Trial Outcomes & Findings for RZL-012 for Dercum's Disease Lipomas (NCT NCT04229030)
NCT ID: NCT04229030
Last Updated: 2024-02-07
Results Overview
Evaluation of the efficacy of RZL-012 following injection into lipomas/nodules of Dercum's Disease (DD) subjects. Efficacy will be determined by ultrasound assessment of the lipoma/nodule dimensions after treatment vs baseline. Lipomas Dimensions will be measured for active vs. placebo at days 28,56,84
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
38 participants
Primary outcome timeframe
0-84 days
Results posted on
2024-02-07
Participant Flow
Unit of analysis: lipomas
Participant milestones
| Measure |
RZL-012
A single-treatment injection, multiple subcutaneous injections of RZL-012 administered into 4-8 lipomas in each subject, preferably 6. Dosing will be done according to lipomas size, as will be determined by Ultrasound. Each injection contains 0.1mL RZL-012 (5mg).
Lipomas in the size of:
2-3.9 cm will be dozed with 0.4mL RZL-012 (20mg). 4-5.9 cm will be dozed with 0.8mL RZL-012 (80mg). 6-7.9 cm will be dozed with 1 mL RZL-012 (100mg). 8-10 cm will be dozed with 1.2 mL RZL-012 (120mg).
RZL-012: small synthetic molecule for the treatment of Dercum's Disease lipomas
|
Vehicle of RZL-012
A single-treatment injection, multiple subcutaneous injections of vehicle administered into 4-8 lipomas in each subject, preferably 6. Dosing will be done according to lipomas size, as will be determined by Ultrasound. Each injection contains 0.1mL vehicle.
Lipomas in the size of:
2-3.9 cm will be dozed with 0.4mL vehicle. 4-5.9 cm will be dozed with 0.8mL vehicle. 6-7.9 cm will be dozed with 1 mL vehicle. 8-10 cm will be dozed with 1.2 mL vehicle.
Vehicle: Vehicle of RZL-012 drug product
|
|---|---|---|
|
Overall Study
STARTED
|
20 125
|
18 109
|
|
Overall Study
COMPLETED
|
20 125
|
18 109
|
|
Overall Study
NOT COMPLETED
|
0 0
|
0 0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
RZL-012 for Dercum's Disease Lipomas
Baseline characteristics by cohort
| Measure |
RZL-012
n=20 Participants
A single-treatment injection, multiple subcutaneous injections of RZL-012 administered into 4-8 lipomas in each subject, preferably 6. Dosing will be done according to lipomas size, as will be determined by Ultrasound. Each injection contains 0.1mL RZL-012 (5mg).
Lipomas in the size of:
2-3.9 cm will be dozed with 0.4mL RZL-012 (20mg). 4-5.9 cm will be dozed with 0.8mL RZL-012 (80mg). 6-7.9 cm will be dozed with 1 mL RZL-012 (100mg). 8-10 cm will be dozed with 1.2 mL RZL-012 (120mg).
RZL-012: small synthetic molecule for the treatment of Dercum's Disease lipomas
|
Vehicle of RZL-012
n=18 Participants
A single-treatment injection, multiple subcutaneous injections of vehicle administered into 4-8 lipomas in each subject, preferably 6. Dosing will be done according to lipomas size, as will be determined by Ultrasound. Each injection contains 0.1mL vehicle.
Lipomas in the size of:
2-3.9 cm will be dozed with 0.4mL vehicle. 4-5.9 cm will be dozed with 0.8mL vehicle. 6-7.9 cm will be dozed with 1 mL vehicle. 8-10 cm will be dozed with 1.2 mL vehicle.
Vehicle: Vehicle of RZL-012 drug product
|
Total
n=38 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
52.3 years
STANDARD_DEVIATION 11.4 • n=5 Participants
|
48 years
STANDARD_DEVIATION 12.4 • n=7 Participants
|
50.15 years
STANDARD_DEVIATION 11.9 • n=5 Participants
|
|
Sex: Female, Male
Female
|
15 Participants
n=5 Participants
|
14 Participants
n=7 Participants
|
29 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
5 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
19 Participants
n=5 Participants
|
17 Participants
n=7 Participants
|
36 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
20 participants
n=5 Participants
|
18 participants
n=7 Participants
|
38 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 0-84 daysEvaluation of the efficacy of RZL-012 following injection into lipomas/nodules of Dercum's Disease (DD) subjects. Efficacy will be determined by ultrasound assessment of the lipoma/nodule dimensions after treatment vs baseline. Lipomas Dimensions will be measured for active vs. placebo at days 28,56,84
Outcome measures
| Measure |
RZL-012
n=20 Participants
A single-treatment injection, multiple subcutaneous injections of RZL-012 administered into 4-8 lipomas in each subject, preferably 6. Dosing will be done according to lipomas size, as will be determined by Ultrasound. Each injection contains 0.1mL RZL-012 (5mg).
