Genetic DisorderNCT00705172
Retrospective Observational Study on Efficacy and Safety of Norditropin® in Children With Prader-Willi Syndrome
An observational study of Genetic Disorder and Prader-Willi Syndrome, run by Novo Nordisk A/S at 3 sites in 3 countries.
Trial summary
This study is conducted in Europe. The aim of this observational study is to collect data from children with Prader-Willi Syndrome, who have been treated off-label with Norditropin® for more than 12 months to seek approval for Norditropin® treatment with Prader-Willi Syndrome. Read more about this study
- Who is it for?
- Ages Up to 15 years
- Conditions the study is for
- Genetic Disorder, Prader-Willi Syndrome
- What is being tested?
- somatropin
- How many people
- 41 participants
- Where
- 3 sitesin 3 countries
- Study dates
- Nov 2008to Nov 2008
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 15 years
- All sexes
- No healthy volunteers
- Child
Study population: Children with Prader-Willi Syndrome
Who may be able to join
- Informed consent obtained before any trial-related activities
- Genetically diagnosed Prader-Willi Syndrome
- Received at least one dose of Norditropin® treatment
- Pre-pubertal at start of treatment; assessed by Tanner stage 1, or testicular volume below 4ml (according to Tanner 1976)
Who cannot join
- Pre-treatment with other Growth Hormone preparation prior to treatment with Norditropin®
Trial locations and contacts
3 sites in 3 countries.
3 sites in 3 countries
About this study
Official title: Efficacy and Safety of Norditropin® (Somatropin) in Children With Prader-Willi Syndrome (PWS)
This study is conducted in Europe. The aim of this observational study is to collect data from children with Prader-Willi Syndrome, who have been treated off-label with Norditropin® for more than 12 months to seek approval for Norditropin® treatment with Prader-Willi Syndrome.
- Conditions
- Genetic Disorder, Prader-Willi Syndrome
- Being tested
- somatropin
- Medical subject headings
- Genetic Diseases, Inborn, Prader-Willi Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
somatropinDrug
Also called Norditropin®
Prader-Willi syndrome children treated with at least one dose of Norditropin®
| Study group | Role | What they receive |
|---|---|---|
| A | Not described Receives: Drug: somatropin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Primary objective is to investigate changes in height Standard Deviation Score (SDS)
Measured: in response to 12 months Norditropin® treatment in children with PWS (referenced to PWS population
Secondary outcomes (8)
Changes in height SDS from start of treatment to last observation during Norditropin treatment (referenced to PWS population1)
Measured: at 12 months- and at last observation during Norditropin treatment
Change in body composition (DEXA, Bio impedance or stable isotope dilution)
Measured: at 12 months- and at last observation during Norditropin treatment
Height velocity (HV) and change in HV
Measured: at 12 months- and at last observation during Norditropin treatment
Glycated Fraction of Haemoglobin (HbA1c)
Measured: at 12 months- and at last observation during Norditropin treatment
Insulin-Like Growth Factor-I (IGF-I)
Measured: at 12 months- and at last observation during Norditropin treatment
Haematology
Measured: at 12 months- and at last observation during Norditropin treatment
Thyroid-stimulating hormone (TSH) and active form of free thyroxin
Measured: at 12 months- and at last observation during Norditropin treatment
Adverse Events.
Measured: at 12 months- and at last observation during Norditropin treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Jun 2008 (estimated)
- Study startNov 2008
- Primary completionNov 2008
- Study completionNov 2008
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Genetic DisorderStudies of the same condition, open and under way.
- More trials run by Novo Nordisk A/SThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Retrospective
- Enrolment
- 41 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novo Nordisk A/S
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Global Clinical Registry (GCR, 1452), Study director, Novo Nordisk A/S
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00705172
- Sponsor's protocol ID
- GHLIQUID-1961
- Status last verified
- Oct 2023
- First submitted
- 24 Jun 2008
- Last updated
- 2 Nov 2023
- Results posted
- No
- Data monitoring committee
- No
Publications
Meinhardt U, Christiansen JS, Farholt S, Lammer C, Ostergaard JR, Schmidt F, Kappelgaard AM, Eiholzer U. The efficacy and safety of long-term Norditropin(R) treatment in children with Prader-Willi syndrome. Horm Metab Res. 2013 Jul;45(7):532-6. doi: 10.1055/s-0033-1343449. Epub 2013 Apr 30.
