Phase 3
N=33
Effectiveness of Paricalcitol in Reducing Parathyroid Hormone (PTH) Levels in X-linked Hypophosphatemic Rickets
Hypophosphatemia, Familial · Hyperparathyroidism
Bottom Line
View on ClinicalTrials.gov: NCT00417612 ↗Enrolled (actual)
33
Serious AEs
0.0%
Results posted
Oct 2014
Primary outcome: Primary: Area Under the Curve for Parathyroid Hormone (PTHauc) Measurement — 1223; 1449; 987; 1616 nlEq*26hr/ml — p=0.007
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Paricalcitol (Drug); Placebo (Other)
- Age
- Pediatric, Adult, Older Adult · 9+ yrs
- Sex
- All
- Sponsor
- Yale University
- Primary completion
- Aug 2012
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Area Under the Curve for Parathyroid Hormone (PTHauc) Measurement |
1223; 1449; 987; 1616 | 0.007 sig |
| PRIMARY Area Under the Curve for Parathyroid Hormone (PTH; Percentage Decrease) |
5; 1; 1; 1; 3; 0 | — |
| PRIMARY Reduction of Parathyroid Hormone Area Under the Curve (PTHauc) of 20% or Greater |
53; 25 | — |
| SECONDARY Static Parameters of Serum Alkaline Phosphatase at Baseline and 1 Year for Paricalcitol and Placebo Arms. |
141; 96; 354; 112; 98; 402 | — |
| SECONDARY Serum Calcium |
9.2; 9.3; 9.4; 9.3 | — |
| SECONDARY Bone Scan Severity Score |
2.18; 2.00; 1.76; 2.12 | — |
| SECONDARY Percent Change in Urinary Calcium Excretion From Baseline to 1 Year |
253; 9 | — |
| SECONDARY Serum Intact Fibroblast Growth Factor 23 (FGF23) |
120; 93; 244; 104 | — |
| SECONDARY Serum 1,25 (OH)2D |
51; 47; 52; 46 | — |
Summary
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.
Eligibility Criteria
Inclusion Criteria
- Diagnosis of XLH rickets
- Fasting serum calcium of 10.7 mg/dl or less
- Fasting PTH greater than 40 nleq/ml and less than 120 nleq/ml in the mid-molecule PTH assay at screening (upper limit of normal is 25 nleq/ml)
- Willing and able to participate in the trial
- Taking stable dose of standard therapy for XLH rickets for at least 2 months prior to study entry
- Concomitant therapy for XLH rickets will not be an exclusion criteria
- Parent or guardian willing to provide informed consent, if applicable
Exclusion Criteria
- Concomitant kidney failure (estimated creatinine clearance less than 60 cc/min or serum creatinine greater than 1.5 mg/dl)
- Serum 25-hydroxy vitamin D less than 20 ng/ml. Participants meeting this criterion will receive vitamin D3 supplementation for 3 months and then be rescreened.
- Unable to comply with protocol and appropriate follow-up visits
- Treatment with agents that may affect skeletal metabolism, such as glucocorticoids and anticonvulsants
Data sourced from ClinicalTrials.gov (NCT00417612). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.