AL AmyloidosisPhase 2NCT03154047
Study in Subjects With Light Chain (AL) Amyloidosis
A Phase 2 study testing NEOD001 in people with AL Amyloidosis, run by Prothena Biosciences Ltd. at 34 sites in 10 countries.
Trial summary
The objective of this study is to evaluate the long-term safety and efficacy of NEOD001 in subjects with AL amyloidosis who have completed Study NEOD001-201. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- AL Amyloidosis
- What is being tested?
- NEOD001
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 80 participants
- Where
- 34 sitesin 10 countries
- Study dates
- 14 Jun 2017to 30 May 2018
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Completed the End of Study Visit in Study NEOD001-201
- Adequate bone marrow reserve, hepatic and renal function, as demonstrated by:
- Absolute neutrophil count (ANC) ≥1.0 × 109/L
- Platelet count ≥75 × 109/L
- Hemoglobin ≥9 g/dL
- Total bilirubin ≤2 × upper limit of normal (ULN)
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- Aspartate aminotransferase (AST) ≤3 × ULN
- Alanine aminotransferase (ALT) ≤3 × ULN
- Alkaline phosphatase (ALP) ≤5 × ULN (except for subjects with hepatomegaly and isozymes specific to liver, rather than bone)
- Estimated glomerular filtration rate (eGFR) ≥25 mL/min/1.73 m2 as estimated by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation, or measured GFR ≥25 mL/min/1.73 m2
- Systolic blood pressure 80-180 mmHg
- Women of childbearing potential must have a negative pregnancy test during Screening and must agree to use highly effective physician-approved contraception from Screening to 90 days following the last study drug administration
- Male subjects must be surgically sterile or must agree to use highly effective physician-approved contraception from Screening to 90 days following the last study drug administration
- Ability to understand and willingness to sign an informed consent form prior to initiation of any study procedures
Who cannot join
- Any new medical contraindication or clinically significant abnormality on physical, neurological, laboratory, vital signs, or electrocardiographic (ECG) examination (e.g., atrial fibrillation; with the exception of subjects for whom the ventricular rate is controlled) that precludes continuation or initiation of treatment with NEOD001 or participation in the study
- Symptomatic orthostatic hypotension that in the medical judgment of the Investigator would interfere with subject's ability to safely receive treatment or complete study assessments
- Myocardial infarction, uncontrolled angina, uncontrolled ventricular arrhythmias, or ECG evidence of acute ischemia, within 6 months prior to the Month 1-Day 1 Visit
- Severe valvular stenosis (e.g., aortic or mitral stenosis with a valve area <1.0 cm2) or severe congenital heart disease
- ECG evidence of acute ischemia or active conduction system abnormalities with the exception of any of the following:
- First degree atrioventricular (AV) block
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- Second degree AV block Type 1 (Mobitz Type 1/ Wenckebach type)
- Right or left bundle branch block
- Atrial fibrillation with a controlled ventricular rate (uncontrolled [i.e., >110 bpm] ventricular rate is not allowed [determined by an average of three beats in Lead II or 3 representative beats if Lead II is not representative of the overall ECG])
- Has not recovered (i.e., equivalent to a Common Terminology Criteria for Adverse Events [CTCAE] ≥Grade 2) from the clinically significant toxic effects of prior anticancer therapy. Exception: subjects who have received treatment with a proteasome inhibitor such as bortezomib may have CTCAE Grade 2 neuropathy.
- Received any of the following within the specified time frame prior to the Month 1-Day 1 Visit:
- Oral or IV antibiotics, antifungals, or antivirals within 1 week, with the exception of prophylactic oral agents. Note: In the event that a subject requires the chronic use of antivirals, Medical Monitor permission is required for entry into the study.
- Hematopoietic growth factors, transfusions of blood or blood products within 1 week
- Chemotherapy, radiotherapy, HDAC inhibitors, or other plasma cell directed therapy within 2 weeks
- ASCT within 4 weeks (i.e., ASCT is allowed if it occurred before enrollment in Study NEOD001-201 or after completion of Study NEOD001-201 if it was at least 4 weeks before Month 1-Day 1 of this study)
- Major surgery within 4 weeks (or within 2 weeks following consultation with and approval of Medical Monitor)
- Planned organ transplant during the study
- Any investigational agent, other than NEOD001, within 4 weeks
- Any experimental imaging agent directed at amyloid within 2 weeks
- Active malignancy with the exception of any of the following:
- Adequately treated basal cell carcinoma, squamous cell carcinoma, or in situ cervical cancer
- Adequately treated Stage I cancer from which the subject is currently in remission and has been in remission for ≥2 years
- Low-risk prostate cancer with Gleason score <7 and prostate-specific antigen <10 mg/mL
- Any other cancer from which the subject has been disease-free for ≥2 years
- History of Grade ≥3 infusion-related adverse events (AEs) or hypersensitivity to NEOD001
- History of severe allergy to any of the components of NEOD001 such as histidine/L-Histidine, Trehalose, or Polysorbate 20
- Currently known uncontrolled bacterial, viral, fungal, HIV, hepatitis B, or hepatitis C infection
- Women who are breastfeeding
- Any condition which could interfere with, or the treatment for which might interfere with, the conduct of the study or which would, in the opinion of the Investigator, unacceptably increase the subject's risk by participating in the study
- Unable or unwilling to adhere to the study-specified procedures and restrictions
- Subject is under legal custodianship
Trial locations and contacts
34 sites in 10 countries.
