Trial Outcomes & Findings for Norditropin NordiFlex® Device Compared to the Device Previously Used by Patients or Parents (NCT NCT01245374)

NCT ID: NCT01245374

Last Updated: 2017-02-27

Results Overview

Analysed for the ITT (intent-to-treat) analysis set: The relative ease of use of the trial injection device compared to the device previously used was assessed using a quantitative scale, ranging from 0 to 10 with 0 = NordiFlex® is far less simple, 5 = equivalent simplicity and 10 = NordiFlex® is far more simple. The participants had to circle the number that represented their perception of the current state.

Recruitment status

COMPLETED

Study phase

PHASE4

Target enrollment

103 participants

Primary outcome timeframe

Week 6

Results posted on

2017-02-27

Participant Flow

The trial was conducted in France (38 sites). Recruitment period occurred from 17-Nov-2010 to 18-Apr-2011.

Eligible participants had to receive training for the use of Nordiflex® system. A member of the investigator's team, physician or nurse, was in charge of this training. At inclusion and before receiving treatment, participants or parents had to complete a questionnaire about the ease of use of the previously used device.

Participant milestones

Participant milestones
Measure
Norditropin NordiFlex®
Individually adjusted dose administered with Norditropin NordiFlex® for 6 weeks. Dosage depended on age, weight, etiologies and according to the summary of product characteristics (SPC).
Overall Study
STARTED
103
Overall Study
COMPLETED
99
Overall Study
NOT COMPLETED
4

Reasons for withdrawal

Reasons for withdrawal
Measure
Norditropin NordiFlex®
Individually adjusted dose administered with Norditropin NordiFlex® for 6 weeks. Dosage depended on age, weight, etiologies and according to the summary of product characteristics (SPC).
Overall Study
Protocol Violation
1
Overall Study
Withdrawal by Subject
3

Baseline Characteristics

Norditropin NordiFlex® Device Compared to the Device Previously Used by Patients or Parents

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Norditropin NordiFlex®
n=103 Participants
Individually adjusted dose administered with Norditropin NordiFlex® for 6 weeks. Dosage depended on age, weight, etiologies and according to the summary of product characteristics (SPC).
Age, Continuous
11.7 years
STANDARD_DEVIATION 2.94 • n=5 Participants
Gender
Female
43 Participants
n=5 Participants
Gender
Male
60 Participants
n=5 Participants
Region of Enrollment
France
103 participants
n=5 Participants
Height
141.0 cm
STANDARD_DEVIATION 16.5 • n=5 Participants
Weight
37.3 kg
STANDARD_DEVIATION 16.0 • n=5 Participants
Body Mass Index (BMI)
18.1 kg/m^2
STANDARD_DEVIATION 4.26 • n=5 Participants
Duration of growth hormone treatment
4.18 years
STANDARD_DEVIATION 2.81 • n=5 Participants

PRIMARY outcome

Timeframe: Week 6

Population: Intent-to-treat (ITT) consists of all participants included in the trial and having signed the informed consent form.

Analysed for the ITT (intent-to-treat) analysis set: The relative ease of use of the trial injection device compared to the device previously used was assessed using a quantitative scale, ranging from 0 to 10 with 0 = NordiFlex® is far less simple, 5 = equivalent simplicity and 10 = NordiFlex® is far more simple. The participants had to circle the number that represented their perception of the current state.

Outcome measures

Outcome measures
Measure
Norditropin NordiFlex®
n=103 Participants
Individually adjusted dose administered with Norditropin NordiFlex® for 6 weeks. Dosage depended on age, weight, etiologies and according to the summary of product characteristics (SPC)
The Relative Ease of Use of NordiFlex® Compared to the Device Previously Used
6.89 units on a scale
Standard Deviation 2.52

PRIMARY outcome

Timeframe: Week 6

Population: Per protocol set (PP): All subjects in the ITT set using NordiFlex® and had completed the trial without any significant violation of the inclusion/exclusion criteria or any other aspect of the protocol considered to potentially affect the efficacy results.

Analysed for the PP (per protocol) analysis set: The relative ease of use of the trial injection device compared to the device previously used was assessed using a quantitative scale, ranging from 0 to 10 with 0 = NordiFlex® is far less simple, 5 = equivalent simplicity and 10 = NordiFlex® is far more simple. The participants had to circle the number that represented their perception of the current state.

