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AL AmyloidosisPhase 1/2NCT01570387

A Phase I/II Trial of Pomalidomide and Dexamethasone in Subjects With Previously-Treated AL Amyloidosis

A Phase 1/2 study testing Pomalidomide and Dexamethasone in people with AL Amyloidosis, run by Boston Medical Center at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

This study seeks to enroll patients with AL amyloidosis, for whom treatment with one of the standard melphalan chemotherapy-based regimens is either not recommended or is not their preference. Pomalidomide (CC-4047) is a drug given by mouth, which can change or regulate the functioning of the immune system. 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?
Pomalidomide and Dexamethasone
Could I receive a placebo?
No, everyone receives an active treatment
How many people
27 participants
Where
1 siteUnited States
Study dates
Jun 2012to Apr 2019
Phase
Phase 1/2
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

  • Understand and voluntarily sign informed consent form.
  • ≥18yrs old
  • Able to adhere to the study visit schedule and other protocol requirements.
  • Biopsy proven tissue amyloid deposits or positive fat aspirate
  • Proof of AL type (a or b)
  • Measurable plasma cell dyscrasia (a or b and c of the following required):
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  • Monoclonal protein in the serum or urine by immunofixation electrophoresis
  • Plasmacytosis of bone marrow (<30% plasma cells) with monoclonal staining for kappa or lambda light-chain isotype
  • dFLC of 50mg/L (dFLC=difference in involved and uninvolved serum free light-chain levels)
  • Must have received ≥1 prior treatment for AL amyloidosis, if it is intensive chemotherapy and an autotransplant it must be ≥6 months prior to enrollment on this study
  • Must have recovered from the reversible side effects of any prior therapy; permanent and stable side effects/changes are acceptable. Prior treatment for AL amyloidosis with chemotherapy, thalidomide, lenalidomide or steroids is not an exclusion
  • Eastern Cooperative Group (ECOG) performance status ≤2 at study entry
  • Lab test results within these ranges:
  • Neutrophil ≥1.5 x10e9/L e. Platelets ≥100x10e9/L f. Total bilirubin <1.5mg/dL g. Aspartate aminotransferase (AST or SGOT) and Alanine Aminotransferase (ALT or SGPT) < 2 x Upper limit of normal h. Serum creatinine <2.5mg/dL
  • Disease free of prior malignancies for at least 5yrs with exception of currently treated basal cell, squamous cell carcinoma of the skin, or carcinoma "in-situ" of the cervix or breast.
  • Females of childbearing potential (FCBP) (a FCBP is a sexually mature woman who has not undergone a hysterectomy or bilateral oophorectomy, or has not been naturally postmenopausal for at least 24 consecutive months) must have a negative serum or urine pregnancy test with a sensitivity ≥ 50 milli-International unit/mL 10-14 days prior to and again ≤ 24 hours of starting pomalidomide and must either commit to continued abstinence from heterosexual intercourse or begin two (2) acceptable methods of birth control, one highly effective method and one additional effective method AT THE SAME TIME, ≥ 28 days before she starts taking pomalidomide. FCBP must also agree to ongoing pregnancy testing. Men must agree to use a latex condom during sexual contact with a FCBP even if they have had a vasectomy. All subjects must be counseled at a minimum of every 28 days about pregnancy precautions and risks of fetal exposure.
  • Able to take aspirin (81 or 325 mg) daily as prophylactic anticoagulation (subjects intolerant to aspirin may use warfarin or low molecular weight heparin).

Who cannot join

  • Secondary or familial amyloidosis
  • Multiple myeloma (≥30% plasma cells in a bone marrow biopsy specimen or lytic bone lesions)
  • Cytotoxic chemo or radiation therapy ≤4 weeks of study entry or following baseline evaluation
  • Symptomatic cardiac arrhythmias or O2-dependent restrictive cardiomyopathy
  • Dialysis-dependent
  • Untreated or uncontrolled infections.
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  • Serious medical conditions, laboratory abnormality, or psychiatric illness that would prevent the subject from signing the informed consent form.
  • Pregnant or breast feeding females (lactating females must agree not to breast feed while taking pomalidomide).
  • Any condition, including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study or confounds the ability to interpret data from the study.
  • Use of any other experimental drug or therapy within 28 days of baseline.
  • Known intolerance to steroids.
  • Known hypersensitivity to thalidomide or lenalidomide
  • The development of erythema nodosum if characterized by a desquamating rash while taking thalidomide or similar drugs.
  • Concurrent use of other anti-cancer agents or treatments.
  • Known HIV positivity is not an exclusion, unless cluster of differentiation 4 (CD4) counts <200/microliter and/or patient has multi-drug resistant HIV infections and/or other concurrent AIDS-defining conditions. HIV b-DNA < 75 copies/mL.

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Boston Medical Center

    Boston, Massachusetts, 02118

Other trials in United States

About this study

This study seeks to enroll patients with AL amyloidosis, for whom treatment with one of the standard melphalan chemotherapy-based regimens is either not recommended or is not their preference.

