Current Clinical Trials Being Conducted in the US
Clinical trials are crucial to the development of safe and effective treatment for our patients. Our goal is to expand our reach in the rare disease space, and further our understanding of how we can improve our products, to better improve our patients’ quality of life. Below is our list of studies currently recruiting in the US.
POST-BARIATRIC HYPOGLYCEMIA
A DOUBLE-BLIND RANDOMIZED PLACEBO-CONTROLLED DOSE-FINDING PHASE II STUDY TO ASSESS THE EFFICACY AND SAFETY OF PASIREOTIDE S.C. IN PATIENTS WITH POST-BARIATRIC HYPOGLYCEMIA
Study Description: The Total duration of trial participation for each participant with post-bariatric hypoglycemia will be a maximum of 59 weeks, with the following duration of trial periods
- 19 weeks for the Core Phase. It is composed of:
- a Screening period: a maximum of 3 weeks
- a Run-in period (no treatment): 4 weeks
- a Blinded Treatment Phase: 12 weeks
- Optional 36 weeks Extension Phase = an open-label Treatment period
- 4 weeks for the safety follow-up period (without any treatment).
Therapeutic area: Endocrinology
Phase: 2
Status: Recruiting
Ages Eligible for the Study: 18 years or older
Sexes Eligible for the Study: Male or non-pregnant female
Locations:
Stanford University School of Medicine
Palo Alto, California, United States
Contact: Jasmine Yang, 650-888-0144, jasminey@stanford.edu
Principal Investigator: Tracey McLaughlin, MD
Georgia Clinical Research, LLC
Lawrenceville, Georgia, United States
Contact: Malede Mangistu, 678-822-5581, mmengistugcr@gmail.com
Principal Investigator: George Rafeedie, MD
Mayo Clinic
Rochester, Minnesota, United States
Contact: Osmundson Kim, (507) 255-9278, Osmundson.Kimberly@mayo.edu
Principal Investigator: Adrian Vella, MD
Montefiore Medical Center
Bronx New York, United States
Contact: Nathalie Zavala, 718-839-7322, nathalie.zavala@einsteinmed.edu
Principal Investigator: Sriram Machineni, MD
University of Texas Health Science Center at San Antonio
San Antonio, Texas, United States
Contact: Nancy Yegge, 210-450-8696, yeggen@uthscsa.edu
Principal Investigator: Marzieh Salehi, MD
Joslin Diabetes Center
Boston, Massachusetts, United States
Contact: Alessandra Amore, 617-309-2400, hypoglycemiastudy@joslin.harvard.edu
Principal Investigator: Mary Elizabeth Patti, MD
ClinicalTrials.gov link: NCT05928390
CUSHING’S DISEASE
A PHASE II, MULTICENTER, OPEN-LABEL, NON-COMPARATIVE STUDY TO EVALUATE THE PHARMACOKINETICS, PHARMACODYNAMICS, AND TOLERABILITY OF OSILODROSTAT IN CHILDREN AND ADOLESCENT PATIENTS WITH CUSHING’S DISEASE. (LINC5)
Study Description: Multicenter, open-label, non-comparative study to evaluate the pharmacokinetics, pharmacodynamics, and tolerability of osilodrostat in children and adolescent patients with Cushing's disease.
Therapeutic area: Endocrinology
Phase: 2
Status: Recruiting
Ages Eligible for the Study: 6 years to 17 years
Sexes Eligible for the Study: All
Locations:
ClinicalTrials.gov link: NCT03708900
HYPERAMMONEMIA DUE TO ORGANIC ACIDEMIAS: PROPIONIC ACIDEMIA, METHYLMALONIC ACIDEMIA
A NON-INTERVENTIONAL POST-AUTHORIZATION SAFETY STUDY (PASS) OF CARBAGLU® FOR THE TREATMENT OF HYPERAMMONEMIA DUE TO METHYLMALONIC ACIDEMIA (MMA) AND PROPIONIC ACIDEMIA (PA) IN ADULT AND PEDIATRIC PATIENT POPULATIONS
Study Description
This study is being conducted to obtain short-term and long-term clinical safety information from adult and pediatric patients treated for hyperammonemia due to Methylmalonic Acidemia (MMA) and Propionic Acidemia (PA). This is an observational/non-interventional study. Patients will be treated per the prescribing information and routine medical practice.
Only available data will be collected as part of the study including developmental outcomes, details of treatment with Carbaglu® and other treatments for hyperammonemia including dietary and protein management, plasma ammonia levels, pregnancy and maternal complications, adverse effects on the developing fetus and neonate, adverse effects on the infant through first year of life.
Therapeutic area: Metabolic
Phase: 4
Status: Recruiting
Ages Eligible for the Study: All
Sexes Eligible for the Study: All
Locations:
Children's National Hospital
Washington, District of Columbia, United States
Contact: Kara Simpson, 202-545-2503, ksimpson@childrensnational.org
Principal Investigator: Nicholas Ah Mew, MD
University of South Florida
Tampa, Florida, United States
Contact: Farideh Oberheu, 813-250-2292, faljalla@usf.edu
Principal Investigator: Amarilis Sanchez-Valle, MD
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
Contact: Michael Sawin, 312-227-2816, Msawin@luriechildrens.org
Principal Investigator: Joshua Baker, MD
Riley Children's Hospital
Indianapolis, Indiana, United States
Contact: Susan Romie, 317-278-6650, sromie@iu.edu
Principal Investigator: Melissa Lah, MD
Icahn School of Medicine at Mt. Sinai
New York, New York, United States
Contact: Colleen Donnelly, 212-241-5983, Colleen.donnelly@mssm.edu
Principal Investigator: Margo Breilyn, MD
ClinicalTrials.gov link: NCT05040178
HYPERAMMONEMIA DUE TO UREA CYCLE DISORDERS: N-ACETYLGLUTAMATE SYNTHASE (NAGS) DEFICIENCY
ORPHAN EUROPE CARBAGLU® SURVEILLANCE PROTOCOL
Study Description
The purpose of this study is to conduct post-marketing surveillance of carglumic acid (Carbaglu) to obtain long-term clinical safety information. Carglumic acid was approved by the United States Food and Drug Administration (FDA) for treatment of acute hyperammonemia due to N-acetylglutamate synthase (NAGS) deficiency. Much of the FDA-required data is already collected through the Longitudinal Study of Urea Cycle Disorders (RDCRN Protocol #5101). This study will collect additional data on adverse events (interim events), adverse reactions, pregnancy, and fetal outcomes.
Therapeutic area: Metabolic
Phase: 4
Status: Recruiting
Ages Eligible for the Study: Child, Adult, Older Adult
Sexes Eligible for the Study: All
Locations:
Children's National Hospital
Washington, District of Columbia, United States
Contact: Kara Simpson, 202-545-2503, ksimpson@childrensnational.org
Principal Investigator: Nicholas Ah Mew, MD
ClinicalTrials.gov link: NCT03409003
INVESTIGATOR SPONSORED STUDIES
There are several Investigator Sponsored Studies conducted in North America with Recordati products in the rare disease space. Please check clinicaltrials.gov for more information.