AmyloidosisPhase 2RAINNCT03168906
Renal AL Amyloid Involvement and NEOD001
A Phase 2 study testing NEOD001 in people with Amyloidosis, run by Tufts Medical Center at 9 sites in United States.
Trial summary
This is a multicenter, Phase 2b, randomized, double-blind, placebo-controlled, two-arm, parallel-group efficacy and safety study of NEOD001 as a single agent administered intravenously in adults with AL amyloidosis who have a maintained hematologic response to their most recent treatment for AL amyloidosis (e.g.… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Amyloidosis
- What is being tested?
- NEOD001
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 12 participants
- Where
- 9 sitesUnited States
- Study dates
- 5 Jul 2017to 6 Feb 2019
- 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
- 18 years of age or older
- Biopsy-proven diagnosis of AL amyloidosis by immunohistochemistry or mass spectroscopy of a tissue biopsy excluding bone marrow
- Screening renal biopsy for RAIN confirming AL amyloidosis as exclusive or dominant cause of renal damage
- Persistent renal involvement from diagnosis with proteinuria (predominantly albumin) > 500mg/day in a 24-hour urine collection
- CKD 1 to 3 (eGFR > 30)
- ≥1 prior systemic hematologic therapy for a free light chain (FLC) producing hematologic malignancy underlying the initial diagnosis of AL amyloidosis with at least a partial FLC response (PR, VGPR, CR) to treatment deemed stable and not requiring further treatment
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- ECOG Performance Status ≤ 2
- Clinical laboratory values:
- Absolute neutrophil count > 1000/μL
- Platelet count > 75,000/μL
- Total bilirubin ≤ 1.5X ULN
- Alkaline phosphatase ≤ 5X ULN
- NT-proBNP < 1800 pg/mL
- Voluntary written consent must be given before performance of any study-related procedure not part of standard medical care with the understanding that consent may be withdrawn by the patient at any time without prejudice to future medical care
Who cannot join
- Amyloidosis due to mutations of the transthyretin gene or presence of other non-AL amyloidosis
- Female patients who are lactating, breastfeeding, or pregnant
- Patients who have not been treated or who have received chemotherapy within 6 months, or SCT within 12 months, for the light-chain producing hematologic disease causing AL amyloidosis, at the time of the first dose of NEOD001 (month 1 day 1)
- Patients who at initial diagnosis or later met the International Myeloma Working Group (IMWG) definition of active multiple myeloma requiring therapy (Appendix 3)
- Patients whose screening renal biopsies for RAIN show dominant causes of renal damage not related to AL amyloidosis
- Medically documented cardiac syncope, uncompensated congestive heart failure, myocardial infarction within the previous 6 months, unstable angina pectoris, clinically significant repetitive atrial or ventricular arrhythmias despite antiarrhythmic treatment, or severe orthostatic hypotension or clinically significant uncompensated autonomic insufficiency
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- Comorbid systemic illnesses or other severe concurrent disease which, in the judgment of the investigator, would make the patient inappropriate for entry into this study or interfere significantly with the proper assessment of safety and toxicity of the prescribed regimens
- Ongoing or active infection, known HIV positive, known to be hepatitis B surface antigen-positive or has known or suspected active hepatitis C infection.
- Psychiatric illness/social situations that would limit compliance with study requirements
Trial locations and contacts
9 sites in 1 country.
9 sites in 1 country
United States9 sites
Tufts Medical Center
Boston, Massachusetts, 02111
Karmanos Cancer Institute
Detroit, Michigan, 48201
Mayo Clinic- Florida
Jacksonville, Florida, 32224
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
Mayo Clinic- Minnesota
Rochester, Minnesota, 55905
University of California San Francisco
San Francisco, California, 94143
Mayo Clinic- Arizona
Scottsdale, Arizona, 85259
About this study
Official title: The RAIN Study, a Multicenter Randomized Double-blind Phase 2b Study of NEOD001 in Previously Treated Subjects With Systemic Light-chain (AL) Amyloidosis and Persistent Renal Involvement
This is a multicenter, Phase 2b, randomized, double-blind, placebo-controlled, two-arm, parallel-group efficacy and safety study of NEOD001 as a single agent administered intravenously in adults with AL amyloidosis who have a maintained hematologic response to their most recent treatment for AL amyloidosis (e.g., chemotherapy, autologous stem cell transplant [ASCT]) and have persistent renal dysfunction.
