Forteo Trial on Idiopathic Osteoporosis in Premenopausal Women

NCT01440803 | Completed Phase 2 Results DMC FDA Drug

• 2 other identifiers: AAAF2251R01FD003902-01
• Study Type: interventional
• Target: 41
• Locations: 1 country
• Sites: 2

Brief Summary

Idiopathic osteoporosis (IOP) is defined as osteoporosis that affects young, otherwise completely healthy individuals with no secondary cause of bone loss. In the course of our prior research with premenopausal women with IOP, the investigators have shown that women with IOP have low areal bone mineral density (aBMD) at the spine, hip and forearm compared to normal women. Additionally, using noninvasive high resolution imaging of the central and peripheral skeleton and detailed analyses of transiliac crest bone biopsies, the investigators identified several features of bone quality in premenopausal women with IOP. There is currently no FDA-approved therapy for IOP in premenopausal women. However, teriparatide (Forteo) has been shown to improve bone mass and microarchitecture in postmenopausal women and is approved for men with primary or idiopathic osteoporosis, as well as men, premenopausal and postmenopausal women with glucocorticoid-induced osteoporosis. Because IOP in premenopausal women is an orphan disease, with an estimated prevalence of about 113,000 in the United States, pharmaceutical companies are unlikely to support development of therapies for this indication. Therefore, the major objective of this protocol is to establish the safety and efficacy of teriparatide in premenopausal women with IOP in a phase 2 clinical trial. All subjects will receive teriparatide as part of the study, but a randomly selected group of patients (10) will receive one year of placebo injections first before starting their two years of treatment. The remainder of subjects (30) will receive active drug only for two years. Funding Source - FDA OOPD

Conditions

Adult Idiopathic Generalized Osteoporosis

Keywords

osteoporosis; premenopausal; forteo; idiopathic; Idiopathic Osteoporosis in Premenopausal Women

Outcome Measures

Primary Outcomes (1)

• Change in Lumbar Spine Bone Mineral Density (LS-BMD) on Active Medication

  Dual Energy X-ray Absorptiometry (DXA) will be used to measuring bone mineral density (BMD).

  Baseline and 12 months

Study Arms (2)

Teriparatide (Forteo)

ACTIVE COMPARATOR

Daily injection of Teriparatide for treatment of idiopathic osteoporosis

Drug: Teriparatide

Placebo saline injection

PLACEBO COMPARATOR

Daily injection of saline placebo for 6 months, followed by 24 months of teriparatide treatment for idiopathic osteoporosis.

Drug: Saline Placebo

Interventions

Teriparatide DRUG

Daily injection of 20 mcg teriparatide for the treatment of idiopathic osteoporosis for 24 months.

Also known as: Forteo, TPTD

Teriparatide (Forteo)

Saline Placebo DRUG

Daily injection of saline placebo for 6 months, followed by teriparatide treatment for 24 months.

Also known as: Placebo

Placebo saline injection

Eligibility Criteria

• Age: 20 Years - 45 Years
• Sex: female
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• Premenopausal women, aged 20-45, with regular menses and no historical or biochemical secondary cause of osteoporosis.
• Documented adult fractures judged to be low-trauma.
• Must be willing to use effective contraception throughout the period of study drug administration.
• Premenopausal women ages 20-35 years must have at least one major osteoporotic fracture (excluding fractures of fingers, toes and face) and low Bone Mineral Density (BMD).
• Premenopausal women above the age of 35 years should have a history of fracture and/or low BMD.