Lipomas in the size of:
2-3.9 cm will be dozed with 0.4mL RZL-012 (20mg). 4-5.9 cm will be dozed with 0.8mL RZL-012 (80mg). 6-7.9 cm will be dozed with 1 mL RZL-012 (100mg). 8-10 cm will be dozed with 1.2 mL RZL-012 (120mg).
RZL-012: small synthetic molecule for the treatment of Dercum's Disease lipomas
|
Vehicle of RZL-012
n=18 Participants
A single-treatment injection, multiple subcutaneous injections of vehicle administered into 4-8 lipomas in each subject, preferably 6. Dosing will be done according to lipomas size, as will be determined by Ultrasound. Each injection contains 0.1mL vehicle.
Lipomas in the size of:
2-3.9 cm will be dozed with 0.4mL vehicle. 4-5.9 cm will be dozed with 0.8mL vehicle. 6-7.9 cm will be dozed with 1 mL vehicle. 8-10 cm will be dozed with 1.2 mL vehicle.
Vehicle: Vehicle of RZL-012 drug product
|
|---|---|---|
|
Efficacy - Change in Lipomas Dimensions
|
-15.13 percentage of change from baseline
Standard Deviation 25.56
|
-15.92 percentage of change from baseline
Standard Deviation 23.96
|
SECONDARY outcome
Timeframe: 0-84 daysAssessment of lipoma/nodule associated pain using the Comparative Pain Scale. 0 is "no pain" and 10 is "worst pain". Pain measured for active vs. placebo at days 28,56,84
Outcome measures
| Measure |
RZL-012
n=20 Participants
A single-treatment injection, multiple subcutaneous injections of RZL-012 administered into 4-8 lipomas in each subject, preferably 6. Dosing will be done according to lipomas size, as will be determined by Ultrasound. Each injection contains 0.1mL RZL-012 (5mg).
Lipomas in the size of:
2-3.9 cm will be dozed with 0.4mL RZL-012 (20mg). 4-5.9 cm will be dozed with 0.8mL RZL-012 (80mg). 6-7.9 cm will be dozed with 1 mL RZL-012 (100mg). 8-10 cm will be dozed with 1.2 mL RZL-012 (120mg).
RZL-012: small synthetic molecule for the treatment of Dercum's Disease lipomas
|
Vehicle of RZL-012
n=18 Participants
A single-treatment injection, multiple subcutaneous injections of vehicle administered into 4-8 lipomas in each subject, preferably 6. Dosing will be done according to lipomas size, as will be determined by Ultrasound. Each injection contains 0.1mL vehicle.
Lipomas in the size of:
2-3.9 cm will be dozed with 0.4mL vehicle. 4-5.9 cm will be dozed with 0.8mL vehicle. 6-7.9 cm will be dozed with 1 mL vehicle. 8-10 cm will be dozed with 1.2 mL vehicle.
Vehicle: Vehicle of RZL-012 drug product
|
|---|---|---|
|
Key Secondary Outcome - Efficacy - Improvement in Pain Assessment of Individual Lipomas
|
-56.15 percentage of change from baseline
Standard Deviation 35.77
|
-34.34 percentage of change from baseline
Standard Deviation 40.15
|
SECONDARY outcome
Timeframe: 0-28 daysSafety will be assessed by by change-from-baseline values for clinical laboratory tests. measured for active vs. placebo at days 1 and 28 following injection. Number of subjects with abnormal laboratory values will be compared in both study arms.
Outcome measures
| Measure |
RZL-012
n=20 Participants
A single-treatment injection, multiple subcutaneous injections of RZL-012 administered into 4-8 lipomas in each subject, preferably 6. Dosing will be done according to lipomas size, as will be determined by Ultrasound. Each injection contains 0.1mL RZL-012 (5mg).
Lipomas in the size of:
2-3.9 cm will be dozed with 0.4mL RZL-012 (20mg). 4-5.9 cm will be dozed with 0.8mL RZL-012 (80mg). 6-7.9 cm will be dozed with 1 mL RZL-012 (100mg). 8-10 cm will be dozed with 1.2 mL RZL-012 (120mg).
RZL-012: small synthetic molecule for the treatment of Dercum's Disease lipomas
|
Vehicle of RZL-012
n=18 Participants
A single-treatment injection, multiple subcutaneous injections of vehicle administered into 4-8 lipomas in each subject, preferably 6. Dosing will be done according to lipomas size, as will be determined by Ultrasound. Each injection contains 0.1mL vehicle.
