A Registry for Participants With Chronic Hypoparathyroidism

NCT01922440 | Active Not Recruiting DMC FDA Drug

• 2 other identifiers: PAR-R13-001EUPAS16927
• Study Type: observational
• Target: 1,339
• Locations: 11 countries
• Sites: 100

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.

Conditions

Hypoparathyroidism

Keywords

Chronic Hypoparathyroidism; rhPTH(1-84); Observational study; parathyroid hormone (PTH); Drug registry; rhPTH(1-34)

Outcome Measures

Primary Outcomes (17)

• Change from Baseline in 24-Hour Urine Calcium

  Change from baseline in 24-hour urine calcium will be evaluated.

  Baseline up to 10 years (follow-up)

• Change from Baseline in Serum Calcium

  Change from baseline in serum calcium will be evaluated.

  Baseline up to 10 years (follow-up)

• Change from Baseline in Serum Albumin

  Change from baseline in serum albumin will be evaluated.

  Baseline up to 10 years (follow-up)

• Change from Baseline in Albumin-Corrected Total Calcium

  Change from baseline in albumin-corrected total calcium will be evaluated.

  Baseline up to 10 years (follow-up)

• Change from Baseline in Serum Ionized Calcium

  Change from baseline in serum ionized calcium will be evaluated.

  Baseline up to 10 years (follow-up)

• Change from Baseline in Serum Magnesium

  Change from baseline in serum magnesium will be evaluated.

  Baseline up to 10 years (follow-up)

• Change from Baseline in Serum Phosphate

  Change from baseline in serum phosphate will be evaluated

  Baseline up to 10 years (follow-up)

• Change from Baseline in 25-Hydroxy (25-OH) Vitamin D results

  Change from baseline in 25-OH vitamin D will be evaluated.

  Baseline up to 10 years (follow-up)

• Change from Baseline in Serum Creatinine

  Change from baseline in serum creatinine will be evaluated.

  Baseline up to 10 years (follow-up)

• Change from Baseline in Estimated Glomerular Filtration Rate (eGFR; calculated)

  Change from baseline in eGFR; calculated will be evaluated.

  Baseline up to 10 years (follow-up)

• Change from Baseline in 24-Hour Urine Protein

  Change from Baseline in 24-hour urine protein will be evaluated.

  Baseline up to 10 years (follow-up)

• Incidence Rate of the Renal Events

  Incidence rate of the renal events will be recorded for nephrolithiasis, nephrocalcinosis, hospitalization/emergency room visits for renal events.

  Baseline up to 10 years (follow-up)

• Incidence Rate of the Soft Tissue Calcifications (site)

  Incidence rate of the soft tissue calcifications (site) will be recorded.

  Baseline up to 10 years (follow-up)

• Incidence Rate of the Cataract

  Incidence rate of the cataract will be recorded by questionnaire (present/not present).

  Baseline up to 10 years (follow-up)

• Incidence Rate of the Bone Fractures (site)

  Incidence rate of the bone fractures (site) will be recorded.

  Baseline up to 10 years (follow-up)

• Incidence Rate of the Cardiovascular Events

  Incidence rate of the cardiovascular events will be calculated for this clinical outcome. Cardiovascular events include myocardial infarction, stroke, arrhythmia.

  Baseline up to 10 years (follow-up)

• Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

  An AE is any untoward medical occurrence in a participant administered a medicinal product and which does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, a new disease or worsening in severity or frequency of a concomitant disease, temporally associated with the use of a medicinal product, whether or not the event is considered causally related to the use of the product. An SAE is any untoward medical occurrence (whether considered to be related to study product or not) that at any dose results in death, life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital abnormality or birth defect, an important medical event.

  Baseline up to 10 years (follow-up)

Secondary Outcomes (3)

• Health-related Quality of Life (HRQoL)

  Baseline up to 10 years (follow-up)

• Disease-specific Patient-reported Outcome Measures

  Baseline up to 10 years (follow-up)

• Rate of Hospitalization/Emergency Room (ER) Visits

  Baseline up to 10 years (follow-up)

Study Arms (2)

Natpar(a)

Participants receiving parathyroid hormone (rhPTH(1-84) (Natpar\[a\]) for treatment of chronic hypoparathyroidism as per standard clinical practice will be enrolled and evaluated during the study period.

Other: No intervention

Conventional Therapy

Participants receiving conventional therapy/standard of care (including calcium supplements, active vitamin D, vitamin D) for treatment of chronic hypoparathyroidism as per standard clinical practice will be enrolled and evaluated during the study period.

Other: No intervention

Interventions

No intervention OTHER

This is a non-interventional study.

Conventional Therapy; Natpar(a)

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

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

You may not qualify if:

• 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).

