Clinical trial NCT06109935: Special Use-results Surveillance on Long Term Use of Sogroya® in Children With Short Stature Due to Growth Hormone Deficiency Where Epiphysial Discs Are Not Closed
Official title: A Multi-centre, Prospective, Open Label, Single-arm, Non-interventional Post-marketing Study to Investigate the Long-term Safety and Clinical Parameters of Sogroya® Treatment in Children With Short Stature Due to Growth Hormone Deficiency Where Epiphysial Discs Are Not Closed Under Normal Clinical Practice Conditions in Japan Special Use-results Surveillance on Long Term Use of Sogroya® in Children With Short Stature Due to Growth Hormone Deficiency (GHD) Where Epiphysial Discs Are Not Closed
Study type: OBSERVATIONAL
Overall status: ENROLLING_BY_INVITATION
Conditions: Growth Hormone Deficiency in Children
Lead sponsor: Novo Nordisk A/S
Sponsor class: INDUSTRY
First posted: 2023-10-31
Enrollment: 200
Sex: ALL
Minimum age (years): 0
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Children with short stature due to Growth Hormone Deficiency (GHD) where epiphysial discs are not closed under normal clinical practice conditions in Japan.

Brief summary: The purpose of the study is to investigate the safety and effectiveness of Sogroya® in children with short stature due to growth hormone deficiency where epiphysial discs are not closed under real-world clinical practice in Japan. The study will last for about 1 year (at shortest) to 3 years (at longest) depending on when the participant takes part in the study. The participant will be asked to answer questionnaire(s) about how they feel about the growth hormone (GH) product treatment once during the study (at about 3 months after starting the Sogroya® treatment) and about 3 months after starting the Sogroya® treatment.


Eligibility criteria: Inclusion Criteria:

1. Signed consent obtained before any study-related activities (study-related activities are any procedure related to recording of data according to the protocol).
2. The decision to initiate treatment with commercially available Sogroya® 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. Both GH treatment naïve and non-naïve children are eligible.
3. Male or female age 0 to 18 years (exclusive) at the time of signing informed consent.
4. Diagnosis with short stature due to GHD where epiphysial discs are not closed according to local normal clinical practice.

Exclusion Criteria:

1. Previous participation in this study. Participation is defined as having given informed consent in this study.
2. Treatment with any investigational drug within 30 days prior to baseline (the starting date of Sogroya® treatment).
3. Mental incapacity, unwillingness or language barriers precluding adequate understanding or cooperation.
4. Contraindication described in approved product labelling in Japan.

   1. Patients with hypersensitivity to the active substance or to any of the excipients
   2. Patients with malignant tumour
   3. Female patients who are either pregnant or likely to be pregnant

