E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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E.1.1.1 | Medical condition in easily understood language |
Acromegaly is a chronic metabolic disorder in which there is too much growth hormone and the body tissues gradually enlarge. |
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E.1.1.2 | Therapeutic area | Diseases [C] - Hormonal diseases [C19] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10000599 |
E.1.2 | Term | Acromegaly |
E.1.2 | System Organ Class | 10014698 - Endocrine disorders |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
Main Part of the Trial: • To assess the overall safety and tolerability of CAM2029 Extension part of the trial: • To assess the overall safety and tolerability of CAM2029 |
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E.2.2 | Secondary objectives of the trial |
Main Part of the Trial: To assess efficacy of CAM2029 based on biochemical characteristics. To assess self-and partner administration To assess plasma concentration of octreotide after administration of CAM2029 To measure patients' satisfaction with CAM2029 To measure the effects of CAM2029 on quality of life (QoL) Extension part of the trial: To assess efficacy of CAM2029 based on biochemical characteristics. To evaluate patients' satisfaction with CAM2029 To evaluate the effects of CAM2029 on QoL To evaluate the effects of CAM2029 on health economic outcomes To evaluate solicited safety assessments after treatment with CAM2029 |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Main Part of the Trial: Male or female patients > =18 years at screening Able to provide written informed consent to participate in the trial Diagnosis of acromegaly by historical evidence (persistent or recurrent) acromegaly Treatment with a stable dose of octreotide LAR or lanreotide ATG for at least 3 months as monotherapy prior to screening IGF-1 levels >1xULN and ≤2.0xULN at screening (adjusted for age and sex; mean value of the first measurement at screening and the second measurement at 2 weeks before Day 1) or IGF-1 levels <=1xULN at screening (adjusted for age and sex; value of the first measurement at screening and the second measurement at 2 weeks before Day 1) either without prior pituitary radiotherapy or with prior pituitary radiotherapy Adequate liver, pancreatic, renal and bone marrow functions Normal ECG Extension Part of the Trial: Continuation Criteria for Patients who Continue Directly to the Extension Part of the Trial Patients who continue directly from the main part of the trial must complete treatment with CAM2029 in the main part of the trial, attend the Week 52 visit, and provide written informed consent to continue treatment in the extension part of the trial before treatment can be continued. Main Inclusion Criteria for Re-invited Patients: Completed treatment with CAM2029 in the main part of the trial and attended the Week 52 visit Adequate liver, pancreatic and renal functions Normal ECG |
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E.4 | Principal exclusion criteria |
Main Part of the Trial: For Roll-over Patients from Trial HS-18-633: • Unresolved, drug-related serious adverse event (SAE) from the preceding trial (HS-18-633) • Patients with a clinically significant or unstable medical or surgical condition that may preclude safe and complete trial participation For New Patients: • 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 prior to screening • Patients who have undergone pituitary surgery within 6 months prior to screening • Patients who have received prior pituitary irradiation within 3 years prior to screening • Patients with poorly controlled diabetes mellitus (hemoglobin A1c [HbA1c] >8.0%) Main Exclusion Criteria for Re-invited Patients -Extension Part of the Trial: • Receiving treatments (other than treatments for acromegaly) known to affect GH or IGF-1 concentration • Patients who have undergone major surgery/surgical therapy (including pituitary surgery) for any cause within 1 month prior to screening • Patients who have received pituitary irradiation since the end of the main part of the trial • Patients with poorly controlled diabetes mellitus (HbA1c >8.0%) |
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E.5 End points |
E.5.1 | Primary end point(s) |
Main Part of the Trial: • Characterization of adverse events (AEs) Extension Part of the Trial: • Characterization of AEs |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
Main Part of the Trial: • Proportion of patients with mean IGF-1 levels ≤1 x upper limit of normal (ULN) and < 1.3xULN at Week 50 and Week 52 (average of the 2 measurements) • Proportion of patients with mean GH levels <2.5 μg/L and <5.0 μg/L at Week 52 • Proportion of patients/partners declared competent by healthcare professional to administer CAM2029 • Octreotide plasma concentrations over time • Treatment Satisfaction Questionnaire for Medication (TSQM) scores over time using all 4 domains of TSQM (effectiveness, side effects, convenience, and satisfaction) • Patient satisfaction scale scores at Week 24 and Week 52 • Change from baseline in Acromegaly Quality of Life Questionnaire (AcroQoL) and EuroQoL 5-dimension 5-level (EQ-5D-5L) scores Extension Part of the Trial: • IGF-1 levels over time • GH levels over time • TSQM scores over time using all 4 domains of TSQM (effectiveness, side effects, convenience, and satisfaction) • AcroQoL, EQ-5D-5L and Short Form-36 (SF-36) scores over time • Work Productivity and Activity Impairment (WPAI) scores over time • Laboratory values, vital signs, ECG readings and gallbladder imaging over time |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Main Part of the Trial: Week 50 and Week 52 Extension Part of the Trial: Through the extension period |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 1 |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 3 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 32 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
United States |
Poland |
Spain |
Germany |
Greece |
Italy |
Hungary |
Russian Federation |
Turkey |
United Kingdom |
Serbia |
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E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 2 |
E.8.9.1 | In the Member State concerned months | 6 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 6 |
E.8.9.2 | In all countries concerned by the trial days | 0 |