Genetic DisorderNCT03435627
Post Marketing Surveillance on Long-term Use With Norditropin® (Short Stature Due to Noonan Syndrome)
An observational study of Genetic Disorder and Noonan Syndrome, run by Novo Nordisk A/S at 22 sites in Japan.
Trial summary
The purpose of this study is to collect information about safety and effectiveness for long term use of Norditropin®. Participants will attend the medical institution according to usual practice and receive medical care, as agreed with the study doctor. Read more about this study
- Who is it for?
- Ages 3 years and older
- Conditions the study is for
- Genetic Disorder, Noonan Syndrome
- What is being tested?
- Somatropin
- How many people
- 71 participants
- Where
- 22 sitesJapan
- Study dates
- 26 Feb 2018to 18 Jan 2022
- 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 3 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: A total of 60 patients (consisting of non-naïve patients who were previously enrolled in study: GHLIQUID-4020 and naïve patients enrolled after approval of the Noonan indication in Japan) are planned to be included in this study.
Who may be able to join
- Signed informed consent obtained before any study-related activities (study-related activities are any procedure related to recording of data according to the protocol).
- The decision to initiate treatment with commercially available Norditropin® has been made by the patient/Legally Acceptable Representative (LAR) and the treating physician before and independently from the decision to include the patient in this study.
- For non-naïve patients; patients who were previously enrolled in study: GHLIQUID-4020.
- For naïve patients; short stature due to Noonan syndrome diagnosed by the physician and a decision to initiate treatment with Norditropin® has been made by the patient/parent and the physician. At study sites, all patients will be registered consecutively from the first patient after approval date (consecutively registered system).
- Male or female, 3 years old or over, bone age: less than 17 years old for male / less than 15 years old for female.
Who cannot join
- Previous participation in this study. Participation is defined as having given informed consent in this study.
- Known or suspected allergy to study products or related products.
- In case of naïve patients, patients who have received growth hormone (GH) products for treatment of indication other than short stature due to Noonan syndrome before approval date of Noonan indication.
Trial locations and contacts
22 sites in 1 country.
22 sites in 1 country
Japan22 sites
Novo Nordisk Investigational Site
Asahikawa, Hokkaido, 078-8510
Novo Nordisk Investigational Site
Fukuoka, 830-0011
Novo Nordisk Investigational Site
Kanagawa, 216-8511
Novo Nordisk Investigational Site
Kanagawa, 232-8555
Novo Nordisk Investigational Site
Kyoto, 602-8566
Novo Nordisk Investigational Site
Kyoto, 629-2261
Novo Nordisk Investigational Site
Maebashi-shi, Gunma, 371-8511
Novo Nordisk Investigational Site
Miyazaki, 889-1692
Novo Nordisk Investigational Site
Nagoya, Aichi, 467-8602
Novo Nordisk Investigational Site
Niigata-shi, Niigata, 951 8520
Novo Nordisk Investigational Site
Ōita, 879-5593
Novo Nordisk Investigational Site
Osaka, 534-0021
Novo Nordisk Investigational Site
Osaka, 594-1101
Novo Nordisk Investigational Site
Saitama-shi, Saitama, 330-8777
Novo Nordisk Investigational Site
Sapporo, Hokkaido, 065-8611
Novo Nordisk Investigational Site
Sendai-shi, Miyagi, 980 8574
Novo Nordisk Investigational Site
Shizuoka, 431-3192
Novo Nordisk Investigational Site
Tochigi, 329-0498
Novo Nordisk Investigational Site
Tokyo, 157 8535
Novo Nordisk Investigational Site
Tokyo, 160-8582
Novo Nordisk Investigational Site
Tokyo, 162-8666
Novo Nordisk Investigational Site
Tokyo, 183-8561
About this study
The purpose of this study is to collect information about safety and effectiveness for long term use of Norditropin®. Participants will attend the medical institution according to usual practice and receive medical care, as agreed with the study doctor.
