Growth Hormone DeficiencyPhase 2NCT00778518
Safety, Tolerability and PK/PD Study in Young Adult Patients With Childhood Onset Growth Hormone Deficiency (GHD).
A Phase 2 study testing ARX201 in people with Growth Hormone Deficiency, run by Ambrx, Inc. at 1 site in Hungary.
Trial summary
Study to find the optimal dose of Growth Hormone Replacement in young adult patients suffering from childhood onset growth hormone deficiency (GHD). Read more about this study
- Who is it for?
- Ages 18 to 30 years, men only
- Conditions the study is for
- Growth Hormone Deficiency
- What is being tested?
- ARX201
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 36 participants
- Where
- 1 siteHungary
- Study dates
- Jul 2008to Oct 2009
- 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 18 to 30 years
- Men only
- No healthy volunteers
- Adult
Who may be able to join
- 18-30 years old
- GHD of childhood onset
- completed growth
- IGF-1 <=2SDS
- rhGH treatment naive
- hGH levels below cut-off
Who cannot join
- History of malignancy or intracranial tumors
- ECG abnormality
- ICH
- hepatic dysfunction
- renal impairment
- major medical conditions
Show all 9Show fewer
- inadequate T4
- positive for HBV, HCV, or HIV
- alcohol or drug abuse
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Phase IIb Study to Evaluate the Safety, Tolerability, Pharmacokinetic and Pharmacodynamic Profile of ARX201 Following Repeated Dosing to Young Adult Patients With Childhood Onset Growth Hormone Deficiency (GHD).
Study to find the optimal dose of Growth Hormone Replacement in young adult patients suffering from childhood onset growth hormone deficiency (GHD).
Read the detailed description
The primary objective of this study is to evaluate the safety, tolerability and pharmacodynamic response of three different ARX201 doses (PEG-ahGH) when administered repeatedly to young adult patients with childhood onset growth hormone deficiency (GHD).
- Conditions
- Growth Hormone Deficiency
- Being tested
- ARX201
- Keywords
- growth hormone, growth hormone deficiency, GHD, PEG, pegylated, IGF-1
- Medical subject headings
- Dwarfism, Pituitary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ARX201Drug
Reconstituted lyophilized drug product at 2.5 mg/wk; 3 periods over six months.
ARX201Drug
Reconstituted lyophilized drug product at 5.0 mg/wk; 3 periods over six months.
ARX201Drug
Reconstituted lyophilized drug product at 10.0 mg/wk; 3 periods over six months.
| Study group | Role | What they receive |
|---|---|---|
| 1 | Active comparator | low dose Receives: Drug: ARX201 |
| 2 | Active comparator | Medium dose Receives: Drug: ARX201 |
| 3 | Active comparator | High Dose Receives: Drug: ARX201 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Temporal profiling of circulating IGF-1 levels.
Measured: 3 period
Secondary outcomes (1)
Body composition measurements at start of study and end of study
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Oct 2008 (estimated)
- Study startJul 2008
- Primary completionOct 2009
- Study completionOct 2009
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Growth Hormone DeficiencyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- Masking
- Single blind
- Who is masked
- Participant
- Enrolment
- 36 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Ambrx, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Merck Serono International SA
- Investigators
- Mihaly Juhasz, MD, Study director, Accelsiors CRO & Consultancy Services
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00778518
- Sponsor's protocol ID
- PRO-ARX201-701
- Other IDs
- EudraCT: 2007-001746-40
- Status last verified
- Oct 2009
- First submitted
- 22 Oct 2008
- Last updated
- 12 Oct 2009
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Oct 2009. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase IIb Study to Evaluate the Safety, Tolerability, Pharmacokinetic and Pharmacodynamic Profile of ARX201 Following Repeated Dosing to Young Adult Patients With Childhood Onset Growth Hormone Deficiency (GHD).
- ClinicalTrials.gov ID
- NCT00778518
- Acronym / sponsor ID
- PRO-ARX201-701
- Conditions
- Growth Hormone Deficiency
- Keywords
- growth hormone, growth hormone deficiency, GHD, PEG, pegylated, IGF-1
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 36 participants (estimated)
- Lead sponsor
- Ambrx, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Merck Serono International SA
- Study start
- Jul 2008
- Primary completion
- Oct 2009 (actual)
- Study completion
- Oct 2009 (actual)
- First posted
- 23 Oct 2008 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Study to find the optimal dose of Growth Hormone Replacement in young adult patients suffering from childhood onset growth hormone deficiency (GHD).
Detailed description
The primary objective of this study is to evaluate the safety, tolerability and pharmacodynamic response of three different ARX201 doses (PEG-ahGH) when administered repeatedly to young adult patients with childhood onset growth hormone deficiency (GHD).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (participant)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| 1 | Active comparator | Drug: ARX201low dose |
| 2 | Active comparator | Drug: ARX201Medium dose |
| 3 | Active comparator | Drug: ARX201High Dose |
Who may qualify
- Ages
- 18 to 30 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (6 listed)
- 18-30 years old
- GHD of childhood onset
- completed growth
- IGF-1 <=2SDS
- rhGH treatment naive
Key exclusion criteria (9 listed)
- History of malignancy or intracranial tumors
- ECG abnormality
- ICH
- hepatic dysfunction
- renal impairment
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ARX201Reconstituted lyophilized drug product at 2.5 mg/wk; 3 periods over six months. | Drug | 1 |
| ARX201Reconstituted lyophilized drug product at 5.0 mg/wk; 3 periods over six months. | Drug | 2 |
| ARX201Reconstituted lyophilized drug product at 10.0 mg/wk; 3 periods over six months. | Drug | 3 |
Primary outcome
Temporal profiling of circulating IGF-1 levels.
Time frame: 3 period
1 secondary outcome
Body composition measurements at start of study and end of study
Time frame: 6 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Oct 2009.
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Growth Hormone Replacement Therapy in Veterans With Mild Traumatic Brain Injury (mTBI) and Adult Growth Hormone Deficiency (AGHD)
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Enrolling by invitationObservational study
Influence of Adherence to Growth Hormone Therapy (GHT) With Norditropin® on Near Final Height in Patients With Growth Hormone Deficiency (GHD) and Born Small for Gestational Age (SGA)
Growth Hormone Deficiency in Children, Born Small for Gestational Age
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Mihaly Juhasz, MD study director
Accelsiors CRO & Consultancy Services
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing ARX201 in people with Growth Hormone Deficiency, run by Ambrx, Inc. at 1 site in Hungary.
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 6 inclusion and 9 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 12 Oct 2009. 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 Hungary. 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?
Temporal profiling of circulating IGF-1 levels. (time frame: 3 period). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 12 Oct 2009.