POSITANO: A Trial to Assess the Efficacy and Safety of Octreotide Subcutaneous Depot in Patients With PLD
Study Details
Study Description
Brief Summary
The purpose of the trial is to compare the effectiveness and safety of 2 treatment regimens of CAM2029 (given weekly or every 2 weeks) to placebo in participants with symptomatic PLD, either isolated as in autosomal dominant PLD (ADPLD) or associated with autosomal dominant polycystic kidney disease (ADPKD).
In the Treatment Period of the trial, participants will be allocated at random to 1 of the 3 treatment arms in a 1:1:1 ratio. After completing the Treatment Period (53 weeks) participants may proceed to a 24-week open-label extension part of the trial and then only receive the same CAM2029 treatment.
The active ingredient in CAM2029, octreotide, is administered as a subcutaneous depot using Camurus' FluidCrystal® technology.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 2/Phase 3 |
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: CAM2029 once weekly 0.5 mL CAM2029 10 mg, subcutaneous (SC) injection, once weekly |
Drug: CAM2029
SC injection using a pre-filled pen
Other Names:
|
| Experimental: CAM2029 once every 2 weeks 0.5 mL CAM2029 10 mg, SC injection, every 2 weeks and 0.5 mL placebo, SC injection, once every 2 weeks (alternating with CAM2029 dosing) |
Drug: CAM2029
SC injection using a pre-filled pen
Other Names:
Drug: Placebo
SC injection using a pre-filled pen
|
| Placebo Comparator: Placebo 0.5 mL placebo, SC injection, once weekly |
Drug: Placebo
SC injection using a pre-filled pen
|
Outcome Measures
Primary Outcome Measures
- Height-adjusted total liver volume (htTLV) [From screening until treatment week 53]
Change from baseline to Week 53 in htTLV as determined by MRI volumetry
Secondary Outcome Measures
- PLD symptom (PLD-S) score [From screening to week 53]
Key secondary endpoint. Change from baseline to Week 53 in the PLD-S measure score
- htTLV [From screening until treatment weeks 13, 25 and 77]
Change from baseline in htTLV as determined by MRI volumetry
- PLD-S [From screening to weeks 13, 21, 25, 39 and 77]
Change from baseline in the PLD-S measure score
- Height-adjusted total kidney volume (htTKV) [From screening until treatment weeks 13, 25, 53 and 77]
Change from baseline in htTKV as determined by MRI volumetry
- Estimated glomerular filtration rate (eGFR) [From treatment week 1 to weeks 13, 25, 53, 65 and 77]
Change from baseline in eGFR, assessed by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) cystatin C equation using serum concentrations of creatinine and cystatin C
- PLD impact (PLD-I) score [From screening to weeks 13, 21, 25, 39, 53 and 77]
Change from baseline in the PLD-I measure score
- Clinical Global Impression of Severity (CGI-S) score [From treatment week 1 to weeks 13, 21, 25, 53 and 77]
Change from baseline in the CGI-S score
- Patient Global Impression of Severity (PGI-S) score [From screening to weeks 13, 21, 25, 39, 53 and 77]
Change from baseline in the PGI-S score
- Patient Global Impression of Change (PGI-C) score [At treatment weeks 13, 21, 25, 39, 53 and 77]
Change from baseline in the PGI-C score
- Short Form-36 (SF-36) score [From treatment week 1 to weeks 25, 53 and 77]
Change from baseline in the SF-36 score
- Polycystic Liver Disease Questionnaire (PLD-Q) [From treatment week 1 to weeks 25, 53 and 77]
Change from baseline in the PLD-Q score
- Adverse events (AEs) [From screening to the safety follow-up, assessed up to approximately 21 months]
Incidence of AEs
Eligibility Criteria
Criteria
Inclusion Criteria:
-
Male or female patient, ≥18 years at screening
-
Diagnosis of PLD (associated with ADPKD or isolated as in ADPLD) as defined by htTLV ≥2500 mL/m at screening
-
Presence of at least 1 of the following PLD-related symptoms within 2 weeks before screening: bloating, fullness in abdomen, lack of appetite, feeling full quickly after beginning to eat, acid reflux, nausea, rib cage pain or pressure, pain in side, abdominal pain, back pain, shortness of breath after physical exertion, limited in mobility, concern about abdomen getting larger, dissatisfied by the size of abdomen
-
Not a candidate for, or not willing to undergo, surgical intervention for hepatic cysts during the trial
Exclusion Criteria:
-
Surgical intervention for PLD within 3 months before screening
-
Treatment with a somatostatin analogue (SSA) within 3 months before screening
-
Non-responsive to previous treatment of PLD with an SSA as per the Investigator's assessment
-
Cholelithiasis within 3 months before screening or previous medical history of cholelithiasis induced by SSAs unless treated with cholecystectomy
-
Presence of extrahepatic cysts that, in the Investigator's opinion, may prevent the patient from safely participating in the trial
-
Severe kidney disease, as defined by eGFR <30 mL/min/1.73^m2
-
Severe liver disease defined as liver cirrhosis of Child-Pugh class C
-
Any other current or prior medical condition that may interfere with the conduct of the trial or the evaluation of its results in the opinion of the Investigator
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Mayo Clinic | Rochester | Minnesota | United States | 55905 |
| 2 | University of Texas Southwestern Medical Center | Dallas | Texas | United States | 75380 |
Sponsors and Collaborators
- Camurus AB
Investigators
- Principal Investigator: Joost Drenth, MD, Department of Gastroenterology and Hepatology, Radboud UMC Nijmegen, The Netherlands
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- HS-20-677
- 2021-003764-27