Trial · NCT04253236
To Assess the Efficacy and Safety of RVT-1401 in the Treatment of Warm Autoimmune Hemolytic Anemia (ASCEND-WAIHA).
NCT04253236, Phase 2, Terminated, Immunovant Sciences GmbH; results first posted 2022-07-28
3 sourcestrials-2026-11-12-0.4.0
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ConductScience Library. To Assess the Efficacy and Safety of RVT-1401 in the Treatment of Warm Autoimmune Hemolytic Anemia (ASCEND-WAIHA). Trials, record NCT04253236. Release trials-2026-11-12-0.4.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04253236
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- To Assess the Efficacy and Safety of RVT-1401 in the Treatment of Warm Autoimmune Hemolytic Anemia (ASCEND-WAIHA).
- NCT number
- NCT04253236
- Status
- Terminated
- Phases
- Phase 2
- Enrollment
- 5 participants, actual
- Lead sponsor
- Immunovant Sciences GmbH
- Conditions
- Warm Autoimmune Hemolytic Anemia
- Interventions
- RVT-1401 680 mg/weekly, RVT-1401 340 mg/weekly
- First posted
- 2020-02-05
- Last updated
- 2022-07-28
- Results first posted
- 2022-07-28
Source ClinicalTrials.gov
Registered outcomes10
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Responders at Week 13 | Week 13 | 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. |
| Primary | Number of Participants With Any Treatment-emergent Adverse Event (TEAE), Serious AE (SAE), Treatment-related Adverse Event (AE), and Death | Up to Week 20 | 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. |
| Secondary | Time to Response | Up to Week 13 | 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). |
| Secondary | Time to Achieving Hb Levels in the Normal Range | Up to Week 13 | Time to achieving Hb levels in the normal range was assessed. |
| Secondary | Number of Participants With Change in Functional Assessment of Chronic Illness Therapy-fatigue (FACIT-F) Score | Up to Week 13 | 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. |
| Secondary | Number of Participants With Change in Medical Research Council (MRC) Breathlessness Scale | Up to Week 13 | 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. |
| Secondary | Number of Participants With Change in Euro Quality-5 Dimension-3 Level (EQ-5D-3L) Score | Up to Week 20 | 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). |
| Secondary | Concentration of RVT-1401 Pre-dose | Pre-dose, Weeks 1, 2, 3, 4, 5, 6, 8, 10, 12 and 13 post-dose | 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. |
| Secondary | Number of Participants With Presence of Anti-RVT 1401 Antibodies | Pre-dose on Weeks 1, 3, 5, 8, 13 and Week 20 | 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 |
| Secondary | Number of Participants With Change in Levels of Total Immunoglobulin (Ig)G and IgG Subclasses (1-4) | Up to Week 20 | 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. |
Source ClinicalTrials.gov
Outcomes10
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Responders at Week 13 | Week 13 | Safety population: All participants who enrolled in the study and received at least 1 dose of study treatment. Data was not collected for Cohort 2 due to early termination of the trial. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Any Treatment-emergent Adverse Event (TEAE), Serious AE (SAE), Treatment-related Adverse Event (AE), and Death | Up to Week 20 | Safety Population. Data was not collected for Cohort 2 due to early termination of the trial. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Time to Response | Up to Week 13 | Safety Population. Only those participants with data available at the specified time points were analyzed. Data was not collected for Cohort 2 due to early termination of the trial. | Weeks | NUMBER |
| SECONDARY | Time to Achieving Hb Levels in the Normal Range | Up to Week 13 | Safety Population. Only those participants with data available at the specified time points were analyzed. Data was not collected for Cohort 2 due to early termination of the trial. | Weeks | NUMBER |
| SECONDARY | Number of Participants With Change in Functional Assessment of Chronic Illness Therapy-fatigue (FACIT-F) Score | Up to Week 13 | Safety Population. Data was not collected for Cohort 2 due to early termination of the trial. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Change in Medical Research Council (MRC) Breathlessness Scale | Up to Week 13 | Safety Population. Data was not collected for Cohort 2 due to early termination of the trial. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Change in Euro Quality-5 Dimension-3 Level (EQ-5D-3L) Score | Up to Week 20 | Safety Population. Data was not collected for Cohort 2 due to early termination of the trial. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Concentration of RVT-1401 Pre-dose | Pre-dose, Weeks 1, 2, 3, 4, 5, 6, 8, 10, 12 and 13 post-dose | Safety Population. Data could not be calculated for Cohort 1 due to high proportion of non-quantifiable values (\>30 percent \[%\] of values were imputed). Data was not collected for Cohort 2 due to early termination of the trial. | Milligrams per liter | GEOMETRIC_MEAN |
