Treatment of Osteogenesis Imperfecta With Parathyroid Hormone and Zoledronic Acid

What Was Tested

In this phase 4 trial, researchers set out to test Teriparatide.

It had two arms (groups): Teriparatide and zoledronic acid (active comparator) and Standard Care (no intervention).

Continuation of existing bone modifying treatment (i.e. bisphosphonate treatment) or no active bone modifying treatment according to the clinical judgement of the local investigator.

The registry classes the interventions as drug.

More on the Plan

The baseline assessment will include dual energy x-ray absorptiometry (DEXA), spine x-rays, safety bloods, medical and fracture history, pain (brief pain inventory, BPI) and quality of life (SF36, HAQ, EQ5D, PSQI).

Blood will be taken for genetic analysis and for analysis of biochemical markers of bone turnover.

In some centres, a high resolution quantitative CT scan (HRQCT) of the wrist and tibia will be performed.

Participants will be seen after 12 months when bloods, questionnaires and HRQTC will be repeated; at 24 months when bloods, questionnaires, DEXA and HRQCT will be repeated.

Who Took Part

The registry lists 350 actual participants took part.

It was open to people of any sex aged from 18 years with no upper age limit.

Healthy volunteers were not accepted; participants needed the condition being studied.

Examples of who could join: Adult patients aged 18 years and over with a clinical diagnosis of Osteogenesis Imperfecta (OI). Patients willing and able to consent and comply with the study protocol.

The full record lists 2 inclusion and 6 exclusion criteria; among the exclusions: current or previous treatment with an investigational (non-licensed experimental) drug with effects on bone metabolism; contraindication to teriparatide or zoledronic acid; women that are breastfeeding.

How the Study Was Run

Design: randomized, parallel-group and single-blind.

Because people in this Teriparatide study were split into groups at random, differences between the groups are less likely to reflect who happened to join each one.

The Teriparatide group and the comparison group ran side by side rather than switching.

In this single-blind Teriparatide study one party knew the assignments and the other did not.

Those kept unaware of assignments: outcomes assessor.

It started in 2016-11-01 with completion listed for 2025-02-28.

It ran at 23 sites in United Kingdom (22) and Ireland (1).

Its stated primary purpose is treatment.

What Was Measured

Primary outcome: Incident clinical fractures validated by x-ray or other imaging (time frame: Through study completion, approximately 5 years).

It also tracked 8 secondary outcomes, for example Total number of incident vertebral fractures, Total number of fractures, Pain assessed by the Brief Pain Inventory (BPI) Short Form measure, Quality of life assessed by the SF-36 (v1) Quality of Life, Sleep quality assessed by the Pittsburgh Sleep Quality Index (PSQI) measure and Functional status assessed by the Health Assessment Questionnaire (HAQ) measure.

What This Means in Plain Language

Put simply, this is a phase 4, randomized trial of Teriparatide in 350 people.

The trial is marked completed; no results are posted to the registry yet, so nothing here describes outcomes.

More context: the Teriparatide compound page, all human trials.