256SM101: Dose Escalation Study of BIIB089 to Adults With Type III SMA
Research type
Research Study
Full title
A Phase 1/2 Open-Label, Multicenter, Dose-Escalation Study to Assess the Safety, Tolerability, and Pharmacokinetics of Single Doses of BIIB089 Delivered Into the Cisterna Magna of Adults With Type III Spinal Muscular Atrophy
IRAS ID
270252
Contact name
Christopher Shaw
Contact email
Sponsor organisation
Biogen Idec Research Limited
Eudract number
2019-000641-12
Duration of Study in the UK
1 years, 11 months, 4 days
Research summary
This is a first-in-human, open-label gene therapy study to evaluate the safety and tolerability of the study drug (BIIB089) in adults with Type III Spinal Muscular Atrophy (SMA). BIIB089 is an Adeno-associated virus (AAV); a small virus that infects humans but does not cause disease. The virus causes a very mild reaction in the body. Gene Therapy is an experimental technique that uses genes to treat or prevent disease. This method may allow doctors to treat a disorder by inserting a gene (unit of DNA) into a patient’s cells.\nThe study drug has been tested in animals but as it has not yet been tested in humans, the safety in humans is unknown. \nParticipants will receive a single dose of the study drug (BIIB089) on Day 1. This will be given as a single injection into a fluid-filled area at the base of the brain, called the “cisterna magna”, at either a low or high dose. After the injection, the participants’ health will be closely observed for at least 24 hours to check for any reactions to the study drug. Participants will then be followed-up for 1 year.\n\nThe study will also look at the following:\n• how the study drug affects physical symptoms of SMA, such as movement (often called “motor function”), lung function, and tiredness\n• what the body does to the study drug (“pharmacokinetics”)\n• how the study drug works in the body (“pharmacodynamics”)\n• how the immune system reacts to the study drug \n• how the body responds to the study drug, by looking at specific cells in the blood and liquid that surrounds the brain and spinal cord (“cerebrospinal fluid”, or CSF). The cells may indicate a change as a result of a disease or treatment.\n\n6 participants will be enrolled and will be enrolled in 1 of 2 treatment groups:\n- Cohort 1, low dose\n- Cohort 2, high dose\n\nAdditional participants may be added to Cohort 1 to support the dose escalation decision.
REC name
London - West London & GTAC Research Ethics Committee
REC reference
19/LO/1947
Date of REC Opinion
20 Dec 2019
REC opinion
Unfavourable Opinion