Growth Hormone Deficiency
Conditions
Brief summary
This is a Phase IV, open label, case-controlled, observational study to evaluate the age-adjusted cumulative height velocity in pre-pubertal isolated growth hormone deficient (IGHD) and idiopathic short stature (ISS) participants receiving daily doses of somatropin (recombinant human growth hormone \[rhGH\]; Nutropin) using NuSpin device compared to historical controls from the national cooperative growth study (NCGS).
Interventions
DEVICENuSpin
Device for administration of doses of somatropin.
DRUGSomatropin
Somatropin using either NuSpin or Nutropin AQ Pen, as per standard dosing.
Sponsors
Genentech, Inc.
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
2 Years to 14 Years
Healthy volunteers
No
Inclusion criteria
* Bone age less than or equal to (\</=) 8 years (females) or \</= 10 years (males) as determined by x-ray of the left hand and wrist obtained within six months before enrollment * Prepubertal males and females by physical exam * Naive to rhGH therapy * Diagnosis of IGHD or ISS by standard pharmacologic testing and no other discernable etiology for short stature * Height standard deviation score (Ht SDS) \</= -1.5 (\</= 5th percentile) for IGHD participants; Ht SDS \</= -2.25 (\</= 1.2 percentile) for ISS participants
Exclusion criteria
* Short stature etiologies other than IGHD or ISS * Participants receiving chronic corticosteroid therapy (greater than \[\>\] 3 months) for other medical conditions * Participants with active malignancy or any other condition that the investigator believes would pose a significant hazard to the participant if rhGH were initiated * Females with turner syndrome * Any previous rhGH treatment * Participation in another simultaneous medical investigation or trial * Pediatric participants with closed epiphyses * Participants prescribed rhGH doses outside the variance of NCGS control participant dosing
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Annualized Height Velocity in Response to rhGH Treatment Using the NuSpin Device After Two Years of Treatment | Baseline up to Month 24 (Year 2) | The annualized height velocity (cm per year \[cm/yr\]) over 2 years was calculated as: \[(height in cm at t24 minus (-) height in cm at t0) divided by (date at t24 - date at t0)\] multiplied by 365.25, where, t0 is the baseline measurement visit and t24 is the 2-year measurement visit. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Annualized Height Velocity in Response to rhGH Treatment Using the NuSpin Device for First Year of Treatment | Baseline up to Month 12 (Year 1) | The annualized height velocity (cm/yr) after 1 year was calculated as: \[(height in cm at t12 - height in cm at t0) divided by (date at t12 - date at t0)\] multiplied by 365.25, where, t0 is the baseline measurement visit and t12 is the 1-year measurement visit. |
| Annualized Height Velocity in Response to rhGH Treatment Using the NuSpin Device During the Second Year of Therapy | Month 12 to Month 24 (Year 1 to Year 2) | The annualized height velocity (cm/yr) for second year was calculated as: \[(height in cm at t24 - height in cm at t12) divided by (date at t24 - date at t12)\] multiplied by 365.25, where, t12 is the 1-year measurement visit and t24 is the 2-year measurement visit. |
Countries
United States
Participant flow
Pre-assignment details
Due to slow enrollment, the study was terminated after screening 33 participants and enrolling only 18 participants, out of 480 planned participants.
Participants by arm
| Arm | Count |
|---|---|
| IGHD Participants IGHD participants who initiated somatropin (DNA origin) (rhGH) using the NuSpin device, were observed for at least 2 years. The choice of initiation of NuSpin treatment and the treatment regimen was as per treating physician's discretion, the study protocol did not enforce or specified any treatment regimen. | 15 |
| ISS Participants ISS participants who initiated somatropin (DNA origin) (rhGH) using the NuSpin device, were observed for at least 2 years. The choice of initiation of NuSpin treatment and the treatment regimen was as per treating physician's discretion, the study protocol did not enforce or specified any treatment regimen. | 3 |
| Total | 18 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Did not meet eligibility criteria | 2 | 0 |
Baseline characteristics
| Characteristic | IGHD Participants | ISS Participants | Total |
|---|---|---|---|
| Age, Continuous | 9.60 Years STANDARD_DEVIATION 2.07 | 8.11 Years STANDARD_DEVIATION 2.6 | 9.35 Years STANDARD_DEVIATION 2.16 |
| Sex: Female, Male Female | 2 Participants | 0 Participants | 2 Participants |
| Sex: Female, Male Male | 13 Participants | 3 Participants | 16 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — |
| other Total, other adverse events | 0 / 15 | 1 / 3 |
| serious Total, serious adverse events | 1 / 15 | 0 / 3 |
Outcome results
Primary
Annualized Height Velocity in Response to rhGH Treatment Using the NuSpin Device After Two Years of Treatment
The annualized height velocity (cm per year \[cm/yr\]) over 2 years was calculated as: \[(height in cm at t24 minus (-) height in cm at t0) divided by (date at t24 - date at t0)\] multiplied by 365.25, where, t0 is the baseline measurement visit and t24 is the 2-year measurement visit.
Time frame: Baseline up to Month 24 (Year 2)
Population: Data for this outcome measure was not collected as the study was terminated prematurely due to slow enrollment.
Secondary
Annualized Height Velocity in Response to rhGH Treatment Using the NuSpin Device During the Second Year of Therapy
The annualized height velocity (cm/yr) for second year was calculated as: \[(height in cm at t24 - height in cm at t12) divided by (date at t24 - date at t12)\] multiplied by 365.25, where, t12 is the 1-year measurement visit and t24 is the 2-year measurement visit.
Time frame: Month 12 to Month 24 (Year 1 to Year 2)
Population: Data for this outcome measure was not collected as the study was terminated prematurely due to slow enrollment.
Secondary
Annualized Height Velocity in Response to rhGH Treatment Using the NuSpin Device for First Year of Treatment
The annualized height velocity (cm/yr) after 1 year was calculated as: \[(height in cm at t12 - height in cm at t0) divided by (date at t12 - date at t0)\] multiplied by 365.25, where, t0 is the baseline measurement visit and t12 is the 1-year measurement visit.
Time frame: Baseline up to Month 12 (Year 1)
Population: Data for this outcome measure was not collected as the study was terminated prematurely due to slow enrollment.