Multiple Treatment Session Study to Assess GSK2398852 Administered Following and Along With GSK2315698
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ClinicalTrials.gov Identifier: NCT03044353 |
Recruitment Status :
Terminated
(Change in benefit/risk profile)
First Posted : February 7, 2017
Results First Posted : October 16, 2019
Last Update Posted : October 16, 2019
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Sponsor:
GlaxoSmithKline
Information provided by (Responsible Party):
GlaxoSmithKline
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Study Type | Interventional |
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Study Design | Allocation: Non-Randomized; Intervention Model: Parallel Assignment; Masking: None (Open Label); Primary Purpose: Treatment |
Condition |
Amyloidosis |
Interventions |
Drug: GSK2315698 (CPHPC) Biological: GSK2398852 (anti-SAP mAb) |
Enrollment | 7 |
Participant Flow
Recruitment Details | This was an open label, non-randomized, monthly repeat anti-serum amyloid p component (anti-SAP) treatment study in systemic amyloidosis participants with cardiac dysfunction caused by cardiac amyloidosis. |
Pre-assignment Details | Twelve participants were screened; seven were enrolled in to study (six were enrolled in Group 1 and one in Group 2). No participant was enrolled in Group 3. The study was terminated by sponsor due to a change in the benefit:risk profile of GSK2315698+GSK2398852 (anti-SAP treatment). |
Arm/Group Title | Group 1: Cardiac TTR Amyloidosis (ATTR-CM) Participants | Group 2: Post-chemotherapy AL Amyloidosis Participants | Group 3: Newly Diagnosed Mayo Stage II/IIIa AL Participants |
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Cardiac transthyretin (TTR) amyloidosis (transthyretin amyloid cardiomyopathy [ATTR-CM]) participants with mutant genotypes primarily associated with familial amyloidotic cardiomyopathy (FAC) and wild-type TTR were included. Participants received 6 anti-SAP treatments, consisting of carboxy pyrrolidine hexanoyl pyrrolidine carboxylate (CPHPC) followed by anti-SAP monoclonal antibody (mAb) at monthly intervals. During each anti-SAP treatment, participants received CPHPC intravenous (IV) infusion once daily for up to 72 hours. After 72 hours of CPHPC administration, participants were administered intravenous infusion of anti-SAP mAb over 6-8 hours each on Days 1 and 3. The starting dose level of anti-SAP mAb was 600 milligrams (mg) (divided into 2 infusions of 300 mg). In each treatment session, CPHPC was administered as subcutaneous (SC) injection for 11 days from the day of first dose of anti-SAP mAb. | Immunoglobin light chain amyloidosis (AL) participants who attained either a very good partial response (VGPR), or complete response (CR), to systemic chemotherapy (including autologous stem cell transplantation) were included. Participants received 6 anti-SAP treatments, consisting of CPHPC followed by anti-SAP mAb at monthly intervals. During each anti-SAP treatment, participants received CPHPC IV infusion once daily for up to 72 hours. After 72 hours of CPHPC administration, participants were administered IV infusion of anti-SAP mAb over 6-8 hours each on Days 1 and 3. The starting dose level of anti-SAP mAb was 600 mg (divided into 2 infusions of 300 mg). In each treatment session, CPHPC was administered by as SC injection for 11 days from the day of first dose of anti-SAP mAb. | Newly diagnosed Mayo stage II/IIIa AL participants who attained a free light chain CR during the first 3 cycles of first-line chemotherapy where the first cycle was cyclophosphamide, bortezomib, dexamethasone (CyBorD) were planned to be included. Participants were planned to receive 6 anti-SAP treatments, consisting of CPHPC followed by anti-SAP mAb at monthly intervals. During each anti-SAP treatment, participants were planned to receive CPHPC IV infusion once daily for up to 72 hours. After 72 hours of CPHPC administration, participants were planned to administer IV infusion of anti-SAP mAb over 6-8 hours each on Days 1 and 3. The starting dose level of anti-SAP mAb was planned to be 600 mg (divided into 2 infusions of 300 mg). In each treatment session, CPHPC was planned to be administered as SC injection for 11 days from the day of first dose of anti-SAP mAb. |
Period Title: Overall Study | |||
Started | 6 | 1 | 0 [1] |
Completed | 6 | 0 | 0 |
Not Completed | 0 | 1 | 0 |
Reason Not Completed | |||
Adverse Event | 0 | 1 | 0 |
[1]
No participant was enrolled in Group-3 due to early termination of the study.
