Primary AmyloidosisPhase 1/2NCT01707264
Phase 1/2, Open Label, Dose Escalation Study of NEOD001 in Subjects With Light Chain (AL) Amyloidosis
A Phase 1/2 study testing NEOD001 in people with Primary Amyloidosis, run by Prothena Biosciences Ltd. at 7 sites in United States.
Trial summary
Dose escalation study to determine the maximum tolerated dose of NEOD001 in approximately 30 subjects with AL amyloidosis. Expansion phase to evaluate safety, efficacy and pharmacokinetics of NEOD001 in 25 additional subjects at the maximum tolerated dose. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Primary Amyloidosis
- What is being tested?
- NEOD001
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 69 participants
- Where
- 7 sitesUnited States
- Study dates
- Apr 2013to 9 Aug 2016
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
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
- Males and females aged ≥18 years;
- ECOG performance status (PS) 0-2;
- Diagnosis of systemic AL amyloidosis (subjects with non-AL amyloidosis are not eligible);
- Received at least one prior systemic therapy, which may include stem cell transplant, for AL amyloidosis;
- Have adequate organ function;
- Ability to understand and willingness to sign informed consent prior to initiation of any study procedures.
Who cannot join
- Secondary or familial amyloidosis;
- Life expectancy of < 3 months;
- Symptomatic multiple myeloma;
- Hypersensitivities to other monoclonal antibodies;
- Known HIV infection;
- Women who are lactating;
- Any other condition or prior therapy, which in the opinion of the PI, would make the subject unsuitable for the study.
Trial locations and contacts
7 sites in 1 country.
7 sites in 1 country
United States7 sites
Tufts Medical Center
Boston, Massachusetts, 02111
Boston University School of Medicine
Boston, Massachusetts, 02118
Karmanos Cancer Institute
Detroit, Michigan, 48201
Memorial Sloan-Kettering Cancer Center
New York, New York, 10065
Stanford University Cancer Center
Palo Alto, California, 94305
Hospital of the University of Pennsylvania
Philadelphia, Pennsylvania, 19104
Mayo Clinic
Rochester, Minnesota, 55905
About this study
Official title: A Phase 1/2, Open Label, Dose Escalation Study of Intravenous Administration of Single Agent NEOD001 in Subjects With Light Chain (AL) Amyloidosis
Dose escalation study to determine the maximum tolerated dose of NEOD001 in approximately 30 subjects with AL amyloidosis. Expansion phase to evaluate safety, efficacy and pharmacokinetics of NEOD001 in 25 additional subjects at the maximum tolerated dose.
Read the detailed description
The purpose of the dose escalation phase of the study is to determine the maximum tolerated dose/Phase 2 recommended dose of NEOD001 when given as a single agent intravenously in approximately 30 subjects with AL amyloidosis.
The purpose of the expansion phase of the study is to evaluate the safety, preliminary efficacy and pharmacokinetics of single agent NEOD001 at the maximum tolerated dose/Phase 2 recommended dose in approximately 25 additional evaluable subjects.
- Conditions
- Primary Amyloidosis
- Being tested
- NEOD001
- Keywords
- AL amyloidosis, Primary amyloidosis
- 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
Monoclonal antibody administered by intravenous infusion every 28 days.
| Study group | Role | What they receive |
|---|---|---|
| NEOD001 | Experimental | NEOD001 will be administered intravenously once every 28 days. The starting dose will be 0.5 mg/kg. Dose escalation will continue until the the maximum tolerated dose is determined for single agent NEOD001. Approximately 20 additional subjects will be treated with the maximum tolerated dose. Receives: Drug: NEOD001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and tolerability
- Adverse event profile
- Dose limiting toxicity and maximum tolerated dose
Measured: 28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator
Maximum tolerated dose
- Adverse event profile
- Dose Limiting Toxicity and maximum tolerated dose
Measured: 28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator
Secondary outcomes (2)
Pharmacokinetics
- Pharmacokinetic parameters including Cmax, Tmax, AUC, Cav, Cmin, t½, CL, and Vz
Measured: 28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator
Immunogenicity
- Measurement of anti-NEOD001 antibodies
Measured: 28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Oct 2012 (estimated)
- Study startApr 2013
- Primary completion9 Aug 2016
- Study completion9 Aug 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Primary 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 1/2
- Primary purpose
- Treatment
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 69 participants (actual)
Sponsor and investigators
- Lead sponsor
- Prothena Biosciences Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Julia Martinisi, Study director, Prothena Biosciences Ltd.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01707264
- Sponsor's protocol ID
- NEOD001-001
- Status last verified
- Aug 2018
- First submitted
- 11 Oct 2012
- Last updated
- 28 Aug 2018
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Gertz MA, Landau H, Comenzo RL, Seldin D, Weiss B, Zonder J, Merlini G, Schonland S, Walling J, Kinney GG, Koller M, Schenk DB, Guthrie SD, Liedtke M. First-in-Human Phase I/II Study of NEOD001 in Patients With Light Chain Amyloidosis and Persistent Organ Dysfunction. J Clin Oncol. 2016 Apr 1;34(10):1097-103. doi: 10.1200/JCO.2015.63.6530. Epub 2016 Feb 8.