Primary outcomes:
measure: Number of adverse reactions (AR)
timeFrame: From baseline (week 0) to end of study (up to 156 weeks)
Secondary outcomes:
measure: Number of adverse events (AEs)
timeFrame: From baseline (week 0) to end of study (up to 156 weeks)
measure: Number of serious adverse events (SAEs)
timeFrame: From baseline (week 0) to end of study (up to 156 weeks)
measure: Number of serious adverse reactions (SARs)
timeFrame: From baseline (week 0) to end of study (up to 156 weeks)
measure: Change in height velocity (HV)
timeFrame: Every 12 months from baseline (week 0) to end of study (up to 156 weeks)
measure: Change in bone age
timeFrame: Every 12 months from baseline (week 0) to end of study (up to 156 weeks)
measure: Change in ratio of bone age/chronological age
timeFrame: Every 12 months from baseline (week 0) to end of study (up to 156 weeks)
measure: Change in height standard deviation score (HSDS)
timeFrame: Every 12 months from baseline (week 0) to end of study (up to 156 weeks)
measure: Change in height velocity standard deviation score (HVSDS)
timeFrame: Every 12 months from baseline (week 0) to end of study (up to 156 weeks)
measure: Change in insulin-like growth factor-I standard deviation score (IGF-I SDS)
timeFrame: Every 12 months from baseline (week 0) to end of study (up to 156 weeks)
measure: Growth hormone device assessment tool (G-DAT)
timeFrame: At 12 weeks
measure: Growth hormone patient preference questionnaire (GH-PPQ)
timeFrame: At 12 weeks
Other outcomes:
Locations:
city: Asahikawa, Hokkaido
country: Japan
facility: Asahikawa Medical Univ. Hospital, Pediatrics
city: Beppu-shi, Oita-ken
country: Japan
facility: Beppu Medical Center
city: Fukuoka-shi, Fukuoka-ken
country: Japan
facility: Fukuoka University Hospital
city: Fukuoka-shi, Fukuoka
country: Japan
facility: Kyushu University Hospital
city: Fukuoka
country: Japan
facility: Kurume University Hospital, Pediatrics
city: Gifu
country: Japan
facility: Gifu University Hospital
city: Gunma
country: Japan
facility: Ota Memorial Hospital_Pediatrics
city: Hachioji-shi, Tokyo-to
country: Japan
facility: Kato Clinic
city: Hamamatsu-shi, Shizuoka
country: Japan
facility: Hamamatsu University Hospital
city: Higashimatsuyama-shi, Saitama-ken
country: Japan
facility: Hello Clinic
city: Himeji-shi, Hyogo-ken
country: Japan
facility: Japanese Red Cross Society Himeji Hospital
city: Hyogo
country: Japan
facility: Hyogo prefectural kobe children's hospital
city: Ichikawa-shi, Chiba-ken
country: Japan
facility: Sunsun Kodomo Clinic
city: Iwaki-shi, Fukushima-ken
country: Japan
facility: Fukushima Seishi Ryougoen
city: Iwate
country: Japan
facility: Iwate Medical University Hospital
city: Izumo-shi, Shimane-ken
country: Japan
facility: Shimane University Hospital_Pediatrics
city: Kamigyo-ku, Kyoto
country: Japan
facility: University Hospital Kyoto Prefectual University of Medicine
city: Kanagawa
country: Japan
facility: Odawara Municipal Hospital
city: Kawasaki-shi, Kanagawa-ken
country: Japan
facility: Nippon Medical School Musashikosugi Hospital
city: Kitakyusyu-shi, Fukuoka
country: Japan
facility: Hospital of the University of Occupational And Environmental Health Japan, Pediatrics
city: Kyoto
country: Japan
facility: Ayabe City Hospital
city: Moka-shi, Tochigi-ken
country: Japan
facility: Haga Red Cross Hospital
city: Morioka-shi, Iwate-ken
country: Japan
facility: Kodomohamirai Morioka Kodomo Clinic
city: Nagoya, Aichi
country: Japan
facility: Nagoya City University Hospital, Pediatric
city: Naha-shi, Okinawa
country: Japan
facility: Naha City Hospital
city: Nakagami-gun, Okinawa-ken
country: Japan
facility: University of the Ryukyus Hospital
city: Nara-shi, Nara
country: Japan
facility: Nara Prefecture General Medical Center
city: Nara
country: Japan
facility: Nara Medical University Hospital_Pediatrics
city: Nishinomiya-shi, Hyogo-ken
country: Japan
facility: Hyogo Medical University Hospital_Pediatrics
city: Obu-shi, Aichi
country: Japan
facility: Aichi Children's Health and Medical Center
city: Okinawa
country: Japan
facility: Chibana Clinic
city: Oosakashi
country: Japan
facility: Kibounomori Clinic
city: Osaka
country: Japan
facility: Osaka City General Hospital
city: Osaka
country: Japan
facility: Saiseikai Suita Hospital
city: Osaka
country: Japan
facility: Osaka University Hospital
city: Osaka
country: Japan
facility: Yasuhara Children's Clinic
city: Osaka
country: Japan
facility: Osaka Women's and Children's Hospital
city: Ota-ku, Tokyo-to
country: Japan
facility: Kojiya Kodomo clinic
city: Ozu-shi, Ehime-ken
country: Japan
facility: Okubo Kids Clinic
city: Sashima-gun, Ibaraki-ken
country: Japan
facility: Sakai Smile Kids Clinic
city: Sendai-shi, Miyagi-ken
country: Japan
facility: JR Sendai Hospital
city: Shizuoka
country: Japan
facility: Hamamatsu Medical Center
city: Tokyo
country: Japan
facility: Tanaka Growth Clinic
city: Toon-shi, Ehime
country: Japan
facility: Ehime University Hospital
city: Toyama-shi, Toyama-ken
country: Japan
facility: Murakami Pediatric and Allergy clinic
city: Toyohashi-shi, Aichi-ken
country: Japan
facility: Toyohashi Municipal Hospital
city: Yamagata-shi, Yamagata-ken
country: Japan
facility: Ikeda Pediatrics Clinic
Study officials:
affiliation: Novo Nordisk A/S
name: Clinical Transparency dept. 2834
role: STUDY_DIRECTOR
References: