AL AmyloidosisPhase 2OLENCT02613182
Open-label Extension Study of NEOD001 in Subjects With Light Chain (AL) Amyloidosis
A Phase 2 study testing NEOD001 in people with AL Amyloidosis, run by Prothena Biosciences Ltd. at 5 sites in United States.
Trial summary
The rationale for this study is to provide additional treatment with NEOD001 for subjects who complete Study NEOD001-001, and to continue to evaluate long term safety and tolerability. All subjects in the current NEOD001 trials are being dosed at 24 mg/kg, which will be continued in this study. Read more about this study
- Who is it for?
- Any age
- 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
- 34 participants
- Where
- 5 sitesUnited States
- Study dates
- Feb 2016to 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Previously enrolled and treated for at least 9 months in Study NEOD001-001
- Ability to understand and willingness to sign an informed consent form prior to initiation of any study procedures
- Has 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
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- Total bilirubin ≤2 times the upper limit of normal (× ULN)
- Aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) ≤3 × ULN
- Estimated glomerular filtration rate ≥30 mL/minute
- Seated systolic blood pressure 90 to 180 mmHg
- ECOG Performance Status 0 to 2
- Women of childbearing potential (WOCBP) must have a negative serum pregnancy test within 28 days prior to the first administration of study drug and agree to use highly effective physician-approved contraception from 30 days prior to the first study drug administration 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 30 days prior to the first study drug administration to 90 days following the last study drug administration
Who cannot join
- Any new medical contraindication or clinically significant abnormality on physical, neurological, laboratory, vital signs, or electrocardiogram (ECG) examination (e.g., atrial fibrillation; with the exception of subjects for whom the ventricular rate is controlled) that precludes continued or initiation of treatment with NEOD001 or participation in the study
- History of Grade ≥3 infusion-associated adverse events (AEs) or hypersensitivities to NEOD001 or any of its excipients
- Treatment with any anticancer therapy (standard or investigational) within the 14 days prior to the first dose of study drug. In addition, subjects must have fully recovered (i.e., National Cancer Institute Common Terminology Criteria for Adverse Events [CTCAE] Grade 1 [exception: subjects with prior bortezomib may have CTCAE Grade 2 neuropathy]) from the clinically significant toxic effects of that treatment
- Received any of the following within the specified time frame prior to the first administration of study drug:
- Hematopoietic growth factors, transfusions of blood or blood products within 1 week
- Major surgery within 2 weeks
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- Radiotherapy within 2 weeks
- Transplant within 8 weeks
- Investigational drug other than NEOD001 within 4 weeks
- Another experimental anti-amyloid therapy other than NEOD001 within 2 years
- Uncontrolled symptomatic orthostatic hypotension
- Myocardial infarction, uncontrolled angina, severe uncontrolled ventricular arrhythmias, or electrocardiographic evidence of acute ischemia, within 6 months prior to the first dose of study drug
- Uncontrolled infection
- Secondary malignancy, with the exception of:
- 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
- Any other cancer from which the subject has been disease-free for ≥3 years
- Uncontrolled human immunodeficiency virus (HIV), hepatitis B, or hepatitis C infection
- Women who are lactating
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
United States5 sites
Tufts Medical Center
Boston, Massachusetts, 02111
Karmanos Cancer Institute
Detroit, Michigan, 48201
Hospital of the University of Pennsylvania
Philadelphia, Pennsylvania, 19104
Mayo Clinic
Rochester, Minnesota, 55905
Stanford Cancer Institute (SCI)
Stanford, California, 94305
About this study
Official title: Open-label Extension Study to Evaluate the Long-term Safety and Tolerability of NEOD001 in Subjects With Light Chain (AL) Amyloidosis
The rationale for this study is to provide additional treatment with NEOD001 for subjects who complete Study NEOD001-001, and to continue to evaluate long term safety and tolerability. All subjects in the current NEOD001 trials are being dosed at 24 mg/kg, which will be continued in this study.