Outcome measures

Outcome measures
Measure
Norditropin NordiFlex®
n=98 Participants
Individually adjusted dose administered with Norditropin NordiFlex® for 6 weeks. Dosage depended on age, weight, etiologies and according to the summary of product characteristics (SPC)
The Relative Ease of Use of NordiFlex® Compared to the Device Previously Used
6.98 units on a scale
Standard Deviation 2.46

SECONDARY outcome

Timeframe: Week 0

Population: Intent-to-treat (ITT) consists of all participants included in the trial and having signed the informed consent form.

The overall simplicity of use was evaluated at every step of the injection preparation (dose selection, injection and dose modification) and using the time spent in the preparation of the injection. Ordinal variables were used for the evaluation (very easy, easy, fairly easy, difficult and very difficult).

Outcome measures

Outcome measures
Measure
Norditropin NordiFlex®
n=103 Participants
Individually adjusted dose administered with Norditropin NordiFlex® for 6 weeks. Dosage depended on age, weight, etiologies and according to the summary of product characteristics (SPC)
Percentage of Participants Evaluating Simplicity of Use: Dose Selection Easiness
Very easy
55.3 percentage of participants
Percentage of Participants Evaluating Simplicity of Use: Dose Selection Easiness
Easy
38.8 percentage of participants
Percentage of Participants Evaluating Simplicity of Use: Dose Selection Easiness
Fairly easy
4.85 percentage of participants
Percentage of Participants Evaluating Simplicity of Use: Dose Selection Easiness
Difficult
0.97 percentage of participants
Percentage of Participants Evaluating Simplicity of Use: Dose Selection Easiness
Very difficult
0 percentage of participants

SECONDARY outcome

Timeframe: Week 0

Population: Intent-to-treat (ITT) consists of all participants included in the trial and having signed the informed consent form. For seven patients, no data available.

The overall simplicity of use was evaluated at every step of the injection preparation (dose selection, injection and dose modification) and using the time spent in the preparation of the injection. Ordinal variables were used for the evaluation (very easy, easy, fairly easy, difficult and very difficult).

Outcome measures

Outcome measures
Measure
Norditropin NordiFlex®
n=96 Participants
Individually adjusted dose administered with Norditropin NordiFlex® for 6 weeks. Dosage depended on age, weight, etiologies and according to the summary of product characteristics (SPC)
Percentage of Participants Evaluating Simplicity of Use: Dose Modification
Very easy
26.0 percentage of participants
Percentage of Participants Evaluating Simplicity of Use: Dose Modification
Easy
40.6 percentage of participants
Percentage of Participants Evaluating Simplicity of Use: Dose Modification
Fairly easy
19.8 percentage of participants
Percentage of Participants Evaluating Simplicity of Use: Dose Modification
Difficult
11.5 percentage of participants
Percentage of Participants Evaluating Simplicity of Use: Dose Modification
Very difficult
2.08 percentage of participants

SECONDARY outcome

Timeframe: Week 0

Population: Intent-to-treat (ITT) consists of all participants included in the trial and having signed the informed consent form. For three patients, no data available.

The overall simplicity of use was evaluated at every step of the injection preparation (dose selection, injection and dose modification) and using the time spent in the preparation of the injection. Ordinal variables were used for the evaluation (very easy, easy, fairly easy, difficult and very difficult).

Outcome measures

Outcome measures
Measure
Norditropin NordiFlex®
n=100 Participants
Individually adjusted dose administered with Norditropin NordiFlex® for 6 weeks. Dosage depended on age, weight, etiologies and according to the summary of product characteristics (SPC)
Percentage of Participants Evaluating Simplicity of Use: Injection Easiness
Very easy
44.7 percentage of participants
Percentage of Participants Evaluating Simplicity of Use: Injection Easiness
Easy
43.7 percentage of participants
Percentage of Participants Evaluating Simplicity of Use: Injection Easiness
Fairly easy
9.71 percentage of participants
Percentage of Participants Evaluating Simplicity of Use: Injection Easiness
Difficult
1.94 percentage of participants
Percentage of Participants Evaluating Simplicity of Use: Injection Easiness
Very difficult
0 percentage of participants

SECONDARY outcome

Timeframe: Week 0

Population: Intent-to-treat (ITT) consists of all participants included in the trial and having signed the informed consent form.

The overall simplicity of use was evaluated at every step of the injection preparation (dose selection, injection and dose modification) and using the time spent in the preparation of the injection. This was evaluated using time intervals: below 5 minutes, 5-10 minutes, 10-20 minutes.