- Conditions
- Genetic Disorder, Noonan Syndrome
- Being tested
- Somatropin
- Medical subject headings
- Genetic Diseases, Inborn, Noonan Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SomatropinDrug
Participants will be treated with commercially available Norditropin® (somatropin) according to routine clinical practice at the discretion of the treating physician.
| Study group | Role | What they receive |
|---|---|---|
| Norditropin® (naïve participants) | The treatment period of Norditropin® for naïve participants will be up to 208 weeks. Receives: Drug: Somatropin | |
| Norditropin® (non-naïve participants) | The treatment period of Norditropin® for non-naïve participants will be up to 442 weeks. Receives: Drug: Somatropin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of adverse drug reactions (ADR)
Count of events
Measured: Weeks 0-208
Secondary outcomes (43)
Number of serious adverse drug reaction (SADR)
Count of events
Measured: Week 0-208
Number of serious adverse event (SAE)
Count of events
Measured: Week 0-208
Number of cardiac adverse event (AE)
Count of events
Measured: Week 0-208
Change in insulin like growth factor I (IGF-I) for non-naïve patients
Measured in ng/mL
Measured: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Change in insulin like growth factor I (IGF-I) for naïve patients
Measured in ng/mL
Measured: Weeks 0, 52, 104, 156, and 208 weeks
Change in insulin like growth factor I standard deviation score (IGF-I SDS) for non-naïve patients
Presented as standard deviation (SD) score
Measured: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Change in insulin like growth factor I standard deviation score (IGF-I SDS) for naïve patients
Presented as SD score
Measured: Weeks 0, 52, 104, 156, and 208 weeks
Change in haemoglobin A1c (HbA1c) for non-naïve patients
Measured in %
Measured: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Change in haemoglobin A1c (HbA1c) for naïve patients
Measured in %
Measured: Weeks 0, 52, 104, 156, and 208 weeks
Change in aspartate aminotransferase (AST) for non-naïve patients
Measured in IU/L
Measured: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Change in aspartate aminotransferase (AST) for naïve patients
Measured in IU/L
Measured: Weeks 0, 52, 104, 156, and 208 weeks
Change in alanine aminotransferase (ALT) for non-naïve patients
Measured in IU/L
Measured: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Change in alanine aminotransferase (ALT) for naïve patients
Measured in IU/L
Measured: Weeks 0, 52, 104, 156, and 208 weeks
Change in total cholesterol (T-CHO) for non-naïve patients
Measured in mg/dL
Measured: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Change in total cholesterol (T-CHO) for naïve patients
Measured in mg/dL
Measured: Weeks 0, 52, 104, 156, and 208 weeks
Change in high-density lipoprotein cholesterol (HDL-C) for non-naïve patients
Measured in mg/dL
Measured: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Change in high-density lipoprotein cholesterol (HDL-C) for naïve patients
Measured in mg/dL
Measured: Weeks 0, 52, 104, 156, and 208 weeks
Change in Low-density lipoprotein cholesterol (LDL-C) for non-naïve patients
Measured in mg/dL
Measured: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Change in Low-density lipoprotein cholesterol (LDL-C) for naïve patients
Measured in mg/dL
Measured: Weeks 0, 52, 104, 156, and 208 weeks
Change in triglyceride (TG) for non-naïve patients
Measured in mg/dL
Measured: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Change in triglyceride (TG) for naïve patients
Measured in mg/dL
Measured: Weeks 0, 52, 104, 156, and 208 weeks
Change in thyroid stimulation hormone(TSH) for non-naïve patients
Measured in μU/mL
Measured: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Change in thyroid stimulation hormone(TSH) for naïve patients
Measured in μU/mL
Measured: Weeks 0, 52, 104, 156, and 208 weeks
Change in free triiodothyronine (FT-3) for non-naïve patients
Measured in pg/mL
Measured: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Change in free triiodothyronine (FT-3) for naïve patients
Measured in pg/mL
Measured: Weeks 0, 52, 104, 156, and 208 weeks
Change in free thyroxine (FT-4) for non-naïve patients
Measured in ng/mL
Measured: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Change in free thyroxine (FT-4) for naïve patients
Measured in ng/mL
Measured: Weeks 0, 52, 104, 156, and 208 weeks
Change in white blood cell (WBC) for non-naïve patients
Measured in μ/L
Measured: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Change in white blood cell (WBC) for naïve patients
Measured in μ/L
Measured: Weeks 0, 52, 104, 156, and 208 weeks
Change in platelet (PLT) for non-naïve patients
Measured in 10^4/μL
Measured: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Change in platelet (PLT) for naïve patients
Measured in 10^4/μL
Measured: Weeks 0, 52, 104, 156, and 208 weeks
Change in bone age (BA) for non-naïve patients
Measured in year
Measured: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Change in bone age (BA) for naïve patients
Measured in year
Measured: Weeks 0, 52, 104, 156, and 208 weeks
Change in bone age/chronological age (BA/CA) for non-naïve patients
Presented as ratio
Measured: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Change in bone age/chronological age (BA/CA) for naïve patients
Presented as ratio
Measured: Weeks 0, 52, 104, 156, and 208 weeks
Change in electrocardiogram (ECG) for non-naïve patients
Assessment of change in ECG is measured by categories recorded in case report forms (CRFs): normal, abnormal not clinically significant and abnormal clinically significant.
