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Abstract: INFO18

Methodology of the POSITANO Clinical Trial: Assessing the Efficacy and Safety of Octreotide Subcutaneous Depot CAM2029 in Patients With Symptomatic Polycystic Liver Disease

Session Information

  • Informational Posters
    November 03, 2022 | Location: Exhibit Hall, Orange County Convention Center‚ West Building
    Abstract Time: 10:00 AM - 12:00 PM

Category: Genetic Diseases of the Kidneys

  • No subcategory defined

Authors

  • Hogan, Marie C., Mayo Clinic Minnesota, Rochester, Minnesota, United States
  • Hjelmstrom, Peter, Camurus AB, Lund, Sweden
  • Svedberg, Agneta, Camurus AB, Lund, Sweden
  • Tiberg, Fredrik, Camurus AB, Lund, Sweden
  • Axling, Ulrika, Camurus AB, Lund, Sweden

Group or Team Name

  • On behalf of Positano Clinical Trial Consortium
Description

Somatostatin receptor analogues (SSAs), including octreotide, have been evaluated in Phase 2 trials in polycystic liver disease (PLD) accompanying autosomal dominant polycystic kidney disease (ADPKD) or autosomal dominant PLD (ADPLD). SSAs have promising effects on total liver volume (TLV) & quality of life in PLD patients not suitable for surgery. However, SSAs are not approved for treatment of PLD & their efficacy on patient reported outcomes (PROs) have not been shown with a regulatory validated qualitative assessment questionnaire instrument. Octreotide depot (CAM2029) is a novel high-exposure, sc formulation administered by a pre-filled pen. Prospective, randomized clinical trials are needed to confirm efficacy of SSAs such as CAM2029 on TLV and PROs in symptomatic PLD.
Methods: POSITANO is a Phase 2/3 randomized, placebo-controlled, double-blind, multi-center trial, aiming to enroll 69 adults with PLD. Key eligibility criteria: PLD (with ADPKD or ADPLD) with height-adjusted TLV (htTLV) ≥2500 mL/m & presence of at least 1 defined PLD-related symptom. Not a candidate for surgery for hepatic cysts during the trial & not received surgery or SSA treatment within 3 mo before screening. Patients will be randomized in a 1:1:1 ratio to 1 of 3 arms: CAM2029 10 mg 1x/wk; CAM2029 10 mg 1x every 2 wk (weekly alternation with placebo) or Placebo 1x wk. Primary EP: Change from baseline to Wk 53 in htTLV as determined by magnetic resonance imaging (MRI) volumetry. The study is powered based on efficacy of SSAs on htTLV in previous trials with SSAs. Key secondary EPs: Change from baseline in the PLD Symptoms (PLD-S) measure score. Other EPs include PLD Impact (PLD-I) measure score as well as other PROs such as, PLD-Q, PGI-S/C, SF-36 and TSQM; change from baseline in height adjusted total kidney volume (htTKV); estimated glomerular filtration rate (eGFR); CGI-S; adverse events. Following the treatment period, patients will continue to a 24-wk, open-label, single-arm, extension period with CAM2029 10 mg /week. This novel head-to-head superiority trial is anticipated to demonstrate the potential efficacy of CAM2029 versus placebo in patients symptomatic PLD. ClinicalTrials.gov ID : NCT05281328.

Funding

  • Camurus Phamaceuticals
Abstract: INFO18

Methodology of the POSITANO Clinical Trial: Assessing the Efficacy and Safety of Octreotide Subcutaneous Depot CAM2029 in Patients With Symptomatic Polycystic Liver Disease

Session Information

  • Informational Posters
    November 04, 2022 | Location: Exhibit Hall, Orange County Convention Center‚ West Building
    Abstract Time: 10:00 AM - 12:00 PM

Category:

  • No subcategory defined

Authors

  • Hogan, Marie C., Mayo Clinic Minnesota, Rochester, Minnesota, United States
  • Hjelmstrom, Peter, Camurus AB, Lund, Sweden
  • Svedberg, Agneta, Camurus AB, Lund, Sweden
  • Tiberg, Fredrik, Camurus AB, Lund, Sweden
  • Axling, Ulrika, Camurus AB, Lund, Sweden
Description

