Growth Hormone Therapy for Wasting in Cystic Fibrosis

NCT00803179 | Terminated Phase 1 Results

• 1 other identifier: 900005
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 1

Brief 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. 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.

Conditions

Cystic Fibrosis

Keywords

CF

Outcome Measures

Primary Outcomes (1)

• Measure Change in Weight in Adults With Cystic Fibrosis (CF) Related Wasting Following Growth Hormone (GH) Therapy

  14 months

Secondary Outcomes (1)

• Evaluate Overall Quality of Life (QOL) in Adults With CF Related Wasting Treated With GH Therapy

  14 months

Study Arms (1)

Growth Hormone Therapy

EXPERIMENTAL

Nutropin Aqueous (AQ): Initiation treatment for adult males is 0.2mg/d and for women 0.4mg/d

Drug: Nutropin AQ

Interventions

Nutropin AQ DRUG

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.

Also known as: Growth Hormone (GH)

Growth Hormone Therapy

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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
• 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.

Sponsors & Collaborators

• University of Massachusetts, Worcester: lead

Study Sites (1)

Umms/Ummhc

Worcester, Massachusetts, 01655, United States

Location

Related Publications (1)

• 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.

  PMID: 34424546 DERIVED

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

Growth Hormone

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Intervention Hierarchy (Ancestors)

Pituitary Hormones, Anterior; Pituitary Hormones; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Peptides; Amino Acids, Peptides, and Proteins

Results Point of Contact

• Title: Michael Stalvey, MD, Principal Investigator
• Organization: UMass Medical School

mstalvey@peds.uab.edu

508-856-4280

Study Officials

• Michael Stalvey, MD

  Unversity of Massachusetts Medical School

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 4, 2008
• First Posted: December 5, 2008
• Study Start: November 1, 2008
• Primary Completion: March 1, 2012
• Study Completion: March 1, 2012
• Last Updated: December 2, 2025
• Results First Posted: January 15, 2013

Record last verified: 2025-11

Locations

US (1)