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

  chris.shaw@kcl.ac.uk

• Sponsor organisation

  Biogen Idec Research Limited

• Eudract number

  2019-000641-12

• Duration of Study in the UK

  2 years, 0 months, 1 days

• Research summary

  This is a first-in-humans, open-label study to evaluate the safety, tolerability and the pharmacokinetics [how a drug is processed](PK) of the study drug (BIIB089) in adults with Type III Spinal Muscular Atrophy (SMA). The study drug has been tested in animals but, since the study drug has not yet been tested in humans, the safety in humans is not known. \n\nParticipants will receive a single dose of the study drug (BIIB089) on Day 1. The study drug 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 study team will monitor participants’ health closely for at least 24 hours and check for any reactions to the study drug. Participants will then be followed-up for 12 months afterwards.\n\nThe study will also look at other things, including:\n•\thow the study drug affects physical symptoms of SMA, such as movement (sometimes called “motor function”), lung function, and tiredness\n•\twhat the body does to the study drug (“pharmacokinetics”)\n•\thow the study drug works in the body (“pharmacodynamics”)\n•\thow the immune system reacts to the study drug \n•\thow the body responds to the study drug, by looking at biomarkers in the blood and in the liquid that surrounds the brain and spinal cord (“cerebrospinal fluid”, or CSF). Biomarkers are substances that occur naturally in the body and can change as a result of a disease or treatment.\n\n6 participants will be enrolled globally and will be enrolled in 1 of 2 treatment groups:\n - Cohort 1, low dose (n=3)\n - Cohort 2, high dose (n=3)\n\nIf needed, additional participants may be added to Cohort 1 to support the dose escalation decision. Participation in the study will last approximately 1 year. \n

• REC name

  London - West London & GTAC Research Ethics Committee

• REC reference

  19/LO/1389

• Date of REC Opinion

  4 Oct 2019

• REC opinion

  Unfavourable Opinion