A Randomized, Blinded, Placebo-Controlled, Phase 1 Single Ascending Dose Study in Healthy Adult Male Volunteers and an Open-Label Multiple Ascending Dose Study in Pediatric SMA Participants Previously Treated with Onasemnogene Abeparvovec (Zolgensma*) to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB115 - 277HV101

Biogen Idec Research Limited0 个研究点目标入组 42 人开始时间: 待定最近更新: 2024年4月9日

适应症

试验速览

阶段: 不适用
状态: 尚未招募
发起方: Biogen Idec Research Limited
入组人数: 42

研究概览入排标准研究者记录与标识符相似试验

研究概览

简要总结

暂无简介。

研究设计

研究类型: Interventional

入排标准

年龄范围: 0 至 64（—）

入选标准

•1. Ability to understand the purpose and risks of the study and provide signed
•and dated informed consent and authorization to use confidential health
•information in accordance with applicable privacy regulations.
•2. Males aged 18 to 55 years, inclusive, at the time of informed consent.
•3. Have a body mass index of 18 to 30 kg/m2, inclusive.
•4. All participants must practice highly effective contraception as described
•in Section 12.5 of the protocol.
•5. Must be in good health as determined by the Investigator, based on medical
•history and Screening evaluations.
•- 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 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

排除标准

•Part A: 1. Any reason, anatomical or otherwise (including abnormal
•hematology/coagulation), that presents increase of risk of complication from
•multiple LP procedures required for dosing and CSF collection, per the
•investigator discretion. 2. 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. 3. History of severe
•allergic or anaphylactic reactions, or of any allergic reactions that in the
•opinion of the Investigator are likely to be exacerbated by any component of
•the study treatment, including LP procedures. 4. History of, or ongoing
•malignancy, carcinoma in situ, or high-grade dysplasia (with the exception of
•no more than 1 basal cell carcinoma or squamous cell carcinoma that was
•completely excised and cured at least 12 months prior to randomization).
•Participants with cancers in remission for greater than 5 years prior to Day -1
•may be included after discussion with the Sponsor. 5. Systolic blood pressure
•> 150 mmHg or < 90 mmHg after resting in a sitting position for at least
•5 minutes at screening or prior to dosing. If out of range, testing may be
•repeated once at screening and once prior to dosing. 6. Clinically significant
•(as determined by the Investigator) 12-lead ECG abnormalities. 7. Confirmed
•demonstration of corrected QT interval, using Fridericia*s correction method,
•of > 450 ms. 8. Plans to undergo elective procedures or surgeries at any
•time after signing the ICF through the follow-up visit. 9. History of a suicide
•attempt within 5 years prior to screening or suicidal ideation in the past 6
•months as indicated by a positive response (*Yes*) to either Question 4 or
•Question 5 of the Screening/Baseline version of the C-SSRS at screening.
•Participants with a history of a suicide attempt spanning more than 5 years
•should be evaluated by a mental health care practitioner before enrollment in
•the study. 10. History of, or positive test result at Screening for, HIV. 11.
•History of hepatitis C infection or positive test result at Screening for HCV
•antibody. 12. Current hepatitis B infection (defined as positive for hepatitis
•B surface antigen [HBsAg] and/or total hepatitis B core antibody [anti-HBc]).
•Participants with immunity to hepatitis B from previous natural infection
•(defined as negative HBsAg, positive anti-HBc, and positive hepatitis B surface
•antibody [anti-HBs]) or vaccination (defined as negative HBsAg, negative
•anti-HBc, and positive anti-HBs) are eligible to participate in the study. 13.
•Chronic, recurrent, or serious infection (e.g., pneumonia, septicemia), as
•determined by the Investigator, within 90 days prior to Screening or between
•Screening and Day -1 14. For the following parameters, if a participant has an
•out-of-range result that is not clinically significant (as determined by the
•Investigator), the test may be repeated once during the Screening period. The
•participant may be enrolled if the repeated result is within the reference
•range. • Any value for ALT, AST, bilirubin, or serum creatinine that is above
•the ULN at Screening or Baseline. • Any value of hemoglobin that is < 7.45
•mmol/L (approx. < 12 g/dL) at Scre

研究者

发起方

Biogen Idec Research Limited

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