A Single Arm, Multicenter Phase III Clinical Trial to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone Injection in Patients With Prader-Willi Syndrome

A Study to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone Injection in Patients With Prader-Willi Syndrome



Sponsors


Source

GeneScience Pharmaceuticals Co., Ltd.

Oversight Info

Has Dmc

No

Is Fda Regulated Drug

No

Is Fda Regulated Device

No


Brief Summary

To evaluate the effectiveness of rhGH (Recombinant human growth hormone) injection for
improving motor development in patients with PWS.

Overall Status

Recruiting

Start Date

2018-04-14

Completion Date

2020-01-01

Primary Completion Date

2020-01-01

Phase

Phase 3

Study Type

Interventional

Primary Outcome

Measure

Time Frame

The change of the total motor quotient calculated by Peabody Developmental Motor Scale before and after treatment
Baseline, 26 weeks, 52 weeks

Secondary Outcome

Measure

Time Frame

Gross motor quotient and fine motor quotient calculated by Peabody Developmental Motor Scale before and after treatment
Baseline, 26 weeks, 52 weeks
Change of height standard deviation score (SDS) by chronological age before and after treatment
Baseline, 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks
Body weight change before and after treatment
Baseline, 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks
Change of BMI (Body mass index) standard deviation score before and after treatment
Baseline, 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks
Global quotient, locomotor quotient, personal-social development quotient, language quotient, eye and hand coordination quotient, performance quotient and practical reasoning change calculated by Griffiths Mental Development Scale
Baseline, 26 weeks, 52 weeks
Bone maturation ( bone age/ chronological age: BA/CA)
Baseline, 52 weeks
Change of IGF-1(Insulin-like growth factor 1) SDS
Baseline, 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks
IGF-1/IGFBP-3 molar ratio
Baseline, 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks

Enrollment

30

Condition


Intervention

Intervention Type

Drug

Intervention Name


Description

Drug: Recombinant Human Growth Hormone Injection /Jintropin AQ, 30IU/10 mg/3ml/kit, 0.5 mg/m2/d for the first 4 weeks, then 1.0 mg/m2/d for subsequent 48 weeks; by subcutaneous injection, once per day for total 52 weeks.

Arm Group Label

rhGH injection/Jintropin AQ


Eligibility

Criteria

Inclusion Criteria:

- 1.Signed informed consent from legal guardian of the subjects;

- Subjects are willing and able to cooperate to complete scheduled visits, treatment
plans and laboratory tests and other procedures;

- Diagnosed as PWS by gene test;

- Age: 1 month (30 days after birth) - 5 years of age;

- Male or female;

- Calculated by Peabody Developmental Motor Scale, total motor quotient, gross motor
quotient or fine motor quotient is less than 90 points;

- Thyroid function is within the normal reference range or remained within the normal
reference range by substitution therapy;

- No history of rhGH therapy before.

Exclusion Criteria:

- Subjects with abnormal liver or kidney function;

- Subjects with obvious central sleep apnea and/or moderate or severe obstructive sleep
apnea, acute lung infection;

- Subjects with chronic diseases that have long-term effects on bone metabolism and body
composition;

- Subjects with congenital skeletal dysplasia, or spine scoliosis with moderate and
above degree requiring treatment or lameness;

- Subjects with history of congenital heart disease, or an echocardiogram showing that
the structural abnormalities require surgery or interventional therapy or that the
left ventricular ejection fraction is <40%, or the abnormal electrocardiogram
requiring intervention;

- Subjects with history of convulsions or epilepsy;

- Subjects with other systemic chronic diseases;

- Subjects with diagnosed tumors;

- Subjects with family history of cancers, a previous history of cancer, or considered
to be a high risk of cancer combinating other information;

- Subjects with mental disease;

- Subjects with diabetes, or abnormal fasting glucose and researchers believe that may
affect the safety of the subject;

- Subjects with severe obesity;

- Subjects with highly allergic constitution or allergy to proteins or investigational
product or its excipient;

- Subjects who took part in other clinical trials within 3 months ;

- Subjects who received drug treatment that may interfere with GH secretion or GH action
within 3 months;

- Other conditions in which the investigator preclude enrollment into the study

Gender

All

Minimum Age

1 Month

Maximum Age

5 Years

Healthy Volunteers

No


Overall Official

Last Name

Role

Affiliation

Feihong Luo, Doctor
Principal Investigator
Children's Hospital of Fudan University

Overall Contact

Last Name

Xiaohua Feng

Phone

0431-85170552

Email



Location

Facility

Status

Tongji medical college huazhong university of science & technology
Wuhan Hubei China
Recruiting
The Children's Hospital,Zhejiang University School of Medicine
Hangzhou Zhejiang China
Recruiting
Beijing Children's Hospital, Capital Medical University
Beijing China
Recruiting
Peking union medical college hospital
Beijing China
Recruiting
Peking university first hospital
Beijing China
Recruiting
Children's Hospital of Fudan University
Shanghai China
Recruiting

Location Countries

Country

China


Verification Date

2018-05-01

Lastchanged Date

N/A

Firstreceived Date

N/A

Responsible Party

Responsible Party Type

Sponsor


Has Expanded Access

No

Condition Browse


Number Of Arms

1

Intervention Browse

Mesh Term

Hormones


Arm Group

Arm Group Label

rhGH injection/Jintropin AQ

Arm Group Type

Experimental

Description

Drug: Recombinant Human Growth Hormone Injection /Jintropin AQ, 30IU/10 mg/3ml/kit, 0.5 mg/m2/d for the first 4 weeks, then 1.0 mg/m2/d for subsequent 48 weeks; by subcutaneous injection, once per day for total 52 weeks.No control.


Firstreceived Results Date

N/A

Firstreceived Results Disposition Date

N/A

Study Design Info

Intervention Model

Single Group Assignment

Primary Purpose

Treatment

Masking

None (Open Label)


Study First Submitted

May 30, 2018

Study First Submitted Qc

May 30, 2018

Study First Posted

June 12, 2018

Last Update Submitted

May 30, 2018

Last Update Submitted Qc

May 30, 2018

Last Update Posted

June 12, 2018


ClinicalTrials.gov processed this data on August 27, 2018

Conditions

Conditions usually refer to a disease, disorder, syndrome, illness, or injury. In ClinicalTrials.gov, conditions include any health issue worth studying, such as lifespan, quality of life, health risks, etc.
Interventions

Interventions refer to the drug, vaccine, procedure, device, or other potential treatment being studied. Interventions can also include less intrusive possibilities such as surveys, education, and interviews.
Study Phase

Most clinical trials are designated as phase 1, 2, 3, or 4, based on the type of questions that study is seeking to answer:

In Phase 1 (Phase I) clinical trials, researchers test a new drug or treatment in a small group of people (20-80) for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.

In Phase 2 (Phase II) clinical trials, the study drug or treatment is given to a larger group of people (100-300) to see if it is effective and to further evaluate its safety.

In Phase 3 (Phase III) clinical trials, the study drug or treatment is given to large groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.

In Phase 4 (Phase IV) clinical trials, post marketing studies delineate additional information including the drug's risks, benefits, and optimal use.

These phases are defined by the Food and Drug Administration in the Code of Federal Regulations.



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