34 sites in 10 countries
United States16 sites
Tufts Medical Center
Boston, Massachusetts, 02111
University of Chicago Medicine
Chicago, Illinois, 60637
Cleveland Clinic
Cleveland, Ohio, 44195
Colorado Blood Cancer Institute
Denver, Colorado, 80218
Karmanos Cancer Institute
Detroit, Michigan, 48201
City of Hope
Duarte, California, 91010
Duke University Medical Center
Durham, North Carolina, 27705
The University of Texas MD Anderson Cancer Center
Houston, Texas, 77030
Indiana University Simon Cancer Center
Indianapolis, Indiana, 46202
Mayo Clinic Hospital - Florida
Jacksonville, Florida, 32224
Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
Vanderbilt University Medical Center
Nashville, Tennessee, 37232
Memorial Sloan-Kettering Cancer Center
New York, New York, 10065
Hospital of the University of Pennsylvania
Philadelphia, Pennsylvania, 19104
Oregon Health & Science University
Portland, Oregon, 97239
Mayo Clinic - Minnesota
Rochester, Minnesota, 48201
Germany4 sites
Charité - Universitätsmedizin Berlin
Berlin, 12200
University of Duisburg-Essen
Essen, 45122
Universitätsklinikum Hamburg-Eppendorf (UKE)
Hamburg, 20246
Universitätsklinikum Heidelberg
Heidelberg, 69120
Australia3 sites
Eastern Health (Box Hill Hospital)
Box Hill, Victoria, 3128
Westmead Hospital
Sydney, New South Wales, 2145
Princess Alexandra Hospital
Woolloongabba, Queensland, 4102
France2 sites
Hôpital Dupuytren - CHU Limoges
Limoges, 87042
Hôpital Pitié-Salpêtrière
Paris, 75651
Greece2 sites
Alexandra General Hospital of Athens
Athens, 11528
University Hospital of Patras
Pátrai
Spain2 sites
Hospital Clínic de Barcelona
Barcelona, 08036
Hospital Universitario Puerta de Hierro - Majadahonda
Majadahonda, 28222
United Kingdom2 sites
Queen Elizabeth Hospital
Birmingham, England, B15 2GW
The Royal Free Hospital
London, England, NW3 2QG
About this study
Official title: A Phase 2b Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of NEOD001 in Subjects With Light Chain (AL) Amyloidosis Who Were Previously Enrolled in Study NEOD001-201 (PRONTO)
The objective of this study is to evaluate the long-term safety and efficacy of NEOD001 in subjects with AL amyloidosis who have completed Study NEOD001-201.
Read the detailed description
Global, multicenter, Phase 2b, open-label extension study of subjects with AL amyloidosis who had a hematologic response to first-line treatment for their amyloidosis (e.g., chemotherapy, autologous stem cell transplant [ASCT]) and completed Study NEOD001-201. Subjects in this study may receive concomitant chemotherapy. Subject screening will occur during the 28 days prior to the first administration of study drug, which may overlap with the last visit in Study NEOD001-201. If all eligibility requirements are met, the subject will be enrolled and Screening assessments will be completed. Study visits will occur every 28 days based on scheduling from Month 1 Day 1. A ±5-day window is allowed for visits starting after Month 1. Subjects who discontinue study drug before the End of Study Visit (EOS) should have an Early Treatment Discontinuation Visit 30 (±5) days after their final administration of study drug. Each subject's study participation may be up to 38 months or until the study is terminated, whichever occurs first. The study consists of a Screening Phase (1 month), Treatment Phase (36 months), and EOS Visit (30 [±5] days after the last dose).
- Conditions
- AL Amyloidosis
- Being tested
- NEOD001
- Medical subject headings
- Immunoglobulin Light-chain Amyloidosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NEOD001Drug
humanized monoclonal antibody
| Study group | Role | What they receive |
|---|---|---|
| Open label | Experimental | Open Label Study Drug NEOD001 Receives: Drug: NEOD001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Adverse Events
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.
