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Study of Liposomal Annamycin for the Treatment of Subjects With Acute Myeloid Leukemia (AML)

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.
 
ClinicalTrials.gov Identifier: NCT03315039
Recruitment Status : Completed
First Posted : October 19, 2017
Results First Posted : February 23, 2022
Last Update Posted : March 10, 2022
Sponsor:
Information provided by (Responsible Party):
Moleculin Biotech, Inc.

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Sequential Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Leukemia, Myeloid, Acute
Intervention Drug: Liposomal Annamycin
Enrollment 7
Recruitment Details  
Pre-assignment Details no participants were enrolled in Phase 2
Arm/Group Title Dose Level 1: 100 mg/m2/Day Liposomal Annamycin Dose Level 2: 120 mg/m2/Day Liposomal Annamycin
Hide Arm/Group Description Liposomal Annamycin: 2-hour intravenous infusion liposomal annamycin daily for 3 consecutive days followed by 18 days off study drug (i.e., one treatment cycle = 21 days). Liposomal Annamycin: 2-hour intravenous infusion liposomal annamycin daily for 3 consecutive days followed by 18 days off study drug (i.e., one treatment cycle = 21 days).
Period Title: Overall Study
Started 4 3
Completed 4 3
Not Completed 0 0
Arm/Group Title Dose Level 1: 100 mg/m2/Day Liposomal Annamycin Dose Level 2: 120 mg/m2/Day Liposomal Annamycin Total
Hide Arm/Group Description Liposomal Annamycin: 2-hour intravenous infusion liposomal annamycin daily for 3 consecutive days followed by 18 days off study drug (i.e., one treatment cycle = 21 days). Liposomal Annamycin: 2-hour intravenous infusion liposomal annamycin daily for 3 consecutive days followed by 18 days off study drug (i.e., one treatment cycle = 21 days). Total of all reporting groups
Overall Number of Baseline Participants 4 3 7
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants 3 participants 7 participants
<=18 years
0
   0.0%
0
   0.0%
0
   0.0%
Between 18 and 65 years
1
  25.0%
1
  33.3%
2
  28.6%
>=65 years
3
  75.0%
2
  66.7%
5
  71.4%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 4 participants 3 participants 7 participants
67.0  (6.73) 57.0  (18.19) 62.7  (12.71)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants 3 participants 7 participants
Female
1
  25.0%
1
  33.3%
2
  28.6%
Male
3
  75.0%
2
  66.7%
5
  71.4%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants 3 participants 7 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
1
  33.3%
1
  14.3%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
0
   0.0%
White
4
 100.0%
2
  66.7%
6
  85.7%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
Region of Enrollment  
Measure Type: Count of Participants
Unit of measure:  Participants
United States Number Analyzed 4 participants 3 participants 7 participants
4
 100.0%
3
 100.0%
7
 100.0%
ECOG Performance Status   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants 3 participants 7 participants
ECOG - 0
0
   0.0%
1
  33.3%
1
  14.3%
ECOG - 1
3
  75.0%
1
  33.3%
4
  57.1%
ECOG- 2
1
  25.0%
1
  33.3%
2
  28.6%
[1]
Measure Description:

ECOG grading scale- 0 - Fully active, able to carry on all pre-disease performance without restriction

1- Restricted in physically strenuous activity but ambulatory and able to carry out work of a light of sedentary nature, e.g., light house work, office work 2 - Ambulatory and capable of all self care but unable to carry out any work activities. Up and about more than 50% of waking hours 3 - Capable of only limited selfcare, confined to bed or chair more than 50% of waking hours 4 - Completely disabled. Cannot carry on any self care. Totally confined to bed or chair.

