effects of age and disease progression. Neuromuscul Disord. 2014 May;24(5):393-401. doi: 10.1016/j.nmd.2013.12.012. Epub 2014 Jan 11.

  PMID: 24491484 BACKGROUND

• Arpan I, Willcocks RJ, Forbes SC, Finkel RS, Lott DJ, Rooney WD, Triplett WT, Senesac CR, Daniels MJ, Byrne BJ, Finanger EL, Russman BS, Wang DJ, Tennekoon GI, Walter GA, Sweeney HL, Vandenborne K. Examination of effects of corticosteroids on skeletal muscles of boys with DMD using MRI and MRS. Neurology. 2014 Sep 9;83(11):974-80. doi: 10.1212/WNL.0000000000000775. Epub 2014 Aug 6.

  PMID: 25098537 BACKGROUND

• Arpan I, Forbes SC, Lott DJ, Senesac CR, Daniels MJ, Triplett WT, Deol JK, Sweeney HL, Walter GA, Vandenborne K. T(2) mapping provides multiple approaches for the characterization of muscle involvement in neuromuscular diseases: a cross-sectional study of lower leg muscles in 5-15-year-old boys with Duchenne muscular dystrophy. NMR Biomed. 2013 Mar;26(3):320-8. doi: 10.1002/nbm.2851. Epub 2012 Oct 9.

  PMID: 23044995 BACKGROUND

• Forbes SC, Willcocks RJ, Triplett WT, Rooney WD, Lott DJ, Wang DJ, Pollaro J, Senesac CR, Daniels MJ, Finkel RS, Russman BS, Byrne BJ, Finanger EL, Tennekoon GI, Walter GA, Sweeney HL, Vandenborne K. Magnetic resonance imaging and spectroscopy assessment of lower extremity skeletal muscles in boys with Duchenne muscular dystrophy: a multicenter cross sectional study. PLoS One. 2014 Sep 9;9(9):e106435. doi: 10.1371/journal.pone.0106435. eCollection 2014.

  PMID: 25203313 BACKGROUND

• Bendixen RM, Lott DJ, Senesac C, Mathur S, Vandenborne K. Participation in daily life activities and its relationship to strength and functional measures in boys with Duchenne muscular dystrophy. Disabil Rehabil. 2014;36(22):1918-23. doi: 10.3109/09638288.2014.883444. Epub 2014 Feb 6.

  PMID: 24499260 BACKGROUND

• Senesac CR, Lott DJ, Forbes SC, Mathur S, Arpan I, Senesac ES, Walter GA, Vandenborne K. Longitudinal Evaluation of Muscle Composition Using Magnetic Resonance in 4 Boys With Duchenne Muscular Dystrophy: Case Series. Phys Ther. 2015 Jul;95(7):978-88. doi: 10.2522/ptj.20140234. Epub 2015 Jan 15.

  PMID: 25592189 BACKGROUND

• Arora H, Willcocks RJ, Lott DJ, Harrington AT, Senesac CR, Zilke KL, Daniels MJ, Xu D, Tennekoon GI, Finanger EL, Russman BS, Finkel RS, Triplett WT, Byrne BJ, Walter GA, Sweeney HL, Vandenborne K. Longitudinal timed function tests in Duchenne muscular dystrophy: ImagingDMD cohort natural history. Muscle Nerve. 2018 Nov;58(5):631-638. doi: 10.1002/mus.26161. Epub 2018 Jul 24.

  PMID: 29742798 BACKGROUND

• Chrzanowski SM, Baligand C, Willcocks RJ, Deol J, Schmalfuss I, Lott DJ, Daniels MJ, Senesac C, Walter GA, Vandenborne K. Multi-slice MRI reveals heterogeneity in disease distribution along the length of muscle in Duchenne muscular dystrophy. Acta Myol. 2017 Sep 1;36(3):151-162. eCollection 2017 Sep.

  PMID: 29774305 BACKGROUND

• Willcocks RJ, Forbes SC, Walter GA, Vandenborne K. Magnetic resonance imaging characteristics of injection site reactions after long-term subcutaneous delivery of drisapersen. Eur J Pediatr. 2019 May;178(5):777-778. doi: 10.1007/s00431-019-03349-0. Epub 2019 Feb 21. No abstract available.

