Beta-ThalassemiaPhase 2/3NCT01016093
Zoledronic Acid for the Prevention of Bone Loss Post-bone Marrow Transplantation for Thalassemia Major Patients
A Phase 2/3 study testing Zoledronic acid in people with Beta-Thalassemia and Bone Marrow Transplantation, run by Tehran University of Medical Sciences at 1 site in Iran.
Trial summary
This is prospective randomized, double blind study designed to evaluate the use of zoledronic acid in the prevention prevention of bone loss post allogenic BMT done for beta-thalassemia major patients. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Beta-Thalassemia, Bone Marrow Transplantation
- What is being tested?
- Zoledronic acid
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 50 participants
- Where
- 1 siteIran
- Study dates
- Nov 2009to Dec 2011
- Phase
- Phase 2/3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Signed written informed consent.
- Patients must be 18 years old and over.
- Diagnosed with beta-thalassemia major
- Scheduled for allogenic bone marrow transplantation
- The date of randomization must be no more than 1 week after BMT.
- Patients must be accessible for follow-up.
- Life expectancy of at least 12 months from randomization.
Who cannot join
- Patients with any clinical or radiological evidence of existing fracture in the lumbar spine and/or total hip.
- Patients with a history of fracture with low-density or no associated trauma.
- Osteoporotic patients with T-score <= -2.5
- Previous treatment with a bisphosphonate.
- Patients with abnormal renal function as evidenced by either a serum creatinine determination 1.5 x or greater above the upper limit of normal or by a calculated creatinine clearance of 30 ml/minute or less.
- Pregnancy and lactation.
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- Women of childbearing potential not on a medically recognized form of contraception.
- Subjects who, in the opinion of the investigator, are unlikely to cooperate fully during the study.
- Subjects participating simultaneously in studies with unapproved drugs, indications or treatment regimens.
- Known hypersensitivity to zoledronic acid or bisphosphonates.
- Patients with prior exposure to anabolic steroids, growth hormone, Parathyroid Hormone (PTH) or other drugs known to affect the skeleton (e.g., calcitonin, mithramycin, or gallium nitrate).
- Serious intercurrent illness
- History of metabolic bone diseases
- History of corticosteroid treatment for other causes
- History of antiepileptic drug treatment
- History or surgery at the lumbosacral spine, with or without implantable devices.
- Any disease of the spine that would preclude the proper acquisition of a lumbar spine DXA.
- Mental illness that precludes the patient from giving informed consent.
- Current active dental problems including infection of the teeth or jawbone (maxilla or mandibular); dental or fixture trauma, or a current or prior diagnosis of osteonecrosis of the jaw (ONJ), of exposed bone in the mouth, or of slow healing after dental procedures.
- Recent (within 6 weeks) or planned dental or jaw surgery (e.g.. extraction, implants)
Trial locations and contacts
1 site in 1 country.
Iran1 site
Hematology-Oncology & SCT Research Center
Tehran, Tehran Province, 14114
About this study
Official title: Zoledronic Acid for the Prevention of Bone Loss Post-bone Marrow Transplantation for Thalassemia Major Patients: A Prospective Pilot Study
This is prospective randomized, double blind study designed to evaluate the use of zoledronic acid in the prevention prevention of bone loss post allogenic BMT done for beta-thalassemia major patients.
- Conditions
- Beta-Thalassemia, Bone Marrow Transplantation
- Being tested
- Zoledronic acid, Placebo
- Keywords
- Thalassemia, BMT, HSCT
- Medical subject headings
- beta-Thalassemia, Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Zoledronic acidDrug
Also called Zometa
Zoledronic acid 4 mg adjusted dose based on renal function IV 15-minute infusion every 3 months for a total of one year ( 4 doses ) beginning as soon as possible after randomization.
PlaceboDrug
Placebo
| Study group | Role | What they receive |
|---|---|---|
| Intervention | Experimental | Patients in this arm received zoledronic acid. Receives: Drug: Zoledronic acid |
| Control | Placebo comparator | Patients in this arm received placebo as control group Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
To describe the percent change in the lumbar spine (L1 to L4) BMD, as measured by dual energy X-ray absorptiometry, at 12 months in patients with beta-thalassemia major undergoing allogenic BMT randomized to zoledronic acid versus placebo.
