Cystic FibrosisPhase 1NCT00803179
Growth Hormone Therapy for Wasting in Cystic Fibrosis
A Phase 1 study testing Nutropin AQ in people with Cystic Fibrosis, run by University of Massachusetts, Worcester at 1 site in United States.
Trial summary
Our hypothesis is that Growth Hormone (GH) will not only target the peripheral tissue to stimulate weight and muscle growth which will maximize nutritional potential and improve overall quality of life. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- Nutropin AQ
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteUnited States
- Study dates
- Nov 2008to Mar 2012
- Phase
- Phase 1
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Ability to provide written informed consent and comply with study assessments for the full duration of the study.
- Age > 18 years
- Cystic fibrosis, diagnosed by either sweat chloride or genetic testing
- Less than 92% ideal body weight based on body mass index (BMI) of 22 for women and 23 for men
- Moderate or better pulmonary function (Forced Expiratory Volume (FEV1) >40% of predicted).
- Agree to use an effective method of birth control to prevent pregnancy during the research study.
- Women should not nurse (breast feed) a baby while on this study because Nutropin AQ may enter breast milk and possibly harm the child.
Who cannot join
- Pregnancy (positive pregnancy test) prior enrollment in the study
- Any other condition that the investigator believes would pose a significant hazard to the subject if the investigational therapy were initiated
- Participation in another simultaneous medical investigation or trial
- Pediatric patients
- Active neoplasm
- History of organ transplantation
Show all 10Show fewer
- Prader Willi Syndrome who are severely obese or have severe respiratory impairment
- Patients with hepatic impairment resulting in abnormal coagulation studies (>1.5 times normal reference range)
- Poorly controlled diabetes as determined by a Hemoglobin A1c greater than or equal to 9.0%.
- Individuals with electrocardiogram abnormality or cardiac pacing.
Trial locations and contacts
1 site in 1 country.
About this study
Our hypothesis is that Growth Hormone (GH) will not only target the peripheral tissue to stimulate weight and muscle growth which will maximize nutritional potential and improve overall quality of life. We theorize that this will occur through a multitude of factors: increased appetite, more constructive utilization of caloric intake and decreased catabolic signaling. The first aim will address changes in weight and lean body mass following the institution of GH therapy in adults with Cystic Fibrosis (CF) related wasting. The second aim will measure impact on quality of life of these individuals. Additionally, the third aim will monitor effects of GH therapy on diabetes and insulin sensitivity. Finally, the fourth aim will observe changes in the subjects underlying diagnosis of CF, specifically lung function, muscle strength and inflammatory state.
Read the detailed description
The following is a more detailed description of the aims listed above:
Specific Aim 1: Measure change in weight in adults with CF related wasting following GH therapy.
1.1) Monitor weight gained or loss from baseline. 1.2) Assess changes in fat free mass from baseline by bioelectrical impedence analysis.
Specific Aim 2: Evaluate overall quality of life (QOL) in adults with CF related wasting treated with GH therapy.
2.1) Perform CF disease-specific and general QOL analysis via CF QOL questionnaires.
2.2) Monitor compliance with therapy via subject report.
Specific Aim 3: Monitor impact of GH therapy in relation to CF related diabetes onset or control.
3.1) Measure impact on insulin sensitivity in non-diabetes subjects 3.2) Observe change in exogenous insulin requirements and glycemic control in subjects with diabetes.
Specific Aim 4: Quantify impact of anabolic therapy on manifestations of underlying diagnosis associated with CF.
4.1) Observe changes in lung function from baseline during GH therapy. 4.2) Determine changes in overall muscle strength via hand grip and six minute walk.
4.3) Evaluate changes in serum markers.
- Conditions
- Cystic Fibrosis
- Being tested
- Nutropin AQ
- Keywords
- CF
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Nutropin AQDrug
Also called Growth Hormone (GH)
Based on recommendations from the 2007 GH Deficiency Consensus Workshop on adult GH deficiency, the recommended initiation of treatment for adult males is 0.2mg/d and for women 0.4mg/d, with a titration upwards based on insulin-like growth factor (IGF-1) (product of GH stimulation at target tissues) levels and patient response. IGF-1 will be monitored at the 3,4,5 and 11 month intervals. For subjects under the age of 25 with an open epiphysis of the hand and/or wrist we will treat with the dose of 0.3mg/kg/week. Subjects will be on growth hormone for 8 months with a baseline visit prior to initiation of therapy and a 3 month follow-up visit after stopping therapy.
| Study group | Role | What they receive |
|---|---|---|
| Growth Hormone Therapy | Experimental | Nutropin Aqueous (AQ):
Initiation treatment for adult males is 0.2mg/d and for women 0.4mg/d Receives: Drug: Nutropin AQ |
What is being measured
The primary outcome is the main question the study is designed to answer.
Measure Change in Weight in Adults With Cystic Fibrosis (CF) Related Wasting Following Growth Hormone (GH) Therapy
Measured: 14 months
Secondary outcomes (1)
Evaluate Overall Quality of Life (QOL) in Adults With CF Related Wasting Treated With GH Therapy
Measured: 14 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Dec 2008 (estimated)
- Study startNov 2008
- Primary completionMar 2012
- Study completionMar 2012
Why it stopped: Poor enrollment, patients were lost to follow up
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- More trials run by University of Massachusetts, WorcesterThe sponsor's other registered studies.