  PMID: 30790036 BACKGROUND

• Batra A, Vohra RS, Chrzanowski SM, Hammers DW, Lott DJ, Vandenborne K, Walter GA, Forbes SC. Effects of PDE5 inhibition on dystrophic muscle following an acute bout of downhill running and endurance training. J Appl Physiol (1985). 2019 Jun 1;126(6):1737-1745. doi: 10.1152/japplphysiol.00664.2018. Epub 2019 Apr 4.

  PMID: 30946638 BACKGROUND

• Barnard AM, Lott DJ, Batra A, Triplett WT, Forbes SC, Riehl SL, Willcocks RJ, Smith BK, Vandenborne K, Walter GA. Imaging respiratory muscle quality and function in Duchenne muscular dystrophy. J Neurol. 2019 Nov;266(11):2752-2763. doi: 10.1007/s00415-019-09481-z. Epub 2019 Jul 26.

  PMID: 31350642 BACKGROUND

• Barnard AM, Riehl SL, Willcocks RJ, Walter GA, Angell AM, Vandenborne K. Characterizing Enrollment in Observational Studies of Duchenne Muscular Dystrophy by Race and Ethnicity. J Neuromuscul Dis. 2020;7(2):167-173. doi: 10.3233/JND-190447.

  PMID: 31929119 BACKGROUND

• Barnard AM, Willcocks RJ, Triplett WT, Forbes SC, Daniels MJ, Chakraborty S, Lott DJ, Senesac CR, Finanger EL, Harrington AT, Tennekoon G, Arora H, Wang DJ, Sweeney HL, Rooney WD, Walter GA, Vandenborne K. MR biomarkers predict clinical function in Duchenne muscular dystrophy. Neurology. 2020 Mar 3;94(9):e897-e909. doi: 10.1212/WNL.0000000000009012. Epub 2020 Feb 5.

  PMID: 32024675 BACKGROUND

• Rooney WD, Berlow YA, Triplett WT, Forbes SC, Willcocks RJ, Wang DJ, Arpan I, Arora H, Senesac C, Lott DJ, Tennekoon G, Finkel R, Russman BS, Finanger EL, Chakraborty S, O'Brien E, Moloney B, Barnard A, Sweeney HL, Daniels MJ, Walter GA, Vandenborne K. Modeling disease trajectory in Duchenne muscular dystrophy. Neurology. 2020 Apr 14;94(15):e1622-e1633. doi: 10.1212/WNL.0000000000009244. Epub 2020 Mar 17.

  PMID: 32184340 BACKGROUND

• Forbes SC, Arora H, Willcocks RJ, Triplett WT, Rooney WD, Barnard AM, Alabasi U, Wang DJ, Lott DJ, Senesac CR, Harrington AT, Finanger EL, Tennekoon GI, Brandsema J, Daniels MJ, Sweeney HL, Walter GA, Vandenborne K. Upper and Lower Extremities in Duchenne Muscular Dystrophy Evaluated with Quantitative MRI and Proton MR Spectroscopy in a Multicenter Cohort. Radiology. 2020 Jun;295(3):616-625. doi: 10.1148/radiol.2020192210. Epub 2020 Apr 14.

  PMID: 32286193 BACKGROUND

• Barnard AM, Willcocks RJ, Finanger EL, Daniels MJ, Triplett WT, Rooney WD, Lott DJ, Forbes SC, Wang DJ, Senesac CR, Harrington AT, Finkel RS, Russman BS, Byrne BJ, Tennekoon GI, Walter GA, Sweeney HL, Vandenborne K. Skeletal muscle magnetic resonance biomarkers correlate with function and sentinel events in Duchenne muscular dystrophy. PLoS One. 2018 Mar 19;13(3):e0194283. doi: 10.1371/journal.pone.0194283. eCollection 2018.

