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Growth Hormone DeficiencyPhase 2NCT03309891

Dose Finding Study of GX-H9 in Paeditaric Patients With Growth Hormone Deficiency

A Phase 2 study testing GX-H9 and Genotropin in people with Growth Hormone Deficiency, run by Genexine, Inc. at 1 site in Ukraine.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

This is a randomized, open-label, active controlled, Phase 2 study designed to assess the safety, tolerability, efficacy, pharmacokinetics, and pharmacodynamics of weekly and semi-monthly doses of GX-H9 in the treatment of Paediatric Growth Hormone Deficiency (PGHD) as compared to the standard of care daily rhGH… Read more about this study

Who is it for?
Ages 3 to 11 years
Conditions the study is for
Growth Hormone Deficiency
What is being tested?
GX-H9 and Genotropin
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
56 participants
Where
1 siteUkraine
Study dates
18 Jan 2016to 15 May 2019
Phase
Phase 2
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 to 11 years
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • Pre-pubertal children with either isolated GHD, or GH insufficiency as part of multiple pituitary hormone insufficiency, idiopathic or organic GH insufficiency (e.g., due to pituitary tumor, pituitary or brain surgery):
  • Boys: 3 years ≤ boy's age ≤ 11 years
  • Girls: 3 years ≤ girl's age ≤ 10 years
  • GHD confirmed by 2 different GH provocation tests with peak GH concentration below 10 ng/mL as described in consensus guidelines. Well documented historical GH provocation tests can be used for study eligibility providing that the tests are performed as defined in Appendix 2 (e.g. the same sampling time points). Data of each historical GH stimulation test will be reviewed by Medical Monitor and Sponsor in order to assess acceptance for the study
  • Without prior exposure to any rhGH therapy
  • Bone age (BA) is not older than chronological age and should not be greater than 9 years for girls and 10 years for boys
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  • Impaired height and height velocity defined as:
  • Height (HT) of at least 2.0 standard deviations (SD) below the mean height for chronological age (CA) and gender according to the standards from Prader et. al 1989, (HT SDS ≤ -2.0)
  • Annualized height velocity (HV) of at least 1 SD below the mean HV for chronological age and gender according to the standards of Prader et al (1989). The interval between two height measurements should be at least 6 months (but not longer than 18 months) before inclusion
  • All subjects must have at least one cranial imaging study [magnetic resonance imaging (MRI) or computed tomography (CT)] prior to randomization:
  • To exclude intracranial causes of GHD in subjects without history of pituitary tumor [obtained within 6 months prior to informed consent signing, or
  • Subjects with a previously treated pituitary tumor must have no tumor progression for at least the past year [obtained within 3 months prior to informed consent signing, compared with a previous MRI or CT performed at least 12 months earlier]
  • If not performed within these specified time frames prior to informed consent signing, may be performed as a part of the screening procedures
  • Body mass Index (BMI) must be within ±2 SD of mean BMI for the chronological age and sex according to the 2000 CDC standards
  • Baseline IGF-1 level of at least 1 SD below the mean IGF-1 level standardized for age and sex (IGF-1 SDS≤ -1.0) according to the central laboratory reference values. One IGF-1 retest is allowed during the Screening period if first results were not higher than
  • -0.85 SDS and if GH stimulation tests results and auxology parameters met eligibility criteria
  • Children with normal fundoscopy (ophthalmoscopy) at screening (without signs/symptoms of intracranial hypertension as assessed by fundoscopy) - it is highly recommended to take a photograph (if equipment is available at the study center)
  • Children with multiple hormonal deficiencies must be on stable replacement therapies for other hypothalamo-pituitary-organ axes for at least 3 months and 6 months for thyroid replacement therapy prior to Screening. Temporary adjustment of glucocorticoid replacement therapy, as appropriate, is acceptable
  • Normal 46 XX karyotype for girls
  • Written informed consent of the parent or legal guardian of the subject and assent of the subject (if the subject can read)
  • Parent or legal guardian who is capable and willing to administer the study drug