Measured: Each subject's study participation may have been up to 36 months or until the study was terminated
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 May 2017
- Study start14 Jun 2017
- Primary completion30 May 2018
- Study completion30 May 2018
Why it stopped: NEOD001 program terminated due to lack of clinical benefit
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for AL AmyloidosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 80 participants (actual)
Sponsor and investigators
- Lead sponsor
- Prothena Biosciences Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03154047
- Sponsor's protocol ID
- NEOD001-OLE251
- Status last verified
- Mar 2019
- First submitted
- 28 Apr 2017
- Last updated
- 26 Mar 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 26 Mar 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2b Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of NEOD001 in Subjects With Light Chain (AL) Amyloidosis Who Were Previously Enrolled in Study NEOD001-201 (PRONTO)
- ClinicalTrials.gov ID
- NCT03154047
- Acronym / sponsor ID
- NEOD001-OLE251
- Conditions
- AL Amyloidosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: NEOD001 program terminated due to lack of clinical benefit
- Enrolment
- 80 participants (actual)
- Lead sponsor
- Prothena Biosciences Ltd.Industry (pharma, biotech, devices)
- Study start
- 14 Jun 2017 (actual)
- Primary completion
- 30 May 2018 (actual)
- Study completion
- 30 May 2018 (actual)
- First posted
- 15 May 2017 (actual)
- Results
- Posted 12 Dec 2018
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The objective of this study is to evaluate the long-term safety and efficacy of NEOD001 in subjects with AL amyloidosis who have completed Study NEOD001-201.
Detailed description
Global, multicenter, Phase 2b, open-label extension study of subjects with AL amyloidosis who had a hematologic response to first-line treatment for their amyloidosis (e.g., chemotherapy, autologous stem cell transplant [ASCT]) and completed Study NEOD001-201. Subjects in this study may receive concomitant chemotherapy. Subject screening will occur during the 28 days prior to the first administration of study drug, which may overlap with the last visit in Study NEOD001-201. If all eligibility requirements are met, the subject will be enrolled and Screening assessments will be completed. Study visits will occur every 28 days based on scheduling from Month 1 Day 1. A ±5-day window is allowed for visits starting after Month 1. Subjects who discontinue study drug before the End of Study Visit (EOS) should have an Early Treatment Discontinuation Visit 30 (±5) days after their final administration of study drug. Each subject's study participation may be up to 38 months or until the study is terminated, whichever occurs first. The study consists of a Screening Phase (1 month), Treatment Phase (36 months), and EOS Visit (30 [±5] days after the last dose).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Open label | Experimental | Drug: NEOD001Open Label Study Drug NEOD001 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (14 listed)
- Completed the End of Study Visit in Study NEOD001-201
- Adequate bone marrow reserve, hepatic and renal function, as demonstrated by:
- Systolic blood pressure 80-180 mmHg
- Women of childbearing potential must have a negative pregnancy test during Screening and must agree to use highly effective physician-approved contraception from Screening to 90 days following the last study drug administration
- Male subjects must be surgically sterile or must agree to use highly effective physician-approved contraception from Screening to 90 days following the last study drug administration
Key exclusion criteria (31 listed)
- Any new medical contraindication or clinically significant abnormality on physical, neurological, laboratory, vital signs, or electrocardiographic (ECG) examination (e.g., atrial fibrillation; with the exception of subjects for whom the ventricular rate is controlled) that precludes continuation or initiation of treatment with NEOD001 or participation in the study
- Symptomatic orthostatic hypotension that in the medical judgment of the Investigator would interfere with subject's ability to safely receive treatment or complete study assessments
- Myocardial infarction, uncontrolled angina, uncontrolled ventricular arrhythmias, or ECG evidence of acute ischemia, within 6 months prior to the Month 1-Day 1 Visit
- Severe valvular stenosis (e.g., aortic or mitral stenosis with a valve area <1.0 cm2) or severe congenital heart disease
- ECG evidence of acute ischemia or active conduction system abnormalities with the exception of any of the following:
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| NEOD001humanized monoclonal antibody | Drug | Open label |
Primary outcome
Number of Participants With Adverse Events
Time frame: Each subject's study participation may have been up to 36 months or until the study was terminated
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.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Mar 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: Undecided
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Venetoclax-Dexamethasone in Relapsed and/or Refractory t(11;14) Amyloidosis
AL Amyloidosis
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
34 sites in 10 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing NEOD001 in people with AL Amyloidosis, run by Prothena Biosciences Ltd. at 34 sites in 10 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 14 inclusion and 31 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 26 Mar 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 34 sites in United States, Germany, Australia, France, Greece and 5 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 12 Dec 2018). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Adverse Events (time frame: Each subject's study participation may have been up to 36 months or until the study was terminated). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Mar 2019.