Clenbuterol to Target DUX4 in FSHD
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age18-75
SponsorJeffrey Statland
About this trial
The purpose of this study is to determine if Clenbuterol is a therapeutic option for FSHD by determining the safety and tolerability of the medication at three different dose levels.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Genetically confirmed diagnosis of FSHD type 1 or 2, or have a clinical diagnosis of FSHD type 1 with a first degree relative with confirmed mutation
between 18 and 75 years of age
with a clinical severity score between 0 and 10
Able to walk 30ft without support of another person
Disqualifiers
Pregnant or planning to become pregnant during the conduct of the study
have a poorly controlled medical condition
Were involved in a study of an experimental agent within 3 months of enrollment
Are taking beta-blockers or anabolic agent or potassium wasting diuretics
Trial design
Design model
Sequential
Treatments tested in this trial
Clenbuterol
DrugBeta-Agonist
Treatment groups
30 Participants
are divided into 3 treatment groupsGroup A: Clenbuterol Cohort 1Experimental treatment 1 intervention
Group B: Clenbuterol Cohort 2Experimental treatment 1 intervention
Group C: Clenbuterol Cohort 3Experimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Frequency of Clenbuterol-related adverse reactions
Patient report and medical records will be used to document adverse events and severe adverse events. Adverse events and severe adverse events will be assessed by the investigator and reported as needed for safety.
Time frame
from baseline to 6 month visit2
change in heart rate
Review of Vitals
Time frame
from Month 1 to Month 6 visit3
change in blood pressure
Review of Vitals
Time frame
from Month 1 to Month 6 visit4
Safety Lab Potassium (K)
patient safety measured by potassium for signs of hypokalemia
Time frame
Baseline to month 6 visitSecondary outcomes
1
MRI
MRI measurements are considered gold standard for lean muscle volume measurement and disease biomarkers (fat volumes, fat faction, and STIR+ presence) that may be modulated by treatment. In addition, we will include analysis to help make needle biopsy based on STIR and quantitative fat fraction more efficient with higher yield using standard fiducial markings and body landmarks.
Time frame
Screening to Month 6 Visit2
Muscle Biopsy for RNA sequencing
change in a set of DUX4-regulated genes as measured in muscle using RNA sequencing. goal is to better understand the molecular changes in muscle tissue effected by the use of Clenbuterol.
Time frame
Baseline and Month 63
FSHD-COM
An 18-item evaluator administered instrument comprised of individually validated functional motor task.the body regions assessed match areas of importance identified by patients and include leg function; shoulder and arm function; trunk function, hand function; and functional balance
Time frame
Screening to Month 6 Visit4
Manual Muscle Testing (MMT)
a modified Medical Research Council 13-point scale with standardized positions for each muscle will help look at subject's change in strength over time.
Time frame
Screening to Month 6 VisitSponsors and contacts
Click on the lead sponsor to view all of their trials.
Jeffrey Statland
Lead sponsor
University of Kansas Medical Center
Sponsor institution
University of Washington
Collaborator
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Collaborator
University of Rochester
Collaborator
Source ClinicalTrials.gov