A Study to Evaluate Higher Dose (HD) Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Previously Treated With Risdiplam (ASCEND)
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|ClinicalTrials.gov Identifier: NCT05067790|
Recruitment Status : Recruiting
First Posted : October 5, 2021
Last Update Posted : May 25, 2023
The primary objective of this study is to evaluate motor function following treatment with HD nusinersen in participants with spinal muscular atrophy (SMA) previously treated with risdiplam.
The secondary objective of this study is to evaluate the safety and tolerability of HD nusinersen in participants with SMA previously treated with risdiplam.
|Condition or disease||Intervention/treatment||Phase|
|Spinal Muscular Atrophy||Drug: Nusinersen||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||135 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase 3b Study to Evaluate Higher Dose Nusinersen (BIIB058) in Patients With Spinal Muscular Atrophy Previously Treated With Risdiplam|
|Actual Study Start Date :||January 21, 2022|
|Estimated Primary Completion Date :||June 14, 2027|
|Estimated Study Completion Date :||June 14, 2027|
Experimental: Higher Dose Nusinersen
There will be two groups of participants previously treated with risdiplam in the study (nusinersen-naive group and nusinersen-experienced group), who will receive HD nusinersen, administered as 2 loading doses of 50 milligrams (mg) each, approximately 2 weeks apart, followed by maintenance doses of 28 mg approximately every 4 months.
Administered as specified in the treatment arm
- Change in Total Revised Upper Limb Module (RULM) Score [ Time Frame: Up to Day 855 ]The RULM is being utilized to assess upper limb functional abilities of participants with SMA. This test consists of upper limb performance items that are reflective of activities of daily living. The RULM is scored from 0 to 37 points, with higher scores indicating better function.
- Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: Up to Day 855 ]An AE is any untoward medical occurrence in a participant administered a pharmaceutical product that does not necessarily have a causal relationship with the treatment. An AE can therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. An SAE is any untoward medical occurrence that at any dose results in death or in the view of the investigator, places the participant at immediate risk of death, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, results in a birth defect, or is a medically important event.
- Number of Participants With Change in Clinical Laboratory Parameters, Electrocardiogram (ECG), Vital Signs and Pulse Oximetry from Baseline [ Time Frame: Up to Day 855 ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05067790
|Contact: US Biogen Clinical Trial Centeremail@example.com|
|Contact: Global Biogen Clinical Trial Centerfirstname.lastname@example.org|
|Study Director:||Medical Director||Biogen|