Outcome measures

Outcome measures
Measure
Norditropin NordiFlex®
n=103 Participants
Individually adjusted dose administered with Norditropin NordiFlex® for 6 weeks. Dosage depended on age, weight, etiologies and according to the summary of product characteristics (SPC)
Percentage of Participants Evaluating Simplicity of Use: Time Spent in the Preparation of the Injection
Below 5 minutes
86.4 percentage of participants
Percentage of Participants Evaluating Simplicity of Use: Time Spent in the Preparation of the Injection
5-10 minutes
11.7 percentage of participants
Percentage of Participants Evaluating Simplicity of Use: Time Spent in the Preparation of the Injection
10-20 minutes
1.94 percentage of participants

SECONDARY outcome

Timeframe: Week 6

Population: Intent-to-treat (ITT) consists of all participants included in the trial and having signed the informed consent form. For one patient, no data available.

The overall simplicity of use was evaluated at every step of the injection preparation (dose selection, injection and dose modification) and using the time spent in the preparation of the injection. Ordinal variables were used for the evaluation (very easy, easy, fairly easy, difficult and very difficult).

Outcome measures

Outcome measures
Measure
Norditropin NordiFlex®
n=102 Participants
Individually adjusted dose administered with Norditropin NordiFlex® for 6 weeks. Dosage depended on age, weight, etiologies and according to the summary of product characteristics (SPC)
Percentage of Participants Evaluating Simplicity of Use: Dose Selection Easiness
Very easy
66.7 percentage of participants
Percentage of Participants Evaluating Simplicity of Use: Dose Selection Easiness
Easy
21.6 percentage of participants
Percentage of Participants Evaluating Simplicity of Use: Dose Selection Easiness
Fairly easy
8.82 percentage of participants
Percentage of Participants Evaluating Simplicity of Use: Dose Selection Easiness
Difficult
2.94 percentage of participants
Percentage of Participants Evaluating Simplicity of Use: Dose Selection Easiness
Very difficult
0 percentage of participants

SECONDARY outcome

Timeframe: Week 6

Population: Intent-to-treat (ITT) consists of all participants included in the trial and having signed the informed consent form. For three patients, no data available.

The overall simplicity of use was evaluated at every step of the injection preparation (dose selection, injection and dose modification) and using the time spent in the preparation of the injection. Ordinal variables were used for the evaluation (very easy, easy, fairly easy, difficult and very difficult).

Outcome measures

Outcome measures
Measure
Norditropin NordiFlex®
n=100 Participants
Individually adjusted dose administered with Norditropin NordiFlex® for 6 weeks. Dosage depended on age, weight, etiologies and according to the summary of product characteristics (SPC)
Percentage of Participants Evaluating Simplicity of Use: Dose Modification
Very easy
84.0 percentage of participants
Percentage of Participants Evaluating Simplicity of Use: Dose Modification
Easy
13.0 percentage of participants
Percentage of Participants Evaluating Simplicity of Use: Dose Modification
Fairly easy
2.0 percentage of participants
Percentage of Participants Evaluating Simplicity of Use: Dose Modification
Difficult
1.0 percentage of participants
Percentage of Participants Evaluating Simplicity of Use: Dose Modification
Very difficult
0.00 percentage of participants

SECONDARY outcome

Timeframe: Week 6

Population: Intent-to-treat (ITT) consists of all participants included in the trial and having signed the informed consent form. For one patient, no data available.

The overall simplicity of use was evaluated at every step of the injection preparation (dose selection, injection and dose modification) and using the time spent in the preparation of the injection. Ordinal variables were used for the evaluation (very easy, easy, fairly easy, difficult and very difficult).

Outcome measures

Outcome measures
Measure
Norditropin NordiFlex®
n=102 Participants
Individually adjusted dose administered with Norditropin NordiFlex® for 6 weeks. Dosage depended on age, weight, etiologies and according to the summary of product characteristics (SPC)
Percentage of Participants Evaluating Simplicity of Use: Injection Easiness
Very easy
62.7 percentage of participants
Percentage of Participants Evaluating Simplicity of Use: Injection Easiness
Easy
22.5 percentage of participants
Percentage of Participants Evaluating Simplicity of Use: Injection Easiness
Fairly easy
12.7 percentage of participants
Percentage of Participants Evaluating Simplicity of Use: Injection Easiness
Difficult
0.98 percentage of participants
Percentage of Participants Evaluating Simplicity of Use: Injection Easiness
Very difficult
0.98 percentage of participants

SECONDARY outcome

Timeframe: Week 6

Population: Intent-to-treat (ITT) consists of all participants included in the trial and having signed the informed consent form. For one patient, no data available.

