Skip to content
Triangle logo TriangleGet started
← Study library

NCT01570387 · REGISTRY CONDITION SEARCH

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

Found through the registry condition index, not selected as a treatment recommendation. This discovery collection was restricted to studies with posted results.

Phase
PHASE1, PHASE2
Status at capture
COMPLETED
Registry last update
2020-09-22

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.

Open the original ClinicalTrials.gov record → · Download preserved record

What did the study report?

Outcomes, safety and baseline populations are separate source sections. Quality-of-life measures appear under their original outcome titles.

flow

Participant flow

→

baseline

Baseline characteristics

→

outcome · POSTED

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

→

outcome · POSTED

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

→

outcome · POSTED

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

→

outcome · POSTED

Response to the Maximal Tolerated Dose

→

adverse events

Adverse events

→

more info

Limitations, agreements, and source contact

→
Who could take part
eligibility Criteria
Inclusion Criteria: 1. Understand and voluntarily sign informed consent form. 2. ≥18yrs old 3. Able to adhere to the study visit schedule and other protocol requirements. 4. Biopsy proven tissue amyloid deposits or positive fat aspirate 5. Proof of AL type (a or b) 6. Measurable plasma cell dyscrasia (a or b and c of the following required): 1. Monoclonal protein in the serum or urine by immunofixation electrophoresis 2. Plasmacytosis of bone marrow (\<30% plasma cells) with monoclonal staining for kappa or lambda light-chain isotype 3. dFLC of 50mg/L (dFLC=difference in involved and uninvolved serum free light-chain levels) 7. 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 8. 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 9. Eastern Cooperative Group (ECOG) performance status ≤2 at study entry 10. Lab test results within these ranges: d. 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 11. 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. 12. 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. 13. Able to take aspirin (81 or 325 mg) daily as prophylactic anticoagulation (subjects intolerant to aspirin may use warfarin or low molecular weight heparin). Exclusion Criteria: 1. Secondary or familial amyloidosis 2. Multiple myeloma (≥30% plasma cells in a bone marrow biopsy specimen or lytic bone lesions) 3. Cytotoxic chemo or radiation therapy ≤4 weeks of study entry or following baseline evaluation 4. Symptomatic cardiac arrhythmias or O2-dependent restrictive cardiomyopathy 5. Dialysis-dependent 6. Untreated or uncontrolled infections. 7. Serious medical conditions, laboratory abnormality, or psychiatric illness that would prevent the subject from signing the informed consent form. 8. Pregnant or breast feeding females (lactating females must agree not to breast feed while taking pomalidomide). 9. 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. 10. Use of any other experimental drug or therapy within 28 days of baseline. 11. Known intolerance to steroids. 12. Known hypersensitivity to thalidomide or lenalidomide 13. The development of erythema nodosum if characterized by a desquamating rash while taking thalidomide or similar drugs. 14. Concurrent use of other anti-cancer agents or treatments. 15. 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.
healthy Volunteers
false
minimum Age
18 Years
sex
ALL
std Ages
  1. ADULT
  2. OLDER_ADULT
Treatment arms and interventions
arm Groups
  1. description
    Pomalidomide 2 mg/day on days 1-28 plus dexamethasone 10-20 mg on days 1-21 of a 28 day cycle
    intervention Names
    1. Drug: Pomalidomide
    2. Drug: Dexamethasone
    label
    Phase I - cohort 1 (Pomalidomide 2mg) plus Dexamethasone
    type
    EXPERIMENTAL
  2. description
    Pomalidomide 3 mg/day on days 1-28 plus dexamethasone 10-20 mg on days 1-21 of a 28 day cycle
    intervention Names
    1. Drug: Pomalidomide
    2. Drug: Dexamethasone
    label
    Phase I - cohort 2 (Pomalidomide 3mg) plus Dexamethasone
    type
    EXPERIMENTAL
  3. description
    Pomalidomide 4 mg/day on days 1-28 plus dexamethasone 10-20 mg on days 1-21 of a 28 day cycle
    intervention Names
    1. Drug: Pomalidomide
    2. Drug: Dexamethasone
    label
    Phase I - cohort 3 (Pomalidomide 4mg) plus Dexamethasone
    type
    EXPERIMENTAL
  4. description
