A Phase 3 interventional study of Paricalcitol and Placebo in Hypophosphatemia, Familial and Hyperparathyroidism, sponsored by Yale University. Completed at 1 site in United States. Open to participants aged 9 Years and older. Per ClinicalTrials.gov, last updated 2020-03-17.
Sponsored by Yale University · Phase 3, Interventional, and Treatment
The purpose of this study is to determine the effectiveness of paricalcitol, a form of synthetic vitamin D, in lowering parathyroid hormone (PTH) levels and reducing disease symptoms in children and adults with X-linked hypophosphatemic (XLH) rickets.
XLH rickets is a rare inherited disorder in which the bones become painfully soft and bend easily because of a phosphate deficiency. This genetic defect causes the kidneys to allow excretion of an inappropriately high amount of phosphate into the urine. The kidneys are also unable to convert vitamin D into a form usable by the body, resulting in inadequate amounts of active vitamin D. Because vitamin D is needed to absorb calcium and phosphate from the intestine, this deficiency further reduces phosphate levels. Without the sufficient phosphate needed for normal bone growth, individuals with XLH rickets typically develop skeletal malformations, bone pain, and abnormally bowed legs. Hyperparathyroidism, a condition in which the parathyroid glands excrete excess amounts of PTH, also occurs frequently in individuals with XLH rickets, and may play a significant role in the skeletal complications associated with XLH rickets. The purpose of this study is to determine the effectiveness of paricalcitol in lowering PTH levels and reducing disease symptoms in individuals with XLH rickets.
This study will last 12 months. Participants will be randomly assigned to receive either paricalcitol or placebo, taken in the form of two pills daily for the duration of the study. During a baseline 3-day inpatient hospital stay, participants will undergo a physical exam, a cardiac ultrasound, a bone scan, blood collection, and a radiographic skeletal survey. The skeletal survey will include x-rays of various body parts. Participants who are 18 years or younger will not undergo the radiographic skeletal survey. Study visits for all participants will occur every 2 months until the end of the study. These visits will include a physical exam, review of disease symptoms, blood and urine collection, and a check of medication compliance.
110 studies on the registry are indexed under Hyperparathyroidism; 12 are open to participants now.
This study's enrollment of 33 is below the median of 53 across 67 interventional studies indexed under Hyperparathyroidism.
Browse Hyperparathyroidism studies →Yale University is the lead sponsor of 1,770 studies on the registry; 294 are open to participants now.
Of its 221 completed or terminated interventional studies of FDA-regulated products, 129 (58%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Creatinine clearance (CrCl)
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Participants given active drug, paricalcitol (Zemplar), in effort to reduce PTH level
Drug: Paricalcitol
Participants given placebo capsule to match for comparison
Other: Placebo
Paricalcitol given first as a dose of 2 capsules once per day. Dose titration as needed per biochemical results at outpatient visits.
Also known as: Zemplar
Placebo sugar pill
Area Under the Curve for Parathyroid Hormone (PTHauc) Measurement
Mean area under the curve for parathyroid hormone levels (PTHauc) sampled during a 26 hour study period, for Paricalcitol (Active Drug) and Placebo groups at Baseline and Month 12 .
Time frame: Measured at baseline and Month 12
Area Under the Curve for Parathyroid Hormone (PTH; Percentage Decrease)
Mean PTHauc (% decrease) for Paricalcitol (Active Drug) and Placebo from Baseline to Month 12.
Time frame: Measured at baseline and Month 12
Reduction of Parathyroid Hormone Area Under the Curve (PTHauc) of 20% or Greater
Clinically significant reduction of Mean PTHauc (% decrease \>/= 20) for Paricalcitol (Active Drug) and Placebo from Baseline to Month 12.
Time frame: Measured at baseline and Month 12
Static Parameters of Serum Alkaline Phosphatase at Baseline and 1 Year for Paricalcitol and Placebo Arms.
Time frame: Measured at baseline and Month 12
Serum Calcium
Time frame: Measured at baseline and Month 12
Bone Scan Severity Score
99-Tc-methylenediphosphonate bone scans were completed and analyzed using the following scale\*: Bone scan severity scale with minimum score of 0 and maximum score of 4 (0 is no disease and 4 is greatest severity of disease). Appearance of lumbar spine and the sacroiliac region were used as internal references to which all suspected lesions were compared, and scored as follows: grade 0, normal scan without suspicious lesions grade 1, lesion(s) less intense than normal lumbar spine grade 2, lesion(s) similar in intensity to normal lumbar spine grade 3, lesions more intense than normal lumbar spine but similar to the normal sacroiliac region grade 4, lesions more intense than the normal sacroiliac region. \*devised by nuclear medicine radiologist at Yale New Haven Hospital
Time frame: Measured at baseline and Month 12
Percent Change in Urinary Calcium Excretion From Baseline to 1 Year
Percent change in daily urinary calcium excretion, which is calculated the measurements of the calcium in the subject's 24-hr urine collections done at baseline and at the 1 year timepoints
Time frame: Measured at baseline and Month 12
Serum Intact Fibroblast Growth Factor 23 (FGF23)
Time frame: Measured at baseline and Month 12
Serum 1,25 (OH)2D
Time frame: Measured at baseline and Month 12
| Milestone | Paricalcitol (Active Drug) | Placebo |
|---|---|---|
| Started | 22 | 11 |
| Completed | 19 | 8 |
| Not completed | 3 | 3 |
| Withdrew: Errant diagnosis | 0 | 1 |
| Withdrew: Adverse event | 2 | 0 |
| Withdrew: Protocol violation | 0 | 2 |
| Withdrew: Lost to follow-up | 1 | 0 |
Mean area under the curve for parathyroid hormone levels (PTHauc) sampled during a 26 hour study period, for Paricalcitol (Active Drug) and Placebo groups at Baseline and Month 12 .
