Trial Outcomes & Findings for To Assess the Efficacy and Safety of RVT-1401 in the Treatment of Warm Autoimmune Hemolytic Anemia (ASCEND-WAIHA). (NCT NCT04253236)

A total of 11 participants were screened, of which 5 participants were enrolled and randomized into the study. The study was terminated early prior to completion of dosing all participants in Cohort 1 and prior to initiating Cohort 2, as efficacy and safety conclusions could not be drawn and Pharmacokinetics/Pharmacodynamics (PK/PD) data were limited, due to the small number of participants (n=5) enrolled in the study.

To Assess the Efficacy and Safety of RVT-1401 in the Treatment of Warm Autoimmune Hemolytic Anemia (ASCEND-WAIHA).

Responders were defined as the participants with level of hemoglobin (Hb) \>=10 grams per deciliter (g/dL) with at least a \>=2 g/dL increase from Baseline without rescue therapy or blood transfusions in the previous two weeks.

AEs were defined as any untoward medical occurrence in a participant or clinical investigation participant, temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. Clinically significant changes determined by the Investigator such as vital signs, Electrocardiograms (ECGs), and clinical laboratory values were also reported as AEs. TEAEs were defined as AEs that either started on or after the date of the first dose of study drug. SAEs were defined as any untoward medical occurrence that, at any dose: resulted in death, was life-threatening, required hospitalization or prolongation of existing hospitalization, resulted in disability/incapacity, was a congenital anomaly/birth defect, or was an important medical event that may have jeopardized the participant or may have required medical or surgical intervention to prevent one of the other outcomes listed in the definition.

The time to response was defined as the amount of time to achieve response (Hb levels \>=10 g/dL with at least a \>=2 g/dL increase from Baseline without rescue therapy or blood transfusions in the previous 2 weeks).

Time to achieving Hb levels in the normal range was assessed.

The FACIT-F scale was a validated scale which measured the physical, emotional and social implications of fatigue, one of the key clinical manifestations of warm autoimmune hemolytic anemia. Scores ranged from 0-52, a higher score indicated a higher quality of life. A score of less than 30 indicated severe fatigue. The scale took approximately 5-10 minutes to complete.

The MRC Breathlessness scale is a questionnaire that consisted of 5 statements about perceived Breathlessness and the focus of the scale was to quantify the disability associated with breathlessness. Score ranged from Grade 0 (limited to no disability) to Grade 4 (severe disability); higher score indicated severe disability.

The EQ-5D-3L is a validated measurement of health-related quality of life. The scale consists of 2 components, the EQ-5D descriptive system and the EQ visual analogue scale (VAS). The descriptive system evaluates mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension has 3 levels: 1=no problems, 2=some problems, and 3=extreme problems; a lower score indicated better quality of life. The EQ VAS records the participant's self-rated health on a vertical visual analogue scale where the endpoints are labelled 'Best imaginable health state' (100) and 'Worst imaginable health state' (0).

Blood samples were planned to be collected at indicated time points to measure the concentration of RVT-1401 pre-dose (Ctrough) as an assessment of the pharmacokinetic (PK) RVT-1401.

Blood samples were collected at indicated time points to determine presence of anti-RVT 1401 antibodies. Participants with presence of anti-RVT 1401 antibodies is reported

Blood samples were collected at indicated time points for pharmacodynamic (PD) analysis of serum total IgG and IgG subclasses (1-4) concentrations. Participants with changes in levels of Total IgG and IgG Subclasses (1-4) is reported.