Phase 3 Muscular Dystrophy Trial, Completed NCT01197300 Sponsor: Novartis Pharmaceuticals Condition: Muscular Dystrophy
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Trial results

AI generated results summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI

According to the results reported on ClinicalTrials.gov, this trial was an extension study involving children with osteoporosis (weakened bones) who were also being treated with steroid medications (glucocorticoids). A total of 25 children took part — 10 who had received zoledronic acid (the study drug) during the earlier core study, and 15 who had received a placebo (a dummy treatment with no active ingredient). All 10 children in the zoledronic acid group completed the study, while 13 of the 15 in the placebo group completed it. The trial was primarily looking at the longer-term safety profile of zoledronic acid over an additional 12 months, and also tracked changes in bone measurements and certain chemical markers in the blood. The reported data shows that, regarding the primary focus of the study — monitoring for unwanted health events — 7 out of 10 children in the zoledronic acid group and 12 out of 13 completing children in the placebo group experienced at least one adverse event (an unwanted or unexpected health occurrence noted during the study). Serious adverse events (more significant health concerns) were reported in 3 children in the zoledronic acid group and none in the placebo group. No deaths were reported in either group. For the secondary measures, both groups showed increases in bone mineral content (a measure of the amount of mineral in the bones) in the spine and across the whole body over 18 and 24 months from the start of the original study. The reported data also shows changes in two blood markers related to bone activity — P1NP and BSAP — with the zoledronic acid group showing larger decreases in these markers compared to the placebo group at both time points. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

These are the results as reported to ClinicalTrials.gov, not medical advice. Verify independently with the trial site and discuss what they mean for you with your doctor.

Phase 3 Muscular Dystrophy Trial, Completed

NCT01197300
Completed Phase 3 🇦🇺 Australian site

Who may be able to join

AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI

Who might be able to join this trial:

Group 1 (patients from the previous related study):

  • Children and teenagers, boys or girls, aged 6 to 19 years old, who took part in the earlier related study (called the "Core study"), took at least one dose of the study medication, and completed a specific visit in that study
  • Patients must join this extension study at a particular follow-up appointment, within 10 months of a specific point in the Core study
  • Patients who followed the required calcium and vitamin D guidelines (through food or supplements) during the Core study

Group 2 (new patients not in the previous study):

  • Children and teenagers, boys or girls, aged 5 to 17 years old, who would have qualified for the earlier related study but were not enrolled because they had significant back pain caused by spinal fractures and their doctor's standard care would involve treating them with a type of bone-strengthening medication called a bisphosphonate
  • Patients who have a non-cancer condition (such as a rheumatic condition, inflammatory bowel disease, Duchenne muscular dystrophy, or nephrotic syndrome) and have been treated with steroid medications (by mouth or through a drip) at any point in the past 12 months
  • Patients whose bone density scan of the lower spine shows results that are at or below a certain low threshold (confirm with trial site)
  • Patients who have had at least one spinal compression fracture confirmed by a central review team, OR at least one low-impact broken bone in an arm or leg (or two such fractures in the upper limbs) within the past 2 years — "low-impact" means a fall from standing height or lower

Who may not be able to join:

  • Patients from Group 1 who seriously broke the rules of the earlier Core study
  • Patients in Group 2 who have previously used bisphosphonates (a type of bone-strengthening medication), or anyone who has used sodium fluoride at doses used for bone conditions (not regular dental hygiene amounts)
  • Patients whose blood tests show that their calcium or phosphate levels are below the normal range for their age
  • Patients whose blood tests show they are low in Vitamin D (below a certain level)
  • Patients whose kidneys are not working well enough, based on a blood test measuring kidney function
  • Girls who could become pregnant, unless they are not pregnant, not breastfeeding, and have been using a reliable form of birth control for more than 2 months before joining — they must also agree to pregnancy tests during the study

Important: Always verify eligibility with the trial site directly before applying.

Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.

This is a simplified plain English summary of the eligibility criteria. Full criteria are set by the trial investigators and may include additional requirements not shown here. Never self-exclude from a trial based on this summary. Contact the trial site directly to confirm your eligibility.
Last synced 27 July 2026
Phase 3: approximately ~65% of drugs entering this phase reach regulatory approval, based on published industry-wide historical data. This is not specific to this trial.
Phase success rates shown are historical industry-wide averages based on published data. They are not a prediction for this specific trial or your individual situation.

Contact this trial

Principal Investigator: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Australian sites

Novartis Investigative Site, Westmead, New South Wales

Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.

GP referral letter

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Trial details

Status
Completed
Phase
Phase 3
Registry
ClinicalTrials.gov
Start date
25 October 2010
Est. completion
27 February 2019

Where this trial is recruiting

🇦🇺 Australia 🇨🇦 Canada 🇭🇺 Hungary 🇷🇺 Russia 🇿🇦 South Africa 🇬🇧 United Kingdom

1 site(s) in Australia. Confirm current status and contact details directly with the trial site.

Primary endpoints

Long-term Safety of Zoledronic Acid for the Treatment of Osteoporotic Children Treated With Glucocorticoids.

Can't join this trial?

Expanded access pathways

If this trial is not available to you, other access pathways may exist. In Australia, the TGA Special Access Scheme allows access to unapproved therapeutic goods for individual patients.

TGA Special Access Scheme information

Find other recruiting trials on ClinicalTrials.gov

Data last synced from ClinicalTrials.gov: 27 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.

Trial recruitment status can change without notice between our nightly data updates. Always contact the trial site directly to confirm current recruitment status before making any decisions or travel arrangements.

View original record on ClinicalTrials.gov