Sponsors & Collaborators

• Takeda: lead
• Takeda Development Center Americas, Inc.: collaborator

Study Sites (100)

University of Alabama at Birmingham

Birmingham, Alabama, 35205, United States

Location

University of Arizona Medical Center

Tucson, Arizona, 85714, United States

Location

Children's Hospital Los Angeles

Los Angeles, California, 90027, United States

Location

Cedars-Sinai Medical Center

Los Angeles, California, 90048, United States

Location

Dr. Joselito Cabaccan

San Jose, California, 95148, United States

Location

University of Colorado Hospital

Aurora, Colorado, 80045, United States

Location

Yale University

New Haven, Connecticut, 06510, United States

Location

Hanson Clinical Research Center

Port Charlotte, Florida, 33952, United States

Location

Thyroid & Endocrine Center of Florida

Sarasota, Florida, 34239, United States

Location

East-West Medical Research Institute

Honolulu, Hawaii, 96814, United States

Location

Suburban Endocrinology & Diabetes

Arlington Heights, Illinois, 60005, United States

Location

University of Chicago Medical Center

Chicago, Illinois, 60637, United States

Location

Northshore University Health System

Evanston, Illinois, 60201, United States

Location

Loyola University Medical Center

Maywood, Illinois, 60153, United States

Location

Indiana University Hospital

Indianapolis, Indiana, 46202, United States

Location

Tilak Mallik, MD F.A.C.E., LCC

Marrero, Louisiana, 70072, United States

Location

Model Clinical Research

Baltimore, Maryland, 21204, United States

Location

Medstar Union Memorial Hospital

Baltimore, Maryland, 21218, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Brigham and Women's Hospital

Boston, Massachusetts, 02115, United States

Location

Harvard Medical School

Boston, Massachusetts, 02115, United States

Location

Boston Medical Center

Boston, Massachusetts, 02118, United States

Location

University of Minnesota

Minneapolis, Minnesota, 55455, United States

Location

Mayo Clinic - Rochester

Rochester, Minnesota, 55905, United States

Location

University of Missouri

Columbia, Missouri, 65212, United States

Location

Diabetes & Endocrine Associates, Methodist Physicians Clinic Diabetes and Endocrine Specialists

Omaha, Nebraska, 68114, United States

Location

Palm Research Center, Inc.

Las Vegas, Nevada, 89128, United States

Location

Northern Nevada Endocrinology

Reno, Nevada, 89511, United States

Location

Rutgers Robert Wood Johnson Medical School

New Brunswick, New Jersey, 08901, United States

Location

Rutgers-Robert Wood Johnson Medical School

New Brunswick, New Jersey, 08901, United States

Location

Albany Med Endocrine Specialists

Albany, New York, 12206, United States

Location

Brokhin Medical PC

Brooklyn, New York, 11223, United States

Location

Gerald Friedman Diabetes Institute

New York, New York, 10022, United States

Location

Columbia University Medical Center

New York, New York, 10032, United States

Location

Endocrine Associates of Long Island, P.C.

Smithtown, New York, 11787, United States

Location

University Physicians Group Research Division

Staten Island, New York, 10301, United States

Location

State University Of New York Upstate Medical University

Syracuse, New York, 13214, United States

Location

Mecklenburg Medical Group

Charlotte, North Carolina, 28211, United States

Location

Physicians East, PA

Greenville, North Carolina, 27834, United States

Location

Cleveland Clinic

Cleveland, Ohio, 44195, United States

Location

Ohio State University Wexner Medical Center

Columbus, Ohio, 43210, United States

Location

Ohio Health Research Institute

Columbus, Ohio, 43214, United States

Location

Endocrinology Associates of Armstrong

Indiana, Pennsylvania, 15701, United States

Location

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Thomas Jefferson University

Philadelphia, Pennsylvania, 76307, United States

Location

Alleghany General Hospital

Pittsburgh, Pennsylvania, 15212, United States

Location

Hallett Center for Diabetes and Endocrinology

East Providence, Rhode Island, 02914, United States

Location

University of South Carolina

Columbia, South Carolina, 29203, United States

Location

Texas Diabetes and Endocrinology, P.A.

Austin, Texas, 78731, United States

Location

Thyroid Endocrinology and Diabetes

Dallas, Texas, 75208, United States

Location

Academy of Diabetes Thyroid and Endocrine

El Paso, Texas, 79935, United States

Location

Diabetes and Thyroid Center of Fort Worth, PLLC

Fort Worth, Texas, 76132, United States

Location

University of Vermont

South Burlington, Vermont, 05403, United States

Location

University of Wisconsin

Madison, Wisconsin, 53792, United States

Location

Medizinische Universitaetsklinik Graz, Universitätsklinik Für Innere Medizin Graz