Who cannot join

  • History of radiation therapy or chemotherapy
  • Malnourished children defined as:
  • Serum albumin below the lower limit of normal (LLN) according to the reference ranges of central laboratory; and
  • Serum iron below the lower limit of normal (LLN) according to the reference ranges of central laboratory; and
  • BMI<-2 SD for age and sex
  • Children with psychosocial dwarfism
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  • Children born small for gestational age (SGA-birth weight and/or birth length < -2 SD for gestational age according to the standards from Niklasson et al., 1991)
  • Presence of anti-hGH antibodies at screening
  • Any clinically significant abnormality likely to affect growth or the ability to evaluate growth, such as, but not limited to, chronic diseases like renal insufficiency, spinal cord irradiation, etc.
  • Subjects with diabetes mellitus
  • Subjects with impaired fasting sugar (based on WHO; fasting blood sugar > 110mg/dl or 6.1 mmol/l) after repeated blood analysis
  • Chromosomal abnormalities and medical syndromes (Turner's syndrome, Laron syndrome, Noonan syndrome, Prader-Willi syndrome, Russell-Silver Syndrome, SHOX mutations/deletions and skeletal dysplasias), with the exception of septo-optic dysplasia
  • Evidence of closed epiphyses
  • Concomitant administration of other treatments that may have an effect on growth such as anabolic steroids and methylphenidate for attention deficit hyperactivity disorder (ADHD), with the exception of hormone replacement therapies [thyroxine, hydrocortisone, desmopressin (DDAVP)]
  • Children requiring glucocorticoid therapy, other than treated for hypothalamo-pituitary-adrenal insufficiency in replacement doses who are taking a dose of greater than 400 μg/d of inhaled budesonide or equivalents for longer than 1 month during a calendar year (e.g. asthma)
  • Major medical conditions and/or presence of contraindication to rhGH treatment
  • Has a history of positive serology results to HIV, HBV and/or HCV
  • Subject who has a known or suspected hypersensitivity to rhGH
  • Other causes of short stature such as coeliac disease, hypothyroidism and rickets
  • The subject and/or the parent/legal guardian are likely to be non-compliant in respect to study conduct
  • Subject who has received an investigational product, or has participated in a clinical study within 60 days before screening

Trial locations and contacts

1 site in 1 country.

Ukraine1 site

  • Odessa National Medical University, Odessa Regional Children's Hospital

    Odesa

Other trials in Ukraine

About this study

Official title: A Phase 2, Randomized, Open-label, Active Controlled, Dose Finding Study of Long-acting Hybrid Fc Fused Recombinant Human Growth Hormone (GX-H9) in Paeditaric Patients With Growth Hormone Deficiency

This is a randomized, open-label, active controlled, Phase 2 study designed to assess the safety, tolerability, efficacy, pharmacokinetics, and pharmacodynamics of weekly and semi-monthly doses of GX-H9 in the treatment of Paediatric Growth Hormone Deficiency (PGHD) as compared to the standard of care daily rhGH treatment.

Read the detailed description

GX-H9 is a new hGH product fused to hybrid Fc in studies as a once-a-week and every other week dosing regimen designed to overcome the inconvenience of daily rhGH injections and is under the studies designed to determine if the safety profile is comparable to currently approved daily rhGH products. Obviating the need for daily injections may increase compliance and therefore efficacy, which would be of great benefit to both paediatric and adult patients with GHD and other disorders with associated growth impairment and need for hGH substitution.

Being tested
GX-H9, Genotropin
Medical subject headings
Dwarfism, Pituitary

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • GX-H9Drug

    subcutaneous injection (weekly or twice-monthly)

  • GenotropinDrug

    subcutaneous injection (daily)

Study groups in NCT03309891
Study groupRoleWhat they receive
Cohort 1ExperimentalGX-H9 subcutaneous injections (weekly)
Receives: Drug: GX-H9
Cohort 2ExperimentalGX-H9 subcutaneous injections (weekly)
Receives: Drug: GX-H9
Cohort 3ExperimentalGX-H9 subcutaneous injections (twice-monthly)
Receives: Drug: GX-H9
Cohort 4Active comparatorGenotropin subcutaneous injections (daily)
Receives: Drug: Genotropin

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Annual height velocity in cm/year

    The primary efficacy variable was the AHV in cm/year at 6 months.

    Measured: 6 months

Secondary outcomes (11)

  1. Annualized Height velocity expressed in SDS

    Measured: 3, 6, 12 and 24 months

  2. Change in height SDS (compared to baseline value)

    Measured: 3, 6, 12 and 24 months

  3. Annualized height velocity expressed in cm/year

    Measured: 3, 12 and 24 months

  4. Change in height expressed in cm

    Measured: 3, 6, 12 and 24 months

  5. Change in absolute IGF-I levels

    Measured: 25 months

  6. Change in IGF-I SDS

    Measured: 25 months

  7. Change in absolute IGFBP-3 levels

    Measured: 25 months

  8. Change in IGFBP-3 SDS

    Measured: 25 months

  9. Bone maturation after 12 and 24 months of treatment;

    Measured: 12 and 24 months

  10. Predicted adult height change from start to 12 and 24 months

    Measured: 12 and 24 months

  11. Number of subjects needing at least one dose modification

    Measured: 25 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted16 Oct 2017
  2. Study start18 Jan 2016
  3. Primary completion27 Oct 2017
  4. Study completion15 May 2019

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Model description
A Phase 2, randomized, open-label, active controlled, dose finding study
Masking
None (open label)
Enrolment
56 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03309891
Sponsor's protocol ID
GX-H9-003
Status last verified
Apr 2020
First submitted
5 Sep 2017
Last updated
20 Apr 2020
Results posted
No
Data monitoring committee
Yes

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing GX-H9 and Genotropin in people with Growth Hormone Deficiency, run by Genexine, Inc. at 1 site in Ukraine.

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 21 inclusion and 21 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 20 Apr 2020. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in Ukraine. 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.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

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?

Annual height velocity in cm/year (time frame: 6 months). 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 NCT03309891.

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 20 Apr 2020.

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