5 - Dead
1.Primary Outcome
Title Dose-limiting Toxicity
Hide Description Number of patients with a dose-limiting toxicity (DLT) at each dose evaluated
Time Frame Day 28
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Dose Level 1: 100 mg/m2/Day Liposomal Annamycin Dose Level 2 : 120 mg/m2/Day Liposomal Annamycin
Hide Arm/Group Description:
Liposomal Annamycin: 2-hour intravenous infusion liposomal annamycin daily for 3 consecutive days followed by 18 days off study drug (i.e., one treatment cycle = 21 days).
Liposomal Annamycin: 2-hour intravenous infusion liposomal annamycin daily for 3 consecutive days followed by 18 days off study drug (i.e., one treatment cycle = 21 days).
Overall Number of Participants Analyzed 4 3
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
0
   0.0%
2.Secondary Outcome
Title Pharmacokinetics - Area Under the Plasma Concentration
Hide Description Area under the plasma concentration - time curve (AUC) of annamycin and its metabolite, annamycinol
Time Frame Pre-dose and at 0.25, 0.5, 1, 2, 4, 8, and 24 hours after the start of liposomal annamycin infusion on Day 1 and Day 3
Hide Outcome Measure Data
Hide Analysis Population Description
One participant excluded from summary statistics due to high Annamycin concentrations
Arm/Group Title Liposomal Annamycin
Hide Arm/Group Description:
Liposomal Annamycin: 2-hour intravenous infusion liposomal annamycin daily for 3 consecutive days followed by 18 days off study drug (i.e., one treatment cycle = 21 days).
Overall Number of Participants Analyzed 6
Geometric Mean (Geometric Coefficient of Variation)
Unit of Measure: hr*ng/ml
Annamycin 100 mg/m2 - Day 1 Number Analyzed 4 participants
1430
(19.6%)
Annamycin 100 mg/m2 - Day 3 Number Analyzed 4 participants
1610
(22.4%)
Annamycin 120 mg/m2 - Day 1 Number Analyzed 2 participants
2330
(42.0%)
Annamycin 120 mg/m2 - Day 3 Number Analyzed 2 participants
2880
(33.3%)
Annamycinol 100 mg/m2 - Day 1 Number Analyzed 4 participants
1400
(43.9%)
Annamycinol 100 mg/m2 - Day 3 Number Analyzed 4 participants
1520
(36.7%)
Annamycinol 120 mg/m2 - Day 1 Number Analyzed 2 participants
1370
(39.5%)
Annamycinol 120 mg/m2 - Day 3 Number Analyzed 2 participants
1610
(67.7%)
3.Secondary Outcome
Title Number of Participants With Anti-leukemic Activity
Hide Description Determined by acute myeloid leukemia (AML) response rate based on the International Working Group (IWG) Response Criteria in AML (Cheson, 2003). Anti-leukemic Activity measured by bone marrow biopsy/aspirate pre and post treatment.
Time Frame 15-35 Days after the start of therapy
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title 100 mg/m2 120 mg/m2
Hide Arm/Group Description:
Best Overall Response for Participants treated at 100 mg/m2 dose level
Best Overall Response for treated at 120 mg/m2 dose level
Overall Number of Participants Analyzed 4 3
Measure Type: Count of Participants
Unit of Measure: Participants
Morphological Leukemia Free State (MLFS)
0
   0.0%
1
  33.3%
Treatment Failure (TF)
4
 100.0%
2
  66.7%
Time Frame 39 days after the last dose of study treatment, a total of about 1 year and 10 months since the start of the study.
Adverse Event Reporting Description

AEs were collected from safety population from the time of study treatment initiation until 39 days after the last dose of study treatment.

Safety population = all participants who received any amount of liposomal annamycin.
 
