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An Open-Label, Dose-Escalation, Phase 1 Study of the Oral Formulation of MLN9708 Administered Weekly in Adult Patients With Relapsed or Refractory Light-Chain Amyloidosis Who Require Further Treatment


Phase 1
18 Years
N/A
Open (Enrolling)
Both
Light-Chain Amyloidosis

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Trial Information

An Open-Label, Dose-Escalation, Phase 1 Study of the Oral Formulation of MLN9708 Administered Weekly in Adult Patients With Relapsed or Refractory Light-Chain Amyloidosis Who Require Further Treatment


Inclusion Criteria:



- Male or female patients 18 years or older

- Biopsy-proven systemic relapsed or refractory light-chain (AL) amyloidosis, which
after at least 1 prior therapy, in the investigator's opinion, requires further
treatment

- If received stem cell transplant, must be at least 3 months posttransplantation and
recovered from side effects

- Must have measurable disease defined as serum differential free light chain
concentration ≥ 40mg/L

- Must have objective measurable organ (heart or kidney) amyloid involvement

- Must have cardiac biomarker risk stage I or II disease

- Must have adequate hematologic, hepatic, and renal function

- Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2

- Female patients who are post menopausal, surgically sterile, or agree to practice 2
effective methods of contraception or agree to abstain from heterosexual intercourse

- Male patients who agree to practice effective barrier contraception or agree to
abstain from heterosexual intercourse

- Voluntary written consent

Exclusion Criteria

- Peripheral neuropathy that is greater or equal to Grade 2

- Cardiac status as described in protocol

- Severe diarrhea (≥ Grade 3) not controllable with medication or requires
administration of total parenteral nutrition

- Known gastrointestinal condition or procedure that could interfere with swallowing or
the oral absorption of tolerance of MLN9708

- Uncontrolled infection requiring systematic antibiotics

- Known human immunodeficiency virus (HIV) positive, hepatitis B surface
antigen-positive status, or known or suspected active hepatitis C infection

- Presence of other active malignancy with the exception of nonmelanoma skin cancer,
cervical cancer, treated early-stage prostate cancer provided that prostate-specific
antigen is within normal limit, or any completely resected carcinoma in situ

- Female patients who are lactating or pregnant

- Major surgery within 14 days before the first dose of study drug

- Serious medical or psychiatric illness that could, in the investigator's opinion,
potentially interfere with the completion of treatment according to the protocol

Type of Study:

Interventional

Study Design:

Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Number of Participants with Adverse Events as a Measure of Safety and Tolerability

Outcome Time Frame:

From start of study through 30 days after last dose or until initiation of subsequent antineoplastic therapy

Safety Issue:

Yes

Principal Investigator

Medical Monitor

Investigator Role:

Study Director

Investigator Affiliation:

Millennium Pharmaceuticals, Inc.

Authority:

United States: Food and Drug Administration

Study ID:

C16007

NCT ID:

NCT01318902

Start Date:

March 2011

Completion Date:

January 2014

Related Keywords:

  • Light-Chain Amyloidosis
  • Amyloidosis

Name

Location

Mayo Clinic Rochester, Minnesota  55905
Fox Chase Cancer Center Philadelphia, Pennsylvania  19111
Mount Sinai School of Medicine New York, New York  10029
Boston Medical Center Boston, Massachusetts  02118
Tufts Medical Center Boston, Massachusetts  02111
Karmanos Cancer Institute Detroit, Michigan  48201