Measured: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Change in electrocardiogram (ECG) for naïve patients
Assessment of change in ECG is measured by categories recorded in CRFs: normal, abnormal not clinically significant and abnormal clinically significant.
Measured: Weeks 0, 52, 104, 156, and 208 weeks
Change in height standard deviation score (HSDS) for non-naïve patients
Presented as SD score
Measured: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Change in height standard deviation score (HSDS) for naïve patients
Presented as SD score
Measured: Weeks 0, 52, 104, 156, and 208 weeks
Height velocity standard deviation score (HVSDS) for non-naïve patients
Presented as SD score
Measured: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Height velocity standard deviation score (HVSDS) for naïve patients
Presented as SD score
Measured: Weeks 0, 52, 104, 156, and 208 weeks
Height velocity (HV) for non-naïve patients
Measured in cm/year
Measured: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Height velocity (HV) for naïve patients
Measured in cm/year
Measured: Weeks 0, 52, 104, 156, and 208 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Feb 2018
- Study start26 Feb 2018
- Primary completion18 Jan 2022
- Study completion18 Jan 2022
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
- Cohort
- Time perspective
- Prospective
- Enrolment
- 71 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novo Nordisk A/S
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Reporting Anchor and Disclosure (1452), Study director, Novo Nordisk A/S
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03435627
- Sponsor's protocol ID
- GHLIQUID-4358
- Other IDs
- U1111-1191-3084
- Status last verified
- Nov 2022
- First submitted
- 31 Jan 2018
- Last updated
- 14 Nov 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: According to the Novo Nordisk disclosure commitment on novonordisk-trials.com
Publications
Muroya K, Kawai M, Yamagishi H, Endo T, Pietropoli A, Ferran JM, Horikawa R. Long-term effectiveness and safety of daily growth hormone therapy in Japanese children with Noonan syndrome: a post-marketing surveillance study. Endocr J. 2026 Feb 2;73(2):229-241. doi: 10.1507/endocrj.EJ25-0116. Epub 2025 Nov 22.
PubMed 41285480
Study at a glance
From the registry record, last updated by the sponsor on 14 Nov 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Post Marketing Surveillance on Long-term Use With Norditropin® (Short Stature Due to Noonan Syndrome)
- ClinicalTrials.gov ID
- NCT03435627
- Acronym / sponsor ID
- GHLIQUID-4358
- Conditions
- Genetic Disorder, Noonan Syndrome
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 71 participants (actual)
- Lead sponsor
- Novo Nordisk A/SIndustry (pharma, biotech, devices)
- Study start
- 26 Feb 2018 (actual)
- Primary completion
- 18 Jan 2022 (actual)
- Study completion
- 18 Jan 2022 (actual)
- First posted
- 19 Feb 2018 (actual)
- 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.
