Clinical trial NCT01922440: A Registry for Participants With Chronic Hypoparathyroidism
Official title: PARADIGHM (Physicians Advancing Disease Knowledge in Hypoparathyroidism): A Registry for Patients With Chronic Hypoparathyroidism
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Hypoparathyroidism
Keywords: Chronic Hypoparathyroidism
rhPTH(1-84)
Observational study
parathyroid hormone (PTH)
Drug registry
rhPTH(1-34)
Lead sponsor: Takeda
Sponsor class: INDUSTRY
Collaborators: name: Takeda Development Center Americas, Inc.
First posted: 2013-08-14
Enrollment: 1339
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: This global registry is expected to enroll a minimum of 900 participants, of whom at least 300 will be participants treated with rhPTH(1-84) and at least 600 will be participants receiving standard of care. Protocol amendment 9 revised the sample size: up to 164 additional participants treated with rhPTH(1-84) (ie, up to a total of 464 participants in that cohort) will be enrolled as needed to ensure the availability of 104 rhPTH(1-84)-treated participants with a full 10 years of follow-up.

Brief summary: The main aim of this study is to find out the long-term safety and effectiveness profile of recombinant human parathyroid hormone (1-84) (rhPTH\[1-84\]) treatment in participants with chronic hypoparathyroidism under conditions of routine clinical practice.

Participants will be treated according to their clinic's standard practice determined by the treating doctors. Each participant will fill out a study questionnaire during a routine doctor visit.


Eligibility criteria: Inclusion Criteria:

* Participants diagnosed with chronic hypoparathyroidism, that is, hypoparathyroidism with a duration of longer than 6 months, including:

  1. Adult participants (greater than or equal to \[\>=\] 18 years of age) who are receiving for chronic hypoparathyroidism any of the following options: standard therapy, standard therapy plus rhPTH(1-84), or rhPTH(1-84) therapy alone.
  2. Pediatric participants (less than \[\<\] 18 years of age) who are receiving for chronic hypoparathyroidism any of the following options: standard therapy, standard therapy plus rhPTH(1-84), or rhPTH(1-84) therapy alone.

Exclusion Criteria:

* Participants or legally acceptable representatives unable to provide informed consent.
* Participants using rhPTH(1-34) or who used rhPTH(1-34) for more than 2 years are excluded. Participants who had been treated with rhPTH(1-34) within 3 months of enrollment are also excluded as are participants currently using rhPTH(1-34).

FOR US SITES ONLY: Participants treated with rhPTH(1-84) prior to the US recall may use rhPTH(1-34) only while rhPTH(1-84) is unavailable due to the recall.

* Participants currently enrolled in an interventional clinical study (whether or not the study is related to hypoparathyroidism); note that this does not include participants enrolled in other observational registries.
* History of hypoparathyroidism resulting from impaired responsiveness to PTH (pseudohypoparathyroidism).