Arm/Group Title 100 mg/m2 Dose Level 120 mg/m2 Dose Level
Hide Arm/Group Description Liposomal Annamycin: 2-hour intravenous infusion liposomal annamycin daily for 3 consecutive days followed by 18 days off study drug (i.e., one treatment cycle = 21 days). Liposomal Annamycin: 2-hour intravenous infusion liposomal annamycin daily for 3 consecutive days followed by 18 days off study drug (i.e., one treatment cycle = 21 days).
All-Cause Mortality
100 mg/m2 Dose Level 120 mg/m2 Dose Level
Affected / at Risk (%) Affected / at Risk (%)
Total   0/4 (0.00%)   0/3 (0.00%) 
Hide Serious Adverse Events
100 mg/m2 Dose Level 120 mg/m2 Dose Level
Affected / at Risk (%) Affected / at Risk (%)
Total   2/4 (50.00%)   0/3 (0.00%) 
General disorders     
Flank Pain † 1  1/4 (25.00%)  0/3 (0.00%) 
Respiratory, thoracic and mediastinal disorders     
Respiratory Failure † 1  1/4 (25.00%)  0/3 (0.00%) 
1
Term from vocabulary, MedDRA 20.0
†
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
100 mg/m2 Dose Level 120 mg/m2 Dose Level
Affected / at Risk (%) Affected / at Risk (%)
Total   4/4 (100.00%)   3/3 (100.00%) 
Blood and lymphatic system disorders     
Anaemia † 1  1/4 (25.00%)  1/3 (33.33%) 
Febrile neutropenia † 1  1/4 (25.00%)  1/3 (33.33%) 
Thrombocytopenia † 1  1/4 (25.00%)  0/3 (0.00%) 
Neutropenia † 1  1/4 (25.00%)  0/3 (0.00%) 
Cardiac disorders     
Tachycardia † 1  0/4 (0.00%)  1/3 (33.33%) 
Eye disorders     
Visual impairment † 1  1/4 (25.00%)  0/3 (0.00%) 
Gastrointestinal disorders     
Diarrhoea † 1  2/4 (50.00%)  1/3 (33.33%) 
Abdominal Pain † 1  1/4 (25.00%)  1/3 (33.33%) 
Constipation † 1  1/4 (25.00%)  1/3 (33.33%) 
Nausea † 1  1/4 (25.00%)  1/3 (33.33%) 
Lip Dry † 1  0/4 (0.00%)  1/3 (33.33%) 
Oral Pain † 1  0/4 (0.00%)  1/3 (33.33%) 
Vomiting † 1  0/4 (0.00%)  1/3 (33.33%) 
General disorders     
Fatigue † 1  2/4 (50.00%)  0/3 (0.00%) 
Oedema peripheral † 1  1/4 (25.00%)  1/3 (33.33%) 
Chills † 1  1/4 (25.00%)  0/3 (0.00%) 
Mucosal iflammation † 1  1/4 (25.00%)  0/3 (0.00%) 
Pyrexia † 1  1/4 (25.00%)  0/3 (0.00%) 
Infections and infestations     
Escherichia bacteraemia † 1  1/4 (25.00%)  1/3 (33.33%) 
Folliculitis † 1  2/4 (50.00%)  0/3 (0.00%) 
Diverticulitis † 1  1/4 (25.00%)  0/3 (0.00%) 
Enterococcal infection † 1  0/4 (0.00%)  1/3 (33.33%) 
Pneumonia fungal † 1  0/4 (0.00%)  1/3 (33.33%) 
Injury, poisoning and procedural complications     
Infusion related reaction † 1  1/4 (25.00%)  0/3 (0.00%) 
Post procedural haemorrhage † 1  0/4 (0.00%)  1/3 (33.33%) 
Skin Wound † 1  0/4 (0.00%)  1/3 (33.33%) 
Investigations     
Platelet count decreased † 1  2/4 (50.00%)  1/3 (33.33%) 
White blood cell count decreased † 1  2/4 (50.00%)  1/3 (33.33%) 
Blood creatinine increased † 1  1/4 (25.00%)  1/3 (33.33%) 
Neutrophil count decreased † 1  1/4 (25.00%)  1/3 (33.33%) 