  PMID: 29554116 BACKGROUND

• Willcocks RJ, Triplett WT, Forbes SC, Arora H, Senesac CR, Lott DJ, Nicholson TR, Rooney WD, Walter GA, Vandenborne K. Magnetic resonance imaging of the proximal upper extremity musculature in boys with Duchenne muscular dystrophy. J Neurol. 2017 Jan;264(1):64-71. doi: 10.1007/s00415-016-8311-0. Epub 2016 Oct 24.

  PMID: 27778157 BACKGROUND

• Forbes SC, Willcocks RJ, Rooney WD, Walter GA, Vandenborne K. MRI quantifies neuromuscular disease progression. Lancet Neurol. 2016 Jan;15(1):26-8. doi: 10.1016/S1474-4422(15)00320-8. Epub 2015 Nov 6. No abstract available.

  PMID: 26549781 BACKGROUND

• Lott DJ, Taivassalo T, Senesac CR, Willcocks RJ, Harrington AM, Zilke K, Cunkle H, Powers C, Finanger EL, Rooney WD, Tennekoon GI, Vandenborne K. Walking activity in a large cohort of boys with Duchenne muscular dystrophy. Muscle Nerve. 2021 Feb;63(2):192-198. doi: 10.1002/mus.27119. Epub 2020 Nov 27.

  PMID: 33188573 BACKGROUND

• Senesac CR, Barnard AM, Lott DJ, Nair KS, Harrington AT, Willcocks RJ, Zilke KL, Rooney WD, Walter GA, Vandenborne K. Magnetic Resonance Imaging Studies in Duchenne Muscular Dystrophy: Linking Findings to the Physical Therapy Clinic. Phys Ther. 2020 Oct 30;100(11):2035-2048. doi: 10.1093/ptj/pzaa140.

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• Barnard AM, Hammers DW, Triplett WT, Kim S, Forbes SC, Willcocks RJ, Daniels MJ, Senesac CR, Lott DJ, Arpan I, Rooney WD, Wang RT, Nelson SF, Sweeney HL, Vandenborne K, Walter GA. Evaluating Genetic Modifiers of Duchenne Muscular Dystrophy Disease Progression Using Modeling and MRI. Neurology. 2022 Nov 22;99(21):e2406-e2416. doi: 10.1212/WNL.0000000000201163. Epub 2022 Sep 2.

  PMID: 36240102 BACKGROUND

• Nair KS, Lott DJ, Forbes SC, Barnard AM, Willcocks RJ, Senesac CR, Daniels MJ, Harrington AT, Tennekoon GI, Zilke K, Finanger EL, Finkel RS, Rooney WD, Walter GA, Vandenborne K. Step Activity Monitoring in Boys with Duchenne Muscular Dystrophy and its Correlation with Magnetic Resonance Measures and Functional Performance. J Neuromuscul Dis. 2022;9(3):423-436. doi: 10.3233/JND-210746.

  PMID: 35466946 BACKGROUND

• Finkel RS, Finanger E, Vandenborne K, Sweeney HL, Tennekoon G, Shieh PB, Willcocks R, Walter G, Rooney WD, Forbes SC, Triplett WT, Yum SW, Mancini M, MacDougall J, Fretzen A, Bista P, Nichols A, Donovan JM. Disease-modifying effects of edasalonexent, an NF-kappaB inhibitor, in young boys with Duchenne muscular dystrophy: Results of the MoveDMD phase 2 and open label extension trial. Neuromuscul Disord. 2021 May;31(5):385-396. doi: 10.1016/j.nmd.2021.02.001. Epub 2021 Feb 4.

  PMID: 33678513 BACKGROUND

• Willcocks RJ, Forbes SC, Walter GA, Sweeney L, Rodino-Klapac LR, Mendell JR, Vandenborne K. Assessment of rAAVrh.74.MHCK7.micro-dystrophin Gene Therapy Using Magnetic Resonance Imaging in Children With Duchenne Muscular Dystrophy. JAMA Netw Open. 2021 Jan 4;4(1):e2031851. doi: 10.1001/jamanetworkopen.2020.31851.