Pomalidomide (CC-4047) is a drug given by mouth, which can change or regulate the functioning of the immune system. So, in theory, it may reduce or prevent the production of the amyloid protein. Pomalidomide is not currently FDA-approved for AL Amyloidosis. Pomalidomide is chemically similar to thalidomide and lenalidomide, both of these drugs have been approved by the FDA for treatment of patients with multiple myeloma (MM), a disease similar to AL Amyloidosis.

Participants in this study will receive pomalidomide and dexamethasone. Phase I is a dose-escalation study and dose escalation will proceed through 3 dose-levels according to standard rules in which dose levels are started sequentially after complete evaluation of the occurrence of dose-limiting toxicities. In the Phase II portion, participants will receive pomalidomide and dexamethasone using the defined maximum tolerated dose.

Read the detailed description

Primary objective:

Determine dose-limiting toxicity (DLT) and the maximal tolerated dose (MTD) of pomalidomide combined with dexamethasone in subjects with previously- treated light-chain (AL)-amyloidosis

Secondary objectives:

Determine the following at the MTD:

  • Hematological complete (CR) very good partial (VGPR) and partial (PR) rates
  • duration of response
  • organ response
  • Time-to-event
  • Survival

Exploratory study objective:

To investigate the relationship of changes in the levels of the biomarkers B-type natriuretic peptide (BNP) and troponin I to frequency of specific adverse events and the occurrence of DLT

Conditions
AL 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.

  • PomalidomideDrug

    Also called pomalyst, imnovist

    Cohort 1 = 2 mg/day, Cohort 2 = 3 mg/day, Cohort 3 = 4 mg/day: Days 1-21 of a 28 day cycle

  • DexamethasoneDrug

    Also called Dexamethasone Acetate

    10-20 mg on days 1, 8, 15, and 22

Study groups in NCT01570387
Study groupRoleWhat they receive
Phase I - cohort 1 (Pomalidomide 2mg) plus DexamethasoneExperimentalPomalidomide 2 mg/day on days 1-28 plus dexamethasone 10-20 mg on days 1-21 of a 28 day cycle
Receives: Drug: Pomalidomide; Drug: Dexamethasone
Phase I - cohort 2 (Pomalidomide 3mg) plus DexamethasoneExperimentalPomalidomide 3 mg/day on days 1-28 plus dexamethasone 10-20 mg on days 1-21 of a 28 day cycle
Receives: Drug: Pomalidomide; Drug: Dexamethasone
Phase I - cohort 3 (Pomalidomide 4mg) plus DexamethasoneExperimentalPomalidomide 4 mg/day on days 1-28 plus dexamethasone 10-20 mg on days 1-21 of a 28 day cycle
Receives: Drug: Pomalidomide; Drug: Dexamethasone
Phase II Expansion- (Pomalidomide 4mg) plus DexamethasoneExperimentalExpansion Phase: Pomalidomide 4 mg/day on days 1-28 plus dexamethasone 10-20 mg on days 1-21 of a 28 day cycle
Receives: Drug: Pomalidomide; Drug: Dexamethasone

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Assessing Dose-limiting Toxicity to Determine Maximal Tolerated Dosage at 2 Milligram Dose

    Number of patients in Phase I cohort 1 experiencing dose-limiting toxicity at the 2 milligram dose of pomalidomide combined with dexamethasone in subjects with previously- treated light-chain amyloidosis

    Measured: one month

  2. Assessing Dose-limiting Toxicity to Determine Maximal Tolerated Dosage at 3 Milligram Dose

    Number of patients in Phase I cohort 2 experiencing dose limiting toxicity in the 3 milligram dose level, cohort 2.

    Measured: One month

  3. Assessing Dose-limiting Toxicity to Determine Maximal Tolerated Dosage at 4 Milligram Dose

    Number of patients in Phase I cohort 3 experiencing dose-limiting toxicity at the 4 milligram dose for participants within the third dose cohort

    Measured: One month

Secondary outcomes (1)

  1. Response to the Maximal Tolerated Dose

    Number of participants with a response to treatment at that maximal tolerated dose (including partial, very good, or complete responses)

    Measured: one year

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted4 Apr 2012 (estimated)
  2. Study startJun 2012
  3. Primary completionNov 2018
  4. Study completionApr 2019

Resources

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
Allocation
Non-randomised
Intervention model
Single group
Masking
None (open label)
Enrolment
27 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01570387
Sponsor's protocol ID
H-31082
Other IDs
PO-AMYL-PI-0024
Status last verified
Sep 2020
First submitted
27 Feb 2012
Last updated
22 Sep 2020
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • Sanchorawala V, Shelton AC, Lo S, Varga C, Sloan JM, Seldin DC. Pomalidomide and dexamethasone in the treatment of AL amyloidosis: results of a phase 1 and 2 trial. Blood. 2016 Aug 25;128(8):1059-62. doi: 10.1182/blood-2016-04-710822. Epub 2016 Jul 5.

    PubMed 27381904

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Pomalidomide and Dexamethasone in people with AL Amyloidosis, run by Boston Medical Center at 1 site 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 17 inclusion and 15 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 22 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 1 site 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?

Yes. Results are posted in the registry (first posted 16 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?

Assessing Dose-limiting Toxicity to Determine Maximal Tolerated Dosage at 2 Milligram Dose (time frame: one month). The study lists 3 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 NCT01570387, 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 22 Sep 2020.

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