Trial Outcomes & Findings for Clinical Trial of Growth Hormone in MPS I, II, and VI (NCT NCT00748969)

NCT ID: NCT00748969

Last Updated: 2018-09-05

Results Overview

• Recruitment status: TERMINATED
• Study phase: PHASE2/PHASE3
• Target enrollment: 2 participants
• Primary outcome timeframe: 12 months
• Results posted on: 2018-09-05

Participant Flow

Participant milestones

Measure	Growth Hormone Treatmen Growth hormone treatment arm. Somatropin (DNA origin) Somatropin (DNA origin): The study starting dose of Nutropin AQ® will be 0.48 mg/kg/week divided into daily SC injections. Nutropin AQ® will be administered by either the subject or, if unable to demonstrate competency in this, then by the guardian. To decrease the risk of increased intracranial hypertension, the dose in the first month of treatment will be decreased by 50% (0.24 mg/kg/week), and then increased to 0.48 mg/kg/week if tolerated well after 1 month.	No Growth Hormone Treatment in Year 1 No growth hormone treatment in year 1; option for treatment in year 2 open-label period.
Overall Study STARTED	1	1
Overall Study COMPLETED	0	0
Overall Study NOT COMPLETED	1	1

Reasons for withdrawal

Measure	Growth Hormone Treatmen Growth hormone treatment arm. Somatropin (DNA origin) Somatropin (DNA origin): The study starting dose of Nutropin AQ® will be 0.48 mg/kg/week divided into daily SC injections. Nutropin AQ® will be administered by either the subject or, if unable to demonstrate competency in this, then by the guardian. To decrease the risk of increased intracranial hypertension, the dose in the first month of treatment will be decreased by 50% (0.24 mg/kg/week), and then increased to 0.48 mg/kg/week if tolerated well after 1 month.	No Growth Hormone Treatment in Year 1 No growth hormone treatment in year 1; option for treatment in year 2 open-label period.
Overall Study Withdrawal by Subject	0	1

Baseline Characteristics

Clinical Trial of Growth Hormone in MPS I, II, and VI

Baseline characteristics by cohort

Measure	Growth Hormone Treatmen n=1 Participants Growth hormone treatment arm. Somatropin (DNA origin) Somatropin (DNA origin): The study starting dose of Nutropin AQ® will be 0.48 mg/kg/week divided into daily SC injections. Nutropin AQ® will be administered by either the subject or, if unable to demonstrate competency in this, then by the guardian. To decrease the risk of increased intracranial hypertension, the dose in the first month of treatment will be decreased by 50% (0.24 mg/kg/week), and then increased to 0.48 mg/kg/week if tolerated well after 1 month.	No Growth Hormone Treatment in Year 1 n=1 Participants No growth hormone treatment in year 1; option for treatment in year 2 open-label period.	Total n=2 Participants Total of all reporting groups
Age, Continuous	10.9 years n=5 Participants	13.5 years n=7 Participants	12.2 years n=5 Participants
Age, Categorical <=18 years	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Sex: Female, Male Male	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Region of Enrollment United States	1 participants n=5 Participants	1 participants n=7 Participants	2 participants n=5 Participants

PRIMARY outcome

Timeframe: 12 months

Population: Zero participants analyzed in the "No growth hormone treatment" group due to subject withdrew from study as soon as informed they were assigned the no treatment group.

Outcome measures

Measure	Growth Hormone Treatment n=1 Participants Growth hormone treatment arm. Somatropin (DNA origin) Somatropin (DNA origin): The study starting dose of Nutropin AQ® will be 0.48 mg/kg/week divided into daily SC injections. Nutropin AQ® will be administered by either the subject or, if unable to demonstrate competency in this, then by the guardian. To decrease the risk of increased intracranial hypertension, the dose in the first month of treatment will be decreased by 50% (0.24 mg/kg/week), and then increased to 0.48 mg/kg/week if tolerated well after 1 month.	No Growth Hormone Treatment Observation only: no growth hormone treatment and no placebo.
Change in Growth Velocity From Baseline to End of Study Year 1.	NA cm/yr insufficient data due to study termination	—

SECONDARY outcome

Timeframe: 1 months

Population: Study stopped early due to inability to enroll participants.

Outcome measures

Measure	Growth Hormone Treatment n=1 Participants Growth hormone treatment arm. Somatropin (DNA origin) Somatropin (DNA origin): The study starting dose of Nutropin AQ® will be 0.48 mg/kg/week divided into daily SC injections. Nutropin AQ® will be administered by either the subject or, if unable to demonstrate competency in this, then by the guardian. To decrease the risk of increased intracranial hypertension, the dose in the first month of treatment will be decreased by 50% (0.24 mg/kg/week), and then increased to 0.48 mg/kg/week if tolerated well after 1 month.	No Growth Hormone Treatment Observation only: no growth hormone treatment and no placebo.
Safety: Number Drug Related SAEs	0 Participants	0 Participants

Adverse Events

GH Treatment

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

No GH Treatment

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	GH Treatment n=1 participants at risk Growth hormone treatment arm. Somatropin (DNA origin) Somatropin (DNA origin): The study starting dose of Nutropin AQ® will be 0.48 mg/kg/week divided into daily SC injections. Nutropin AQ® will be administered by either the subject or, if unable to demonstrate competency in this, then by the guardian. To decrease the risk of increased intracranial hypertension, the dose in the first month of treatment will be decreased by 50% (0.24 mg/kg/week), and then increased to 0.48 mg/kg/week if tolerated well after 1 month.	No GH Treatment No placebo/no treatment
Nervous system disorders Headache	100.0% 1/1 • Number of events 1 Zero participants analyzed in the "No growth hormone treatment" group due to subject withdrew from study as soon as informed they were assigned the no treatment group.	— 0/0 Zero participants analyzed in the "No growth hormone treatment" group due to subject withdrew from study as soon as informed they were assigned the no treatment group.
Respiratory, thoracic and mediastinal disorders worsening OSA	100.0% 1/1 • Number of events 1 Zero participants analyzed in the "No growth hormone treatment" group due to subject withdrew from study as soon as informed they were assigned the no treatment group.	— 0/0 Zero participants analyzed in the "No growth hormone treatment" group due to subject withdrew from study as soon as informed they were assigned the no treatment group.

Additional Information

Dr. Lynda Polgreen

LA Biomed at Harbor-UCLA Medical Center

Phone: 310-222-1972

Email: lpolgreen@labiomed.org

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place