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Baseline Characteristics
Arm/Group Title | Group 1: Cardiac TTR Amyloidosis (ATTR-CM) Participants | Group 2: Post-chemotherapy AL Amyloidosis Participants | Group 3: Newly Diagnosed Mayo Stage II/IIIa AL Participants | Total | |
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Cardiac transthyretin (TTR) amyloidosis (transthyretin amyloid cardiomyopathy [ATTR-CM]) participants with mutant genotypes primarily associated with familial amyloidotic cardiomyopathy (FAC) and wild-type TTR were included. Participants received 6 anti-SAP treatments, consisting of carboxy pyrrolidine hexanoyl pyrrolidine carboxylate (CPHPC) followed by anti-SAP monoclonal antibody (mAb) at monthly intervals. During each anti-SAP treatment, participants received CPHPC intravenous (IV) infusion once daily for up to 72 hours. After 72 hours of CPHPC administration, participants were administered intravenous infusion of anti-SAP mAb over 6-8 hours each on Days 1 and 3. The starting dose level of anti-SAP mAb was 600 milligrams (mg) (divided into 2 infusions of 300 mg). In each treatment session, CPHPC was administered as subcutaneous (SC) injection for 11 days from the day of first dose of anti-SAP mAb. | Immunoglobin light chain amyloidosis (AL) participants who attained either a very good partial response (VGPR), or complete response (CR), to systemic chemotherapy (including autologous stem cell transplantation) were included. Participants received 6 anti-SAP treatments, consisting of CPHPC followed by anti-SAP mAb at monthly intervals. During each anti-SAP treatment, participants received CPHPC IV infusion once daily for up to 72 hours. After 72 hours of CPHPC administration, participants were administered IV infusion of anti-SAP mAb over 6-8 hours each on Days 1 and 3. The starting dose level of anti-SAP mAb was 600 mg (divided into 2 infusions of 300 mg). In each treatment session, CPHPC was administered by as SC injection for 11 days from the day of first dose of anti-SAP mAb. | Newly diagnosed Mayo stage II/IIIa AL participants who attained a free light chain CR during the first 3 cycles of first-line chemotherapy where the first cycle was cyclophosphamide, bortezomib, dexamethasone (CyBorD) were planned to be included. Participants were planned to receive 6 anti-SAP treatments, consisting of CPHPC followed by anti-SAP mAb at monthly intervals. During each anti-SAP treatment, participants were planned to receive CPHPC IV infusion once daily for up to 72 hours. After 72 hours of CPHPC administration, participants were planned to administer IV infusion of anti-SAP mAb over 6-8 hours each on Days 1 and 3. The starting dose level of anti-SAP mAb was planned to be 600 mg (divided into 2 infusions of 300 mg). In each treatment session, CPHPC was planned to be administered as SC injection for 11 days from the day of first dose of anti-SAP mAb. | Total of all reporting groups | |
Overall Number of Baseline Participants | 6 | 1 | 0 | 7 | |
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No participant was enrolled in "Group 3: Newly diagnosed Mayo stage II/IIIa AL participant" due to early termination of the study.
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Age, Continuous
Mean (Standard Deviation) Unit of measure: Years |
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Number Analyzed | 6 participants | 1 participants | 0 participants | 7 participants | |
74.3 (3.27) | 67.0 [1] (NA) | 73.3 (4.07) | |||
[1]
Standard deviation could not be calculated due to insufficient number of participants (N=1).
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Sex: Female, Male
Measure Type: Count of Participants Unit of measure: Participants |
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Number Analyzed | 6 participants | 1 participants | 0 participants | 7 participants | |
Female |
0 0.0%
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0 0.0%
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0 |
0 0.0%
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Male |
6 100.0%
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1 100.0%
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0 |
7 100.0%
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Race/Ethnicity, Customized
Measure Type: Count of Participants Unit of measure: Participants |
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Race | Number Analyzed | 6 participants | 1 participants | 0 participants | 7 participants |
White/Caucasian/European Heritage |
6 100.0%
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1 100.0%
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0 |
7 100.0%
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