Trial Outcomes & Findings for Long-term Safety of BCX9930 in Participants With Paroxysmal Nocturnal Hemoglobinuria (NCT NCT05741346)
NCT ID: NCT05741346
Last Updated: 2025-10-24
Results Overview
An adverse event (AE) is any untoward medical occurrence in a clinical study participant. No causal relationship with study intervention or with the clinical study itself is implied. An AE could be an unfavorable and unintended sign, symptom (including an abnormal laboratory finding), syndrome, or illness that developed or worsened during the clinical study. A serious adverse event (SAE) is defined as any untoward medical occurrence that results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, or other medically important event. An AE is considered treatment emergent if its start date was on or after the date of first dose of study treatment in Study 205 or if the AE was ongoing from the prior study. TEAEs included both serious TEAEs and non-serious TEAEs.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
28 participants
Primary outcome timeframe
From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Results posted on
2025-10-24
Participant Flow
The study was conducted in France, Hungary, Malaysia, South Africa, South Korea, Spain, and the United Kingdom.
A total of 28 participants were enrolled. The participants were enrolled from previous studies BCX9930-201 (2020-000501-93), BCX9930-202 (2020-004438-39), or BCX9930-203 (2020-004403-14).
Participant milestones
| Measure |
BCX9930
Participants who had completed at least 12 weeks of treatment with BCX9930 in studies BCX9930-201, BCX9930-202, or BCX9930-203, and in the opinion of the investigator, had benefited from treatment with BCX9930 and were expected to continue benefiting from BCX9930, with no other effective treatment options, continued to receive BCX9930 tablets at a dose of 400 milligram (mg) twice daily (BID) for up to 96 weeks. For participants who were permanently discontinuing BCX9930, in the absence of alternative complement inhibitor therapy, and if medically appropriate, the dose of BCX9930 was tapered based on investigator medical judgement.
|
|---|---|
|
Overall Study
STARTED
|
28
|
|
Overall Study
COMPLETED
|
0
|
|
Overall Study
NOT COMPLETED
|
28
|
Reasons for withdrawal
| Measure |
BCX9930
Participants who had completed at least 12 weeks of treatment with BCX9930 in studies BCX9930-201, BCX9930-202, or BCX9930-203, and in the opinion of the investigator, had benefited from treatment with BCX9930 and were expected to continue benefiting from BCX9930, with no other effective treatment options, continued to receive BCX9930 tablets at a dose of 400 milligram (mg) twice daily (BID) for up to 96 weeks. For participants who were permanently discontinuing BCX9930, in the absence of alternative complement inhibitor therapy, and if medically appropriate, the dose of BCX9930 was tapered based on investigator medical judgement.
|
|---|---|
|
Overall Study
Withdrawn due to termination of study
|
28
|
Baseline Characteristics
Long-term Safety of BCX9930 in Participants With Paroxysmal Nocturnal Hemoglobinuria
Baseline characteristics by cohort
| Measure |
BCX9930
n=28 Participants
Participants who had completed at least 12 weeks of treatment with BCX9930 in studies BCX9930-201, BCX9930-202, or BCX9930-203, and in the opinion of the investigator, had benefited from treatment with BCX9930 and were expected to continue benefiting from BCX9930, with no other effective treatment options, continued to receive BCX9930 tablets at a dose of 400 mg BID for up to 96 weeks. For participants who were permanently discontinuing BCX9930, in the absence of alternative complement inhibitor therapy, and if medically appropriate, the dose of BCX9930 was tapered based on investigator medical judgement.
|
|---|---|
|
Age, Continuous
|
41.1 years
STANDARD_DEVIATION 14.51 • n=5 Participants
|
|
Sex: Female, Male
Female
|
13 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
15 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Ethnicity: Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Ethnicity: Not Hispanic or Latino
|
25 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Ethnicity: Not Reported
|
1 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Ethnicity: Unknown
|
2 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Race: American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Race: Asian
|
5 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Race: Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Race: Black or African American
|
13 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Race: White
|
9 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Race: Other
|
1 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: From Day 1 up to 30 days after last dose (up to approximately 100 weeks)Population: Safety Population was defined as all participants who received at least 1 dose.
