Don't get left behind! The modernized is coming. Check it out now.
Say goodbye to!
The new site is coming soon - go to the modernized
Working… Menu

A Evaluation of the Safety of Oncocort IV Pegylated Liposomal Dexamethasone Phosphate in Patients With Progressive Multiple Myeloma (AMETHYST)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT03033316
Recruitment Status : Terminated (Patient enrolment issues)
First Posted : January 26, 2017
Last Update Posted : November 6, 2020
University Hospital, Aachen
Information provided by (Responsible Party):
Enceladus Pharmaceuticals BV

Brief Summary:
Oncocort Dexamethasone sodium phosphate encapsulated in so-called 'long-circulating' PEGylated liposomes is in development with the prospect of providing enhanced and prolonged lesion uptake of dexamethasone and therefore increased therapeutic benefit over existing formulations of dexamethasone in Multiple Myeloma. The current trial is a first-in-man study with Oncocort monotherapy with the objective to assess safety, tolerability, efficacy and dose response after short-term treatment with repeated infusions.

Condition or disease Intervention/treatment Phase
Multiple Myeloma Drug: Dexamethasone Phase 1 Phase 2

Detailed Description:
This open label multicenter study will evaluate the safety and efficacy of I.V. Oncocort Dexamethasone sodium phosphate given as a short term induction therapy in Multiple Myeloma patients. It consist of a Phase 1 part with a maximum number of 5 ascending dose levels while additional patients will be treated in the Phase 2a part with the recommended phase 2 dose to obtain additional safety data and preliminary efficacy data in multiple myeloma patients. For the dose escalation phase 1 part, 3 patients per dose level will be enrolled. If one patient reaches DLT, another 3 patients will be enrolled in the same dose level. If two or more patients reach DLT in one dose level, maximum tolerated dose (MTD) is defined at the last dose level beneath DLT. Not more than 5 dose level groups are anticipated for this part of the study. MTD will be the Recommended Phase II Dose (RPTD) for the phase IIa part of the study. For the phase 2a part, 7 additional patients will be enrolled at the RPTD dose level, bringing the total number of patients monitored for responses at this dose level to at least 10.

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 7 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase I-IIa, Open Label, Multi-Center, Dose Escalating Study to Evaluate the Safety of Intravenous Pegylated Liposomal Dexamethasone Sodium Phosphate (Oncocort) as Monotherapy in Patients With Previously Treated Progressive Multiple Myeloma
Actual Study Start Date : January 2017
Actual Primary Completion Date : August 2020
Actual Study Completion Date : August 2020

Arm Intervention/treatment
Experimental: IV Liposomal Dexamethasone
IV Liposomal Dexamethasone given 1 to 4 times in total over period of maximally 4 weeks
Drug: Dexamethasone

Primary Outcome Measures :
  1. Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 [ Time Frame: Week 8 ]

Secondary Outcome Measures :
  1. Response according to IMWG Criteria [ Time Frame: 4 and 8 weeks after 1st dose ]
  2. Percentage change of myeloma-related measures [ Time Frame: up to 8 weeks after 1st dose ]
  3. Quality of life questionnaire [ Time Frame: up to 8 weeks after 1st dose ]
  4. Peak Plasma Concentration (Cmax) [ Time Frame: during 1st 4 weeks ]
  5. Area under the plasma concentration versus time curve (AUC) [ Time Frame: during 1st 4 weeks ]

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

Layout table for eligibility information
Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria

  1. Previously diagnosed symptomatic MM according to International Myeloma Working Group (IMWG) criteria 1.1. Previously treated with at least two therapy lines including at least one proteasome-inhibitor and at least one immunomodulatory drug 1.2. Relapse after or progressive disease under last therapy according to IMWG criteria.
  2. At least 18 years old
  3. Measurable disease (M-protein and/or free light chains) in serum and/or urine
  4. Willing and able to comply the study protocol visits and assessments
  5. Willing to use highly effective methods of birth control (see section 7.1.3)
  6. Written informed consent prior to study participation

