Clinical Trial Results:
A Phase 2, Open-Label, Repeat Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Proof-of-Concept of Intravenous ANX0005 in Subjects with Warm Autoimmune Hemolytic Anemia
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Summary
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EudraCT number |
2020-003675-18 |
Trial protocol |
DE AT FR CZ BG IT |
Global end of trial date |
17 Jan 2023
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Results information
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Results version number |
v1(current) |
This version publication date |
18 Oct 2026
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First version publication date |
18 Oct 2026
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Other versions |
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Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
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Trial identification
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Sponsor protocol code |
ANX005-wAIHA-02
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
NCT04691570 | ||
WHO universal trial number (UTN) |
- | ||
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Sponsors
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Sponsor organisation name |
Annexon, Inc.
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Sponsor organisation address |
1400 Sierra Point Parkway, Building C, 2nd Floor, Brisbane, CA, United States, 94005
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Public contact |
Study Coordinator, Annexon, Inc., 1 650-822-5500, clinicaltrials@annexonbio.com
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Scientific contact |
Study Coordinator, Annexon, Inc., 1 650-822-5500, clinicaltrials@annexonbio.com
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Paediatric regulatory details
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Is trial part of an agreed paediatric investigation plan (PIP) |
No
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Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Results analysis stage
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Analysis stage |
Final
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Date of interim/final analysis |
19 Jan 2024
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Is this the analysis of the primary completion data? |
Yes
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Primary completion date |
17 Jan 2023
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Global end of trial reached? |
Yes
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Global end of trial date |
17 Jan 2023
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Was the trial ended prematurely? |
No
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General information about the trial
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Main objective of the trial |
The main objective of the study is to evaluate the safety and tolerability of two once-weekly intravenous (IV) infusions of ANX005 in participants with warm autoimmune hemolytic anemia (wAIHA).
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Protection of trial subjects |
This study was conducted in accordance with the protocol and consensus ethical principles derived from international guidelines including the Declaration of Helsinki, Council for International Organizations of Medical Sciences (CIOMS) International Ethical Guidelines, applicable International Council for Harmonization of Technical Requirements for Pharmaceuticals for Human Use (ICH) GCP Guidelines, local IRB/IEC requirements and other applicable laws and regulations.
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Background therapy |
- | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
10 Nov 2021
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Long term follow-up planned |
No
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Independent data monitoring committee (IDMC) involvement? |
No
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Population of trial subjects
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Number of subjects enrolled per country |
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Country: Number of subjects enrolled |
Austria: 3
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Country: Number of subjects enrolled |
Bulgaria: 2
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Country: Number of subjects enrolled |
Australia: 1
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Worldwide total number of subjects |
6
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EEA total number of subjects |
5
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Number of subjects enrolled per age group |
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In utero |
0
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Preterm newborn - gestational age < 37 wk |
0
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Newborns (0-27 days) |
0
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Infants and toddlers (28 days-23 months) |
0
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Children (2-11 years) |
0
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Adolescents (12-17 years) |
0
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Adults (18-64 years) |
5
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From 65 to 84 years |
1
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85 years and over |
0
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Recruitment
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Recruitment details |
- | ||||||||
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Pre-assignment
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Screening details |
Participants with wAIHA who met eligibility criteria were enrolled. One participant was enrolled in the study but not dosed and data are not included for this participants | ||||||||
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Period 1
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Period 1 title |
Overall Study (overall period)
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Is this the baseline period? |
Yes | ||||||||
Allocation method |
Not applicable
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Blinding used |
Not blinded | ||||||||
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Arms
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Arm title
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ANX005 | ||||||||
Arm description |
Participants were administered intravenous infusion of ANX005 on Day 1 and Day 8. | ||||||||
Arm type |
Experimental | ||||||||
Investigational medicinal product name |
ANX005
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Infusion
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Routes of administration |
Intravenous use
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Dosage and administration details |
Two one-weekly intravenous infusions
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Baseline characteristics reporting groups
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Reporting group title |
ANX005
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Reporting group description |
Participants were administered intravenous infusion of ANX005 on Day 1 and Day 8. | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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End points reporting groups
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Reporting group title |
ANX005
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Reporting group description |
Participants were administered intravenous infusion of ANX005 on Day 1 and Day 8. | ||
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End point title |
Number of Participants with Treatment-emergent Adverse Events (TEAEs) [1] | ||||||||
End point description |
An adverse event (AE) was any untoward medical occurrence in a participant who had been administered a pharmaceutical product. An AE did not necessarily have a causal relationship with the product and therefore could be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a pharmaceutical product. An AE could arise with any use, route of administration, formulation, dose (including an overdose), or when used in combination with another pharmaceutical product. A TEAE was an AE with an onset date/time after the first infusion of ANX005 until the end of the study. A summary of serious and all other non-serious adverse events regardless of causality is located in the Adverse Events module.