PubMed 23632904
Study at a glance
From the registry record, last updated by the sponsor on 2 Nov 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy and Safety of Norditropin® (Somatropin) in Children With Prader-Willi Syndrome (PWS)
- ClinicalTrials.gov ID
- NCT00705172
- Acronym / sponsor ID
- GHLIQUID-1961
- Conditions
- Genetic Disorder, Prader-Willi Syndrome
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 41 participants (actual)
- Lead sponsor
- Novo Nordisk A/SIndustry (pharma, biotech, devices)
- Study start
- Nov 2008
- Primary completion
- Nov 2008 (actual)
- Study completion
- Nov 2008 (actual)
- First posted
- 25 Jun 2008 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study is conducted in Europe. The aim of this observational study is to collect data from children with Prader-Willi Syndrome, who have been treated off-label with Norditropin® for more than 12 months to seek approval for Norditropin® treatment with Prader-Willi Syndrome.
Study design
- Observational model
- Other
- Time perspective
- Retrospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| A | – | Drug: somatropin |
Who may qualify
- Ages
- Up to 15 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- Children with Prader-Willi Syndrome
Key inclusion criteria (4 listed)
- Informed consent obtained before any trial-related activities
- Genetically diagnosed Prader-Willi Syndrome
- Received at least one dose of Norditropin® treatment
- Pre-pubertal at start of treatment; assessed by Tanner stage 1, or testicular volume below 4ml (according to Tanner 1976)
Key exclusion criteria (1 listed)
- Pre-treatment with other Growth Hormone preparation prior to treatment with Norditropin®
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| somatropinAlso: Norditropin®Prader-Willi syndrome children treated with at least one dose of Norditropin® | Drug | A |
Primary outcome
Primary objective is to investigate changes in height Standard Deviation Score (SDS)
Time frame: in response to 12 months Norditropin® treatment in children with PWS (referenced to PWS population
8 secondary outcomes
Changes in height SDS from start of treatment to last observation during Norditropin treatment (referenced to PWS population1)
Time frame: at 12 months- and at last observation during Norditropin treatment
Change in body composition (DEXA, Bio impedance or stable isotope dilution)
Time frame: at 12 months- and at last observation during Norditropin treatment
Height velocity (HV) and change in HV
Time frame: at 12 months- and at last observation during Norditropin treatment
Glycated Fraction of Haemoglobin (HbA1c)
Time frame: at 12 months- and at last observation during Norditropin treatment
Insulin-Like Growth Factor-I (IGF-I)
Time frame: at 12 months- and at last observation during Norditropin treatment
Haematology
Time frame: at 12 months- and at last observation during Norditropin treatment
Thyroid-stimulating hormone (TSH) and active form of free thyroxin
Time frame: at 12 months- and at last observation during Norditropin treatment
Adverse Events.
Time frame: at 12 months- and at last observation during Norditropin treatment
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Nov 2023.
- Derived from this study: PubMed 23632904Meinhardt U, Christiansen JS, Farholt S, Lammer C, Ostergaard JR, Schmidt F, Kappelgaard AM, Eiholzer U. The efficacy and safety of long-term Norditropin(R) treatment in children with Prader-Willi syndrome. Horm Metab Res. 2013 Jul;45(7):532-6. doi: 10.1055/s-0033-1343449. Epub 2013 Apr 30.
- Clinical Trials at Novo NordiskLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Global Clinical Registry (GCR, 1452) study director
Novo Nordisk A/S
3 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Genetic Disorder and Prader-Willi Syndrome, run by Novo Nordisk A/S at 3 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 2 Nov 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in Denmark, Germany, Switzerland. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Primary objective is to investigate changes in height Standard Deviation Score (SDS) (time frame: in response to 12 months Norditropin® treatment in children with PWS (referenced to PWS population). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00705172, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Nov 2023.