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Recurrent Plasma Cell MyelomaPhase 1NCT02697344

Gossypol Acetic Acid With Lenalidomide and Dexamethasone in Treating Patients With Relapsed Symptomatic Multiple Myeloma

A Phase 1 study testing Dexamethasone, Laboratory Biomarker Analysis and Lenalidomide in people with Recurrent Plasma Cell Myeloma, run by Mayo Clinic 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 phase I trial studies the side effects and best dose of R-(-)-gossypol acetic acid when given together with lenalidomide and dexamethasone and to see how well it works in treating patients with multiple myeloma, also known as plasma cell myeloma, that has come back after a period of improvement or has gotten… Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Recurrent Plasma Cell Myeloma
What is being tested?
Dexamethasone, Laboratory Biomarker Analysis and Lenalidomide
Could I receive a placebo?
No, everyone receives an active treatment
How many people
10 participants
Where
1 siteUnited States
Study dates
14 Apr 2016to 20 Dec 2023
Phase
Phase 1
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

  • Calculated creatinine clearance (using Cockcroft-Gault equation) >= 60 mL/min =<14 days prior to registration
  • Absolute neutrophil count (ANC) >= 1000/mm^3 =<14 days prior to registration
  • Platelet count >= 75000/mm^3 =<14 days prior to registration
  • Hemoglobin >= 8.0 g/dL =<14 days prior to registration
  • Patient must have relapsed and symptomatic multiple myeloma
  • Measurable disease of multiple myeloma as defined by at least ONE of the following:
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  • Serum monoclonal protein >= 1.0 g/dL
  • >= 200 mg of monoclonal protein in the urine on 24 hour electrophoresis
  • Serum immunoglobulin free light chain >= 10 mg/dL AND abnormal serum immunoglobulin kappa to lambda free light chain ratio
  • Eastern Cooperative Oncology Group (ECOG) performance status (PS) 0, 1 or 2
  • Patients must have received ≥3 prior regimens for multiple myeloma
  • Provide informed written consent
  • Negative pregnancy test done =< 7 days prior to registration, for women of childbearing potential only
  • Willing to return to enrolling institution for follow-up (during the active monitoring phase of the study)
  • Arm A Only (Closed): Willing to provide bone marrow and blood samples for correlative research purposes

Who cannot join

  • Monoclonal gammopathy of undetermined significance (MGUS) or smoldering myeloma
  • Patients who received daratumumab, pomalidomide and dexamethasone as the most immediate prior line of therapy prior to trial enrollment (prior therapy with a daratumumab-based or a pomalidomide-based regimen are allowed and prior daratumumab-pomalidomide-dexamethasone is allowed so long as it was not given as the most immediate prior line of therapy prior to trial enrollment).
  • Other malignancy requiring active therapy EXCEPTIONS: non-melanotic skin cancer or carcinoma-in-situ of the cervix; NOTE: if there is a history of prior malignancy, they must not be receiving other specific treatment for their cancer
  • Any of the following because this study involves an investigational agent whose genotoxic, mutagenic and teratogenic effects on the developing fetus and newborn are unknown:
  • Pregnant women
  • Nursing women
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  • Men or women of childbearing potential who are unwilling to employ adequate contraception
  • Co-morbid 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 (e.g., uncontrolled infection, uncompensated heart or lung disease)
  • Other concurrent chemotherapy, radiotherapy, or any ancillary therapy considered investigational; NOTE: Bisphosphonates are considered to be supportive care rather than therapy, and are thus allowed while on protocol treatment
  • Prior severe skin reaction (toxic epidermal necrosis) with immunomodulating agents
  • Major surgery =< 14 days before study registration
  • Concurrent medical problems that preclude use of deep vein thrombosis (DVT) prophylaxis with lenalidomide treatment
  • Evidence of current uncontrolled cardiovascular conditions, including cardiac arrhythmias, congestive heart failure, angina, or myocardial infarction =< 6 months prior to registration
  • Immunocompromised patients and patients known human immunodeficiency virus (HIV) positive and currently receiving antiretroviral therapy NOTE: Patients known to be HIV positive, but without clinical evidence of an immunocompromised state, are eligible for this trial
  • Uncontrolled intercurrent illness including, but not limited to
  • ongoing or active infection
  • symptomatic congestive heart failure
  • unstable angina pectoris
  • cardiac arrhythmia
  • or psychiatric illness/social situations that would limit compliance with study requirements
  • Known allergy to any of the study medications, their analogues or excipients in the various formulations

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Mayo Clinic in Florida

    Jacksonville, Florida, 32224-9980

Other trials in United States

About this study

Official title: Phase I/II Trial of AT-101 in Combination With Lenalidomide and Dexamethasone in Patients With Relapsed Symptomatic Multiple Myeloma

This phase I trial studies the side effects and best dose of R-(-)-gossypol acetic acid when given together with lenalidomide and dexamethasone and to see how well it works in treating patients with multiple myeloma, also known as plasma cell myeloma, that has come back after a period of improvement or has gotten worse after treatment. R-(-)-gossypol acetic acid may stop the growth of cancer cells by recognizing certain proteins and stimulating programmed cell death. Lenalidomide may stimulate or suppress the immune system in different ways and stop cancer cells from growing. Drugs used in chemotherapy, such as dexamethasone, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving R-(-)-gossypol acetic acid with lenalidomide and dexamethasone may work better in treating patients with multiple myeloma.

