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A Registry for Participants With Chronic Hypoparathyroidism (PARADIGHM)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01922440
Recruitment Status : Recruiting
First Posted : August 14, 2013
Last Update Posted : March 7, 2022
Sponsor:
Collaborator:
Takeda Development Center Americas, Inc.
Information provided by (Responsible Party):
Takeda

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.


Condition or disease Intervention/treatment
Hypoparathyroidism Other: No intervention

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Study Type : Observational [Patient Registry]
Estimated Enrollment : 1064 participants
Observational Model: Other
Time Perspective: Prospective
Target Follow-Up Duration: 10 Years
Official Title: PARADIGHM (Physicians Advancing Disease Knowledge in Hypoparathyroidism): A Registry for Patients With Chronic Hypoparathyroidism
Actual Study Start Date : July 1, 2013
Estimated Primary Completion Date : November 30, 2034
Estimated Study Completion Date : November 30, 2034

Resource links provided by the National Library of Medicine


Group/Cohort Intervention/treatment
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
This is a non-interventional study.

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
This is a non-interventional study.




Primary Outcome Measures :
  1. Change from Baseline in 24-Hour Urine Calcium [ Time Frame: Baseline up to 10 years (follow-up) ]
    Change from baseline in 24-hour urine calcium will be evaluated.

  2. Change from Baseline in Serum Calcium [ Time Frame: Baseline up to 10 years (follow-up) ]
    Change from baseline in serum calcium will be evaluated.

  3. Change from Baseline in Serum Albumin [ Time Frame: Baseline up to 10 years (follow-up) ]
    Change from baseline in serum albumin will be evaluated.

  4. Change from Baseline in Albumin-Corrected Total Calcium [ Time Frame: Baseline up to 10 years (follow-up) ]
    Change from baseline in albumin-corrected total calcium will be evaluated.

  5. Change from Baseline in Serum Ionized Calcium [ Time Frame: Baseline up to 10 years (follow-up) ]
    Change from baseline in serum ionized calcium will be evaluated.

  6. Change from Baseline in Serum Magnesium [ Time Frame: Baseline up to 10 years (follow-up) ]
    Change from baseline in serum magnesium will be evaluated.

  7. Change from Baseline in Serum Phosphate [ Time Frame: Baseline up to 10 years (follow-up) ]
    Change from baseline in serum phosphate will be evaluated

  8. Change from Baseline in 25-Hydroxy (25-OH) Vitamin D results [ Time Frame: Baseline up to 10 years (follow-up) ]
    Change from baseline in 25-OH vitamin D will be evaluated.

  9. Change from Baseline in Serum Creatinine [ Time Frame: Baseline up to 10 years (follow-up) ]
    Change from baseline in serum creatinine will be evaluated.

  10. Change from Baseline in Estimated Glomerular Filtration Rate (eGFR; calculated) [ Time Frame: Baseline up to 10 years (follow-up) ]
    Change from baseline in eGFR; calculated will be evaluated.

  11. Change from Baseline in 24-Hour Urine Protein [ Time Frame: Baseline up to 10 years (follow-up) ]
    Change from Baseline in 24-hour urine protein will be evaluated.

  12. Incidence Rate of the Renal Events [ Time Frame: Baseline up to 10 years (follow-up) ]
    Incidence rate of the renal events will be recorded for nephrolithiasis, nephrocalcinosis, hospitalization/emergency room visits for renal events.

  13. Incidence Rate of the Soft Tissue Calcifications (site) [ Time Frame: Baseline up to 10 years (follow-up) ]
    Incidence rate of the soft tissue calcifications (site) will be recorded.

  14. Incidence Rate of the Cataract [ Time Frame: Baseline up to 10 years (follow-up) ]
    Incidence rate of the cataract will be recorded by questionnaire (present/not present).

  15. Incidence Rate of the Bone Fractures (site) [ Time Frame: Baseline up to 10 years (follow-up) ]
    Incidence rate of the bone fractures (site) will be recorded.

  16. Incidence Rate of the Cardiovascular Events [ Time Frame: Baseline up to 10 years (follow-up) ]
    Incidence rate of the cardiovascular events will be calculated for this clinical outcome. Cardiovascular events include myocardial infarction, stroke, arrhythmia.

  17. Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: Baseline up to 10 years (follow-up) ]
    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.


Secondary Outcome Measures :
  1. Health-related Quality of Life (HRQoL) [ Time Frame: Baseline up to 10 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 10 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 10 years (follow-up) ]
    The rate of hospitalizations and ER visits during follow-up will be summarized.



Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.
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).

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
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Contact: Takeda Contact +1-877-825-3327 medinfoUS@takeda.com

Locations
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Sponsors and Collaborators
Takeda
Takeda Development Center Americas, Inc.
Investigators
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Study Director: Study Director Takeda
Additional Information:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Takeda
ClinicalTrials.gov Identifier: NCT01922440    
Other Study ID Numbers: PAR-R13-001
EUPAS16927 ( Other Identifier: ENCePP EU PAS )
First Posted: August 14, 2013    Key Record Dates
Last Update Posted: March 7, 2022
Last Verified: March 2022

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Studies a U.S. FDA-regulated Drug Product: Yes
Keywords provided by Takeda:
Chronic Hypoparathyroidism
rhPTH(1-84)
Observational study
parathyroid hormone (PTH)
Drug registry
rhPTH(1-34)
Additional relevant MeSH terms:
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Hypoparathyroidism
Parathyroid Diseases
Endocrine System Diseases