Graz, 8036, Austria

Location

AKH Wien, Universitaetsklinik fuer Innere Medizin III

Vienna, 1090, Austria

Location

Nova Scotia Health Authority

Halifax, Nova Scotia, B3H 2Y9, Canada

Location

McMaster University

Oakville, Ontario, L6J IX8, Canada

Location

Aallborg University Hospital

Aalborg, 9000, Denmark

Location

Aarhus Universitetshospital

Aarhus N, 8200, Denmark

Location

Nordsjaellands Hospital - Hillerod

Hilleroed, 3400, Denmark

Location

Regions Hospitalet Viborg

Viborg, 8800, Denmark

Location

Diakonie-Klinikum Stuttgart

Stuttgart, Baden-Wurttemberg, 70176, Germany

Location

Medizinische Hochschule Hannover

Hanover, Lower Saxony, 30625, Germany

Location

University Hospital Duesseldorf

Düsseldorf, North Rhine-Westphalia, 40225, Germany

Location

University of Leipzig

Leipzig, Saxony, 04103, Germany

Location

Universitaetsklinik Aachen

Aachen, 52074, Germany

Location

Charite-Universitiitsmedizin Berlin (CCM)

Berlin, 10117, Germany

Location

Praxis an der Kaiserreiche

Berlin, 12159, Germany

Location

Medizinische Fakultaet Carl Gustav Carus Technische Universitaet Dresden

Dresden, 01307, Germany

Location

Endokrinologikum Frankfurt

Frankfurt, 60596, Germany

Location

MVZ endokrinologikum Göttingen

Göttingen, 37075, Germany

Location

Universitaet Zur Luebeck

Lübeck, 23538, Germany

Location

Medicover Neuroendokrinologie MVZ

München, 81667, Germany

Location

Medicover Neu-Ulm Mvz

Neu-Ulm, 89231, Germany

Location

Medicover Oldenburg MVZ

Oldenburg, 26122, Germany

Location

Medicover Saarbuecken Mvz

Saarbrücken, 66111, Germany

Location

Endokrinologie Zentrum Ulm

Ulm, 89075, Germany

Location

Universitaetsklinikum Wuerzburg

Würzburg, 97080, Germany

Location

General Hospital Of Athens - Korgialenio-Benakio E.E.S.

Athens, 11526, Greece

Location

General Hospital of Athens Georgios Gennimatas

Athens, 11527, Greece

Location

General Hospital of Athens Alexandra

Athens, 11528, Greece

Location

AHEPA University General Hospital of Thessaloniki

Thessaloniki, 54636, Greece

Location

Hippokrateion General Hospital of Thessaloniki

Thessaloniki, 54642, Greece

Location

Azienda Ospedaliero-Universitaria Careggi

Florence, FI 50139, Italy

Location

Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico

Milan, MI 20122, Italy

Location

Azienda Ospedaliera San Giovanni Addolorata

Roma, RM 00184, Italy

Location

Azienda Ospedaliero-Universitaria Citta della Salute e della Scienza - Ospedale Molinette

Torino, TO 10126, Italy

Location

Haukeland University Hospital

Bergen, 5012, Norway

Location

Spesialistsentret Pilestredet Park

Oslo, 0176, Norway

Location

Hospital Del Mar

Barcelona, 08003, Spain

Location

Hospital Universitario San Cecilio

Granada, 18016, Spain

Location

Hospital Universitario 12 de Octubre

Madrid, 28041, Spain

Location

Universitetssjukhuset I Linkoping

Linköping, 58185, Sweden

Location

Universitetssjukhuset I Orebro

Örebro, 70185, Sweden

Location

Karolinska Universitetssjukhuset Solna

Stockholm, 17176, Sweden

Location

Akademiska Sjukhuset - Uppsala University Hospital

Uppsala, 75185, Sweden

Location

Queen Elizabeth Hospital-Mindelsohn Way

Birmingham, England, B15, United Kingdom

Location

University of Hospitals of Leicester

Leicester, LE1 5WW, United Kingdom

Location

Norfolk And Norwich University Hospital - Norwich Medical School

Norwich, NR4 7UQ, United Kingdom

Location

Related Publications (1)

• 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.

  PMID: 34801015 DERIVED

Related Links

• To obtain more information on the study, click here/on this link

MeSH Terms

Conditions

Hypoparathyroidism

Condition Hierarchy (Ancestors)

Parathyroid Diseases; Endocrine System Diseases

Study Officials

• Study Director

  Takeda

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: PROSPECTIVE
• Target Duration: 10 Years
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 1, 2013
• First Posted: August 14, 2013
• Study Start: July 30, 2013
• Primary Completion (Estimated): November 30, 2034
• Study Completion (Estimated): November 30, 2034
• Last Updated: September 29, 2025

Record last verified: 2025-09

Locations

DK (4); DE (17); SE (4); CA (2); GR (5); US (54); NO (2); ES (3); IT (4); GB (3); AU (2)