Measured: 1 year
Secondary outcomes (5)
To describe the percent change in total hip BMD at 12 months in the two study arms.
Measured: 1 year
To compare the changes in bone turn over markers at 3, 6, and 12 months in the two treatment arms.
Measured: 1 year
To describe the incidence rate of all clinical fractures at 1 year in the two study arms.
Measured: 1 year
To describe the general safety of zoledronic acid.
Measured: 1 year
To describe the percent change in lumbar spine (L1 to L4) BMD and total hip BMD at 6 months when available.
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Nov 2009 (estimated)
- Study startNov 2009
- Primary completionNov 2011 (estimated)
- Study completionDec 2011 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Beta-ThalassemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Tehran University of Medical Sciences
- Sponsor type
- University, hospital or other
- Collaborators
- Novartis
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01016093
- Sponsor's protocol ID
- HORCSCT-0601
- Status last verified
- Jul 2011
- First submitted
- 17 Nov 2009
- Last updated
- 26 Jul 2011
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 26 Jul 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Zoledronic Acid for the Prevention of Bone Loss Post-bone Marrow Transplantation for Thalassemia Major Patients: A Prospective Pilot Study
- ClinicalTrials.gov ID
- NCT01016093
- Acronym / sponsor ID
- HORCSCT-0601
- Conditions
- Beta-Thalassemia, Bone Marrow Transplantation
- Keywords
- Thalassemia, BMT, HSCT
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Tehran University of Medical SciencesUniversity, hospital or other
- Collaborators
- Novartis
- Study start
- Nov 2009
- Primary completion
- Nov 2011 (estimated)
- Study completion
- Dec 2011 (estimated)
- First posted
- 18 Nov 2009 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is prospective randomized, double blind study designed to evaluate the use of zoledronic acid in the prevention prevention of bone loss post allogenic BMT done for beta-thalassemia major patients.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Intervention | Experimental | Drug: Zoledronic acidPatients in this arm received zoledronic acid. |
| Control | Placebo comparator | Drug: PlaceboPatients in this arm received placebo as control group |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Signed written informed consent.
- Patients must be 18 years old and over.
- Diagnosed with beta-thalassemia major
- Scheduled for allogenic bone marrow transplantation
- The date of randomization must be no more than 1 week after BMT.
Key exclusion criteria (20 listed)
- Patients with any clinical or radiological evidence of existing fracture in the lumbar spine and/or total hip.
- Patients with a history of fracture with low-density or no associated trauma.
- Osteoporotic patients with T-score <= -2.5
- Previous treatment with a bisphosphonate.
- Patients with abnormal renal function as evidenced by either a serum creatinine determination 1.5 x or greater above the upper limit of normal or by a calculated creatinine clearance of 30 ml/minute or less.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Zoledronic acidAlso: ZometaZoledronic acid 4 mg adjusted dose based on renal function IV 15-minute infusion every 3 months for a total of one year ( 4 doses ) beginning as soon as possible after randomization. | Drug | Intervention |
| PlaceboPlacebo | Drug | Control |
Primary outcome
To describe the percent change in the lumbar spine (L1 to L4) BMD, as measured by dual energy X-ray absorptiometry, at 12 months in patients with beta-thalassemia major undergoing allogenic BMT randomized to zoledronic acid versus placebo.
Time frame: 1 year
5 secondary outcomes
To describe the percent change in total hip BMD at 12 months in the two study arms.
Time frame: 1 year
To compare the changes in bone turn over markers at 3, 6, and 12 months in the two treatment arms.
Time frame: 1 year
To describe the incidence rate of all clinical fractures at 1 year in the two study arms.
Time frame: 1 year
To describe the general safety of zoledronic acid.
Time frame: 1 year
To describe the percent change in lumbar spine (L1 to L4) BMD and total hip BMD at 6 months when available.
Time frame: 1 year
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Jul 2011.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Zoledronic acid in people with Beta-Thalassemia and Bone Marrow Transplantation, run by Tehran University of Medical Sciences at 1 site in Iran.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 20 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 26 Jul 2011. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Iran. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To describe the percent change in the lumbar spine (L1 to L4) BMD, as measured by dual energy X-ray absorptiometry, at 12 months in patients with beta-thalassemia major undergoing allogenic BMT randomized to zoledronic acid versus placebo. (time frame: 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Jul 2011.