Trial Outcomes & Findings for Study in Subjects With Light Chain (AL) Amyloidosis (NCT NCT03154047)
NCT ID: NCT03154047
Last Updated: 2019-03-26
Results Overview
AEs are defined as any unfavorable and unintended diagnosis, symptom, sign (including an abnormal laboratory finding), syndrome, or disease which either occurs during study, having been absent at baseline, or, if present at baseline, appears to worsen. Serious adverse events are any untoward medical occurrences that result in death, are life threatening, require (or prolong) hospitalization, cause persistent or significant disability/incapacity, result in congenital anomalies or birth defects, or are other conditions which in judgment of investigators represent significant hazards.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
80 participants
Primary outcome timeframe
Each subject's study participation may have been up to 36 months or until the study was terminated
Results posted on
2019-03-26
Participant Flow
Participant milestones
| Measure |
NEOD001 24 mg/kg
NEOD001, 24 mg/kg IV every 4 weeks for 36 months
|
|---|---|
|
Overall Study
STARTED
|
80
|
|
Overall Study
Number of Patients Dosed
|
80
|
|
Overall Study
COMPLETED
|
0
|
|
Overall Study
NOT COMPLETED
|
80
|
Reasons for withdrawal
| Measure |
NEOD001 24 mg/kg
NEOD001, 24 mg/kg IV every 4 weeks for 36 months
|
|---|---|
|
Overall Study
Death
|
1
|
|
Overall Study
Study terminated by sponsor
|
76
|
|
Overall Study
Withdrawal by Subject
|
3
|
Baseline Characteristics
Study in Subjects With Light Chain (AL) Amyloidosis
Baseline characteristics by cohort
| Measure |
NEOD001 24 mg/kg
n=80 Participants
NEOD001, 24 mg/kg IV every 4 weeks for 36 months
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
36 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
44 Participants
n=5 Participants
|
|
Age, Continuous
|
64.5 years
STANDARD_DEVIATION 8.62 • n=5 Participants
|
|
Sex: Female, Male
Female
|
30 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
50 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Asian
|
1 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Black or African American
|
2 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
White
|
74 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Other
|
3 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Not Hispanic or Latino
|
79 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Not Reported
|
1 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Each subject's study participation may have been up to 36 months or until the study was terminatedPopulation: OLE Safety Population
AEs are defined as any unfavorable and unintended diagnosis, symptom, sign (including an abnormal laboratory finding), syndrome, or disease which either occurs during study, having been absent at baseline, or, if present at baseline, appears to worsen. Serious adverse events are any untoward medical occurrences that result in death, are life threatening, require (or prolong) hospitalization, cause persistent or significant disability/incapacity, result in congenital anomalies or birth defects, or are other conditions which in judgment of investigators represent significant hazards.
Outcome measures
| Measure |
NEOD001 24 mg/kg
n=80 Participants
NEOD001, 24 mg/kg IV every 4 weeks
|
|---|---|
|
Number of Participants With Adverse Events
Serious Adverse Events
|
13 Participants
|
|
Number of Participants With Adverse Events
Non-Serious Adverse Events
|
57 Participants
|
|
Number of Participants With Adverse Events
Death (all causes)
|
1 Participants
|
|
Number of Participants With Adverse Events
Death Resulting from Adverse Events
|
1 Participants
|
|
Number of Participants With Adverse Events
Overall Number Baseline Subjects
|
80 Participants
|
Adverse Events
NEOD001 24 mg/kg
Serious events: 13 serious events
Other events: 28 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
NEOD001 24 mg/kg
n=80 participants at risk
NEOD001, 24 mg/kg IV every 4 weeks for 36 months
|
|---|---|
|
Infections and infestations
Device related infection
|
1.2%
1/80 • Initiation of study drug through study completion, up to 11 months
AE that newly appears, increases in frequency, or worsens in severity following initiation of study drug and through the last study visit or up to 30 days after date of last dose, whichever is later.
|
|
Infections and infestations
Endocarditis bacterial
|
1.2%
1/80 • Initiation of study drug through study completion, up to 11 months
AE that newly appears, increases in frequency, or worsens in severity following initiation of study drug and through the last study visit or up to 30 days after date of last dose, whichever is later.
|
|
Infections and infestations
Influenza
|
1.2%
1/80 • Initiation of study drug through study completion, up to 11 months
AE that newly appears, increases in frequency, or worsens in severity following initiation of study drug and through the last study visit or up to 30 days after date of last dose, whichever is later.
|
|
Infections and infestations
Lung Infection
|
1.2%
1/80 • Initiation of study drug through study completion, up to 11 months
AE that newly appears, increases in frequency, or worsens in severity following initiation of study drug and through the last study visit or up to 30 days after date of last dose, whichever is later.
|
|
Infections and infestations
Pneumonia
|
1.2%
1/80 • Initiation of study drug through study completion, up to 11 months
AE that newly appears, increases in frequency, or worsens in severity following initiation of study drug and through the last study visit or up to 30 days after date of last dose, whichever is later.