The purpose of this study is to collect information about safety and effectiveness for long term use of Norditropin®. Participants will attend the medical institution according to usual practice and receive medical care, as agreed with the study doctor.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Norditropin® (naïve participants) | – | Drug: SomatropinThe treatment period of Norditropin® for naïve participants will be up to 208 weeks. |
| Norditropin® (non-naïve participants) | – | Drug: SomatropinThe treatment period of Norditropin® for non-naïve participants will be up to 442 weeks. |
Who may qualify
- Ages
- 3 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- A total of 60 patients (consisting of non-naïve patients who were previously enrolled in study: GHLIQUID-4020 and naïve patients enrolled after approval of the Noonan indication in Japan) are planned to be included in this study.
Key inclusion criteria (5 listed)
- Signed informed consent obtained before any study-related activities (study-related activities are any procedure related to recording of data according to the protocol).
- The decision to initiate treatment with commercially available Norditropin® has been made by the patient/Legally Acceptable Representative (LAR) and the treating physician before and independently from the decision to include the patient in this study.
- For non-naïve patients; patients who were previously enrolled in study: GHLIQUID-4020.
- For naïve patients; short stature due to Noonan syndrome diagnosed by the physician and a decision to initiate treatment with Norditropin® has been made by the patient/parent and the physician. At study sites, all patients will be registered consecutively from the first patient after approval date (consecutively registered system).
- Male or female, 3 years old or over, bone age: less than 17 years old for male / less than 15 years old for female.
Key exclusion criteria (3 listed)
- Previous participation in this study. Participation is defined as having given informed consent in this study.
- Known or suspected allergy to study products or related products.
- In case of naïve patients, patients who have received growth hormone (GH) products for treatment of indication other than short stature due to Noonan syndrome before approval date of Noonan indication.
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 |
|---|---|---|
| SomatropinParticipants will be treated with commercially available Norditropin® (somatropin) according to routine clinical practice at the discretion of the treating physician. | Drug | Norditropin® (naïve participants), Norditropin® (non-naïve participants) |
Primary outcome
Number of adverse drug reactions (ADR)
Time frame: Weeks 0-208
Count of events
43 secondary outcomes
Number of serious adverse drug reaction (SADR)
Time frame: Week 0-208
Number of serious adverse event (SAE)
Time frame: Week 0-208
Number of cardiac adverse event (AE)
Time frame: Week 0-208
Change in insulin like growth factor I (IGF-I) for non-naïve patients
Time frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Change in insulin like growth factor I (IGF-I) for naïve patients
Time frame: Weeks 0, 52, 104, 156, and 208 weeks
Change in insulin like growth factor I standard deviation score (IGF-I SDS) for non-naïve patients
Time frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Change in insulin like growth factor I standard deviation score (IGF-I SDS) for naïve patients
Time frame: Weeks 0, 52, 104, 156, and 208 weeks
Change in haemoglobin A1c (HbA1c) for non-naïve patients
Time frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Change in haemoglobin A1c (HbA1c) for naïve patients
Time frame: Weeks 0, 52, 104, 156, and 208 weeks
Change in aspartate aminotransferase (AST) for non-naïve patients
Time frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Change in aspartate aminotransferase (AST) for naïve patients
Time frame: Weeks 0, 52, 104, 156, and 208 weeks
Change in alanine aminotransferase (ALT) for non-naïve patients
Time frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Change in alanine aminotransferase (ALT) for naïve patients
Time frame: Weeks 0, 52, 104, 156, and 208 weeks
Change in total cholesterol (T-CHO) for non-naïve patients
Time frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Change in total cholesterol (T-CHO) for naïve patients
Time frame: Weeks 0, 52, 104, 156, and 208 weeks
Change in high-density lipoprotein cholesterol (HDL-C) for non-naïve patients
Time frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Change in high-density lipoprotein cholesterol (HDL-C) for naïve patients
Time frame: Weeks 0, 52, 104, 156, and 208 weeks
