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PARADIGHM (Physicians Advancing Disease Knowledge in Hypoparathyroidism): A Registry for Subjects With Chronic Hypoparathyroidism (PARADIGHM)

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ClinicalTrials.gov Identifier: NCT01922440
Recruitment Status : Recruiting
First Posted : August 14, 2013
Last Update Posted : November 29, 2017
Sponsor:
Information provided by (Responsible Party):

Study Description
Brief Summary:
This is a prospective, observational disease and drug registry designed to observe the clinical course of chronic hypoparathyrodism and to evaluate the safety and efficacy profile of rhPTH(1-84) under conditions of routine clinical practice. No treatment is provided as a result of participating in this registry and all decisions on the participant's treatment are determined by his or her physician.

Condition or disease Intervention/treatment
Chronic Hypoparathyroidism Other: None [Disease and drug registry]

Study Design

Study Type : Observational [Patient Registry]
Estimated Enrollment : 900 participants
Observational Model: Other
Time Perspective: Prospective
Target Follow-Up Duration: 17 Years
Official Title: PARADIGHM (Physicians Advancing Disease Knowledge in Hypoparathyroidism): A Registry for Subjects With Chronic Hypoparathyroidism
Actual Study Start Date : July 1, 2013
Estimated Primary Completion Date : June 1, 2030
Estimated Study Completion Date : June 1, 2032

Resource links provided by the National Library of Medicine

U.S. FDA Resources

Groups and Cohorts

Group/Cohort Intervention/treatment
Chronic Hypoparathyroidism Other: None [Disease and drug registry]


Outcome Measures

Primary Outcome Measures :
  1. Hypoparathyroidism Laboratory Test [ Time Frame: Baseline up to 17 years (follow-up) ]
    Hypoparathyroidism laboratory tests will be evaluated as change over time for 24-hour urine calcium, serum calcium, serum magnesium, serum phosphate and 25-OH vitamin D.

  2. Renal Function [ Time Frame: Baseline up to 17 years (follow-up) ]
    Renal function will be evaluated as change over time for serum creatinine, estimated glomerular filtration rate (eGFR; calculated), 24-hour urine calcium and 24 hour urine protein.

  3. Incidence Rate of the Renal Events [ Time Frame: Baseline up to 17 years (follow-up) ]
    Incidence rate of the renal events will be recorded for nephrolithiasis, nephrocalcinosis, hospitalization/emergency room (er) visits for renal events.

  4. Incidence Rate of the Soft Tissue Calcifications (site) [ Time Frame: Baseline up to 17 years (follow-up) ]
    Incidence rate of the soft tissue calcifications (site) will be recorded.

  5. Incidence Rate of the Cataract [ Time Frame: Baseline up to 17 years (follow-up) ]
    Incidence rate of the cataract will be recorded by questionnaire (present/not present).

  6. Incidence Rate of the Bone Fractures (site) [ Time Frame: Baseline up to 17 years (follow-up) ]
    Incidence rate of the bone fractures (site) will be recorded.

  7. Incidence Rate of the Cardiovascular Events [ Time Frame: Baseline up to 17 years (follow-up) ]
    Incidence rate of the cardiovascular events will be calculated for this clinical outcome. Cardiovascular events include myocardial infarction, stroke, arrhythmia.

  8. Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: Baseline up to 17 years (follow-up) ]
    An adverse event (AE) is any noxious, pathologic, or unintended change in anatomical, physiologic, or metabolic function as indicated by physical signs, symptoms, or laboratory changes occurring in the registry, whether or not considered product-related. This includes an exacerbation of a pre - existing condition. An AE or ADR that meets one or m ore of the following criteria/outcomes is classified as SAE whether considered to be related to the pharmaceutical product or not: death, life-threatening, requires in patient hospitalization or prolongation of existing hospitalizations, a persistent or significant disability or incapacity, a congenital anomaly or birth defect and important medical events.

  9. Number of Participants With Adverse Drug Reactions Specific to rhPTH(1-84) [ Time Frame: Baseline up to 17 years (follow-up) ]
    An ADR is a response to a medicinal product which is noxious and unintended and which occurs at doses normally used in man for prophylaxis, diagnosis, and treatment of disease or for the restoration, correction, or modification of physiological function. Response in this context means that a causal relationship between a medicinal product and an AE is at least a reasonable possibility. Adverse events considered to be related to rhPTH(1-84) and/or other treatments are considered ADRs and will be documented in the electronic AE form in the registry database.


Secondary Outcome Measures :
  1. Health-related Quality of Life (HRQoL) [ Time Frame: Baseline up to 17 years (follow-up) ]
    Health-Related Quality of Life (HRQoL), as measured by the short-form-10 (SF-10) for pediatrics, short-form-36 (SF-36) for adults will be examined longitudinally using methods for continuous data.

  2. Disease-specific Patient-reported Outcome Measures [ Time Frame: Baseline up to 17 years (follow-up) ]
    Chronic hypoparathyroidism patient-reported outcome will be recorded as measured by the hypoparathyroidism multi-symptom diary (HPT-SD).

  3. Rate of Hospitalization/emergency Room (ER) Visits [ Time Frame: Baseline up to 17 years (follow-up) ]
    The rate of hospitalizations and ER visits during follow-up will be summarized. The event rate is defined as the total number of hospitalizations, ER visits, or physician visits/cumulative follow-up time for chronic hypoparathyroidism or for any other reason.


Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
This registry is designed to enroll globally at least 900 participants with chronic hypoparathyroidism, including a minimum of 300 participants who are receiving treatment with recombinant human parathyroid hormone (rhPTH) (1-84).
Criteria

Inclusion Criteria:

1. Subjects diagnosed with chronic hypoparathyroidism, ie, hypoparathyroidism with a duration of longer than 6 months, including:

  1. Adult subjects (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 subjects (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:

  1. Subjects unable to provide informed consent.
  2. Subjects using rhPTH(1-34) or used rhPTH(1-34) for more than 2 years and in the last 3 months.
  3. Subjects currently enrolled in an interventional clinical study (whether or not the study is related to hypoparathyroidism). Note that this does not include subjects enrolled in other observational registries.
  4. History of hypoparathyroidism resulting from a known activating mutation in the CaSR gene.
  5. History of hypoparathyroidism resulting from impaired responsiveness to PTH (pseudohypoparathyroidism).
Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01922440


Contacts
Contact: Shire Contact clinicaltransparency@shire.com

  Show 48 Study Locations
Sponsors and Collaborators
Shire
Investigators
Study Director: Shire Study Physician Shire
More Information

Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT01922440     History of Changes
Other Study ID Numbers: PAR-R13-001
First Posted: August 14, 2013    Key Record Dates
Last Update Posted: November 29, 2017
Last Verified: November 2017

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Shire:
Chronic Hypoparathyroidism
rhPTH(1-84)
Observational study
parathyroid hormone (PTH)
Drug registry

Additional relevant MeSH terms:
Hypoparathyroidism
Parathyroid Diseases
Endocrine System Diseases