Read the detailed description
This is a multicenter, Phase 2b, randomized, double-blind, placebo-controlled, two-arm, parallel-group efficacy and safety study of NEOD001 as a single agent administered intravenously in adults with AL amyloidosis who have a maintained hematologic response to their most recent treatment for AL amyloidosis (e.g., chemotherapy, autologous stem cell transplant [SCT]) and have persistent renal dysfunction. Subject screening will occur during the 28 days prior to the first administration of study drug (i.e. month 1 day 1). If screening assessments are completed and all eligibility requirements are met, the subject will be enrolled. 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 may receive up to 12 infusions of study drug. Subjects who discontinue study drug before the initial End of Study (EOS) visit should have an Early Treatment Discontinuation (ETD) Visit 30 (±5) days after their final administration of study drug. After completing 12 months of treatment and the confirmatory EOS visit, a subject may enter an open-label extension (OLE) study, during which subjects will receive active treatment with NEOD001 for 12 months and may receive concurrent chemotherapy.
- Conditions
- Amyloidosis
- Being tested
- NEOD001, Placebo
- Keywords
- Amyloidosis, NEOD001, RAIN
- Medical subject headings
- Amyloidosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NEOD001Drug
NEOD001 is a monoclonal antibody directed at soluble and insoluble light chain aggregates
PlaceboDrug
Saline bag
| Study group | Role | What they receive |
|---|---|---|
| NEOD001 | Experimental | Study Drug given IV every 28 days at 24mg/kg Receives: Drug: NEOD001 |
| Placebo | Placebo comparator | Placebo Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Confirmed Renal Response After Treatment With NEOD001
A renal response is a ≥ 30% reduction in proteinuria in the absence of a ≥ 25% decrease in eGFR. A confirmed renal response is one that has been documented as present one month after 12 monthly treatments.
Measured: Baseline to 13 Months
Secondary outcomes (7)
Measured GFR at Study Entry
The aim of this study is to assess the performance of CKD-EPI (Chronic Kidney Disease Epidemiology Collaboration) creatinine in comparison to iothalamate clearance measured in AL amyloidosis patients. Iothalamate will be given subcutaneously. Urine and plasma samples will then be obtained. All laboratory tests for samples obtained for GFR measurement will be performed at Mayo Clinic Rochester. Quantification of iothalamate in urine and plasma will be performed using a tandem mass spectrometric method.
Measured: Baseline
Time to CKD 4 or 5
Months to Chronic Kidney Disease level 4 or 5
Measured: Baseline to 13 Months
Time to eGFR ≤ 15 or Dialysis
Months to estimated Glomerular Filtration Rate ≤ 15 or dialysis
Measured: Baseline to 13 Months
Time to Doubling of Creatinine
Months to doubling of serum creatinine
Measured: Baseline to 13 Months
Time to ≥ 40% Reduction in eGFR
Months to ≥ 40% reduction in estimated glomerular filtration rate
Measured: Baseline to 13 Months
Renal Response in Patients With Maintained Hematologic Responses After 24 Monthly Treatments.
A renal response is a ≥ 30% decrease in proteinuria or drop of proteinuria below 0.5 g/24h in the absence of renal progression.
Measured: Baseline to 26 months
All Cause of Mortality at 26 Months
Death at 26 months from Baseline due to any cause
Measured: Baseline to 26 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 May 2017
- Study start5 Jul 2017
- Primary completion23 Apr 2018
- Study completion6 Feb 2019
Why it stopped: Futility
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 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Tufts Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Raymond Comenzo, MD, Study chair, Tufts Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03168906
- Sponsor's protocol ID
- NEOD001-RAIN
- Acronym
- RAIN
- Status last verified
- Aug 2020
- First submitted
- 26 Apr 2017
- Last updated
- 9 Sep 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Sep 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The RAIN Study, a Multicenter Randomized Double-blind Phase 2b Study of NEOD001 in Previously Treated Subjects With Systemic Light-chain (AL) Amyloidosis and Persistent Renal Involvement
- ClinicalTrials.gov ID
- NCT03168906
- Acronym / sponsor ID
- RAIN / NEOD001-RAIN
- Conditions
- Amyloidosis
- Keywords
- Amyloidosis, NEOD001, RAIN
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Futility
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Tufts Medical CenterUniversity, hospital or other
- Study start
- 5 Jul 2017 (actual)
- Primary completion
- 23 Apr 2018 (actual)
- Study completion
- 6 Feb 2019 (actual)
- First posted
- 30 May 2017 (actual)
- Results
- Posted 24 Dec 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a multicenter, Phase 2b, randomized, double-blind, placebo-controlled, two-arm, parallel-group efficacy and safety study of NEOD001 as a single agent administered intravenously in adults with AL amyloidosis who have a maintained hematologic response to their most recent treatment for AL amyloidosis (e.g., chemotherapy, autologous stem cell transplant [ASCT]) and have persistent renal dysfunction.
