Trial Outcomes & Findings for Open-label Extension Study of NEOD001 in Subjects With Light Chain (AL) Amyloidosis (NCT NCT02613182)
NCT ID: NCT02613182
Last Updated: 2019-05-16
Results Overview
Adverse events 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
34 participants
Primary outcome timeframe
From initiation of study drug through the last study visit or up to 30 days after date of last dose, whichever came first, assessed up to 24 months
Results posted on
2019-05-16
Participant Flow
Participant milestones
| Measure |
NEOD001 24 mg/kg
NEOD001, 24 mg/kg IV every 28 days for 22 months
|
|---|---|
|
Overall Study
STARTED
|
34
|
|
Overall Study
Number of Patients Dosed
|
34
|
|
Overall Study
COMPLETED
|
0
|
|
Overall Study
NOT COMPLETED
|
34
|
Reasons for withdrawal
| Measure |
NEOD001 24 mg/kg
NEOD001, 24 mg/kg IV every 28 days for 22 months
|
|---|---|
|
Overall Study
Death
|
6
|
|
Overall Study
Physician Decision
|
1
|
|
Overall Study
Study Terminated by Sponsor
|
21
|
|
Overall Study
Withdrawal by Subject
|
6
|
Baseline Characteristics
Open-label Extension Study of NEOD001 in Subjects With Light Chain (AL) Amyloidosis
Baseline characteristics by cohort
| Measure |
NEOD001 24 mg/kg
n=34 Participants
NEOD001, 24 mg/kg IV every 28 days for 22 months (open-label study drug)
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
17 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
17 Participants
n=5 Participants
|
|
Age, Continuous
|
65.1 years
STANDARD_DEVIATION 8.13 • n=5 Participants
|
|
Sex: Female, Male
Female
|
14 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
20 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
34 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
32 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: From initiation of study drug through the last study visit or up to 30 days after date of last dose, whichever came first, assessed up to 24 monthsAdverse events 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=34 Participants
NEOD001, 24 mg/kg IV every 28 days for 22 months
|
|---|---|
|
Long-term Safety and Tolerability of NEOD001
Death (all causes)
|
6 Participants
|
|
Long-term Safety and Tolerability of NEOD001
Death (from Adverse Events)
|
1 Participants
|
|
Long-term Safety and Tolerability of NEOD001
Overall number of baseline subjects
|
34 Participants
|
|
Long-term Safety and Tolerability of NEOD001
Serious Adverse Events
|
12 Participants
|
|
Long-term Safety and Tolerability of NEOD001
Non-Serious Adverse Events
|
34 Participants
|
Adverse Events
NEOD001 24 mg/kg
Serious events: 12 serious events
Other events: 34 other events
Deaths: 6 deaths
Serious adverse events
| Measure |
NEOD001 24 mg/kg
n=34 participants at risk
24 mg/kg IV every 28 days for 22 months
|
|---|---|
|
Infections and infestations
Infection
|
2.9%
1/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Infections and infestations
Lung Infection
|
2.9%
1/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Infections and infestations
Respiratory syncytial virus infection
|
2.9%
1/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Infections and infestations
Skin infection
|
2.9%
1/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Infections and infestations
Urinary tract infection
|
2.9%
1/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Cardiac disorders
Cardiac failure
|
5.9%
2/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Cardiac disorders
Acute myocardial infarction
|
2.9%
1/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Injury, poisoning and procedural complications
Fall
|
2.9%
1/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Injury, poisoning and procedural complications
Lower limb fracture
|
2.9%
1/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Metabolism and nutrition disorders
Hypervolaemia
|
2.9%
1/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Metabolism and nutrition disorders
Hyponatraemia
|
2.9%
1/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Nervous system disorders
Cerebrovascular accident
|
2.9%
1/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Nervous system disorders
Seizure
|
2.9%
1/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
2.9%
1/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Endocrine disorders
Adrenal insufficiency
|
2.9%
1/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
General disorders
Asthenia
|
2.9%
1/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Myelodysplastic syndrome
|
2.9%
1/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Renal and urinary disorders
Acute kidney injury
|
2.9%
1/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
2.9%
1/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
2.9%
1/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Vascular disorders
Hypotension
|
2.9%
1/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Infections and infestations
Viral pharyngitis
|
2.9%
1/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
Other adverse events
| Measure |
NEOD001 24 mg/kg
n=34 participants at risk
24 mg/kg IV every 28 days for 22 months
|
|---|---|
|
Infections and infestations
Upper respiratory tract infection
|
44.1%
15/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Infections and infestations
Urinary tract infection
|
8.8%
3/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Infections and infestations
Pneumonia
|
5.9%
2/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Gastrointestinal disorders
Oedema peripheral
|
26.5%
9/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
General disorders
Fatigue
|
17.6%
6/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
General disorders
Pyrexia
|
8.8%
3/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
General disorders
Chest discomfort
|
5.9%
2/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Gastrointestinal disorders
Constipation
|
23.5%
8/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Gastrointestinal disorders
Diarrhoea
|
17.6%
6/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Gastrointestinal disorders
Abdominal pain
|
14.7%
5/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Gastrointestinal disorders
Nausea
|
14.7%
5/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Gastrointestinal disorders
Abdominal distension
|
8.8%
3/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Gastrointestinal disorders
Abdominal discomfort
|
5.9%
2/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Gastrointestinal disorders
Dyspepsia
|
5.9%
2/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Gastrointestinal disorders
Dysphagia
|
5.9%
2/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Gastrointestinal disorders
Eructation
|
5.9%
2/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Gastrointestinal disorders
Vomiting
|
5.9%
2/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
23.5%
8/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
14.7%
5/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
5.9%
2/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
5.9%
2/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
5.9%
2/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Investigations
Weight increased
|
8.8%
3/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Investigations
Blood creatine phosphokinase increased
|
5.9%
2/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Investigations
Blood creatinine increased
|
5.9%
2/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Investigations
Weight decreased
|
5.9%
2/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Injury, poisoning and procedural complications
Contusion
|
11.8%
4/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Injury, poisoning and procedural complications
Fall
|
8.8%
3/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Injury, poisoning and procedural complications
Infusion related reaction
|
5.9%
2/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Nervous system disorders
Dizziness
|
14.7%
5/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Nervous system disorders
Headache
|
8.8%
3/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Skin and subcutaneous tissue disorders
Ecchymosis
|
17.6%
6/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Skin and subcutaneous tissue disorders
Rash
|
8.8%
3/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Blood and lymphatic system disorders
Anaemia
|
11.8%
4/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Blood and lymphatic system disorders
Neutropenia
|
8.8%
3/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Metabolism and nutrition disorders
Hypocalcaemia
|
8.8%
3/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Metabolism and nutrition disorders
Decreased appetite
|
5.9%
2/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
8.8%
3/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
8.8%
3/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
8.8%
3/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
5.9%
2/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Psychiatric disorders
Insomnia
|
20.6%
7/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Vascular disorders
Hypertension
|
8.8%
3/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Vascular disorders
Hypotension
|
5.9%
2/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Cardiac disorders
Palpitations
|
5.9%
2/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
|
Renal and urinary disorders
Dysuria
|
5.9%
2/34 • Initiation of study drug through the last study visit or up to 30 days after date of last dose
AE that newly appears, increases in frequency, or worsens in severity following initiation of study
|
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