Treatment of Hypoparathyroidism With Synthetic Human Parathyroid Hormone 1-34

NCT00001304 | Completed Phase 2 Results DMC

• 2 other identifiers: 92001192-CH-0011
• Study Type: interventional
• Target: 27
• Locations: 1 country
• Sites: 1

Brief Summary

This study has been important in establishing synthetic human parathyroid hormone 1-34 (PTH) as a beneficial treatment for hypoparathyroidism, superior to conventional therapy with calcium and calcitriol. Providing synthetic human parathyroid hormone 1-34 (PTH) to patients who are unresponsive to conventional therapy has enabled severe cases of hypoparathyroidism to be managed effectively with the investigational drug, PTH. The primary goals of this study are to (1) provide long-term PTH therapy to patients who do not respond to conventional therapy; (2) understand the long-term effect of therapeutic PTH replacement on kidney function and bone mineral density; (3) study and track linear growth and bone accrual in children with hypoparathyroidism. (4) determine if subjects reach a normal level of peak bone mass and if the timing of this is comparable to normal age-matched healthy controls.

Conditions

Hypoparathyroidism

Keywords

Hypoparathyroidism; Parathyroid Hormone; Vitamin D; Calcium Metabolism; PTH 1-34; Bone

Outcome Measures

Primary Outcomes (2)

• Serum Calcium Level

  Measurements were taken 1 hour before the morning dose of PTH or, calcitriol and calcium; UOM = mmol/liter, normal range 2.05-2.5. Measurements were obtained on three successive days (three separate measures) semiannually at the NIH CC for each protocol subject. The average data are the average of these three semi-annual data points for each subject which are then averaged across all the semi-annual means for all subjects within each arm over the three years of study.

  3 years

• Urine Calcium Excretion Level

  Measurements were taken1 hour before the morning dose of PTH or, calcitriol and calcium; UOM = mmol/24 h, normal range 1.25-6.25. Measurements were obtained on three successive days (three separate measures) semiannually at the NIH CC for each protocol subject. The average data are the average of these three semi-annual data points for each subject which are then averaged across all the semi-annual means for all subjects within each arm over the three years of study.

  3 years

Secondary Outcomes (5)

• Serum 1,25-hydroxyvitamin D Level

  3 years

• Serum 25-hydroxyvitamin D Level

  3 years

• Serum Magnesium Level

  3 years

• Serum Phosphorus Level

  3 years

• Urinary Creatinine Clearance

  3 years

Study Arms (2)

PTH 1-34

EXPERIMENTAL

All patients received twice daily synthetic Human Parathyroid Hormone 1-34.

Drug: Synthetic Human Parathyroid Hormone 1-34

Calcitriol & Calcium

EXPERIMENTAL

All patients received twice daily Calcitriol and Calcium 1000mg divided into four doses daily.

Drug: Calcitriol & Calcium

Interventions

Synthetic Human Parathyroid Hormone 1-34 DRUG

twice daily subcutaneous injections

Also known as: teriperitide

PTH 1-34

Calcitriol & Calcium DRUG

Twice daily oral calcitriol with 1000 mg Calcium carbonate supplementation

Also known as: rocaltrol; 1,25 OH Vitamin D

Calcitriol & Calcium

Eligibility Criteria

• Age: 17 Years - 69 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• This study will include patients (ages 17-69) with biochemically confirmed hypoparathyroidism.

You may not qualify if:

• Women who are pregnant will be excluded.

Sponsors & Collaborators

• Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD): lead

Study Sites (1)

National Institutes of Health Clinical Center, 9000 Rockville Pike

Bethesda, Maryland, 20892, United States

Location

Related Publications (5)

• Winer KK, Yanovski JA, Cutler GB Jr. Synthetic human parathyroid hormone 1-34 vs calcitriol and calcium in the treatment of hypoparathyroidism. JAMA. 1996 Aug 28;276(8):631-6.

  PMID: 8773636 BACKGROUND

• Winer KK, Yanovski JA, Sarani B, Cutler GB Jr. A randomized, cross-over trial of once-daily versus twice-daily parathyroid hormone 1-34 in treatment of hypoparathyroidism. J Clin Endocrinol Metab. 1998 Oct;83(10):3480-6. doi: 10.1210/jcem.83.10.5185.

  PMID: 9768650 BACKGROUND

• Winer KK, Zhang B, Shrader JA, Peterson D, Smith M, Albert PS, Cutler GB Jr. Synthetic human parathyroid hormone 1-34 replacement therapy: a randomized crossover trial comparing pump versus injections in the treatment of chronic hypoparathyroidism. J Clin Endocrinol Metab. 2012 Feb;97(2):391-9. doi: 10.1210/jc.2011-1908. Epub 2011 Nov 16.

  PMID: 22090268 BACKGROUND

• Winer KK, Ko CW, Reynolds JC, Dowdy K, Keil M, Peterson D, Gerber LH, McGarvey C, Cutler GB Jr. Long-term treatment of hypoparathyroidism: a randomized controlled study comparing parathyroid hormone-(1-34) versus calcitriol and calcium. J Clin Endocrinol Metab. 2003 Sep;88(9):4214-20. doi: 10.1210/jc.2002-021736.

  PMID: 12970289 BACKGROUND

• Winer KK, Sinaii N, Peterson D, Sainz B Jr, Cutler GB Jr. Effects of once versus twice-daily parathyroid hormone 1-34 therapy in children with hypoparathyroidism. J Clin Endocrinol Metab. 2008 Sep;93(9):3389-95. doi: 10.1210/jc.2007-2552. Epub 2008 May 20.

  PMID: 18492754 BACKGROUND

MeSH Terms

Conditions

Hypoparathyroidism

Interventions

Calcitriol; Calcium

Condition Hierarchy (Ancestors)

Parathyroid Diseases; Endocrine System Diseases

Intervention Hierarchy (Ancestors)

Dihydroxycholecalciferols; Hydroxycholecalciferols; Cholecalciferol; Cholestenes; Cholestanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Sterols; Vitamin D; Secosteroids; Membrane Lipids; Lipids; Metals, Alkaline Earth; Elements; Inorganic Chemicals; Metals; Blood Coagulation Factors; Biological Factors

Limitations and Caveats

Study reported here was the primary long-term study for adults with hypoparathyroidism. Subsequent studies included additional patients.

Results Point of Contact

• Title: WINER, Karen
• Organization: National Institute of Child Health and Human Development

winerk@mail.nih.gov

+1 301 435 6877

Study Officials

• Karen K Winer, M.D.

  Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: NIH
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: medical officer

Study Record Dates

• First Submitted: November 3, 1999
• First Posted: November 4, 1999
• Study Start: October 1, 1991
• Primary Completion: March 1, 2014
• Study Completion: April 1, 2014
• Last Updated: December 15, 2015
• Results First Posted: December 15, 2015

Record last verified: 2015-11

Locations

US (1)