SMA Patients Being Recruited for Phase 2 Study of Skeletal Muscle Therapy

Malika Ammam, PhD avatar

by Malika Ammam, PhD |

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03/Junio/2016 Madrid, Arganda del Rey. Álvaro prueba el primer exoesqueleto diseñado para niños en el Centro de Automática y Robótica, CAT, del CSIC. © JOAN COSTA / CSIC

A Phase 2 clinical trial investigating a potential treatment, CK-2127107, in people with spinal muscular atrophy (SMA), ages 12 years and older, is currently recruiting patients in the U.S. with varying degrees of disease severity (Types 2, 3, or 4).

CK-2127107 is an investigational drug candidate designed for skeletal muscle and other debilitating neuromuscular and non-neuromuscular diseases linked to muscle weakness and/or fatigue. The drug works through slowing down the rate of calcium released from the skeletal muscle fibers, and, as such, may improve muscle function and physical performance in patients with SMA.

The safety, tolerability, bioavailability, pharmacokinetics, and pharmacodynamics of CK-107 was evaluated in five completed Phase 1 clinical trials involving healthy volunteers. The primary goal of this Phase 2 trial (NCT02644668) is to evaluate the pharmacodynamic effect of oral CK-2127107 on measures of skeletal muscle function or fatigue in SMA patients. Patient will randomly receive either a placebo or CK-2127107 at two dose levels (150 mg or 450 mg), administrated as a liquid by mouth, and be monitored for a period of eight weeks of dosing.

Muscle function and fatigue in patients with ambulatory and non-ambulatory SMA symptoms will be assessed through tests that include measures of Forced Vital Capacity (FVC), Maximum Inspiratory Pressure (MIP)/Maximum Expiratory Pressure (MEP), the Hammersmith Functional Motor Scale-Expanded, and the 6-Minute Walk Test. Furthermore, plasma concentration of CK-2127107 will be quantified to gain a better understanding of patients’ response at the different dosages.

Other criteria related to safety and tolerability will also be examined in the trial, taking place at nine sites across the United States.

SMA is a hereditary disease identified by progressive loss of functionality of motor neurons that ultimately leads to muscle wasting and mobility impairment. The disease is categorized by different degrees of severity (with Type 1 being most severe), based on ages of onset, severity, and progression of symptoms.  Types 1-3 affect infants and children; type 4 is adult-onset SMA.

There are no available pharmacological treatments designed to spinal muscular atrophy. The management and care of the disease is often symptomatic and performed through orthopedics, mobility support, respiratory care, nutrition, cardiology, and mental health.

Further information about this trial, including how to participate and the exact location of actively recruiting sites, is available through the study’s  clinical website.

This trial is sponsored by Cytokinetics in partnership with Astellas.