| nlEq*26hr/ml | Paricalcitol (Active Drug) | Placebo |
|---|---|---|
| Baseline | 1223 ± 426 | 1449 ± 865 |
| Month 12 | 987 ± 372 | 1616 ± 945 |
| mg/dl | Paricalcitol (Active Drug) | Placebo | Paricalcitol (Children Ages 9-17) |
|---|---|---|---|
| Serum Alkaline Phosphatase (Baseline) | 141 ± 96 | 96 ± 28 | 354 ± 2 |
| Serum Alkaline Phosphatase (1 Year) | 112 ± 70 | 98 ± 34 | 402 ± 211 |
| mg/dl | Paricalcitol (Active Drug) | Placebo |
|---|---|---|
| Serum Calcium (Baseline) | 9.2 ± 0.5 | 9.3 ± 0.5 |
| Serum Calcium (1 Year) | 9.4 ± 0.5 | 9.3 ± 0.5 |
99-Tc-methylenediphosphonate bone scans were completed and analyzed using the following scale\*: Bone scan severity scale with minimum score of 0 and maximum score of 4 (0 is no disease and 4 is greatest severity of disease). Appearance of lumbar spine and the sacroiliac region were used as internal references to which all suspected lesions were compared, and scored as follows: grade 0, normal scan without suspicious lesions grade 1, lesion(s) less intense than normal lumbar spine grade 2, lesion(s) similar in intensity to normal lumbar spine grade 3, lesions more intense than normal lumbar spine but similar to the normal sacroiliac region grade 4, lesions more intense than the normal sacroiliac region. \*devised by nuclear medicine radiologist at Yale New Haven Hospital
| units on a scale | Paricalcitol (Active Drug) | Placebo |
|---|---|---|
| Baseline Scan Score | 2.18 ± 1.19 | 2.00 ± 1.51 |
| 12 Month Scan Score | 1.76 ± 1.25 | 2.12 ± 1.55 |
Percent change in daily urinary calcium excretion, which is calculated the measurements of the calcium in the subject's 24-hr urine collections done at baseline and at the 1 year timepoints
| percent change | Paricalcitol (Active Drug) | Placebo |
|---|---|---|
| Percent Change in Urinary Calcium Excretion From Baseline to 1 Year | 253 ± 388 | 9 ± 36 |
| pg/ml | Paricalcitol (Active Drug) | Placebo |
|---|---|---|
| Serum Intact FGF23 (Baseline) | 120 (72 to 203) | 93 (46 to 188) |
| Serum Intact FGF23 (1 Year) | 244 (145 to 411) | 104 (47 to 227) |
| pg/ml | Paricalcitol (Active Drug) | Placebo |
|---|---|---|
| Serum 1,25(OH)2D (Baseline) | 51 ± 18 | 47 ± 9 |
| Serum 1,25 (OH)2D (1 Year) | 52 ± 17 | 46 ± 14 |
Mean PTHauc (% decrease) for Paricalcitol (Active Drug) and Placebo from Baseline to Month 12.
| participants | Paricalcitol (Active Drug) | Placebo |
|---|---|---|
| 20-29% | 5 | 1 |
| 30-39% | 1 | 1 |
| >/= 40% | 3 | 0 |
Clinically significant reduction of Mean PTHauc (% decrease \>/= 20) for Paricalcitol (Active Drug) and Placebo from Baseline to Month 12.
| percentage of participants | Paricalcitol (Active Drug) | Placebo |
|---|---|---|
| Reduction of Parathyroid Hormone Area Under the Curve (PTHauc) of 20% or Greater | 53 | 25 |
Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Paricalcitol (Active Drug) | — | 0/22 (0%) | 2/22 (9.1%) |
| Placebo | — | 0/11 (0%) | 0/11 (0%) |
| Event | Paricalcitol (Active Drug) | Placebo |
|---|---|---|
| Elevated Serum CreatinineRenal and urinary disorders | 1/22 | 0/11 |
| HypercalcemiaBlood and lymphatic system disorders | 1/22 | 0/11 |
| Age, Categorical(Participants) | Paricalcitol (Active Drug) | Placebo | Total |
|---|---|---|---|
| <=18 years | 2 | 1 | 3 |
| Between 18 and 65 years | 19 | 10 | 29 |
| >=65 years | 1 | 0 | 1 |
| Sex: Female, Male(Participants) | Paricalcitol (Active Drug) | Placebo | Total |
|---|---|---|---|
| Female | 17 | 9 | 26 |
| Male | 5 | 2 | 7 |
| Region of Enrollment(participants) | Paricalcitol (Active Drug) | Placebo | Total |
|---|---|---|---|
| United States | 22 | 11 | 33 |
This study is completed, as verified in Mar 2020. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Paricalcitol