Safety Population: all participants who received any amount of ANX005.
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End point type |
Primary
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End point timeframe |
Day 1 through Day 71
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| Notes [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: The analysis was descriptive in nature. |
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| No statistical analyses for this end point | |||||||||
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End point title |
Maximum Change from Baseline in Hemoglobin Levels [2] | ||||||||||||
End point description |
Maximum change from Baseline was calculated as the maximum post-Baseline value observed up to Day 71 minus the Baseline value. Baseline was defined as the last non-missing observation recorded before the first dose of ANX005.
Safety Population: all participants who received any amount of ANX005.
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End point type |
Primary
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End point timeframe |
Baseline up to Day 71
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| Notes [2] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: The analysis was descriptive in nature. |
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| No statistical analyses for this end point | |||||||||||||
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End point title |
Change from Baseline in Lactate Dehydrogenase Levels at Day 71 [3] | ||||||||||||
End point description |
Change from Baseline was calculated as the post-Baseline value at Day 71 minus the Baseline value. Baseline was defined as the last non-missing observation recorded before the first dose of ANX005.
Safety Population: all participants who received any amount of ANX005.
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End point type |
Primary
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End point timeframe |
Baseline, Day 71
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| Notes [3] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: The analysis was descriptive in nature. |
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| No statistical analyses for this end point | |||||||||||||
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End point title |
Change from Baseline in Percentage of Reticulocytes/Total Cells Count at Day 71 [4] | ||||||||||||
End point description |
Change from Baseline was calculated as the post-Baseline value at Day 71 minus the Baseline value.
Baseline was defined as the last non-missing observation recorded before the first dose of ANX005.
Safety Population: all participants who received any amount of ANX005.
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End point type |
Primary
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End point timeframe |
Baseline, Day 71
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| Notes [4] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: The analysis was descriptive in nature. |
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| No statistical analyses for this end point | |||||||||||||
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End point title |
Change from Baseline in Haptoglobin Levels at Day 71 [5] | ||||||||||||
End point description |
Change from Baseline was calculated as the post-Baseline value at Day 71 minus the Baseline value. Baseline was defined as the last non-missing observation recorded before the first dose of ANX005.
Safety Population: all participants who received any amount of ANX005.
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End point type |
Primary
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End point timeframe |
Baseline, Day 71
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| Notes [5] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: The analysis was descriptive in nature. |
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| No statistical analyses for this end point | |||||||||||||
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End point title |
Change from Baseline in Total Bilirubin Levels at Day 71 [6] | ||||||||||||
End point description |
Change from Baseline was calculated as the post-Baseline value at Day 71 minus the Baseline value. Baseline was defined as the last non-missing observation recorded before the first dose of ANX005.
Safety Population: all participants who received any amount of ANX005.
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End point type |
Primary
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End point timeframe |
Baseline, Day 71
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| Notes [6] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: The analysis was descriptive in nature. |
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| No statistical analyses for this end point | |||||||||||||
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End point title |
Change from Baseline in Indirect Bilirubin Levels at Day 71 [7] | ||||||||||||
End point description |
Change from Baseline was calculated as the post-Baseline value at Day 71 minus the Baseline value. Baseline was defined as the last non-missing observation recorded before the first dose of ANX005.