You may not qualify if:

• History of any condition that increases the risk of osteosarcoma
• Early follicular phase serum
• Disorders of mineral metabolism
• Suspicion of osteomalacia
• Vitamin D deficiency
• Pregnancy or lactation within past 12 months
• Prolonged amenorrhea (\> 6 months) during reproductive years (except pregnancy or lactation)
• Prior eating disorder
• Malignancy, except cured basal or squamous cell skin carcinoma
• Endocrinopathy: new onset untreated hyperthyroidism, hypothyroidism, Cushing's syndrome, prolactinoma
• Renal insufficiency
• Liver disease
• Intestinal disorders
• History/current glucocorticoids (GCs), anticonvulsants, anticoagulants, methotrexate, depot progesterone, Gonadotrophin-releasing hormone (GnRH) agonists
• Oral glucocorticoid use (subject will not be excluded if used dose equivalent to less than prednisone 5 mg for \<3 months).

Sponsors & Collaborators

• Elizabeth Shane: lead
• Food and Drug Administration (FDA): collaborator

Study Sites (2)

Creighton University

Omaha, Nebraska, 68131, United States

Location

Columbia University Medical Center

New York, New York, 10032, United States

Location

Related Publications (4)

• Cohen A, Shiau S, Nair N, Recker RR, Lappe JM, Dempster DW, Nickolas TL, Zhou H, Agarwal S, Kamanda-Kosseh M, Bucovsky M, Williams JM, McMahon DJ, Stubby J, Shane E. Effect of Teriparatide on Bone Remodeling and Density in Premenopausal Idiopathic Osteoporosis: A Phase II Trial. J Clin Endocrinol Metab. 2020 Oct 1;105(10):e3540-56. doi: 10.1210/clinem/dgaa489.

  PMID: 32876328 DERIVED

• Cohen A, Kamanda-Kosseh M, Recker RR, Lappe JM, Dempster DW, Zhou H, Cremers S, Bucovsky M, Stubby J, Shane E. Bone Density After Teriparatide Discontinuation in Premenopausal Idiopathic Osteoporosis. J Clin Endocrinol Metab. 2015 Nov;100(11):4208-14. doi: 10.1210/jc.2015-2829. Epub 2015 Sep 10.

  PMID: 26358172 DERIVED

• Nishiyama KK, Cohen A, Young P, Wang J, Lappe JM, Guo XE, Dempster DW, Recker RR, Shane E. Teriparatide increases strength of the peripheral skeleton in premenopausal women with idiopathic osteoporosis: a pilot HR-pQCT study. J Clin Endocrinol Metab. 2014 Jul;99(7):2418-25. doi: 10.1210/jc.2014-1041. Epub 2014 Mar 31.

  PMID: 24684466 DERIVED

• Cohen A, Stein EM, Recker RR, Lappe JM, Dempster DW, Zhou H, Cremers S, McMahon DJ, Nickolas TL, Muller R, Zwahlen A, Young P, Stubby J, Shane E. Teriparatide for idiopathic osteoporosis in premenopausal women: a pilot study. J Clin Endocrinol Metab. 2013 May;98(5):1971-81. doi: 10.1210/jc.2013-1172. Epub 2013 Mar 29.

  PMID: 23543660 DERIVED

Related Links

• Columbia University, Dept of Medicine, Division of Endocrinology website

MeSH Terms

Conditions

Osteoporosis

Interventions

Teriparatide

Condition Hierarchy (Ancestors)

Bone Diseases, Metabolic; Bone Diseases; Musculoskeletal Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Parathyroid Hormone; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Peptides; Amino Acids, Peptides, and Proteins

Results Point of Contact

• Title: Elizabeth Shane, MD
• Organization: Columbia University

es54@cumc.columbia.edu

212-305-7225

Study Officials

• Elizabeth Shane, MD

  Columbia University

  PRINCIPAL INVESTIGATOR

• Adi Cohen, MD

  Columbia University

  STUDY DIRECTOR

• Emily M Stein, MD

  Columbia University

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor of Medicine

Study Record Dates

• First Submitted: September 23, 2011
• First Posted: September 27, 2011
• Study Start: August 2, 2012
• Primary Completion: January 1, 2018
• Study Completion: February 1, 2019
• Last Updated: December 11, 2019
• Results First Posted: May 9, 2019

Record last verified: 2019-12

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)