|
|
Infections and infestations
Sepsis
|
1.2%
1/80 • Initiation of study drug through study completion, up to 11 months
AE that newly appears, increases in frequency, or worsens in severity following initiation of study drug and through the last study visit or up to 30 days after date of last dose, whichever is later.
|
|
Infections and infestations
Septic embolus
|
1.2%
1/80 • Initiation of study drug through study completion, up to 11 months
AE that newly appears, increases in frequency, or worsens in severity following initiation of study drug and through the last study visit or up to 30 days after date of last dose, whichever is later.
|
|
Cardiac disorders
Atrial fibrillation
|
1.2%
1/80 • Initiation of study drug through study completion, up to 11 months
AE that newly appears, increases in frequency, or worsens in severity following initiation of study drug and through the last study visit or up to 30 days after date of last dose, whichever is later.
|
|
Cardiac disorders
Cardiac failure
|
1.2%
1/80 • Initiation of study drug through study completion, up to 11 months
AE that newly appears, increases in frequency, or worsens in severity following initiation of study drug and through the last study visit or up to 30 days after date of last dose, whichever is later.
|
|
Metabolism and nutrition disorders
Fluid overload
|
1.2%
1/80 • Initiation of study drug through study completion, up to 11 months
AE that newly appears, increases in frequency, or worsens in severity following initiation of study drug and through the last study visit or up to 30 days after date of last dose, whichever is later.
|
|
Metabolism and nutrition disorders
Hyponatraemia
|
1.2%
1/80 • Initiation of study drug through study completion, up to 11 months
AE that newly appears, increases in frequency, or worsens in severity following initiation of study drug and through the last study visit or up to 30 days after date of last dose, whichever is later.
|
|
Nervous system disorders
Cerebrovascular accident
|
2.5%
2/80 • Initiation of study drug through study completion, up to 11 months
AE that newly appears, increases in frequency, or worsens in severity following initiation of study drug and through the last study visit or up to 30 days after date of last dose, whichever is later.
|
|
Gastrointestinal disorders
Gastrointestinal angiodysplasia haemorrhagic
|
1.2%
1/80 • Initiation of study drug through study completion, up to 11 months
AE that newly appears, increases in frequency, or worsens in severity following initiation of study drug and through the last study visit or up to 30 days after date of last dose, whichever is later.
|
|
Injury, poisoning and procedural complications
Contusion
|
1.2%
1/80 • Initiation of study drug through study completion, up to 11 months
AE that newly appears, increases in frequency, or worsens in severity following initiation of study drug and through the last study visit or up to 30 days after date of last dose, whichever is later.
|
|
Respiratory, thoracic and mediastinal disorders
Pneumothorax
|
1.2%
1/80 • Initiation of study drug through study completion, up to 11 months
AE that newly appears, increases in frequency, or worsens in severity following initiation of study drug and through the last study visit or up to 30 days after date of last dose, whichever is later.
|
Other adverse events
| Measure |
NEOD001 24 mg/kg
n=80 participants at risk
NEOD001, 24 mg/kg IV every 4 weeks for 36 months
|
|---|---|
|
Infections and infestations
Upper respiratory tract infection
|
12.5%
10/80 • Initiation of study drug through study completion, up to 11 months
AE that newly appears, increases in frequency, or worsens in severity following initiation of study drug and through the last study visit or up to 30 days after date of last dose, whichever is later.
|
|
Gastrointestinal disorders
Fatigue
|
13.8%
11/80 • Initiation of study drug through study completion, up to 11 months
AE that newly appears, increases in frequency, or worsens in severity following initiation of study drug and through the last study visit or up to 30 days after date of last dose, whichever is later.
|
|
General disorders
Oedema peripheral
|
5.0%
4/80 • Initiation of study drug through study completion, up to 11 months
AE that newly appears, increases in frequency, or worsens in severity following initiation of study drug and through the last study visit or up to 30 days after date of last dose, whichever is later.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
5.0%
4/80 • Initiation of study drug through study completion, up to 11 months
AE that newly appears, increases in frequency, or worsens in severity following initiation of study drug and through the last study visit or up to 30 days after date of last dose, whichever is later.
|
|
Gastrointestinal disorders
Nausea
|
8.8%
7/80 • Initiation of study drug through study completion, up to 11 months
AE that newly appears, increases in frequency, or worsens in severity following initiation of study drug and through the last study visit or up to 30 days after date of last dose, whichever is later.
|
|
Nervous system disorders
Headache
|
6.2%
5/80 • Initiation of study drug through study completion, up to 11 months
AE that newly appears, increases in frequency, or worsens in severity following initiation of study drug and through the last study visit or up to 30 days after date of last dose, whichever is later.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The PI, institution and the sponsor have agreed that the PI and institution may publish or disclose study results from their site after the earlier of (a) publication of the complete multicenter study results or (b) 18 months after database lock for the multicenter study. The sponsor has at least 45 days to review a proposed publication and may request deletion of confidential information and up to 60 days additional delay to obtain patent protection.
- Publication restrictions are in place
Restriction type: OTHER