An adverse event (AE) is any untoward medical occurrence in a clinical study participant. No causal relationship with study intervention or with the clinical study itself is implied. An AE could be an unfavorable and unintended sign, symptom (including an abnormal laboratory finding), syndrome, or illness that developed or worsened during the clinical study. A serious adverse event (SAE) is defined as any untoward medical occurrence that results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, or other medically important event. An AE is considered treatment emergent if its start date was on or after the date of first dose of study treatment in Study 205 or if the AE was ongoing from the prior study. TEAEs included both serious TEAEs and non-serious TEAEs.
Outcome measures
| Measure |
BCX9930
n=28 Participants
Participants who had completed at least 12 weeks of treatment with BCX9930 in studies BCX9930-201, BCX9930-202, or BCX9930-203, and in the opinion of the investigator, had benefited from treatment with BCX9930 and were expected to continue benefiting from BCX9930, with no other effective treatment options, continued to receive BCX9930 tablets at a dose of 400 mg BID for up to 96 weeks. For participants who were permanently discontinuing BCX9930, in the absence of alternative complement inhibitor therapy, and if medically appropriate, the dose of BCX9930 was tapered based on investigator medical judgement.
|
|---|---|
|
Number of Participants With Treatment-emergent Events (TEAEs)
|
27 Participants
|
Adverse Events
BCX9930
Serious events: 16 serious events
Other events: 27 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
BCX9930
n=28 participants at risk
Participants who had completed at least 12 weeks of treatment with BCX9930 in studies BCX9930-201, BCX9930-202, or BCX9930-203, and in the opinion of the investigator, had benefited from treatment with BCX9930 and were expected to continue benefiting from BCX9930, with no other effective treatment options, continued to receive BCX9930 tablets at a dose of 400 mg BID for up to 96 weeks. For participants who were permanently discontinuing BCX9930, in the absence of alternative complement inhibitor therapy, and if medically appropriate, the dose of BCX9930 was tapered based on investigator medical judgement.
|
|---|---|
|
Renal and urinary disorders
Acute kidney injury
|
17.9%
5/28 • Number of events 6 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Renal and urinary disorders
Tubulointerstitial nephritis
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Infections and infestations
Escherichia urinary tract infection
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Infections and infestations
Urinary tract infection
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Infections and infestations
Pneumonia
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Investigations
Blood lactate dehydrogenase increased
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Investigations
C-reactive protein increased
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Vascular disorders
Hypotension
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Vascular disorders
Thrombophlebitis
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Blood and lymphatic system disorders
Haemolysis
|
35.7%
10/28 • Number of events 10 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Blood and lymphatic system disorders
Anaemia
|
7.1%
2/28 • Number of events 2 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Blood and lymphatic system disorders
Intravascular haemolysis
|
10.7%
3/28 • Number of events 5 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Gastrointestinal disorders
Diarrhoea
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary embolism
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
Other adverse events
| Measure |
BCX9930
n=28 participants at risk
Participants who had completed at least 12 weeks of treatment with BCX9930 in studies BCX9930-201, BCX9930-202, or BCX9930-203, and in the opinion of the investigator, had benefited from treatment with BCX9930 and were expected to continue benefiting from BCX9930, with no other effective treatment options, continued to receive BCX9930 tablets at a dose of 400 mg BID for up to 96 weeks. For participants who were permanently discontinuing BCX9930, in the absence of alternative complement inhibitor therapy, and if medically appropriate, the dose of BCX9930 was tapered based on investigator medical judgement.
