Historically Controlled Trial of Corticosteroids in Young Boys With Duchenne Muscular Dystrophy

NCT02167217 | Completed Phase 2 Results DMC

• 1 other identifier: 201308062
• Study Type: interventional
• Target: 25
• Locations: 1 country
• Sites: 6

Brief Summary

While it has been known for many years that corticosteroid use benefits boys with Duchenne Muscular dystrophy (DMD), most clinicians do not consider treating until after age 3 or 4 years of age. The primary reason for the delay is that daily corticosteroid use has many side effects including short stature, obesity, and osteoporosis. A recent randomized blinded study of weekend oral corticosteroid use over one year showed equal improvement in strength with fewer side effects, particularly as related to growth and cushingoid changes. The investigators will test the efficacy of oral weekend corticosteroid use in infants and young boys with DMD who are under age 30 months. The investigators have demonstrated that the Bayley-III Scales of Infant development shows that infants and young boys in this age group who are untreated decline in abilities when compared to their peers. Here, in this Phase 2 historically controlled trial, the investigators will use these two measures and treat boys at five Muscular Dystrophy Association-DMD centers

Conditions

Duchenne Muscular Dystrophy

Keywords

Duchenne Muscular Dystrophy; Corticosteroids; Infants; Young Children; Toddlers

Outcome Measures

Primary Outcomes (1)

• Bayley III Gross Motor Scaled Score (Change From Baseline to 12 Month)

  Bayley III Gross Motor Scaled Score measures motor development. This is normed for typically developing children and follow a bell shaped curve. The scale has mean of 10 +/-3 for children at all ages and is bell shaped. Therefore the two standard deviation range is 16 to 4 with higher values indicated better performance. Lower values have been shown to be common in boys with DMD and it this study the baseline average score was 4.2.

  One year

Study Arms (1)

Oral Prednisolone

EXPERIMENTAL

Oral Prednisolone 5mg/kg/ day on two consecutive days, Friday and Saturday with breakfast

Drug: Prednisolone

Interventions

Prednisolone DRUG

Prednisolone (5mg per kg) will be taken on two consecutive days, Friday and Saturday mornings each week with breakfast

Also known as: prednisolone (Morton Grove Pharm.) National Drug Code is 60432-212-08

Oral Prednisolone

Eligibility Criteria

• Age: 1 Month - 30 Months
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Appropriate degree of weakness for age, creatine kinase greater than 20 times the upper limit of normal, and genetic mutation known to be causative for Duchenne muscular dystrophy .
• Appropriate degree of weakness for age, creatine kinase greater than 20 times the upper limit of normal and genetic or biopsy confirmation of Duchenne muscular dystrophy in a primary relative (e.g. brother or maternal uncle).
• De-identified, genetic studies will be reviewed by collaborator Kevin Flanigan, MD prior to enrollment of subjects.
• Age at entry: one month through 30 months.

You may not qualify if:

• Prior treatment with corticosteroids

Sponsors & Collaborators

• Washington University School of Medicine: lead
• Nationwide Children's Hospital: collaborator
• Northwestern University Feinberg School of Medicine: collaborator
• University of Texas Southwestern Medical Center: collaborator
• University of California, Davis: collaborator
• Nemours Children's Hospital: collaborator

Study Sites (6)

University of California Davis

Sacramento, California, 95817, United States

Location

Nemours Hospital

Orlando, Florida, 32827, United States

Location

Laurie Children's Hospital Of Chicago

Chicago, Illinois, 60611-2605, United States

Location

Washington University in Saint Louis

St Louis, Missouri, 63109, United States

Location

Research Institute Center for Gene Therapy at Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Location

University of Texas South Western Medical Center of Dallas

Dallas, Texas, 75390, United States

Location

Related Publications (4)

• Connolly AM, Florence JM, Cradock MM, Malkus EC, Schierbecker JR, Siener CA, Wulf CO, Anand P, Golumbek PT, Zaidman CM, Philip Miller J, Lowes LP, Alfano LN, Viollet-Callendret L, Flanigan KM, Mendell JR, McDonald CM, Goude E, Johnson L, Nicorici A, Karachunski PI, Day JW, Dalton JC, Farber JM, Buser KK, Darras BT, Kang PB, Riley SO, Shriber E, Parad R, Bushby K, Eagle M; MDA DMD Clinical Research Network. Motor and cognitive assessment of infants and young boys with Duchenne Muscular Dystrophy: results from the Muscular Dystrophy Association DMD Clinical Research Network. Neuromuscul Disord. 2013 Jul;23(7):529-39. doi: 10.1016/j.nmd.2013.04.005. Epub 2013 May 28.

  PMID: 23726376 BACKGROUND

• Connolly AM, Schierbecker J, Renna R, Florence J. High dose weekly oral prednisone improves strength in boys with Duchenne muscular dystrophy. Neuromuscul Disord. 2002 Dec;12(10):917-25. doi: 10.1016/s0960-8966(02)00180-3.

  PMID: 12467746 BACKGROUND

• Escolar DM, Hache LP, Clemens PR, Cnaan A, McDonald CM, Viswanathan V, Kornberg AJ, Bertorini TE, Nevo Y, Lotze T, Pestronk A, Ryan MM, Monasterio E, Day JW, Zimmerman A, Arrieta A, Henricson E, Mayhew J, Florence J, Hu F, Connolly AM. Randomized, blinded trial of weekend vs daily prednisone in Duchenne muscular dystrophy. Neurology. 2011 Aug 2;77(5):444-52. doi: 10.1212/WNL.0b013e318227b164. Epub 2011 Jul 13.

  PMID: 21753160 BACKGROUND

• Connolly AM, Zaidman CM, Golumbek PT, Cradock MM, Flanigan KM, Kuntz NL, Finkel RS, McDonald CM, Iannaccone ST, Anand P, Siener CA, Florence JM, Lowes LP, Alfano LN, Johnson LB, Nicorici A, Nelson LL, Mendell JR; MDA DMD Clinical Research Network. Twice-weekly glucocorticosteroids in infants and young boys with Duchenne muscular dystrophy. Muscle Nerve. 2019 Jun;59(6):650-657. doi: 10.1002/mus.26441. Epub 2019 Feb 23.

  PMID: 30706490 DERIVED

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Interventions

Prednisolone

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

Intervention Hierarchy (Ancestors)

Pregnadienetriols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds

Results Point of Contact

• Title: Anne M. Connolly, MD FAAN
• Organization: Washington University School of Medicine

connollya@wustl.edu

314-362-6981

Study Officials

• Anne M Connolly, MD

  Washington University School of Medicine

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 3, 2014
• First Posted: June 19, 2014
• Study Start: April 17, 2014
• Primary Completion: February 22, 2017
• Study Completion: March 22, 2017
• Last Updated: December 21, 2018
• Results First Posted: May 18, 2018

Record last verified: 2018-12

Locations

US (6)