Alanine aminotransferase increased † 1  0/4 (0.00%)  1/3 (33.33%) 
Asparate aminotransferase increased † 1  0/4 (0.00%)  1/3 (33.33%) 
Blood alkaline phosphatase increased † 1  0/4 (0.00%)  1/3 (33.33%) 
Blood bilirubin increased † 1  1/4 (25.00%)  0/3 (0.00%) 
Lymphocyte count decreased † 1  0/4 (0.00%)  1/3 (33.33%) 
Weight Increased † 1  0/4 (0.00%)  1/3 (33.33%) 
Metabolism and nutrition disorders     
Hypokalaemia † 1  1/4 (25.00%)  2/3 (66.67%) 
Decreased apetite † 1  1/4 (25.00%)  1/3 (33.33%) 
Hypomagnesaemia † 1  2/4 (50.00%)  0/3 (0.00%) 
Hypocalcaemia † 1  1/4 (25.00%)  1/3 (33.33%) 
Hypophosphataemia † 1  1/4 (25.00%)  1/3 (33.33%) 
Fluid Overload † 1  1/4 (25.00%)  0/3 (0.00%) 
Hyperuricaemia † 1  1/4 (25.00%)  0/3 (0.00%) 
Hypoalbuminaemia † 1  0/4 (0.00%)  1/3 (33.33%) 
Hyponatraemia † 1  1/4 (25.00%)  0/3 (0.00%) 
Musculoskeletal and connective tissue disorders     
Arthralgia † 1  1/4 (25.00%)  1/3 (33.33%) 
Back Pain † 1  0/4 (0.00%)  1/3 (33.33%) 
Flank Pain † 1  1/4 (25.00%)  0/3 (0.00%) 
Muscular weakness † 1  1/4 (25.00%)  0/3 (0.00%) 
Nervous system disorders     
Dizziness † 1  1/4 (25.00%)  1/3 (33.33%) 
Dysgeusia † 1  1/4 (25.00%)  0/3 (0.00%) 
Psychiatric disorders     
Insomnia † 1  2/4 (50.00%)  1/3 (33.33%) 
Anxiety † 1  1/4 (25.00%)  0/3 (0.00%) 
Depression † 1  0/4 (0.00%)  1/3 (33.33%) 
Renal and urinary disorders     
Urinary hesitation † 1  1/4 (25.00%)  0/3 (0.00%) 
Reproductive system and breast disorders     
Vaginal haemorrhage † 1  1/4 (25.00%)  0/3 (0.00%) 
Respiratory, thoracic and mediastinal disorders     
Dyspnoea † 1  3/4 (75.00%)  1/3 (33.33%) 
Epistaxis † 1  0/4 (0.00%)  3/3 (100.00%) 
Cough † 1  1/4 (25.00%)  1/3 (33.33%) 
Pulmonary oedema † 1  1/4 (25.00%)  0/3 (0.00%) 
Respiratory Failure † 1  1/4 (25.00%)  0/3 (0.00%) 
Tachypnoea † 1  0/4 (0.00%)  1/3 (33.33%) 
Skin and subcutaneous tissue disorders     
Alopecia † 1  1/4 (25.00%)  0/3 (0.00%) 
Pruritus † 1  0/4 (0.00%)  1/3 (33.33%) 
Rash maculo-papular † 1  0/4 (0.00%)  1/3 (33.33%) 
Drug eruption † 1  1/4 (25.00%)  0/3 (0.00%) 
Night Sweats † 1  1/4 (25.00%)  0/3 (0.00%) 
Pain of Skin † 1  0/4 (0.00%)  1/3 (33.33%) 
Vascular disorders     
Hypertension † 1  0/4 (0.00%)  1/3 (33.33%) 
1
Term from vocabulary, MedDRA 20.0
†
Indicates events were collected by systematic assessment
[Not Specified]
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Chief Scientific Officer
Organization: Moleculin Biotech Inc
Phone: 862-273-1432
EMail: DPicker@moleculin.com
Layout table for additonal information
Responsible Party: Moleculin Biotech, Inc.
ClinicalTrials.gov Identifier: NCT03315039    
Other Study ID Numbers: MB-104
First Submitted: October 16, 2017
First Posted: October 19, 2017
Results First Submitted: December 7, 2021
Results First Posted: February 23, 2022
Last Update Posted: March 10, 2022