Acromegaly
Conditions
Keywords
Acromegaly, octreotide, CAM2029, phase 3
Brief summary
The purpose of this trial is to assess the efficacy and safety of CAM2029 in patients with acromegaly. Patients will be randomized to either CAM2029 or placebo administered subcutaneously once monthly during 6 months.
Interventions
Octreotide subcutaneous depot for monthly injections in acromegaly patients
DRUGMatching placebo
Matching placebo for CAM2029
Sponsors
Camurus AB
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Caregiver, Investigator)
Masking description
Double-blind
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Male or female patients, ≥18 years at screening * Able to provide written informed consent to participate in the trial prior to any trial related procedures are performed * Diagnosis of acromegaly by historical evidence of (persistent or recurrent) acromegaly * Treatment with a stable dose of octreotide long-acting repeatable (LAR) or lanreotide autogel (ATG) for at least 3 months as monotherapy prior to screening * Insulin-like growth factor-1 (IGF-1) levels ≤1x upper limit of normal (ULN) at screening * Adequate liver, pancreatic, renal and bone marrow functions * Normal ECG
Exclusion criteria
* Growth hormone (GH) ≥2.5 μg/L at screening (cycle) * Have received medical treatment for acromegaly with pasireotide (within 6 months prior to screening), pegvisomant (within 3 months prior to screening), dopamine agonists (within 3 months prior to screening) or other investigational agents (within 30 days or 5 half-lives prior to screening \[whichever is longer\] * Patients who usually take octreotide LAR or lanreotide ATG less frequently than every 4 weeks (e.g. every 6 weeks or 8 weeks) * Patients with compression of the optic chiasm causing any visual field defect for whom surgical intervention is indicated * Patients who have undergone major surgery/surgical therapy for any cause within 1 month from screening * Patients who have undergone pituitary surgery within 6 months prior to screening * Patients who have received prior pituitary irradiation * Patients with poorly controlled diabetes mellitus (hemoglobin A1c \>8.0%)
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Proportion of Participants With Mean Insulin-like Growth Factor -1 (IGF-1) Levels ≤1 x Upper Limit of Normal (ULN) at Week 22/24 | Week 22 and 24 | If one of the IGF-1 values at Week 22 or Week 24 was missing, the other value was used to define a responder/non-responder in the analysis. The variable of interest was considered missing only if no IGF-1 value could be obtained from either the Week 22 or the Week 24 sample. A composite strategy was assumed for intercurrent events, and a participant was considered as a non-responder if they discontinued IMP, or had the dose reduced prior to Week 22 (regardless of IGF-1 values), and/or was switched to rescue medication. ULN was derived from the participant's sex and age at screening. In order to account for all participants in the intention-to-treat (ITT) analysis set, multiple imputation was applied. The mean proportion of responders was calculated as an average of responders across the imputed datasets. The resulting proportion was multiplied by 100 to present "Percentage of participants". |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Proportion of Participants With Mean IGF-1 Levels ≤1x Upper Limit or Normal (ULN) at Week 22/24, Including Participants With Dose Reduction | Week 22 and 24 | First key secondary endpoint. If one of the IGF-1 values at Week 22 or Week 24 was missing, the other value was used to define a responder/non-responder in the analysis. The variable of interest was considered missing only if no IGF-1 value could be obtained from either the Week 22 or the Week 24 sample. A composite strategy was assumed for intercurrent events, and a participant was considered as a non-responder if they discontinued IMP and/or was switched to rescue medication. For this endpoint, a patient who had their dose reduced was not directly classified as a non-responder. No participant had their dose reduced during the trial. ULN was derived from the participant's sex and age at screening. In order to account for all participants in the ITT analysis set, multiple imputation was applied. The mean proportion of responders was calculated as an average of responders across the imputed datasets. The resulting proportion was multiplied by 100 to present "Percentage of participants" |
| Proportion of Participants With Mean IGF-1 Levels ≤1xULN at Week 22/Week 24 and Mean Growth Hormone (GH) Levels <2.5 µg/L at Week 24 | Week 22 and 24 | Second key secondary endpoint. If one of the IGF-1 values at Week 22 or Week 24 was missing, the other value was used to define a responder/non-responder in the analysis. The variable of interest was considered missing only if no IGF-1 value could be obtained from either the Week 22 or the Week 24 sample or no GH value at Week 24. A composite strategy was assumed for intercurrent events, and a patient was considered as a non-responder if they discontinued IMP or had the dose reduced prior to Week 22 (regardless of IGF-1 values), and/or was switched to rescue medication. ULN was based on the patient's sex and age at screening. In order to account for all participants in the ITT analysis set, multiple imputation was applied. The mean proportion of responders was calculated as an average of responders across the imputed datasets. The resulting proportion was multiplied by 100 to present "Percentage of participants". |
| Proportion of Participants With Mean GH Levels <2.5 µg/L at Week 24 | Week 24 | For the responder analysis of mean GH \<2.5 µg/L at Week 24, a composite strategy was assumed for intercurrent events. A participant was considered as a non-responder if they discontinued IMP, or had the dose reduced prior to Week 24 (regardless of mean GH values), and/or was switched to rescue medication. In order to account for all participants in the ITT analysis set, multiple imputation was applied. The mean proportion of participants was calculated as an average of responders across the imputed datasets. The resulting proportion was multiplied by 100 to present "Percentage of participants". |
| Proportion of Participants With Mean GH Levels <1.0 µg/L at Week 24 | Week 24 | For the responder analysis of mean GH \<1.0 µg/L at Week 24, a composite strategy was assumed for intercurrent events. A participant was considered as a non-responder if they discontinued IMP, or had the dose reduced prior to Week 24 (regardless of mean GH values), and/or was switched to rescue medication. In order to account for all participants in the ITT analysis set, multiple imputation was applied. The mean proportion of participants was calculated as an average of responders across the imputed datasets. The resulting proportion was multiplied by 100 to present "Percentage of participants". |
| Number of Participants With Treatment Emergent Adverse Events | Week 0 to 24 | A treatment emergent adverse event was defined as an adverse event that occurred during or after the first administration of the IMP to the end of trial visit or the next dose of any acromegaly treatment, whichever came first. |
| Proportion of Participants/Partners Declared Competent by a Healthcare Professional to Administer Intervention | Week 0 to 20 and Week 24 | During participants'/partners' first three attempts during the trial period of 24 weeks whenever these visits took place. Percentages were based on those who opted for self-/partner-administration. |
| Octreotide Plasma Concentrations Over Time | Week 0 to 24 | Plasma samples were taken pre-dose on dosing days. |
Countries
Germany, Greece, Hungary, Italy, Poland, Russia, Spain, Turkey (Türkiye), United Kingdom, United States
Contacts
PRINCIPAL_INVESTIGATORPamela Freda, M.D
Columbia University
Baseline characteristics
| Characteristic | — |
|---|---|
| Age, Continuous | 57.0 years STANDARD_DEVIATION 11.2 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 1 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 69 Participants |
| Sex: Female, Male Female | 28 Participants |
| Sex: Female, Male Male | 12 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 47 | 0 / 24 |
| other Total, other adverse events | 36 / 47 | 19 / 24 |
| serious Total, serious adverse events | 4 / 47 | 2 / 24 |
Outcome results
None listed