Lipomas in the size of:
2-3.9 cm will be dozed with 0.4mL vehicle. 4-5.9 cm will be dozed with 0.8mL vehicle. 6-7.9 cm will be dozed with 1 mL vehicle. 8-10 cm will be dozed with 1.2 mL vehicle.
Vehicle: Vehicle of RZL-012 drug product
|
|---|---|---|
|
Safety Number of Participants With One or More Abnormal Laboratory Values up to Day 28
|
0 Participants
|
0 Participants
|
OTHER_PRE_SPECIFIED outcome
Timeframe: 0-84 DaysImprovement in Quality of Life - QOL questionnaire. Improvement will be measured for active vs. placebo at Day 0 and Day 84 following treatment.
Outcome measures
| Measure |
RZL-012
n=20 Participants
A single-treatment injection, multiple subcutaneous injections of RZL-012 administered into 4-8 lipomas in each subject, preferably 6. Dosing will be done according to lipomas size, as will be determined by Ultrasound. Each injection contains 0.1mL RZL-012 (5mg).
Lipomas in the size of:
2-3.9 cm will be dozed with 0.4mL RZL-012 (20mg). 4-5.9 cm will be dozed with 0.8mL RZL-012 (80mg). 6-7.9 cm will be dozed with 1 mL RZL-012 (100mg). 8-10 cm will be dozed with 1.2 mL RZL-012 (120mg).
RZL-012: small synthetic molecule for the treatment of Dercum's Disease lipomas
|
Vehicle of RZL-012
n=18 Participants
A single-treatment injection, multiple subcutaneous injections of vehicle administered into 4-8 lipomas in each subject, preferably 6. Dosing will be done according to lipomas size, as will be determined by Ultrasound. Each injection contains 0.1mL vehicle.
Lipomas in the size of:
2-3.9 cm will be dozed with 0.4mL vehicle. 4-5.9 cm will be dozed with 0.8mL vehicle. 6-7.9 cm will be dozed with 1 mL vehicle. 8-10 cm will be dozed with 1.2 mL vehicle.
Vehicle: Vehicle of RZL-012 drug product
|
|---|---|---|
|
Exploratory
|
0 Participants
|
0 Participants
|
Adverse Events
RZL-012
Serious events: 0 serious events
Other events: 20 other events
Deaths: 0 deaths
Vehicle of RZL-012
Serious events: 0 serious events
Other events: 18 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
RZL-012
n=20 participants at risk
A single-treatment injection, multiple subcutaneous injections of RZL-012 administered into 4-8 lipomas in each subject, preferably 6. Dosing will be done according to lipomas size, as will be determined by Ultrasound. Each injection contains 0.1mL RZL-012 (5mg).
Lipomas in the size of:
2-3.9 cm will be dozed with 0.4mL RZL-012 (20mg). 4-5.9 cm will be dozed with 0.8mL RZL-012 (80mg). 6-7.9 cm will be dozed with 1 mL RZL-012 (100mg). 8-10 cm will be dozed with 1.2 mL RZL-012 (120mg).
RZL-012: small synthetic molecule for the treatment of Dercum's Disease lipomas
|
Vehicle of RZL-012
n=18 participants at risk
A single-treatment injection, multiple subcutaneous injections of vehicle administered into 4-8 lipomas in each subject, preferably 6. Dosing will be done according to lipomas size, as will be determined by Ultrasound. Each injection contains 0.1mL vehicle.
Lipomas in the size of:
2-3.9 cm will be dozed with 0.4mL vehicle. 4-5.9 cm will be dozed with 0.8mL vehicle. 6-7.9 cm will be dozed with 1 mL vehicle. 8-10 cm will be dozed with 1.2 mL vehicle.
Vehicle: Vehicle of RZL-012 drug product
|
|---|---|---|
|
General disorders
Injection site bruising
|
30.0%
6/20 • 6 months
|
16.7%
3/18 • 6 months
|
|
General disorders
Injection site discoloration
|
25.0%
5/20 • 6 months
|
0.00%
0/18 • 6 months
|
|
General disorders
Injection site erythema
|
100.0%
20/20 • 6 months
|
94.4%
17/18 • 6 months
|
|
General disorders
Injection site hypesthesia
|
10.0%
2/20 • 6 months
|
0.00%
0/18 • 6 months
|
|
General disorders
Injection site edema
|
100.0%
20/20 • 6 months
|
100.0%
18/18 • 6 months
|
|
General disorders
Injection site pain
|
55.0%
11/20 • 6 months
|
50.0%
9/18 • 6 months
|
|
General disorders
Injection site pruritus
|
35.0%
7/20 • 6 months
|
0.00%
0/18 • 6 months
|
Additional Information
Racheli Gueta, Director of Clinical trials
Raziel Therapeutics
Phone: 97289126941
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place