E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Non-Small Cell Lung Cancer related Cachexia (NSCLC-C) |
|
E.1.1.1 | Medical condition in easily understood language |
Weight-loss and muscle mass loss due to NSCLC-C |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Cancer [C04] |
MedDRA Classification |
E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To evaluate the safety and tolerability of Anamorelin HCl |
|
E.2.2 | Secondary objectives of the trial |
1. To evaluate the effect of Anamorelin HCl on body weight
2. To evaluate the effect of Anamorelin HCl on muscle strength as measured by hand grip strength 3. To evaluate the effect of Anamorelin HCl on quality of life as assessed using the FAACT (Functional Assessment of Anorexia/Cachexia Treatment) and the FACIT F (Functional Assessment of Chronic Illness Therapy – Fatigue) (see Appendix II)
|
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E.2.3 | Trial contains a sub-study | Yes |
E.2.3.1 | Full title, date and version of each sub-study and their related objectives |
Title: Cost and cost effectiveness analysis alongside randomized,
double-blind placebo-controlled trials of Anamorelin HCL in the
treatment of non-small cell lung cancer – cachexia (HT-ANAM-301
[ROMANA 1], HT-ANAM-302 [ROMANA 2] and HT-ANAM-303 [ROMANA
3]).
Protocol No.: HE-01 version of 27.09.2012
The proposed economic analysis is to estimate the differences in costs
and health outcomes that are likely to translate into Anamorelin HCI
being a cost-effective way of controlling cachexia. The primary objective
of this economic study is to measure the costs and cost-effectiveness of
Anamorelin HCI from the societal perspective.
The secondary objectives are as follows: To estimate the financial and
economic costs of providing Anamorelin HCI from the country in analysis
perspective; To estimate the cost of cachexia events to the patient and
their caregiver(s); To evaluate the effect of Anamorelin HCI vs. placebo
on medical resource utilisation, including the frequency of outpatient
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and inpatient hospitalisations, concomitant medications and additional
investigations; To obtain utility values from the patients in the ROMANA
studies through the EuroQoL-5 dimension questionnaire (EQ-5D)1 in
order to estimate Quality Adjusted Life Years (QALYs) as an intermediate
effectiveness measure of these trials and; To assess the incremental
costs and cost-effectiveness of Anamorelin HCI compared to placebo
after one year based on survival data. |
|
E.3 | Principal inclusion criteria |
• The patient has completed the Day 85 Visit in the original trial (Study HT ANAM-301 or HT-ANAM-302) and the Investigator considers the patient to be appropriate to continue to receive an additional 12 weeks of study drug administration. The patient must start dosing on the extension study within 5 days of completing dosing on the original trial.
• Females and males at least 18 years of age
• ECOG performance status less than / equal to 2 |
|
E.4 | Principal exclusion criteria |
• Women who are pregnant or breast-feeding
• Had major surgery (central venous access placement and tumor biopsies are not considered major surgery) within 4 weeks prior to enrollment into the extension study. Patients must be well recovered from acute effects of surgery prior to screening. Patients should not have plans to undergo major surgical procedures during the treatment period.
• Patients unable to readily swallow oral tablets. Patients with severe gastointestinal disease (including esophagitis, gastritis, malaborption, or obstructive symptoms) or intractable or frequent vomiting are excluded.
• Has known or symptomatic brain metastases
|
|
E.5 End points |
E.5.1 | Primary end point(s) |
1. Changes in body weight from baseline of the HT-ANAM-301 or HT-ANAM-302 trials
2. Change in Hand Grip Strength of the non-dominant hand
3. Change in HGS of the dominant hand
4. Quality of life |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
1. Changes in body weight at Weeks 4, 8, 12
2. Changes in Hand Grip Strength of non-dominant hand at Weeks 8, 12
3. Changes in Hand Grip Strength of dominant hand at Weeks 8, 12
4. Quality of life as assessed by FAACT and FACIT-F at Weeks 4, 8, 12 |
|
E.5.2 | Secondary end point(s) |
No secondary endpoints specified. All endpoints listed above as primary are the efficacy endpoints for the study |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
|
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 | No |
E.6.7 | Pharmacodynamic | No |
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 | Yes |
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 | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
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 | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 2 |
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 | 8 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 48 |
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 |
Australia |
Belgium |
Canada |
Czech Republic |
France |
Germany |
Hungary |
Israel |
Italy |
Netherlands |
Poland |
Russian Federation |
Spain |
United Kingdom |
United States |
|
E.8.7 | Trial has a data monitoring committee | Yes |
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
|
A follow-up visit will be completed 28 days (±7 days) after the last dose of study drug for adverse event assessment. The patient will continue in the survival follow-up for the original trial in which they participated (HT-ANAM-301 or HT-ANAM-302). |
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 3 |
E.8.9.1 | In the Member State concerned months | 1 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 3 |
E.8.9.2 | In all countries concerned by the trial months | 1 |
E.8.9.2 | In all countries concerned by the trial days | 0 |