Change in Low-density lipoprotein cholesterol (LDL-C) for non-naïve patients
Time frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Change in Low-density lipoprotein cholesterol (LDL-C) for naïve patients
Time frame: Weeks 0, 52, 104, 156, and 208 weeks
Change in triglyceride (TG) for non-naïve patients
Time frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Change in triglyceride (TG) for naïve patients
Time frame: Weeks 0, 52, 104, 156, and 208 weeks
Change in thyroid stimulation hormone(TSH) for non-naïve patients
Time frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Change in thyroid stimulation hormone(TSH) for naïve patients
Time frame: Weeks 0, 52, 104, 156, and 208 weeks
Change in free triiodothyronine (FT-3) for non-naïve patients
Time frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Change in free triiodothyronine (FT-3) for naïve patients
Time frame: Weeks 0, 52, 104, 156, and 208 weeks
Change in free thyroxine (FT-4) for non-naïve patients
Time frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Change in free thyroxine (FT-4) for naïve patients
Time frame: Weeks 0, 52, 104, 156, and 208 weeks
Change in white blood cell (WBC) for non-naïve patients
Time frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Change in white blood cell (WBC) for naïve patients
Time frame: Weeks 0, 52, 104, 156, and 208 weeks
Change in platelet (PLT) for non-naïve patients
Time frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Change in platelet (PLT) for naïve patients
Time frame: Weeks 0, 52, 104, 156, and 208 weeks
Change in bone age (BA) for non-naïve patients
Time frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Change in bone age (BA) for naïve patients
Time frame: Weeks 0, 52, 104, 156, and 208 weeks
Change in bone age/chronological age (BA/CA) for non-naïve patients
Time frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Change in bone age/chronological age (BA/CA) for naïve patients
Time frame: Weeks 0, 52, 104, 156, and 208 weeks
Change in electrocardiogram (ECG) for non-naïve patients
Time frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Change in electrocardiogram (ECG) for naïve patients
Time frame: Weeks 0, 52, 104, 156, and 208 weeks
Change in height standard deviation score (HSDS) for non-naïve patients
Time frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Change in height standard deviation score (HSDS) for naïve patients
Time frame: Weeks 0, 52, 104, 156, and 208 weeks
Height velocity standard deviation score (HVSDS) for non-naïve patients
Time frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
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 14 Nov 2022.
- Derived from this study: PubMed 41285480Muroya K, Kawai M, Yamagishi H, Endo T, Pietropoli A, Ferran JM, Horikawa R. Long-term effectiveness and safety of daily growth hormone therapy in Japanese children with Noonan syndrome: a post-marketing surveillance study. Endocr J. 2026 Feb 2;73(2):229-241. doi: 10.1507/endocrj.EJ25-0116. Epub 2025 Nov 22.
- Individual participant dataSharing planned: Yes. According to the Novo Nordisk disclosure commitment on novonordisk-trials.com
Related trials for Genetic Disorder
Active, not recruitingPhase 1/2
Selumetinib in Treating Young Patients With Recurrent or Refractory Low Grade Glioma
Low Grade Glioma, Recurrent Childhood Pilocytic Astrocytoma
Recruiting
Methylation Profiling in Self-Samples for Identification of (Epi)Genetic Biomarkers for Gynecological Cancers
Gynaecological Cancers, Ovarian Cancer
Not yet recruitingPhase 1
A Phase I Study of GLR2053 Tablets in Chinese Healthy Adult Participants
Atopic Dermatitis
RecruitingPhase 1/2
A First-in-human Study of KK8123 in Adults With X-linked Hypophosphatemia
X-linked Hypophosphatemia
RecruitingObservational study
Blood, Urine, and Tissue Collection for Cutaneous Lymphoma, Eczema, and Atopic Dermatitis Research
Cutaneous T-cell Lymphoma, Sezary Syndrome
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.
Clinical Reporting Anchor and Disclosure (1452) study director
Novo Nordisk A/S
22 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Genetic Disorder and Noonan Syndrome, run by Novo Nordisk A/S at 22 sites in Japan.
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 5 inclusion and 3 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 14 Nov 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 22 sites in Japan. 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?
Number of adverse drug reactions (ADR) (time frame: Weeks 0-208). 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 NCT03435627, 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 14 Nov 2022.