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Spironolactone Versus Prednisolone in DMD

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StatusRekrutierung
Sponsoren
Kevin Flanigan
Mitarbeiter
Muscular Dystrophy Association

Schlüsselwörter

Abstrakt

This is a randomized, open-label, pilot clinical trial of spironolactone suspension versus oral prednisolone for use in Duchenne muscular dystrophy. The goals are to determine the safety of 6 months of treatment with spironolactone treatment int he steroid-naive DMD population as well as to determine if either spironolactone or a standard clinical dose of corticosteroids results in equivalent improvement in time to complete the 100 meter timed test (100M).

Beschreibung

Until recently, the only treatment shown to improve strength and preserve ambulation in DMD patients was the use of glucocorticoids, which are accompanied by significant side effects including obesity, cushingoid features, osteoporosis, and behavioral disturbances. Spironolactone is an aldosterone antagonist primarily used as a potassium sparing diuretic that is widely used in the pediatric population, with limited side-effects including gynecomastia and hyperkalemia. Recent studies by Dr. Rafael-Fortney have evaluated the effect of spironolactone treatment in several different mouse models of DMD. Her results show that treatment of these mice demonstrates increased muscle membrane stabilization while reducing the negative side-effects typically associated with standard of care glucocorticoids. This pilot study is designed to determine whether this commonly used medication, spironolactone, may have similar beneficial effects with a lower side effect profile and be applicable to a wider population of DMD patients.

The hypothesis for this controlled pilot trial is that spironolactone and prednisolone are of equal efficacy in improving skeletal muscle function over a 6-month period, and that spironolactone will be well tolerated in this patient population.

One outcome is that both drugs demonstrate equal efficacy in motor function. This would then serve as pilot data for a longer term study.

Termine

Zuletzt überprüft: 01/31/2020
Zuerst eingereicht: 12/09/2018
Geschätzte Einschreibung eingereicht: 12/12/2018
Zuerst veröffentlicht: 12/16/2018
Letztes eingereichtes Update: 02/17/2020
Letztes Update veröffentlicht: 02/19/2020
Tatsächliches Startdatum der Studie: 12/04/2018
Geschätztes primäres Abschlussdatum: 11/30/2021
Voraussichtliches Abschlussdatum der Studie: 11/30/2021

Zustand oder Krankheit

Muscular Dystrophy, Duchenne

Intervention / Behandlung

Drug: Spironolactone

Drug: Prednisolone

Phase

Phase 1

Armgruppen

ArmIntervention / Behandlung
Experimental: Spironolactone
Twelve subjects will be prescribed a standard clinical dose of spironolactone of 1 mg/kg/day. The spironolactone will be provided as suspension.
Drug: Spironolactone
Spironolactone will be prescribed for 6 months, after which the family and primary care physician will determine to either remain on spironolactone or transfer to prednisolone.
Active Comparator: Prednisolone
Twelve subjects will be prescribed a standard clinical dose of prednisolone of 0.75 mg/kg/day or weekend dosing of 5 mg/kg/day as per sites standard of care. The prednisolone will be provided will be provided as suspension.
Drug: Prednisolone
Prednisolone will be prescribed for 6 months as the clinical standard of care.

Zulassungskriterien

Altersberechtigt für das Studium 4 Years Zu 4 Years
Studienberechtigte GeschlechterMale
Akzeptiert gesunde FreiwilligeJa
Kriterien

Inclusion Criteria:

- Duchenne muscular dystrophy (DMD) patients ≥4 to ≤7 years of age

- Clinical features of DMD that include proximal predominant weakness and/or gait disturbance

- Presence of a truncating mutation of the DMD gene OR a muscle biopsy that demonstrates <5% dystrophin in the patient or an affected relative

- Normal left ventricular ejection fraction by screening echocardiogram

- Ability to cooperate for testing

- No prior glucocorticoid treatment

- No concomitant experimental therapies

Exclusion Criteria:

- Subject amenable to or currently being treated with eteplirsen

- Hyperkalemia at screening

- History of or ongoing renal failure (elevated creatinine, oliguria, anuria)

- Hypersensitivity to spironolactone (rash, respiratory distress, arrhythmia, numbness or tingling of extremities)

- Current treatment with an ACEi

- Severe peptic ulcer disease or recent gastrointestinal perforations

Ergebnis

Primäre Ergebnismaße

1. Efficacy: Change in time to complete a 100 meter timed test. [6 months]

The determination of whether spironolactone has similar efficacy to glucocorticoids in improving muscle strength in steroid naïve DMD patients. This will be determined by measuring the time to complete a 100 meter timed test (100M).

2. Safety will be monitored through regular review of electrolytes. [6 months]

Electrolytes (Sodium, Potassium, Cloride and Carbon dioxide, mmol/L) will be measured on a monthly basis following initiation of either spironolactone or prednisolone.

Sekundäre Ergebnismaße

1. Efficacy: Dynamometry score [6 months]

Secondary outcome measures will be Dynamometry score, which is a summation of maximum voluntary isometric contraction test values for knee flexion, knee extension, elbow flexion, and elbow extension; 4-stair climb; North Star Ambulatory Assessment (NSAA); and time to arise from the floor.

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