PubMed 26858336
Study at a glance
From the registry record, last updated by the sponsor on 28 Aug 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2, Open Label, Dose Escalation Study of Intravenous Administration of Single Agent NEOD001 in Subjects With Light Chain (AL) Amyloidosis
- ClinicalTrials.gov ID
- NCT01707264
- Acronym / sponsor ID
- NEOD001-001
- Conditions
- Primary Amyloidosis
- Keywords
- AL amyloidosis, Primary amyloidosis
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 69 participants (actual)
- Lead sponsor
- Prothena Biosciences Ltd.Industry (pharma, biotech, devices)
- Study start
- Apr 2013 (actual)
- Primary completion
- 9 Aug 2016 (actual)
- Study completion
- 9 Aug 2016 (actual)
- First posted
- 16 Oct 2012 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Dose escalation study to determine the maximum tolerated dose of NEOD001 in approximately 30 subjects with AL amyloidosis. Expansion phase to evaluate safety, efficacy and pharmacokinetics of NEOD001 in 25 additional subjects at the maximum tolerated dose.
Detailed description
The purpose of the dose escalation phase of the study is to determine the maximum tolerated dose/Phase 2 recommended dose of NEOD001 when given as a single agent intravenously in approximately 30 subjects with AL amyloidosis.
The purpose of the expansion phase of the study is to evaluate the safety, preliminary efficacy and pharmacokinetics of single agent NEOD001 at the maximum tolerated dose/Phase 2 recommended dose in approximately 25 additional evaluable subjects.
Study design
- Primary purpose
- Treatment
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| NEOD001 | Experimental | Drug: NEOD001NEOD001 will be administered intravenously once every 28 days. The starting dose will be 0.5 mg/kg. Dose escalation will continue until the the maximum tolerated dose is determined for single agent NEOD001. Approximately 20 additional subjects will be treated with the maximum tolerated dose. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Males and females aged ≥18 years;
- ECOG performance status (PS) 0-2;
- Diagnosis of systemic AL amyloidosis (subjects with non-AL amyloidosis are not eligible);
- Received at least one prior systemic therapy, which may include stem cell transplant, for AL amyloidosis;
- Have adequate organ function;
Key exclusion criteria (7 listed)
- Secondary or familial amyloidosis;
- Life expectancy of < 3 months;
- Symptomatic multiple myeloma;
- Hypersensitivities to other monoclonal antibodies;
- Known HIV infection;
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 |
|---|---|---|
| NEOD001Monoclonal antibody administered by intravenous infusion every 28 days. | Drug | NEOD001 |
Primary outcome
Safety and tolerability
Time frame: 28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator
* Adverse event profile * Dose limiting toxicity and maximum tolerated dose
Maximum tolerated dose
Time frame: 28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator
* Adverse event profile * Dose Limiting Toxicity and maximum tolerated dose
2 secondary outcomes
Pharmacokinetics
Time frame: 28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator
Immunogenicity
Time frame: 28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Aug 2018.
- Derived from this study: PubMed 26858336Gertz MA, Landau H, Comenzo RL, Seldin D, Weiss B, Zonder J, Merlini G, Schonland S, Walling J, Kinney GG, Koller M, Schenk DB, Guthrie SD, Liedtke M. First-in-Human Phase I/II Study of NEOD001 in Patients With Light Chain Amyloidosis and Persistent Organ Dysfunction. J Clin Oncol. 2016 Apr 1;34(10):1097-103. doi: 10.1200/JCO.2015.63.6530. Epub 2016 Feb 8.
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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.
Julia Martinisi study director
Prothena Biosciences Ltd.
7 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing NEOD001 in people with Primary Amyloidosis, run by Prothena Biosciences Ltd. at 7 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 6 inclusion and 7 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: finished, as of the sponsor's last update on 28 Aug 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Safety and tolerability (time frame: 28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01707264, which also lists 1 linked publication.
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 28 Aug 2018.