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• Barnard AM, Lott DJ, Batra A, Triplett WT, Willcocks RJ, Forbes SC, Rooney WD, Daniels MJ, Smith BK, Vandenborne K, Walter GA. Characterizing Expiratory Respiratory Muscle Degeneration in Duchenne Muscular Dystrophy Using MRI. Chest. 2022 Mar;161(3):753-763. doi: 10.1016/j.chest.2021.08.078. Epub 2021 Sep 15.

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• Willcocks RJ, Barnard AM, Wortman RJ, Senesac CR, Lott DJ, Harrington AT, Zilke KL, Forbes SC, Rooney WD, Wang DJ, Finanger EL, Tennekoon GI, Daniels MJ, Triplett WT, Walter GA, Vandenborne K. Development of Contractures in DMD in Relation to MRI-Determined Muscle Quality and Ambulatory Function. J Neuromuscul Dis. 2022;9(2):289-302. doi: 10.3233/JND-210731.

  PMID: 35124659 BACKGROUND

• Mercuri E, Vilchez JJ, Boespflug-Tanguy O, Zaidman CM, Mah JK, Goemans N, Muller-Felber W, Niks EH, Schara-Schmidt U, Bertini E, Comi GP, Mathews KD, Servais L, Vandenborne K, Johannsen J, Messina S, Spinty S, McAdam L, Selby K, Byrne B, Laverty CG, Carroll K, Zardi G, Cazzaniga S, Coceani N, Bettica P, McDonald CM; EPIDYS Study Group. Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet Neurol. 2024 Apr;23(4):393-403. doi: 10.1016/S1474-4422(24)00036-X.

  PMID: 38508835 BACKGROUND

• Willcocks RJ, Barnard AM, Daniels MJ, Forbes SC, Triplett WT, Brandsema JF, Finanger EL, Rooney WD, Kim S, Wang DJ, Lott DJ, Senesac CR, Walter GA, Sweeney HL, Vandenborne K. Clinical importance of changes in magnetic resonance biomarkers for Duchenne muscular dystrophy. Ann Clin Transl Neurol. 2024 Jan;11(1):67-78. doi: 10.1002/acn3.51933. Epub 2023 Nov 6.

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• Kim S, Willcocks RJ, Daniels MJ, Morales JF, Yoon DY, Triplett WT, Barnard AM, Conrado DJ, Aggarwal V, Belfiore-Oshan R, Martinez TN, Walter GA, Rooney WD, Vandenborne K. Multivariate modeling of magnetic resonance biomarkers and clinical outcome measures for Duchenne muscular dystrophy clinical trials. CPT Pharmacometrics Syst Pharmacol. 2023 Oct;12(10):1437-1449. doi: 10.1002/psp4.13021. Epub 2023 Aug 27.

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• Yoon DY, Daniels MJ, Willcocks RJ, Triplett WT, Morales JF, Walter GA, Rooney WD, Vandenborne K, Kim S; DMD MR Biomarker Steering Committee. Five multivariate Duchenne muscular dystrophy progression models bridging six-minute walk distance and MRI relaxometry of leg muscles. J Pharmacokinet Pharmacodyn. 2024 Dec;51(6):671-683. doi: 10.1007/s10928-024-09910-1. Epub 2024 Apr 12.

  PMID: 38609673 BACKGROUND

Related Links

• active website

Biospecimen

Retention: SAMPLES WITH DNA

Blood, Urine, Saliva samples collected, and a one time Muscle Biopsy in Becker MD subjects and matched controls only

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• William Rooney, PhD

  Oregan Health and Science University

  PRINCIPAL INVESTIGATOR

• H. Lee Sweeney, PhD

  University of Florida

  PRINCIPAL INVESTIGATOR

• Krista Vandenborne, PhD

  University of Florida

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Krista Vandenborne, PhD

CONTACT

352-273-6100; kvandenb@phhp.ufl.edu

Kelly Rock, PhD

CONTACT

352-294-5798; k.rock@phhp.ufl.edu

Study Design

• Study Type: observational
• Observational Model: CASE CONTROL
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 10, 2011
• First Posted: December 2, 2011
• Study Start: September 1, 2020
• Primary Completion (Estimated): August 31, 2026
• Study Completion (Estimated): August 31, 2026
• Last Updated: October 15, 2025

Record last verified: 2025-10

Locations

US (3)