Clinical Trials

The Patient-Centered Outcomes Research Institute has funded a grant (PCORI) to engage cystic fibrosis (CF) stakeholders in discussions on reproductive health issues and life course experiences facing women with CF. We are looking for reproductive-aged women with CF of all ages, from teenagers to those already in menopause, who wish to participate in a partnership with researchers, physicians and other CF stakeholders to develop research questions related to the unique needs of women with CF including: sex and sexuality, decisions not to pursue motherhood, pregnancy and pregnancy prevention, infertility treatment, alternatives to biological motherhood, breastfeeding and child care, periods and menstrual cycle symptoms, perimenopause and menopause, and sexually transmitted diseases.

If you are interested, please email Kelly Gilmore, Research Coordinator at: 206-685-1855; The principal investigator is Dr. Emily Godfrey, a family physician at the University of Washington. We have scheduled our first participant session on August 31 so contacting Kelly by August 26 would be ideal. Participants will receive a small stipend of $25 per discussion session.

PTI: A Phase I Study Assessing PTI-428 Safety, Tolerability, and Pharmacokinetics in Subjects with Cystic Fibrosis with any mutation.

Pharmaxis: A Safety and Efficacy Trial of Inhaled Mannitol (Bronchitol) in Adult Cystic Fibrosis Patients

Abbvie: Long-Term Study In U.S. Cystic Fibrosis Patients Receiving Digestive Enzyme Supplements to Assess Narrowing of the Large Intestine Causing Adverse Intestinal Symptoms (Fibrosing Colonpathy)

Prevention of Cystic Fibrosis Related Diabetes (CFRD) Using Sitagliptin

Improving Treatment of Nontuberculous Mycobacteria Infection in Cystic Fibrosis

Bayer: Early Signs of Efficacy Study with Riociguat in Adult Homozygous Delta F508 Cystic Fibrosis Patients

Anthera: Solution Study of Oral Liprotamase Unit-Matched Therapy of Non-Porcine Origin in Patients with Cystic Fibrosis

ProQR: Exploratory Study to Evaluate QR-010 in Cystic Fibrosis Subjects Homozygous and Heterozygous DeltaF08 Mutation

Nivalis: Study of N91115 in Patients Homozygous for the F508del-CFTR Mutation (SNO-6)

Role of Glucagon in Glucose Control in Cystic Fibrosis Related Diabetes

PET Assessment of Acute of Lung Transplant Rejection

Pro QR Therapeutics: Dose Escalation Study of QR-010 in Homozygous ΔF508 in CF Patients

Prospective Observational Study to Evaluate Biomarkers of Aminoglycoside Nephrotoxicity in Patients With CF

Redox Imbalance and the Development of CF Related Diabetes (RedoxyMeal)

Predictive Value of PIIINP Urinary for the Development of Chronic Renal Failure in Patients With Cystic Fibrosis After Lung Transplantation (MUCO-IRC)

Parion Sciences: Clearing Lungs with ENAC Inhibition in CF (CLEAN-CF):

Effect of Chronic Incretin-Based Therapy in CF (CF Related Diabetes Study)

Blood Flow and Vascular Function in CF (Exercise, Blood Flow & Artery Function):

Comparison of Absorption of Vitamin D (Powdered Pill Vit D versus Oil Form):

Gender Disparity and Hormones in CF (Investigating Impact of Hormones in CF):

Ataluren: Phase III Study of Ataluren in “Nonsense” Cystic Fibrosis Mutations
Trial Sites: North America, South America, Europe, Australia

Hypertonic Saline: A Phase I Study of Lung Clearance After Hypertonic Saline Delivery Using the tPAD (transnasal Pulmonary Aerosol Delivery) Device

Phase I Study of N91115 in Patients With Cystic Fibrosis Homozygous F508del-CFTR Mutation

Vitamin D for Enhancing the Immune System in Cystic Fibrosis (Phase III DISC Study)

Vertex: A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Phase III Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor in CF Patients Homozygous F508del

Clinical Trials for Kalydeco Mutations G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, and G1349D
The new cystic fibrosis medication, ivacaftor (Kalydeco), has been shown to drastically change the course of cystic fibrosis lung disease for some people. Currently it is approved only for use in CF patients with the following Class II mutations: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, and G1349D. However, there are reasons to believe that some CF patients with other mutations also will respond to and benefit from ivacaftor. We believe you might be in this group of ivacaftor responders, and therefore, we invite you to participate in a brief trial of ivacaftor for other CF mutations.

Ivacaftor has shown corrective activity with many different CF channels. Having a lower sweat chloride (55 to 85 mmol), not needing pancreatic enzymes, or having better than expected lung health, are all signs of CF channel presence and function. We believe CF patients with one or more of these characteristics might respond to and benefit from ivacaftor.

Your participation in this study will help further our understanding of how to best use ivacaftor in the treatment of CF. To participate in this study, you must be 6 years old or older, have a diagnosis of cystic fibrosis, and not have any other chronic lung disorders.

If you choose to participate, the SING study will last about 7-12 weeks, during which time, you will take ivacaftor for 2 weeks and placebo for 2 weeks. Participants will not be charged or reimbursed for participating. Please see the enclosed document for more details.

Each participant has the right to refuse to participate or to leave the study at any time. Every effort will be made to guard privacy. The study is sponsored by UCSF and has been approved by the UCSF Committee on Human Research.

Thank you for considering participation. We would be glad to answer any questions and can be reached at 415-502-2880.

Dennis W. Nielson, M.D., Ph.D.
Director, UCSF Cystic Fibrosis Center
Professor, UCSF School of Medicine

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