Somatostatin receptor analogues (SSAs), including octreotide, have been evaluated in Phase 2 trials in polycystic liver disease (PLD) accompanying autosomal dominant polycystic kidney disease (ADPKD) or autosomal dominant PLD (ADPLD). SSAs have promising effects on total liver volume (TLV) & quality of life in PLD patients not suitable for surgery. However, SSAs are not approved for treatment of PLD & their efficacy on patient reported outcomes (PROs) have not been shown with a regulatory validated qualitative assessment questionnaire instrument. Octreotide depot (CAM2029) is a novel high-exposure, sc formulation administered by a pre-filled pen. Prospective, randomized clinical trials are needed to confirm efficacy of SSAs such as CAM2029 on TLV and PROs in symptomatic PLD.
Methods: POSITANO is a Phase 2/3 randomized, placebo-controlled, double-blind, multi-center trial, aiming to enroll 69 adults with PLD. Key eligibility criteria: PLD (with ADPKD or ADPLD) with height-adjusted TLV (htTLV) ≥2500 mL/m & presence of at least 1 defined PLD-related symptom. Not a candidate for surgery for hepatic cysts during the trial & not received surgery or SSA treatment within 3 mo before screening. Patients will be randomized in a 1:1:1 ratio to 1 of 3 arms: CAM2029 10 mg 1x/wk; CAM2029 10 mg 1x every 2 wk (weekly alternation with placebo) or Placebo 1x wk. Primary EP: Change from baseline to Wk 53 in htTLV as determined by magnetic resonance imaging (MRI) volumetry. The study is powered based on efficacy of SSAs on htTLV in previous trials with SSAs. Key secondary EPs: Change from baseline in the PLD Symptoms (PLD-S) measure score. Other EPs include PLD Impact (PLD-I) measure score as well as other PROs such as, PLD-Q, PGI-S/C, SF-36 and TSQM; change from baseline in height adjusted total kidney volume (htTKV); estimated glomerular filtration rate (eGFR); CGI-S; adverse events. Following the treatment period, patients will continue to a 24-wk, open-label, single-arm, extension period with CAM2029 10 mg /week. This novel head-to-head superiority trial is anticipated to demonstrate the potential efficacy of CAM2029 versus placebo in patients symptomatic PLD. ClinicalTrials.gov ID : NCT05281328.

Abstract: INFO18

Methodology of the POSITANO Clinical Trial: Assessing the Efficacy and Safety of Octreotide Subcutaneous Depot CAM2029 in Patients With Symptomatic Polycystic Liver Disease

Session Information

  • Informational Posters
    November 05, 2022 | Location: Exhibit Hall, Orange County Convention Center‚ West Building
    Abstract Time: 10:00 AM - 12:00 PM

Category:

  • No subcategory defined

Authors

  • Hogan, Marie C., Mayo Clinic Minnesota, Rochester, Minnesota, United States
  • Hjelmstrom, Peter, Camurus AB, Lund, Sweden
  • Svedberg, Agneta, Camurus AB, Lund, Sweden
  • Tiberg, Fredrik, Camurus AB, Lund, Sweden
  • Axling, Ulrika, Camurus AB, Lund, Sweden
Description

Somatostatin receptor analogues (SSAs), including octreotide, have been evaluated in Phase 2 trials in polycystic liver disease (PLD) accompanying autosomal dominant polycystic kidney disease (ADPKD) or autosomal dominant PLD (ADPLD). SSAs have promising effects on total liver volume (TLV) & quality of life in PLD patients not suitable for surgery. However, SSAs are not approved for treatment of PLD & their efficacy on patient reported outcomes (PROs) have not been shown with a regulatory validated qualitative assessment questionnaire instrument. Octreotide depot (CAM2029) is a novel high-exposure, sc formulation administered by a pre-filled pen. Prospective, randomized clinical trials are needed to confirm efficacy of SSAs such as CAM2029 on TLV and PROs in symptomatic PLD.
Methods: POSITANO is a Phase 2/3 randomized, placebo-controlled, double-blind, multi-center trial, aiming to enroll 69 adults with PLD. Key eligibility criteria: PLD (with ADPKD or ADPLD) with height-adjusted TLV (htTLV) ≥2500 mL/m & presence of at least 1 defined PLD-related symptom. Not a candidate for surgery for hepatic cysts during the trial & not received surgery or SSA treatment within 3 mo before screening. Patients will be randomized in a 1:1:1 ratio to 1 of 3 arms: CAM2029 10 mg 1x/wk; CAM2029 10 mg 1x every 2 wk (weekly alternation with placebo) or Placebo 1x wk. Primary EP: Change from baseline to Wk 53 in htTLV as determined by magnetic resonance imaging (MRI) volumetry. The study is powered based on efficacy of SSAs on htTLV in previous trials with SSAs. Key secondary EPs: Change from baseline in the PLD Symptoms (PLD-S) measure score. Other EPs include PLD Impact (PLD-I) measure score as well as other PROs such as, PLD-Q, PGI-S/C, SF-36 and TSQM; change from baseline in height adjusted total kidney volume (htTKV); estimated glomerular filtration rate (eGFR); CGI-S; adverse events. Following the treatment period, patients will continue to a 24-wk, open-label, single-arm, extension period with CAM2029 10 mg /week. This novel head-to-head superiority trial is anticipated to demonstrate the potential efficacy of CAM2029 versus placebo in patients symptomatic PLD. ClinicalTrials.gov ID : NCT05281328.