Safety Population: all participants who received any amount of ANX005.
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End point type |
Primary
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End point timeframe |
Baseline, Day 71
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| Notes [7] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: The analysis was descriptive in nature. |
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| No statistical analyses for this end point | |||||||||||||
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End point title |
Change in Percent Inhibition Complement CH50 from Baseline Through Day 71 | ||||||||||||||||||||||||||||||||
End point description |
Change in percent inhibition complement CH50 from Baseline was calculated as the post-Baseline value minus the Baseline value. Baseline was defined as the last non-missing observation recorded before the first dose of ANX005. A decrease from baseline indicated a better outcome.
Safety Population: all participants who received any amount of ANX005. 'Number analyzed' = participants evaluable at specified timepoint.
Baseline: n=5; Days 2, 4, 8, 15, 22, 36, 43, 50, and 71: n=4; Days 29 and 57: n=3
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End point type |
Secondary
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End point timeframe |
Baseline, Days 2, 4, 8, 15, 22, 29, 36, 43, 50, 57, and 71
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| No statistical analyses for this end point | |||||||||||||||||||||||||||||||||
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End point title |
Change from Baseline in Complement C4 Level through Day 71 | ||||||||||||||||||||||||||||||||||
End point description |
Change from Baseline was calculated as the post-Baseline value minus the Baseline value. Baseline was defined as the last non-missing observation recorded before the first dose of ANX005.
Safety Population: all participants who received any amount of ANX005. 'Number analyzed' = participants evaluable at specified timepoint.
Baseline and Days 4, 8 (predose), 15, 22, 50, Day 71: n=6; Days 2, 8 (end of infusion), 29, 36, and 43: n=5; Day 57: n=4
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End point type |
Secondary
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End point timeframe |
Baseline, Days 2, 4, 8 (pre-dose and end of infusion), 15, 22, 29, 36, 43, 50, 57, and 71
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| No statistical analyses for this end point | |||||||||||||||||||||||||||||||||||
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End point title |
Change from Baseline in Complement C1q Level through Day 71 | ||||||||||||||||||||||||||||||||||||||
End point description |
Change from Baseline was calculated as the post-Baseline value minus the Baseline value. Baseline was defined as the last non-missing observation recorded before the first dose of ANX005.
Safety Population: all participants who received any amount of ANX005. 'Number analyzed' = participants evaluable at specified timepoint.
Baseline, Day 4, 8 (pre-dose), 15, 22,: n=6; Days 2 (end of infusion), 2 (4 hours after infusion), 8 (end of infusion), 8 (4 hours after Infusion), 29, 36, 43: n =5; and Days 50, 57, and 71: n=4
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End point type |
Secondary
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End point timeframe |
Baseline, Days 2 (4 hours [hr] after Infusion and end of infusion), 4, 8 (pre-dose, 4 hr after Infusion, and end of infusion), 15, 22, 29, 36, 43, 50, 57, and 71
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| No statistical analyses for this end point | |||||||||||||||||||||||||||||||||||||||
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Adverse events information
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Timeframe for reporting adverse events |
Day 1 through Day 71
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Adverse event reporting additional description |
Safety Population: all participants who received any amount of ANX005.
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Assessment type |
Systematic | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Dictionary used for adverse event reporting
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Dictionary name |
MedDRA | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary version |
24.1
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Reporting groups
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Reporting group title |
ANX005
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Reporting group description |
Participants were administered intravenous infusion of ANX005 on Day 1 and Day 8. | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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| Frequency threshold for reporting non-serious adverse events: 0% | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Substantial protocol amendments (globally) |
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| Were there any global substantial amendments to the protocol? Yes | |||
Date |
Amendment |
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06 May 2021 |
Amendment 1, protocol version 2.0 |
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23 Mar 2022 |
Amendment 2, protocol version 3.0 |
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Interruptions (globally) |
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| Were there any global interruptions to the trial? No | |||
Limitations and caveats |
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| Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data. | |||
| None reported | |||