|
|---|---|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Squamous cell carcinoma of skin
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Vascular disorders
Thrombophlebitis
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
General disorders
Fatigue
|
17.9%
5/28 • Number of events 5 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
General disorders
Pain
|
10.7%
3/28 • Number of events 3 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
General disorders
Pyrexia
|
7.1%
2/28 • Number of events 2 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
General disorders
Asthenia
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
General disorders
Inflammation
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Reproductive system and breast disorders
Breast mass
|
7.7%
1/13 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Reproductive system and breast disorders
Vaginal haemorrhage
|
7.7%
1/13 • Number of events 2 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Reproductive system and breast disorders
Erectile dysfunction
|
6.7%
1/15 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
7.1%
2/28 • Number of events 2 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory disorder
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary embolism
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Psychiatric disorders
Insomnia
|
7.1%
2/28 • Number of events 2 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Psychiatric disorders
Major depression
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Investigations
Blood cholesterol increased
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Investigations
Blood lactate dehydrogenase increased
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Investigations
Heart rate increased
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Investigations
Lung diffusion test decreased
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Investigations
SARS-CoV-2 test positive
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Injury, poisoning and procedural complications
Epicondylitis
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Injury, poisoning and procedural complications
Overdose
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Injury, poisoning and procedural complications
Skin laceration
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Cardiac disorders
Tachycardia
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Nervous system disorders
Headache
|
17.9%
5/28 • Number of events 6 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Nervous system disorders
Dizziness
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Nervous system disorders
Hypersomnia
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Nervous system disorders
Multiple sclerosis
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Nervous system disorders
Radiculopathy
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Blood and lymphatic system disorders
Haemolysis
|
32.1%
9/28 • Number of events 15 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Blood and lymphatic system disorders
Anaemia
|
7.1%
2/28 • Number of events 3 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Blood and lymphatic system disorders
Splenomegaly
|
3.6%
1/28 • Number of events 2 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Ear and labyrinth disorders
Vertigo
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Gastrointestinal disorders
Diarrhoea
|
14.3%
4/28 • Number of events 4 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Gastrointestinal disorders
Nausea
|
14.3%
4/28 • Number of events 5 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Gastrointestinal disorders
Abdominal pain
|
7.1%
2/28 • Number of events 2 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Gastrointestinal disorders
Toothache
|
7.1%
2/28 • Number of events 2 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Gastrointestinal disorders
Vomiting
|
7.1%
2/28 • Number of events 4 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Gastrointestinal disorders
Abdominal pain upper
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Gastrointestinal disorders
Dyspepsia
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Gastrointestinal disorders
Glossodynia
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Gastrointestinal disorders
Dysphagia
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Gastrointestinal disorders
Oesophageal spasm
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
10.7%
3/28 • Number of events 3 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Skin and subcutaneous tissue disorders
Acne
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Skin and subcutaneous tissue disorders
Rash pruritic
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Skin and subcutaneous tissue disorders
Skin lesion
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Skin and subcutaneous tissue disorders
Rash
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Renal and urinary disorders
Acute kidney injury
|
14.3%
4/28 • Number of events 5 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Renal and urinary disorders
Haemoglobinuria
|
10.7%
3/28 • Number of events 5 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Renal and urinary disorders
Haematuria
|
7.1%
2/28 • Number of events 2 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Renal and urinary disorders
Chronic kidney disease
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Renal and urinary disorders
Micturition urgency
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Renal and urinary disorders
Pollakiuria
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Renal and urinary disorders
Sterile pyuria
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Renal and urinary disorders
Urinary tract pain
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Renal and urinary disorders
Dysuria
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
14.3%
4/28 • Number of events 5 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
10.7%
3/28 • Number of events 3 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Musculoskeletal and connective tissue disorders
Gouty arthritis
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Musculoskeletal and connective tissue disorders
Muscle fatigue
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Infections and infestations
Upper respiratory tract infection
|
21.4%
6/28 • Number of events 11 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Infections and infestations
Nasopharyngitis
|
7.1%
2/28 • Number of events 2 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Infections and infestations
Urinary tract infection
|
7.1%
2/28 • Number of events 2 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Infections and infestations
Acute sinusitis
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Infections and infestations
COVID-19
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Infections and infestations
Candida infection
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Infections and infestations
Folliculitis
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Infections and infestations
Herpes simplex reactivation
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Infections and infestations
Influenza
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Infections and infestations
Periorbital cellulitis
|
3.6%
1/28 • Number of events 2 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Infections and infestations
Pulmonary tuberculosis
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Infections and infestations
Rhinovirus infection
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Infections and infestations
Viral upper respiratory tract infection
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Metabolism and nutrition disorders
Gout
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Metabolism and nutrition disorders
Hypercalcaemia
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Metabolism and nutrition disorders
Hyperuricaemia
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Metabolism and nutrition disorders
Hypoglycaemia
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
|
Metabolism and nutrition disorders
Hypomagnesaemia
|
3.6%
1/28 • Number of events 1 • From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place