Read the detailed description
The rationale for this study is to provide additional treatment with NEOD001 for subjects who complete Study NEOD001-001, and to continue to evaluate long term safety and tolerability. All subjects in the current NEOD001 trials are being dosed at 24 mg/kg, which will be continued in this study. The primary objective of the study is to evaluate long-term safety and tolerability of NEOD001 and the secondary objectives are to assess the immunogenicity of NEOD001 and to incorporate serum NEOD001 concentrations in a population pharmacokinetic (PK) analysis.
- 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
NEOD001
| 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.
Long-term Safety and Tolerability of NEOD001
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.
Measured: 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
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Nov 2015 (estimated)
- Study startFeb 2016
- Primary completionMay 2018
- Study completionMay 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
- 34 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
- NCT02613182
- Sponsor's protocol ID
- NEOD001-OLE001
- Acronym
- OLE
- Status last verified
- May 2019
- First submitted
- 18 Nov 2015
- Last updated
- 16 May 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 May 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Open-label Extension Study to Evaluate the Long-term Safety and Tolerability of NEOD001 in Subjects With Light Chain (AL) Amyloidosis
- ClinicalTrials.gov ID
- NCT02613182
- Acronym / sponsor ID
- OLE / NEOD001-OLE001
- Conditions
- AL Amyloidosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: NEOD001 program terminated due to lack of clinical benefit
- Enrolment
- 34 participants (actual)
- Lead sponsor
- Prothena Biosciences Ltd.Industry (pharma, biotech, devices)
- Study start
- Feb 2016
- Primary completion
- May 2018 (actual)
- Study completion
- May 2018 (actual)
- First posted
- 24 Nov 2015 (estimated)
- Results
- Posted 16 May 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The rationale for this study is to provide additional treatment with NEOD001 for subjects who complete Study NEOD001-001, and to continue to evaluate long term safety and tolerability. All subjects in the current NEOD001 trials are being dosed at 24 mg/kg, which will be continued in this study.
Detailed description
The rationale for this study is to provide additional treatment with NEOD001 for subjects who complete Study NEOD001-001, and to continue to evaluate long term safety and tolerability. All subjects in the current NEOD001 trials are being dosed at 24 mg/kg, which will be continued in this study. The primary objective of the study is to evaluate long-term safety and tolerability of NEOD001 and the secondary objectives are to assess the immunogenicity of NEOD001 and to incorporate serum NEOD001 concentrations in a population pharmacokinetic (PK) analysis.
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
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (13 listed)
- Previously enrolled and treated for at least 9 months in Study NEOD001-001
- Ability to understand and willingness to sign an informed consent form prior to initiation of any study procedures
- Has adequate bone marrow reserve, hepatic and renal function, as demonstrated by:
- Seated systolic blood pressure 90 to 180 mmHg
- ECOG Performance Status 0 to 2
Key exclusion criteria (19 listed)
- Any new medical contraindication or clinically significant abnormality on physical, neurological, laboratory, vital signs, or electrocardiogram (ECG) examination (e.g., atrial fibrillation; with the exception of subjects for whom the ventricular rate is controlled) that precludes continued or initiation of treatment with NEOD001 or participation in the study
- History of Grade ≥3 infusion-associated adverse events (AEs) or hypersensitivities to NEOD001 or any of its excipients
- Treatment with any anticancer therapy (standard or investigational) within the 14 days prior to the first dose of study drug. In addition, subjects must have fully recovered (i.e., National Cancer Institute Common Terminology Criteria for Adverse Events [CTCAE] Grade 1 [exception: subjects with prior bortezomib may have CTCAE Grade 2 neuropathy]) from the clinically significant toxic effects of that treatment
- Received any of the following within the specified time frame prior to the first administration of study drug:
- Uncontrolled symptomatic orthostatic hypotension
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 |
|---|---|---|
| NEOD001NEOD001 | Drug | Open Label |
Primary outcome
Long-term Safety and Tolerability of NEOD001
Time frame: 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
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.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 May 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
5 sites in 1 country: 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 5 sites in United States.
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 13 inclusion and 19 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 16 May 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States. 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 16 May 2019). 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?
Long-term Safety and Tolerability of NEOD001 (time frame: 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). 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 16 May 2019.