Biogen, a Cambridge, Massachusetts based developer, manufacturer and marketer of innovative therapies for patients living with serious neurological, autoimmune and hematologic disorders, in collaboration with Isis Pharmaceuticals — a leader in discovery and and development of drugs based on antisense and RNAi technology and focused on RNA as a therapeutic target., has announced a new Phase 2 clinical trial, called EMBRACE — “A Study to Assess the Safety and Tolerability of ISIS 396443 (ISIS SMNRx) in Participants With Spinal Muscular Atrophy (SMA)”.
The primary objective of this multi-center, Phase 2 clinical study is to evaluate the safety and exploratory efficacy of the investigational drug, ISIS-SMNRx, (ISIS 396443) in a small subset of patients with infantile or childhood-onset Spinal Muscular Atrophy (SMA) who do not meet the age and other criteria of the ongoing Phase 3 studies ENDEAR & CHERISH. (see below). The secondary objective of this study is to examine the pharmacokinetics of ISIS 396443 in participants with SMA.
The study will be conducted at clinical centers in the U.S. and Europe, and a listing of clinical centers participating in the study will be posted on http://clinicaltrials.gov (NCT02462759) as they become available. Because of the complexity of the medical criteria involved in this study, it is very important for prospective subjects’ parents/guardians to contact a participating clinical center in order to have a full medical evaluation by expert clinical staff knowledgeable about the study. All patients who enroll in the study must meet all criteria as evaluated by a study doctor ISIS-SMNRx is administered intrathecally. It is injected with a thin needle into a space in the lower back at the end of the spinal cord — a medical procedure is commonly referred to as a “lumbar puncture”.
The study is double-blind (meaning none of the patients, the parents or the blinded study staff will know if the patient is assigned to the drug or placebo group), randomized, and sham-procedure controlled (A sham control mimics all aspects of the procedure to deliver the experimental medication with the exception of the actual delivery of the investigational drug to the patient. In the case of this study, the sham-procedure will involve a needle prick on the skin at the site of the lower back where an injection would occur. It is blinded so that patients, families and other study staff will not know whether the participant actually receives the study drug). It is designed to examine the safety and exploratory efficacy of ISIS-SMNRx in approximately 20 patients with SMA over a 14 month period. see the EMBRACE Q&A for details on enrollment criteria, or contact one of the participating trial centers for more information.
EMBRACE is the fourth ongoing trial testing ISIS-SMNRx. The others are:
ENDEAR, a Phase 3 clinical trial testing infants with SMA type I.
CHERISH, a Phase 3 clinical trial testing children age 2-12, with onset of symptoms at age six months or later.
NURTURE, a Phase 2 clinical trial testing very young infants who are genetically predisposed to SMA but not yet showing symptoms.
The EMBRACE trial is not yet open for participant recruitment.
Primary outcome measures of the EMBRACE trial are:
– Number of participants with adverse events and serious adverse events
– Change from Baseline in clinical laboratory parameters
-Assessed by the following laboratory tests: Blood chemistry: total protein, albumin, creatinine, cystatin C, creatine phosphokinase, blood urea nitrogen, total bilirubin (direct and indirect), alkaline phosphatase, alanine aminotransferase, aspartate aminotransferase, glucose, calcium, phosphorus, chloride, sodium, potassium. Hematology: red blood cells, hemoglobin, hematocrit, platelets, white blood cells, white blood cell differential. Urinalysis: specific gravity, pH, protein, glucose, ketones, bilirubin, red blood cells, white blood cells, epithelial cells, bacteria, casts, crystals
– Change from Baseline in electrocardiograms (ECGs)
– Change from Baseline in vital signs. Vital signs will be assessed by: Resting systolic and diastolic blood pressure, pulse rate, respiratory rate, temperature, pulse oximetry, and transcutaneous carbon dioxide
– Change from Baseline in neurological examination outcomes
assessed by the following neurological examination: mental status, level of consciousness, sensory motor function, cranial nerve function, and reflexes
Secondary outcome measures:
– ISIS 396443 plasma concentration
– ISIS 396443 cerebrospinal fluid (CSF) concentration
Estimated Enrollment in the study will be approximately 21 subjects with the projected study start date being June 2015, and estimated study completion date of October 2017 and a preliminaty estimated primary completion date in July 2017 (Final data collection date for primary outcome measure).
Choosing to participate in a study is an important personal decision, and the study’s designers recommend consulting with your doctor and family members or friends about deciding to join a study.
This study’s ClinicalTrials.gov identifier is: NCT02462759
For more information, visit:
Keywords provided by Biogen:
Spinal Muscular Atrophy