The overall simplicity of use was evaluated at every step of the injection preparation (dose selection, injection and dose modification) and using the time spent in the preparation of the injection. This was evaluated using time intervals: below 5 minutes, 5-10 minutes, 10-20 minutes.

Outcome measures

Outcome measures
Measure
Norditropin NordiFlex®
n=102 Participants
Individually adjusted dose administered with Norditropin NordiFlex® for 6 weeks. Dosage depended on age, weight, etiologies and according to the summary of product characteristics (SPC)
Percentage of Participants Evaluating Simplicity of Use: Time Spent in the Preparation of the Injection
Below 5 minutes
93.1 percentage of participants
Percentage of Participants Evaluating Simplicity of Use: Time Spent in the Preparation of the Injection
5-10 minutes
5.88 percentage of participants
Percentage of Participants Evaluating Simplicity of Use: Time Spent in the Preparation of the Injection
10-20 minutes
0.98 percentage of participants

SECONDARY outcome

Timeframe: Week 6

Population: Intent-to-treat (ITT) consists of all participants included in the trial and having signed the informed consent form.

The person in charge, physician or nurse, of giving all the instruction of use to patients or parents assessed the ease of learning of the Nordiflex® device. Ease of learning was evaluated using ordinal variables (very easy, easy and difficult).

Outcome measures

Outcome measures
Measure
Norditropin NordiFlex®
n=103 Participants
Individually adjusted dose administered with Norditropin NordiFlex® for 6 weeks. Dosage depended on age, weight, etiologies and according to the summary of product characteristics (SPC)
Ease of Learning Assessed by the Physician or the Nurse: Ease of Training
Very easy
72.8 percentage of participants
Ease of Learning Assessed by the Physician or the Nurse: Ease of Training
Easy
26.2 percentage of participants
Ease of Learning Assessed by the Physician or the Nurse: Ease of Training
Difficult
0.97 percentage of participants

SECONDARY outcome

Timeframe: Week 6

Population: Intent-to-treat (ITT) consists of all participants included in the trial and having signed the informed consent form. For three patients, no data available.

The person in charge, physician or nurse, of giving all the instruction of use to patients or parents assessed the ease of learning of the Nordiflex® device. Time to learning was assessed using time intervals: 6-10 minutes, 11-15 minutes, 16-30 minutes and 31 minutes to 1 hour.

Outcome measures

Outcome measures
Measure
Norditropin NordiFlex®
n=100 Participants
Individually adjusted dose administered with Norditropin NordiFlex® for 6 weeks. Dosage depended on age, weight, etiologies and according to the summary of product characteristics (SPC)
Ease of Learning Assessed by the Physician or the Nurse: Time Learning
6-10 minutes
40.0 percentage of participants
Ease of Learning Assessed by the Physician or the Nurse: Time Learning
11-15 minutes
26.0 percentage of participants
Ease of Learning Assessed by the Physician or the Nurse: Time Learning
16-30 minutes
30.0 percentage of participants
Ease of Learning Assessed by the Physician or the Nurse: Time Learning
31 minutes - 1 hour
4.0 percentage of participants

SECONDARY outcome

Timeframe: Week 6

Population: Intent-to-treat (ITT) consists of all participants included in the trial and having signed the informed consent form.

The person in charge, physician or nurse, of giving all the instruction of use to patients or parents assessed the ease of learning of the Nordiflex® device. An added value of device was evaluated by the physician or the nurse using categorical variables.

Outcome measures

Outcome measures
Measure
Norditropin NordiFlex®
n=103 Participants
Individually adjusted dose administered with Norditropin NordiFlex® for 6 weeks. Dosage depended on age, weight, etiologies and according to the summary of product characteristics (SPC)
Ease of Learning Assessed by the Physician or the Nurse: Added Values of the Products
Overall simplicity
82.5 percentage of participants
Ease of Learning Assessed by the Physician or the Nurse: Added Values of the Products
Dose selection
35.0 percentage of participants
Ease of Learning Assessed by the Physician or the Nurse: Added Values of the Products
Maximum available dose
15.5 percentage of participants
Ease of Learning Assessed by the Physician or the Nurse: Added Values of the Products
Dose adjustment
64.1 percentage of participants
Ease of Learning Assessed by the Physician or the Nurse: Added Values of the Products
Easiness of injection
26.2 percentage of participants
Ease of Learning Assessed by the Physician or the Nurse: Added Values of the Products
Improvement of compliance or motivation
27.2 percentage of participants
Ease of Learning Assessed by the Physician or the Nurse: Added Values of the Products
No added values
4.85 percentage of participants

SECONDARY outcome

Timeframe: Week 0

Population: Intent-to-treat (ITT) consists of all participants included in the trial and having signed the informed consent form.