Exclusion Criteria:

  1. Types of Multiple Myeloma previously shown to be irresponsive to dexamethasone monotherapy during the last 6 months
  2. Plasma cell leukemia
  3. Subject with positive hepatitis panel (including hepatitis B surface antigen [HBsAg], and / or anti-hepatitis B core antibodies, and / or hepatitis C virus antibody [anti-HCV]), and / or a positive HIV antibody screen
  4. Severe abnormal organ function function or laboratory results at the time of the Screening Visit: WBC <3.0 g/L, ANC <1.5 G/L, PLT <50 G/L, Sodium <135 mmol/L or >150 mmol/L, potassium <3.5 mmol/L or >5.5 mmol/L, calcium <2.0 mmol/L or > 2.9mmol/L, phosphorus <0.8 mmol/L, total bilirubin >1.5x ULN, AP >2.5x ULN, gammaGT >3x ULN, ALT >3x ULN, CK >2.5x ULN, Creatinine >2x ULN, fasting glucose >250 mg/dL, albumin <3 g/dL, cholesterol > 300 mg/dL
  5. Treatment with oral or injectable (including intra-articular) corticosteroids (CS) within 4 weeks prior to Screening Visit. Inhaled and dermal corticosteroid formulations are allowed
  6. Subjects who have received a chemotherapeutic treatment cycle including dexamethasone within 4 weeks prior to the Screening Visit
  7. Known sensitivity to any component of the study drug or previous hypersensitivity reaction or other clinically significant reaction to intravenous medications, biologic therapy or IV radiocontrast agents
  8. Active infection requiring systemic treatment
  9. Planned major surgery during the study period or had undergone major surgery within 30 days prior to the Screening visit
  10. Pregnancy or breastfeeding
  11. Any clinically significant hepatic, renal, cardiac, pulmonary, gastrointestinal, metabolic or endocrine disturbances, other medical or psychiatric condition, or clinically relevant abnormal values on any investigation, in the opinion of the Investigator, that could make the subject unsuitable for the study, could compromise subject safety, limit the subject's ability to complete the study, and/or compromise the objectives of the study, including severe neuropathies or other painful conditions that might interfere with pain evaluation
  12. Participation in another clinical investigation less than 4 weeks prior to inclusion
  13. The subject has a history of any other illness, which, in the opinion of the Investigator, might pose an unacceptable risk by administering study medication.
  14. The subject has any current or past medical condition and/or required medication to treat a condition that could affect the evaluation of the study
  15. The subject is unwilling or unable to follow the procedures outlined in the protocol
  16. The subject is mentally or legally incapacitated
  17. Persons who are in a relationship of dependence to the Investigator or the Sponsor

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03033316

Sponsors and Collaborators
Enceladus Pharmaceuticals BV
University Hospital, Aachen
Layout table for investigator information
Principal Investigator: Stefan Wilop, MD RWTH University Clinic Aachen
Layout table for additonal information
Responsible Party: Enceladus Pharmaceuticals BV Identifier: NCT03033316    
Other Study ID Numbers: 14-097
First Posted: January 26, 2017    Key Record Dates
Last Update Posted: November 6, 2020
Last Verified: November 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Keywords provided by Enceladus Pharmaceuticals BV:
Multiple Myeloma
Liposomal Dexamethasone
Targeted Drug Delivery
Additional relevant MeSH terms:
Layout table for MeSH terms
Multiple Myeloma
Neoplasms, Plasma Cell
Neoplasms by Histologic Type
Hemostatic Disorders
Vascular Diseases
Cardiovascular Diseases
Blood Protein Disorders
Hematologic Diseases
Hemorrhagic Disorders
Lymphoproliferative Disorders
Immunoproliferative Disorders
Immune System Diseases
Anti-Inflammatory Agents
Autonomic Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Gastrointestinal Agents
Hormones, Hormone Substitutes, and Hormone Antagonists
Antineoplastic Agents, Hormonal
Antineoplastic Agents