Primary outcomes:
measure: Change from Baseline in 24-Hour Urine Calcium
timeFrame: Baseline up to 10 years (follow-up)
measure: Change from Baseline in Serum Calcium
timeFrame: Baseline up to 10 years (follow-up)
measure: Change from Baseline in Serum Albumin
timeFrame: Baseline up to 10 years (follow-up)
measure: Change from Baseline in Albumin-Corrected Total Calcium
timeFrame: Baseline up to 10 years (follow-up)
measure: Change from Baseline in Serum Ionized Calcium
timeFrame: Baseline up to 10 years (follow-up)
measure: Change from Baseline in Serum Magnesium
timeFrame: Baseline up to 10 years (follow-up)
measure: Change from Baseline in Serum Phosphate
timeFrame: Baseline up to 10 years (follow-up)
measure: Change from Baseline in 25-Hydroxy (25-OH) Vitamin D results
timeFrame: Baseline up to 10 years (follow-up)
measure: Change from Baseline in Serum Creatinine
timeFrame: Baseline up to 10 years (follow-up)
measure: Change from Baseline in Estimated Glomerular Filtration Rate (eGFR; calculated)
timeFrame: Baseline up to 10 years (follow-up)
measure: Change from Baseline in 24-Hour Urine Protein
timeFrame: Baseline up to 10 years (follow-up)
measure: Incidence Rate of the Renal Events
timeFrame: Baseline up to 10 years (follow-up)
measure: Incidence Rate of the Soft Tissue Calcifications (site)
timeFrame: Baseline up to 10 years (follow-up)
measure: Incidence Rate of the Cataract
timeFrame: Baseline up to 10 years (follow-up)
measure: Incidence Rate of the Bone Fractures (site)
timeFrame: Baseline up to 10 years (follow-up)
measure: Incidence Rate of the Cardiovascular Events
timeFrame: Baseline up to 10 years (follow-up)
measure: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
timeFrame: Baseline up to 10 years (follow-up)
Secondary outcomes:
measure: Health-related Quality of Life (HRQoL)
timeFrame: Baseline up to 10 years (follow-up)
measure: Disease-specific Patient-reported Outcome Measures
timeFrame: Baseline up to 10 years (follow-up)
measure: Rate of Hospitalization/Emergency Room (ER) Visits
timeFrame: Baseline up to 10 years (follow-up)
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama at Birmingham
state: Alabama
city: Tucson
country: United States
facility: University of Arizona Medical Center
state: Arizona
city: Los Angeles
country: United States
facility: Children's Hospital Los Angeles
state: California
city: Los Angeles
country: United States
facility: Cedars-Sinai Medical Center
state: California
city: San Jose
country: United States
facility: Dr. Joselito Cabaccan
state: California
city: Aurora
country: United States
facility: University of Colorado Hospital
state: Colorado
city: New Haven
country: United States
facility: Yale University
state: Connecticut
city: Port Charlotte
country: United States
facility: Hanson Clinical Research Center
state: Florida
city: Sarasota
country: United States
facility: Thyroid & Endocrine Center of Florida
state: Florida
city: Honolulu
country: United States
facility: East-West Medical Research Institute
state: Hawaii
city: Arlington Heights
country: United States
facility: Suburban Endocrinology & Diabetes
state: Illinois
city: Chicago
country: United States
facility: University of Chicago Medical Center
state: Illinois
city: Evanston
country: United States
facility: Northshore University Health System
state: Illinois
city: Maywood
country: United States
facility: Loyola University Medical Center
state: Illinois
city: Indianapolis
country: United States
facility: Indiana University Hospital
state: Indiana
city: Marrero
country: United States
facility: Tilak Mallik, MD F.A.C.E., LCC
state: Louisiana
city: Baltimore
country: United States
facility: Model Clinical Research
state: Maryland
city: Baltimore
country: United States
facility: Medstar Union Memorial Hospital
state: Maryland
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Boston
country: United States
facility: Brigham and Women's Hospital
state: Massachusetts
city: Boston
country: United States
facility: Harvard Medical School
state: Massachusetts
city: Boston
country: United States
facility: Boston Medical Center
state: Massachusetts
city: Minneapolis
country: United States
facility: University of Minnesota
state: Minnesota
city: Rochester
country: United States
facility: Mayo Clinic - Rochester
state: Minnesota
city: Columbia
country: United States
facility: University of Missouri
state: Missouri
city: Omaha
country: United States
facility: Diabetes & Endocrine Associates, Methodist Physicians Clinic Diabetes and Endocrine Specialists
state: Nebraska
city: Las Vegas
country: United States
facility: Palm Research Center, Inc.
state: Nevada
city: Reno
country: United States
facility: Northern Nevada Endocrinology
state: Nevada
city: New Brunswick
country: United States
facility: Rutgers Robert Wood Johnson Medical School
state: New Jersey
city: New Brunswick
country: United States
facility: Rutgers-Robert Wood Johnson Medical School
state: New Jersey
city: Albany
country: United States
facility: Albany Med Endocrine Specialists
state: New York
city: Brooklyn
country: United States
facility: Brokhin Medical PC
state: New York
city: New York
country: United States
facility: Gerald Friedman Diabetes Institute
state: New York
city: New York
country: United States
facility: Columbia University Medical Center
state: New York
city: Smithtown
country: United States
facility: Endocrine Associates of Long Island, P.C.
state: New York
city: Staten Island
country: United States
facility: University Physicians Group Research Division
state: New York
city: Syracuse
country: United States
facility: State University Of New York Upstate Medical University
state: New York
city: Charlotte
country: United States
facility: Mecklenburg Medical Group
state: North Carolina
city: Greenville
country: United States
facility: Physicians East, PA
