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Universitatsklinikum Essen | Ruhrlandklinik - Zentrum fur Schlaf- und Telemedizin

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A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB115

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Biogen

Status and phase

Enrolling
Phase 1

Conditions

Muscular Atrophy, Spinal
Healthy Volunteer

Treatments

Drug: BIIB115
Drug: BIIB115-Matching Placebo

Study type

Interventional

Funder types

Industry

Identifiers

NCT05575011
277HV101
2022-000956-12 (EudraCT Number)

Details and patient eligibility

About

The primary objective of the study is to evaluate the safety and tolerability of single ascending dose of BIIB115 administered via intrathecal (IT) bolus injection to healthy male participants in Part A and multiple ascending doses of BIIB115 administered via IT bolus injection to pediatric Spinal Muscular Atrophy (SMA) participants previously treated with onasemnogene abeparvovec in Part B. The secondary objective of the study is to evaluate the pharmacokinetics (PK) of single-dose of BIIB115 administered via IT bolus injection to healthy male participants in Part A and multiple ascending doses of BIIB115 administered via IT bolus injection to pediatric SMA participants who previously received onasemnogene abeparvovec in Part B.

Full description

This is a first-in-human (FIH) study to evaluate the safety, tolerability, and PK of BIIB115, and consists two parts.

Part A includes cohorts 1 through 4, where healthy volunteer (HV) participants will be assigned to receive a single dose of either BIIB115 or placebo. This part will have a 4-week Screening Period, a Treatment Period, and a 13-month Follow-up Period. The study duration for each participant will be approximately 14 months.

Part B includes cohorts 5 and 6, where pediatric participants with SMA will be assigned to receive 2 doses of BIIB115. This part will have a 4-week Screening Period, a Treatment Period, and a 24-month Follow-up Period. The study duration for each participant will be approximately 25 months.

The study will be conducted in approximately 20 sites globally.

Enrollment

62 estimated patients

Sex

All

Ages

6 months to 55 years old

Volunteers

Accepts Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

Part A:

  • Male healthy participants aged 18 to 55 years, inclusive
  • Have a body mass index of 18 to 30 kilograms per meter square (kg/m^2), inclusive
  • Must be in good health as determined by the investigator, based on medical history and screening evaluations

Part B:

  • Age 0.5 to 12 years old, inclusive, at the time of informed consent
  • Weight ≥7 kg at the time of informed consent
  • Genetic diagnosis of SMA (5q SMA homozygous survival motor neuron 1 (SMN1) gene deletion or mutation or compound heterozygous mutation)
  • Survival motor neuron 2 (SMN2) copy number ≥1
  • Must have received intravenous (IV) onasemnogene abeparvovec per the approved label or per guidelines including the steroid regimen and monitoring specified therein
  • Treatment with onasemnogene abeparvovec ≥180 days prior to first BIIB115 dose
  • Potential for improvement due to suboptimal clinical status secondary to SMA, as determined by the Investigator

Key Exclusion Criteria:

Part A:

  • Any reason, anatomical or otherwise (including abnormal hematology/coagulation), that presents increase of risk of complication from multiple lumbar puncture (LP) procedures required for dosing and CSF collection, per the investigator discretion
  • History of any clinically significant cardiac, endocrine, gastrointestinal, hematologic, hepatic, immunologic, metabolic, urologic, pulmonary, neurologic, dermatologic, psychiatric, or renal disease, or other major disease, as determined by the Investigator
  • Chronic, recurrent, or serious infection, as determined by the investigator, within 90 days prior to screening or between screening and Day -1
  • Current enrollment or a plan to enroll in any interventional clinical study of a drug, biologic, or device, in which an investigational treatment or approved therapy for investigational use is administered within 3 months (or 5 half-lives of the agent, whichever is longer) prior to randomization

Part B:

  • Severe or serious AEs related to onasemnogene abeparvovec therapy that are ongoing during Screening
  • Interval of <180 days between onasemnogene abeparvovec therapy and first BIIB115 dose
  • Ongoing steroid treatment following onasemnogene abeparvovec at time of screening
  • History of drug induced liver injury or liver failure per Hy's law definition
  • History of thrombotic micrangiopathy
  • Treatment with any SMN2-splicing modifier (nusinersen or risdiplam) after receiving onasemnogene abeparvovec. Treatment with nusinersen <12 months from the first dose of BIIB115.
  • Any reason, anatomical or otherwise (including abnormal hematology/coagulation), that presents increase of risk of complication from the LP procedures, CSF circulation, or safety assessments, including a history of hydrocephalus or implanted shunt for CSF drainage.
  • Permanent ventilation, defined as tracheostomy or ≥16 hours ventilation /day continuously for >21 days in the absence of an acute reversible event

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

Trial design

Primary purpose

Treatment

Allocation

Randomized

Interventional model

Sequential Assignment

Masking

Quadruple Blind

62 participants in 7 patient groups, including a placebo group

Part A: Cohort 1: BIIB115 Dose 1
Experimental group
Description:
Participants will receive a single dose of BIIB115, Dose 1, via IT bolus injection, on Day 1.
Treatment:
Drug: BIIB115
Part A: Cohort 2: BIIB115 Dose 2
Experimental group
Description:
Participants will receive a single dose of BIIB115, Dose 2, via IT bolus injection, on Day 1.
Treatment:
Drug: BIIB115
Part A: Cohort 3: BIIB115 Dose 3
Experimental group
Description:
Participants will receive a single dose of BIIB115, Dose 3, via IT bolus injection, on Day 1.
Treatment:
Drug: BIIB115
Part A: Cohort 4: BIIB115 Dose 4
Experimental group
Description:
Participants will receive a single dose of BIIB115, Dose 4, via IT bolus injection, on Day 1.
Treatment:
Drug: BIIB115
Part A: Cohorts 1-4: BIIB115-Matching Placebo
Placebo Comparator group
Description:
Participants will receive a single dose of BIIB115-matching placebo, via IT bolus injection, on Day 1.
Treatment:
Drug: BIIB115-Matching Placebo
Part B: Cohort 5: BIIB115 Dose 3
Experimental group
Description:
Pediatric SMA participants previously treated with onasemnogene abeparvovec will receive two doses of BIIB115, Dose 3, via IT bolus injection at two separate time points.
Treatment:
Drug: BIIB115
Part B: Cohort 6: BIIB115 Dose 4
Experimental group
Description:
Pediatric SMA participants previously treated with onasemnogene abeparvovec will receive two doses of BIIB115, Dose 4, via IT bolus injectionat two separate time points.
Treatment:
Drug: BIIB115

Trial contacts and locations

18

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Central trial contact

Global Biogen Clinical Trial Center; US Biogen Clinical Trial Center

Data sourced from clinicaltrials.gov

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