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PTC596 for Leiomyosarcoma

Phase-Based Progress Estimates
1
Effectiveness
1
Safety
LeiomyosarcomaPTC596 - Drug
Eligibility
18+
All Sexes
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Study Summary

A Study of Unesbulin (PTC596) in Combination With Dacarbazine in Participants With Advanced Leiomyosarcoma (LMS)

Treatment Effectiveness

Effectiveness Progress

1 of 3

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Study Objectives

3 Primary · 20 Secondary · Reporting Duration: From Baseline until the date of objectively documented progression per RECIST v1.1 or the date of initiation of subsequent therapy or palliative local therapy, whichever occurs first (up to approximately 1.5 years)

Week 6
MTD and RP2D of PTC596 in Combination With Dacarbazine
MTD and RP2D of Unesbulin in Combination With Dacarbazine
Year 5
Time to Response as Determined by the Investigator Using RECIST v1.1
Year 5
Best Overall Response Rate (Disease Control Rate) (Percentage of Participants With Best Overall Response)
Objective Response Rate (ORR) (Percentage of Participants With Objective Response) as Determined by the Investigator Using Response Evaluation Criteria in Solid Tumors Version 1.1 (RECIST v1.1)
Year 5
Number of Participants With Adverse Events
Day 21
Maximum Observed Plasma Concentration (Cmax) of PTC596, Dacarbazine, and Inactive Metabolite of Dacarbazine (5-amino-imidazole-4-carboxamide [AIC])
Day 21
Accumulation Ratio (R) of PTC596, Dacarbazine, and Inactive Metabolite of Dacarbazine (AIC)
Apparent Clearance (CL/F) of PTC596, Dacarbazine, and Inactive Metabolite of Dacarbazine (AIC)
Apparent Volume of Distribution (Vz/F) of PTC596, Dacarbazine, and Inactive Metabolite of Dacarbazine (AIC)
Area Under the Plasma Concentration-Time Curve (AUC) of PTC596, Dacarbazine, and Inactive Metabolite of Dacarbazine (AIC)
Half-Life (t1/2) of PTC596, Dacarbazine, and Inactive Metabolite of Dacarbazine (AIC)
Time to Reach Maximum Plasma Concentration (Tmax) of PTC596, Dacarbazine, and Inactive Metabolite of Dacarbazine (AIC)
Year 5
Duration of Response (DOR)
Year 5
Overall Survival (OS)
Year 5
Progression-Free Survival (PFS)
Day 21
Maximum Observed Plasma Concentration (Cmax) of Unesbulin, Dacarbazine, and Inactive Metabolite of Dacarbazine (5-amino-imidazole-4-carboxamide [AIC])
Day 21
Accumulation Ratio (R) of Unesbulin, Dacarbazine, and Inactive Metabolite of Dacarbazine (AIC)
Dacarbazine
Dacarbazine
Area Under the Plasma Concentration-Time Curve (AUC) of Unesbulin, Dacarbazine, and Inactive Metabolite of Dacarbazine (AIC)
Half-Life (t1/2) of Unesbulin, Dacarbazine, and Inactive Metabolite of Dacarbazine (AIC)
Day 21
Time to Reach Maximum Plasma Concentration (Tmax) of Unesbulin, Dacarbazine, and Inactive Metabolite of Dacarbazine (AIC)

Trial Safety

Safety Progress

1 of 3

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Side Effects for

Arm B (Stanford V)
98%Anemia
93%Leukocytes decreased
90%Lymphopenia
84%Neutrophils decreased
78%Neuropathy-sensory
75%Alopecia
74%Fatigue
67%Nausea
60%Hyperglycemia
52%Constipation
46%Hypoalbuminemia
40%Myalgia
34%Stomatitis
33%Insomnia
32%Vomiting
27%Platelets decreased
26%Alkaline phosphatase increased
26%Aspartate aminotransferase increased
23%Dyspnea
20%Dyspepsia
19%Dysphagia
19%Headache
16%Arthralgia
16%Anorexia
15%Abdominal pain
15%Neuropathy-motor
14%Cough
14%Fever
14%Infection w/o neutropenia
13%Diarrhea w/o prior colostomy
13%Rash/desquamation
12%Bone pain
11%Weight gain
11%Taste disturbance
11%Anxiety/agitation
10%Sweating
10%Radiation dermatitis
9%Dizziness/lightheadedness
9%Rigors/chills
9%Injection site reaction
8%Pain-other
8%Phlebitis
8%Chest pain
8%Blood bilirubin increased
8%Dysphagia-esophageal radiation
8%Hypoglycemia
7%Edema
7%Pruritus
7%Creatinine increased
6%Infection w/ grade 3 or 4 neutropenia
6%Hot flashes
6%Weight loss
5%Mouth dryness
5%Depression
5%Muscle weakness
4%Pneumonitis/pulmonary infiltrates
4%Transfusion: pRBCs
3%Irregular menses
3%Febrile neutropenia
3%Nail changes
3%Thrombosis/embolism
2%Allergic rhinitis
1%Syncope
1%Sinus tachycardia
1%Infection w/ unknown ANC
1%Neuropathic pain
1%Allergic reaction
1%Dehydration
This histogram enumerates side effects from a completed 2016 Phase 3 trial (NCT00003389) in the Arm B (Stanford V) ARM group. Side effects include: Anemia with 98%, Leukocytes decreased with 93%, Lymphopenia with 90%, Neutrophils decreased with 84%, Neuropathy-sensory with 78%.

Trial Design

2 Treatment Groups

PTC596 and Dacarbazine
1 of 2
Unesbulin and Dacarbazine
1 of 2

Experimental Treatment

41 Total Participants · 2 Treatment Groups

Primary Treatment: PTC596 · No Placebo Group · Phase 1

PTC596 and DacarbazineExperimental Group · 2 Interventions: PTC596, Dacarbazine · Intervention Types: Drug, Drug
Unesbulin and DacarbazineExperimental Group · 2 Interventions: Unesbulin, Dacarbazine · Intervention Types: Drug, Drug
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
PTC596
2016
Completed Phase 1
~40
Dacarbazine
FDA approved

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: from baseline until the date of objectively documented progression per recist v1.1 or the date of initiation of subsequent therapy or palliative local therapy, whichever occurs first (up to approximately 1.5 years)

Who is running the clinical trial?

PTC TherapeuticsLead Sponsor
70 Previous Clinical Trials
6,008 Total Patients Enrolled
1 Trials studying Leiomyosarcoma
345 Patients Enrolled for Leiomyosarcoma
Brian Van TinePrincipal InvestigatorWashington University Medical Campus
Christian MeyerPrincipal InvestigatorJohns Hopkins University
1 Previous Clinical Trials
142 Total Patients Enrolled
Steven AttiaPrincipal InvestigatorMayo Clinic
1 Previous Clinical Trials
16 Total Patients Enrolled
Matthew InghamPrincipal InvestigatorColumbia University
3 Previous Clinical Trials
127 Total Patients Enrolled
2 Trials studying Leiomyosarcoma
95 Patients Enrolled for Leiomyosarcoma
Suzanne GeorgePrincipal InvestigatorDana-Farber Cancer Institute
2 Previous Clinical Trials
68 Total Patients Enrolled
1 Trials studying Leiomyosarcoma
20 Patients Enrolled for Leiomyosarcoma

Eligibility Criteria

Age 18+ · All Participants · 10 Total Inclusion Criteria

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References

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