- 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Massachusetts, Worcester
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Michael Stalvey, MD, Principal investigator, Unversity of Massachusetts Medical School
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00803179
- Sponsor's protocol ID
- 900005
- Status last verified
- Nov 2025
- First submitted
- 4 Dec 2008
- Last updated
- 2 Dec 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
Thaker V, Carter B, Putman M. Recombinant growth hormone therapy for cystic fibrosis in children and young adults. Cochrane Database Syst Rev. 2021 Aug 23;8(8):CD008901. doi: 10.1002/14651858.CD008901.pub5.
PubMed 34424546
Study at a glance
From the registry record, last updated by the sponsor on 2 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Growth Hormone Therapy for Wasting in Cystic Fibrosis
- ClinicalTrials.gov ID
- NCT00803179
- Acronym / sponsor ID
- 900005
- Conditions
- Cystic Fibrosis
- Keywords
- CF
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Poor enrollment, patients were lost to follow up
- Enrolment
- 5 participants (actual)
- Lead sponsor
- University of Massachusetts, WorcesterUniversity, hospital or other
- Study start
- Nov 2008
- Primary completion
- Mar 2012 (actual)
- Study completion
- Mar 2012 (actual)
- First posted
- 5 Dec 2008 (estimated)
- Results
- Posted 15 Jan 2013
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Our hypothesis is that Growth Hormone (GH) will not only target the peripheral tissue to stimulate weight and muscle growth which will maximize nutritional potential and improve overall quality of life. We theorize that this will occur through a multitude of factors: increased appetite, more constructive utilization of caloric intake and decreased catabolic signaling. The first aim will address changes in weight and lean body mass following the institution of GH therapy in adults with Cystic Fibrosis (CF) related wasting. The second aim will measure impact on quality of life of these individuals. Additionally, the third aim will monitor effects of GH therapy on diabetes and insulin sensitivity. Finally, the fourth aim will observe changes in the subjects underlying diagnosis of CF, specifically lung function, muscle strength and inflammatory state.
Detailed description
The following is a more detailed description of the aims listed above:
Specific Aim 1: Measure change in weight in adults with CF related wasting following GH therapy.
1.1) Monitor weight gained or loss from baseline. 1.2) Assess changes in fat free mass from baseline by bioelectrical impedence analysis.
Specific Aim 2: Evaluate overall quality of life (QOL) in adults with CF related wasting treated with GH therapy.
2.1) Perform CF disease-specific and general QOL analysis via CF QOL questionnaires.
2.2) Monitor compliance with therapy via subject report.
Specific Aim 3: Monitor impact of GH therapy in relation to CF related diabetes onset or control.
3.1) Measure impact on insulin sensitivity in non-diabetes subjects 3.2) Observe change in exogenous insulin requirements and glycemic control in subjects with diabetes.
Specific Aim 4: Quantify impact of anabolic therapy on manifestations of underlying diagnosis associated with CF.
4.1) Observe changes in lung function from baseline during GH therapy. 4.2) Determine changes in overall muscle strength via hand grip and six minute walk.
4.3) Evaluate changes in serum markers.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Growth Hormone Therapy | Experimental | Drug: Nutropin AQNutropin Aqueous (AQ): Initiation treatment for adult males is 0.2mg/d and for women 0.4mg/d |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Ability to provide written informed consent and comply with study assessments for the full duration of the study.
- Age > 18 years
- Cystic fibrosis, diagnosed by either sweat chloride or genetic testing
- Less than 92% ideal body weight based on body mass index (BMI) of 22 for women and 23 for men
- Moderate or better pulmonary function (Forced Expiratory Volume (FEV1) >40% of predicted).
Key exclusion criteria (10 listed)
- Pregnancy (positive pregnancy test) prior enrollment in the study
- Any other condition that the investigator believes would pose a significant hazard to the subject if the investigational therapy were initiated
- Participation in another simultaneous medical investigation or trial
- Pediatric patients
- Active neoplasm
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 |
|---|---|---|
| Nutropin AQAlso: Growth Hormone (GH)Based on recommendations from the 2007 GH Deficiency Consensus Workshop on adult GH deficiency, the recommended initiation of treatment for adult males is 0.2mg/d and for women 0.4mg/d, with a titration upwards based on insulin-like growth factor (IGF-1) (product of GH stimulation at target tissues) levels and patient response. IGF-1 will be monitored at the 3,4,5 and 11 month intervals. For subjects under the age of 25 with an open epiphysis of the hand and/or wrist we will treat with the dose of 0.3mg/kg/week. Subjects will be on growth hormone for 8 months with a baseline visit prior to initiation of therapy and a 3 month follow-up visit after stopping therapy. | Drug | Growth Hormone Therapy |
Primary outcome
Measure Change in Weight in Adults With Cystic Fibrosis (CF) Related Wasting Following Growth Hormone (GH) Therapy
Time frame: 14 months
1 secondary outcome
Evaluate Overall Quality of Life (QOL) in Adults With CF Related Wasting Treated With GH Therapy
Time frame: 14 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Dec 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 34424546Thaker V, Carter B, Putman M. Recombinant growth hormone therapy for cystic fibrosis in children and young adults. Cochrane Database Syst Rev. 2021 Aug 23;8(8):CD008901. doi: 10.1002/14651858.CD008901.pub5.
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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.
Michael Stalvey, MD principal investigator
Unversity of Massachusetts Medical School
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Nutropin AQ in people with Cystic Fibrosis, run by University of Massachusetts, Worcester at 1 site in United States.
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 7 inclusion and 10 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: stopped early, as of the sponsor's last update on 2 Dec 2025. 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 United States. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 15 Jan 2013). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Measure Change in Weight in Adults With Cystic Fibrosis (CF) Related Wasting Following Growth Hormone (GH) Therapy (time frame: 14 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 NCT00803179, which also lists 1 linked publication.
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 2 Dec 2025.