    Expansion Phase: Pomalidomide 4 mg/day on days 1-28 plus dexamethasone 10-20 mg on days 1-21 of a 28 day cycle
    intervention Names
    1. Drug: Pomalidomide
    2. Drug: Dexamethasone
    label
    Phase II Expansion- (Pomalidomide 4mg) plus Dexamethasone
    type
    EXPERIMENTAL
interventions
  1. arm Group Labels
    1. Phase I - cohort 1 (Pomalidomide 2mg) plus Dexamethasone
    2. Phase I - cohort 2 (Pomalidomide 3mg) plus Dexamethasone
    3. Phase I - cohort 3 (Pomalidomide 4mg) plus Dexamethasone
    4. Phase II Expansion- (Pomalidomide 4mg) plus Dexamethasone
    description
    Cohort 1 = 2 mg/day, Cohort 2 = 3 mg/day, Cohort 3 = 4 mg/day: Days 1-21 of a 28 day cycle
    name
    Pomalidomide
    other Names
    1. pomalyst
    2. imnovist
    type
    DRUG
  2. arm Group Labels
    1. Phase I - cohort 1 (Pomalidomide 2mg) plus Dexamethasone
    2. Phase I - cohort 2 (Pomalidomide 3mg) plus Dexamethasone
    3. Phase I - cohort 3 (Pomalidomide 4mg) plus Dexamethasone
    4. Phase II Expansion- (Pomalidomide 4mg) plus Dexamethasone
    description
    10-20 mg on days 1, 8, 15, and 22
    name
    Dexamethasone
    other Names
    1. Dexamethasone Acetate
    type
    DRUG
Study design
allocation
NON_RANDOMIZED
intervention Model
SINGLE_GROUP
masking Info
masking
NONE
primary Purpose
TREATMENT
Enrollment
count
27
type
ACTUAL
Registered outcome plans (not posted results)
primary Outcomes
  1. description
    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
    measure
    Assessing Dose-limiting Toxicity to Determine Maximal Tolerated Dosage at 2 Milligram Dose
    time Frame
    one month
  2. description
    Number of patients in Phase I cohort 2 experiencing dose limiting toxicity in the 3 milligram dose level, cohort 2.
    measure
    Assessing Dose-limiting Toxicity to Determine Maximal Tolerated Dosage at 3 Milligram Dose
    time Frame
    One month
  3. description
    Number of patients in Phase I cohort 3 experiencing dose-limiting toxicity at the 4 milligram dose for participants within the third dose cohort
    measure
    Assessing Dose-limiting Toxicity to Determine Maximal Tolerated Dosage at 4 Milligram Dose
    time Frame
    One month
secondary Outcomes
  1. description
    Number of participants with a response to treatment at that maximal tolerated dose (including partial, very good, or complete responses)
    measure
    Response to the Maximal Tolerated Dose
    time Frame
    one year
Full study description
brief 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. 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.
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
Source references
references
  1. citation
    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.
    pmid
    27381904
    type
    DERIVED
Source notices and limitations
    Discovery and provenance
    1. release id
      ctgov-registry-1336d216cbd1bbff780dac70
      edge id
      nct-edge-3c0ba3785719040682f2
      requested nct id
      NCT01570387
      primary nct id
      NCT01570387
      source kind
      ctgov_condition_query
      source record id
      Source null
      source file sha256
      2314ce01316b2d7044d298c6d25eda12e99cc5f44273bc75efed6cd5e03a8c14
      payload sha256
      331cd9a274a44c3c26524ec6593fc434106cea6e2a406e5a2b1b2d2e5a2cf705
      payload
      clinical indication matching status
      condition_index_match_asserted_by_registry_not_reviewed
      end exclusive
      true
      id
      nct-edge-3c0ba3785719040682f2
      link basis
      returned_by_explicit_condition_and_posted_results_filter_query
      nct id
      NCT01570387
      occurrences
      1. context exact
        NCT01570387
        end
        11
        exact text
        NCT01570387
        start
        0
      offset unit
      unicode_code_points
      query cond
      Plasma Cell Neoplasms
      query disease state id
      ds-plasma-cell-neoplasms
      source file
      /Users/arunverma/Documents/ChatGPT/Cancer Statistics Website/research-data/2026-09-10/clinicaltrials-condition/round-0000/raw/cond-plasma-cell-neoplasms-000.json
      source file sha256
      2314ce01316b2d7044d298c6d25eda12e99cc5f44273bc75efed6cd5e03a8c14
      source json pointer
      /studies/267/protocolSection/identificationModule/nctId
      source kind
      ctgov_condition_query
      source record id
      Source null
      source string basis
      parsed_JSON_string_unmodified
      source string sha256
      36717da32eeb7993af67191bb4cab66dd903033a2b9595b0f5e5da7cf107801c

    Preserved source evidence · Not medical advice