| SECONDARY | Number of Participants With Presence of Anti-RVT 1401 Antibodies | Pre-dose on Weeks 1, 3, 5, 8, 13 and Week 20 | Safety population. Data was not collected for Cohort 2 due to early termination of the trial. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Change in Levels of Total Immunoglobulin (Ig)G and IgG Subclasses (1-4) | Up to Week 20 | Safety Population. Data was not collected for Cohort 2 due to early termination of the trial. | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group14
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Responders at Week 13 | Cohort 1: RVT-1401 680 mg/Week | 1 | Participants | ||
| Number of Participants With Any Treatment-emergent Adverse Event (TEAE), Serious AE (SAE), Treatment-related Adverse Event (AE), and Death | Cohort 1: RVT-1401 680 mg/Week | TEAEs | 5 | Participants | |
| Number of Participants With Any Treatment-emergent Adverse Event (TEAE), Serious AE (SAE), Treatment-related Adverse Event (AE), and Death | Cohort 1: RVT-1401 680 mg/Week | SAEs | 1 | Participants | |
| Number of Participants With Any Treatment-emergent Adverse Event (TEAE), Serious AE (SAE), Treatment-related Adverse Event (AE), and Death | Cohort 1: RVT-1401 680 mg/Week | Treatment-related AEs | 4 | Participants | |
| Number of Participants With Any Treatment-emergent Adverse Event (TEAE), Serious AE (SAE), Treatment-related Adverse Event (AE), and Death | Cohort 1: RVT-1401 680 mg/Week | Deaths | 0 | Participants | |
| Time to Response | Cohort 1: RVT-1401 680 mg/Week | 3 | Weeks | ||
| Time to Achieving Hb Levels in the Normal Range | Cohort 1: RVT-1401 680 mg/Week | 5 | Weeks | ||
| Number of Participants With Change in Functional Assessment of Chronic Illness Therapy-fatigue (FACIT-F) Score | Cohort 1: RVT-1401 680 mg/Week | 3 | Participants | ||
| Number of Participants With Change in Medical Research Council (MRC) Breathlessness Scale | Cohort 1: RVT-1401 680 mg/Week | 4 | Participants | ||
| Number of Participants With Change in Euro Quality-5 Dimension-3 Level (EQ-5D-3L) Score | Cohort 1: RVT-1401 680 mg/Week | 4 | Participants |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Cohort 1: RVT-1401 680 mg/Week | 5 | |
| Overall Study | STARTED | Cohort 2: RVT-1401 340 mg/Week | 0 | |
| Overall Study | COMPLETED | Cohort 1: RVT-1401 680 mg/Week | 2 | |
| Overall Study | COMPLETED | Cohort 2: RVT-1401 340 mg/Week | 0 | |
| Overall Study | NOT COMPLETED | Cohort 1: RVT-1401 680 mg/Week | 3 | |
| Overall Study | NOT COMPLETED | Cohort 2: RVT-1401 340 mg/Week | 0 |
Source ClinicalTrials.gov
Adverse events32
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Oedema peripheral | General disorders | Cohort 1: RVT-1401 680 mg/Week | 2 | 5 | 2 |
| other | Oedema peripheral | General disorders | Cohort 2: RVT-1401 340 mg/Week | 0 | 0 | 0 |
| other | Hypoalbuminaemia | Metabolism and nutrition disorders | Cohort 1: RVT-1401 680 mg/Week | 3 | 5 | 3 |
| other | Hypoalbuminaemia | Metabolism and nutrition disorders | Cohort 2: RVT-1401 340 mg/Week | 0 | 0 | 0 |
| other | Fatigue | General disorders | Cohort 1: RVT-1401 680 mg/Week | 2 | 5 | 3 |
| other | Fatigue | General disorders | Cohort 2: RVT-1401 340 mg/Week | 0 | 0 | 0 |
| other | Rhinitis | Infections and infestations | Cohort 1: RVT-1401 680 mg/Week | 1 | 5 | 1 |
| other | Rhinitis | Infections and infestations | Cohort 2: RVT-1401 340 mg/Week | 0 | 0 | 0 |
| other | Paraesthesia | Nervous system disorders | Cohort 1: RVT-1401 680 mg/Week | 1 | 5 | 1 |
| other | Paraesthesia | Nervous system disorders | Cohort 2: RVT-1401 340 mg/Week | 0 | 0 | 0 |
1 to 10 of 32Source ClinicalTrials.gov
See all 32 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Cohort 1: RVT-1401 680 mg/Week | 0 | 5 |
| Total, all-cause mortality | Cohort 2: RVT-1401 340 mg/Week | 0 | 0 |
| Total, other adverse events | Cohort 1: RVT-1401 680 mg/Week | 5 | 5 |
| Total, other adverse events | Cohort 2: RVT-1401 340 mg/Week | 0 | 0 |
| Total, serious adverse events | Cohort 1: RVT-1401 680 mg/Week | 1 | 5 |
| Total, serious adverse events | Cohort 2: RVT-1401 340 mg/Week | 0 | 0 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Source
- Release
- trials-2026-11-12-0.4.0
- Release manifest SHA-256
- 0f4a8d8c48aee2a7573ae0fa94ecbeab70c1adbe449bbc95c424bc7d3e97b9d4
- Registry capture
- 2026-11-12
- Results copy, AACT
- 2026-03-23
- NIH clinical studies file
- 2026-11-13
Source ClinicalTrials.gov
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNone stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04253236
GET https://conductscience.org/api/trials/v1/NCT04253236/events?page=2