Detailed description
This is a multicenter, Phase 2b, randomized, double-blind, placebo-controlled, two-arm, parallel-group efficacy and safety study of NEOD001 as a single agent administered intravenously in adults with AL amyloidosis who have a maintained hematologic response to their most recent treatment for AL amyloidosis (e.g., chemotherapy, autologous stem cell transplant [SCT]) and have persistent renal dysfunction. Subject screening will occur during the 28 days prior to the first administration of study drug (i.e. month 1 day 1). If screening assessments are completed and all eligibility requirements are met, the subject will be enrolled. 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 may receive up to 12 infusions of study drug. Subjects who discontinue study drug before the initial End of Study (EOS) visit should have an Early Treatment Discontinuation (ETD) Visit 30 (±5) days after their final administration of study drug. After completing 12 months of treatment and the confirmatory EOS visit, a subject may enter an open-label extension (OLE) study, during which subjects will receive active treatment with NEOD001 for 12 months and may receive concurrent chemotherapy.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| NEOD001 | Experimental | Drug: NEOD001Study Drug given IV every 28 days at 24mg/kg |
| Placebo | Placebo comparator | Drug: PlaceboPlacebo |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (14 listed)
- 18 years of age or older
- Biopsy-proven diagnosis of AL amyloidosis by immunohistochemistry or mass spectroscopy of a tissue biopsy excluding bone marrow
- Screening renal biopsy for RAIN confirming AL amyloidosis as exclusive or dominant cause of renal damage
- Persistent renal involvement from diagnosis with proteinuria (predominantly albumin) > 500mg/day in a 24-hour urine collection
- CKD 1 to 3 (eGFR > 30)
Key exclusion criteria (9 listed)
- Amyloidosis due to mutations of the transthyretin gene or presence of other non-AL amyloidosis
- Female patients who are lactating, breastfeeding, or pregnant
- Patients who have not been treated or who have received chemotherapy within 6 months, or SCT within 12 months, for the light-chain producing hematologic disease causing AL amyloidosis, at the time of the first dose of NEOD001 (month 1 day 1)
- Patients who at initial diagnosis or later met the International Myeloma Working Group (IMWG) definition of active multiple myeloma requiring therapy (Appendix 3)
- Patients whose screening renal biopsies for RAIN show dominant causes of renal damage not related to AL amyloidosis
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 is a monoclonal antibody directed at soluble and insoluble light chain aggregates | Drug | NEOD001 |
| PlaceboSaline bag | Drug | Placebo |
Primary outcome
Confirmed Renal Response After Treatment With NEOD001
Time frame: Baseline to 13 Months
A renal response is a ≥ 30% reduction in proteinuria in the absence of a ≥ 25% decrease in eGFR. A confirmed renal response is one that has been documented as present one month after 12 monthly treatments.
7 secondary outcomes
Measured GFR at Study Entry
Time frame: Baseline
Time to CKD 4 or 5
Time frame: Baseline to 13 Months
Time to eGFR ≤ 15 or Dialysis
Time frame: Baseline to 13 Months
Time to Doubling of Creatinine
Time frame: Baseline to 13 Months
Time to ≥ 40% Reduction in eGFR
Time frame: Baseline to 13 Months
Renal Response in Patients With Maintained Hematologic Responses After 24 Monthly Treatments.
Time frame: Baseline to 26 months
All Cause of Mortality at 26 Months
Time frame: Baseline to 26 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Sep 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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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.
Raymond Comenzo, MD study chair
Tufts Medical Center
9 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 Amyloidosis, run by Tufts Medical Center at 9 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 14 inclusion and 9 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 9 Sep 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 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. Some participants in this study may receive a placebo. 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 24 Dec 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?
Confirmed Renal Response After Treatment With NEOD001 (time frame: Baseline to 13 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 9 Sep 2020.