Read the detailed description

PRIMARY OBJECTIVES:

  1. To determine the maximum tolerated dose of R-(-)-gossypol acetic acid (AT-101) that can be combined with lenalidomide and dexamethasone in patients with relapsed symptomatic multiple myeloma (MM). (Phase I) II. To determine the overall response rate (partial response or better) of AT-101 when used in combination with lenalidomide and dexamethasone in patients with relapsed symptomatic MM. (Phase II)

SECONDARY OBJECTIVES:

  1. To determine the progression free survival and overall survival among patients with relapsed symptomatic MM following treatment with AT-101 in combination with lenalidomide and dexamethasone.

II. To determine the toxicities associated with AT-101 in combination with lenalidomide and dexamethasone in patients with relapsed symptomatic MM.

TERTIARY OBJECTIVES:

  1. Determine in vivo the anti-myeloma effect of AT-101 alone by assessing extent of decrease in M-protein (serum and/or urine) after 1 cycle of AT-101 alone.

II. Determine the pharmacodynamics effect of AT-101 in combination with lenalidomide and dexamethasone treatment on primary myeloma cell in vivo.

III. Explore potential mechanism of resistance to AT-101 in combination with lenalidomide and dexamethasone therapy and the role of B-cell CLL/lymphoma 2 (Bcl-2).

OUTLINE: This is a phase I dose-escalation study of R-(-)-gossypol acetic acid followed by a phase II study.

Patients receive R-(-)-gossypol acetic acid orally (PO) once a day (QD) on days 1-21. Beginning in course 2, patients also receive lenalidomide PO QD on days 1-21 and dexamethasone PO QD on days 1, 8, and 15. Treatment repeats every 28 days for 12 courses in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed up for up to 3 years.

Being tested
Dexamethasone, Laboratory Biomarker Analysis, Lenalidomide, Pharmacological Study, R-(-)-Gossypol Acetic Acid
Medical subject headings
Multiple Myeloma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • DexamethasoneDrug

    Also called Aacidexam, Adexone, Aknichthol Dexa, Alba-Dex

    Given PO

  • Laboratory Biomarker AnalysisOther

    Correlative studies

  • LenalidomideDrug

    Also called CC-5013, CC5013, CDC 501, Revlimid

    Given PO

  • Pharmacological StudyOther

    Correlative studies

  • R-(-)-Gossypol Acetic AcidDrug

    Also called (-)-Gossypol Acetic Acid, AT-101, GOSSYPOL ACETIC ACID, (R)-

    Given PO

Study groups in NCT02697344
Study groupRoleWhat they receive
Treatment (AT-101, lenalidomide, and dexamethasone)ExperimentalPatients receive R-(-)-gossypol acetic acid PO QD on days 1-21. Beginning in course 2, patients also receive lenalidomide PO QD on days 1-21 and dexamethasone PO QD on days 1, 8, and 15 of courses 2-12. Treatment repeats every 28 days for 12 courses in the absence of disease progression or unacceptable toxicity.
Receives: Drug: Dexamethasone; Other: Laboratory Biomarker Analysis; Drug: Lenalidomide; Other: Pharmacological Study; Drug: R-(-)-Gossypol Acetic Acid

What is being measured

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

  1. Maximally tolerated dose of AT-101 in combination with lenalidomide and dexamethasone defined as the dose level below the lowest dose that induces dose-limiting toxicity in at least one-third of patients (Phase I)

    The number and severity of all adverse events (overall and by dose-level) will be tabulated and summarized in this patient population. The Grade 3+ adverse events will also be described and summarized in a similar fashion.

    Measured: Up to day 28 of course 2

  2. Overall response rate, with response defined to be a stringent complete response, complete response, very good partial response, or partial response (Phase II)

    Responses will be summarized by simple descriptive summary statistics delineating complete and partial responses as well as stable and progressive disease in this patient population. The proportion of successes will be estimated by the number of successes divided by the total number of evaluable patients. Confidence intervals for the true success proportion will be calculated according to the approach of Duffy and Santner.

    Measured: Up to 3 years

Secondary outcomes (3)

  1. Incidence of adverse events

    The maximum grade for each type of adverse event, regardless of causality, will be recorded and reported for each patient, and frequency tables will be reviewed to determine adverse event patterns.

    Measured: Up to 30 days after the last day of study drug treatment

  2. Overall survival (Phase II)

    Estimated using the method of Kaplan-Meier.

    Measured: Time from registration to death due to any cause, assessed up to 3 years

  3. Progression free survival (Phase II)

    Estimated using the method of Kaplan-Meier.

    Measured: Time from registration to the earliest date of documentation of disease progression or death due to any cause, assesse up to 3 years

Timeline

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

  1. First posted3 Mar 2016 (estimated)
  2. Study start14 Apr 2016
  3. Primary completion26 Oct 2018
  4. Study completion20 Dec 2023

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
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
10 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02697344
Sponsor's protocol ID
MC1384B
Other IDs
NCI-2016-00073, 15-003501
Status last verified
Dec 2023
First submitted
22 Feb 2016
Last updated
28 Dec 2023
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Dexamethasone, Laboratory Biomarker Analysis and Lenalidomide in people with Recurrent Plasma Cell Myeloma, run by Mayo Clinic 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 15 inclusion and 21 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 Dec 2023. 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 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.

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?

Maximally tolerated dose of AT-101 in combination with lenalidomide and dexamethasone defined as the dose level below the lowest dose that induces dose-limiting toxicity in at least one-third of patients (Phase I) (time frame: Up to day 28 of course 2). 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 NCT02697344.

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 Dec 2023.

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