The patient autonomy was evaluated using percentage of participants who performed all the operations of the treatment administration including dose selection, dose modification in case of error and injection.

Outcome measures

Outcome measures
Measure
Norditropin NordiFlex®
n=103 Participants
Individually adjusted dose administered with Norditropin NordiFlex® for 6 weeks. Dosage depended on age, weight, etiologies and according to the summary of product characteristics (SPC)
Patient Autonomy: Percentage of Patients Performing Operations for Treatment Injection
Yes
28.2 percentage of participants
Patient Autonomy: Percentage of Patients Performing Operations for Treatment Injection
No
71.8 percentage of participants

SECONDARY outcome

Timeframe: Week 6

Population: Intent-to-treat (ITT) consists of all participants included in the trial and having signed the informed consent form.

The patient autonomy was evaluated using percentage of participants who performed all the operations of the treatment administration including dose selection, dose modification in case of error and injection.

Outcome measures

Outcome measures
Measure
Norditropin NordiFlex®
n=103 Participants
Individually adjusted dose administered with Norditropin NordiFlex® for 6 weeks. Dosage depended on age, weight, etiologies and according to the summary of product characteristics (SPC)
Patient Autonomy: Percentage of Patients Performing Operations for Treatment Injection
Yes
41.2 percentage of participants
Patient Autonomy: Percentage of Patients Performing Operations for Treatment Injection
No
58.8 percentage of participants

SECONDARY outcome

Timeframe: Week 6

Population: Intent-to-treat (ITT) consists of all participants included in the trial and having signed the informed consent form. For two patients, no data available.

The patient preference was evaluated using the percentage of participants preferring the new system, old system or none of them for their growth hormone therapy.

Outcome measures

Outcome measures
Measure
Norditropin NordiFlex®
n=101 Participants
Individually adjusted dose administered with Norditropin NordiFlex® for 6 weeks. Dosage depended on age, weight, etiologies and according to the summary of product characteristics (SPC)
Patient Preference: Percentage of Participants Preferring System for Continuation of Growth Hormone Treatment
Nordiflex®
64.36 percentage of participants
Patient Preference: Percentage of Participants Preferring System for Continuation of Growth Hormone Treatment
Previous system
34.65 percentage of participants
Patient Preference: Percentage of Participants Preferring System for Continuation of Growth Hormone Treatment
None of them
0.99 percentage of participants

SECONDARY outcome

Timeframe: Weeks 0 - 6

Population: The safety set consisted of all participants included in the trial and having taken at least one dose of trial treatment. The adverse events and technical complaints were collected during the treatment period (6 weeks after inclusion).

Outcome measures

Outcome measures
Measure
Norditropin NordiFlex®
n=103 Participants
Individually adjusted dose administered with Norditropin NordiFlex® for 6 weeks. Dosage depended on age, weight, etiologies and according to the summary of product characteristics (SPC)
Number of Participants Reporting Adverse Events, Medical Events of Special Interest (MESI) and Technical Problems With Devices Used in Trial
Serious adverse events
0 participants
Number of Participants Reporting Adverse Events, Medical Events of Special Interest (MESI) and Technical Problems With Devices Used in Trial
Medical event of special interest (MESI)
0 participants
Number of Participants Reporting Adverse Events, Medical Events of Special Interest (MESI) and Technical Problems With Devices Used in Trial
At least one technical complaint
14 participants
Number of Participants Reporting Adverse Events, Medical Events of Special Interest (MESI) and Technical Problems With Devices Used in Trial
NordiFlex® pen technical complaint
8 participants
Number of Participants Reporting Adverse Events, Medical Events of Special Interest (MESI) and Technical Problems With Devices Used in Trial
PenMate technical complaint
10 participants
Number of Participants Reporting Adverse Events, Medical Events of Special Interest (MESI) and Technical Problems With Devices Used in Trial
Other adverse events greater than or equal to 5%
0 participants

Adverse Events

Norditropin NordiFlex®

Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Public Access to Clinical Trials

Novo Nordisk A/S

Results disclosure agreements

  • Principal investigator is a sponsor employee Novo Nordisk reserves the right to not release data until specified milestones, including the right to not release interim results of clinical trials. At the end of the trial, one or more manuscripts for publication will be prepared collaboratively between Investigator(s) and Novo Nordisk. Novo Nordisk reserves the right to postpone publication and/or communication for less than 60 days to protect intellectual property.
  • Publication restrictions are in place

Restriction type: OTHER