NCT00527228

Brief Summary

The present study is designed to assess the natural history in a one year pre-phase of the trial and evaluate therapeutic efficacy and side effects of deflazacort in LGMD2B/MM patients in a placebo-controlled trial. Furthermore, long-term development of the disease under naturalistic conditions will be documented in a 2-year follow-up after the end of the double-blind treatment phase.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
25

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Sep 2003

Longer than P75 for phase_2

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Start

First participant enrolled

September 1, 2003

Completed
4 years until next milestone

First Submitted

Initial submission to the registry

September 6, 2007

Completed
4 days until next milestone

First Posted

Study publicly available on registry

September 10, 2007

Completed
7 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 1, 2008

Completed
5 months until next milestone

Study Completion

Last participant's last visit for all outcomes

September 1, 2008

Completed
Last Updated

September 2, 2015

Status Verified

August 1, 2015

Enrollment Period

4.6 years

First QC Date

September 6, 2007

Last Update Submit

August 31, 2015

Conditions

LGMD2BMiyoshi MyopathyDysferlinopathy

Keywords

Muscular dystrophytherapysteroidsdeflazacortdysferlinopathyLGMDMM

Outcome Measures

Primary Outcomes (1)

  • Muscle strength according to Medical Research Council Scales (MRC) and quantitative strength measurement evaluated by hand-held dynamometry (Citec, Groningen, The Netherlands)in the same muscle groups.

    each 6 months

Secondary Outcomes (1)

  • Quantitative strength measurement (QSM, M3diagnos, Fa. Schnell, Germany), Neuromuscular Symptoms Score (NSS), timed function tests, Clinical Global Impressions (CGI) of change and quality of life assessment(SF-36 scale).

    each 6 months

Study Arms (2)

A

ACTIVE COMPARATOR

After 6 months of treatment, and a 3-months wash-out, there is cross-over to Arm B

Drug: deflazacort

B

PLACEBO COMPARATOR

After 6 months of treatment, and a 3-months wash-out, there is cross-over to Arm A

Drug: placebo

Interventions

deflazacortDRUG

In the first 12 months, patients will receive no treatment to assess the natural history of the disease. Afterwards, patients will be treated with deflazacort 1mg/kg/day or placebo for the first month on treatment, from the second month on deflazacort or placebo will be administered on an alternate day regimen). Patients will be randomized to six months verum or placebo each, after a 3-months wash-out patients cross over to the alternate treatment for six months. In a 2-years follow-up phase after the double-blind treatment phase, long-term development of the disorder will be documented.

Also known as: Calcort, Corticosteroid
A
placeboDRUG
B

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Clinically, histologically, immunohistochemically and genetically defined muscular dystrophy with dysferlin-deficiency (LGMD2B/MM).
  • Patients should fulfill clinical, morphological, immunohistochemical and immunoblot criteria of LGMD 2B and definite mutation in dysferlin gene.

You may not qualify if:

  • Patients confined to bed or wheelchair.
  • Patients with other neurologic or internistic diseases and patients with former or current steroid treatment will not be included.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Friedrich-Baur-Institute, Dept. of Neurology, Ludwig-Maximilians-University of Munich

Munich, 80801, Germany

Location

Related Publications (1)

  • Walter MC, Reilich P, Thiele S, Schessl J, Schreiber H, Reiners K, Kress W, Muller-Reible C, Vorgerd M, Urban P, Schrank B, Deschauer M, Schlotter-Weigel B, Kohnen R, Lochmuller H. Treatment of dysferlinopathy with deflazacort: a double-blind, placebo-controlled clinical trial. Orphanet J Rare Dis. 2013 Feb 14;8:26. doi: 10.1186/1750-1172-8-26.

    PMID: 23406536DERIVED

Related Links

MeSH Terms

Conditions

Limb-girdle muscular dystrophy, type 2BMiyoshi myopathyDysferlinopathyMuscular Dystrophies

Interventions

deflazacortAdrenal Cortex Hormones

Condition Hierarchy (Ancestors)

Muscular Disorders, AtrophicMuscular DiseasesMusculoskeletal DiseasesNeuromuscular DiseasesNervous System DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

HormonesHormones, Hormone Substitutes, and Hormone Antagonists

Study Officials

  • Maggie C. Walter, MD

    Friedrich-Baur-Institute, Dept. of Neurology, Ludwig-Maximilians-University of Munich, Germany

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
CROSSOVER
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Prof. Dr. Maggie C. Walter, MD, MA

Study Record Dates

First Submitted

September 6, 2007

First Posted

September 10, 2007

Study Start

September 1, 2003

Primary Completion

April 1, 2008

Study Completion

September 1, 2008

Last Updated

September 2, 2015

Record last verified: 2015-08

Locations

DE(1)