state: North Carolina
city: Cleveland
country: United States
facility: Cleveland Clinic
state: Ohio
city: Columbus
country: United States
facility: Ohio State University Wexner Medical Center
state: Ohio
city: Columbus
country: United States
facility: Ohio Health Research Institute
state: Ohio
city: Indiana
country: United States
facility: Endocrinology Associates of Armstrong
state: Pennsylvania
city: Philadelphia
country: United States
facility: The Children's Hospital of Philadelphia
state: Pennsylvania
city: Philadelphia
country: United States
facility: Thomas Jefferson University
state: Pennsylvania
city: Pittsburgh
country: United States
facility: Alleghany General Hospital
state: Pennsylvania
city: East Providence
country: United States
facility: Hallett Center for Diabetes and Endocrinology
state: Rhode Island
city: Columbia
country: United States
facility: University of South Carolina
state: South Carolina
city: Austin
country: United States
facility: Texas Diabetes and Endocrinology, P.A.
state: Texas
city: Dallas
country: United States
facility: Thyroid Endocrinology and Diabetes
state: Texas
city: El Paso
country: United States
facility: Academy of Diabetes Thyroid and Endocrine
state: Texas
city: Fort Worth
country: United States
facility: Diabetes and Thyroid Center of Fort Worth, PLLC
state: Texas
city: South Burlington
country: United States
facility: University of Vermont
state: Vermont
city: Madison
country: United States
facility: University of Wisconsin
state: Wisconsin
city: Graz
country: Austria
facility: Medizinische Universitaetsklinik Graz, Universitätsklinik Für Innere Medizin Graz
city: Wien
country: Austria
facility: AKH Wien, Universitaetsklinik fuer Innere Medizin III
city: Halifax
country: Canada
facility: Nova Scotia Health Authority
state: Nova Scotia
city: Oakville
country: Canada
facility: McMaster University
state: Ontario
city: Aalborg
country: Denmark
facility: Aallborg University Hospital
city: Aarhus N
country: Denmark
facility: Aarhus Universitetshospital
city: Hilleroed
country: Denmark
facility: Nordsjaellands Hospital - Hillerod
city: Viborg
country: Denmark
facility: Regions Hospitalet Viborg
city: Stuttgart
country: Germany
facility: Diakonie-Klinikum Stuttgart
state: Baden-Wuerttemberg
city: Hannover
country: Germany
facility: Medizinische Hochschule Hannover
state: Lower Saxony
city: Dusseldorf
country: Germany
facility: University Hospital Duesseldorf
state: Nordrhein-Westfalen
city: Leipzig
country: Germany
facility: University of Leipzig
state: Sachsen
city: Aachen
country: Germany
facility: Universitaetsklinik Aachen
city: Berlin
country: Germany
facility: Charite-Universitiitsmedizin Berlin (CCM)
city: Berlin
country: Germany
facility: Praxis an der Kaiserreiche
city: Dresden
country: Germany
facility: Medizinische Fakultaet Carl Gustav Carus Technische Universitaet Dresden
city: Frankfurt
country: Germany
facility: Endokrinologikum Frankfurt
city: Göttingen
country: Germany
facility: MVZ endokrinologikum Göttingen
city: Luebeck
country: Germany
facility: Universitaet Zur Luebeck
city: Muenchen
country: Germany
facility: Medicover Neuroendokrinologie MVZ
city: Neu-Ulm
country: Germany
facility: Medicover Neu-Ulm Mvz
city: Oldenburg
country: Germany
facility: Medicover Oldenburg MVZ
city: Saarbruecken
country: Germany
facility: Medicover Saarbuecken Mvz
city: Ulm
country: Germany
facility: Endokrinologie Zentrum Ulm
city: Wuerzburg
country: Germany
facility: Universitaetsklinikum Wuerzburg
city: Athens
country: Greece
facility: General Hospital Of Athens - Korgialenio-Benakio E.E.S.
city: Athens
country: Greece
facility: General Hospital of Athens Georgios Gennimatas
city: Athens
country: Greece
facility: General Hospital of Athens Alexandra
city: Thessaloniki
country: Greece
facility: AHEPA University General Hospital of Thessaloniki
city: Thessaloniki
country: Greece
facility: Hippokrateion General Hospital of Thessaloniki
city: Firenze
country: Italy
facility: Azienda Ospedaliero-Universitaria Careggi
city: Milano
country: Italy
facility: Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico
city: Roma
country: Italy
facility: Azienda Ospedaliera San Giovanni Addolorata
city: Torino
country: Italy
facility: Azienda Ospedaliero-Universitaria Citta della Salute e della Scienza - Ospedale Molinette
city: Bergen
country: Norway
facility: Haukeland University Hospital
city: Oslo
country: Norway
facility: Spesialistsentret Pilestredet Park
city: Barcelona
country: Spain
facility: Hospital Del Mar
city: Granada
country: Spain
facility: Hospital Universitario San Cecilio
city: Madrid
country: Spain
facility: Hospital Universitario 12 de Octubre
city: Linkoping
country: Sweden
facility: Universitetssjukhuset I Linkoping
city: Orebro
country: Sweden
facility: Universitetssjukhuset I Orebro
city: Stockholm
country: Sweden
facility: Karolinska Universitetssjukhuset Solna
city: Uppsala
country: Sweden
facility: Akademiska Sjukhuset - Uppsala University Hospital
city: Birmingham
country: United Kingdom
facility: Queen Elizabeth Hospital-Mindelsohn Way
state: England
city: Leicester
country: United Kingdom
facility: University of Hospitals of Leicester
city: Norwich
country: United Kingdom
facility: Norfolk And Norwich University Hospital - Norwich Medical School
Study officials:
affiliation: Takeda
name: Study Director
role: STUDY_DIRECTOR
References:
citation: Gittoes N, Rejnmark L, Ing SW, Brandi ML, Bjornsdottir S, Hahner S, Hofbauer LC, Houillier P, Khan AA, Levine MA, Mannstadt M, Shoback DM, Vokes TJ, Zhang P, Marelli C, Germak J, Clarke BL. The PARADIGHM (physicians advancing disease knowledge in hypoparathyroidism) registry for patients with chronic hypoparathyroidism: study protocol and interim baseline patient characteristics. BMC Endocr Disord. 2021 Nov 20;21(1):232. doi: 10.1186/s12902-021-00888-2.