Gaucher's DiseasePhase 2NCT00004488
Phase II Randomized Study of Alendronate Sodium for Osteopenia in Patients With Gaucher's Disease
A Phase 2 study testing alendronate sodium, calcium carbonate and cholecalciferol in people with Gaucher's Disease and Osteopenia, run by Children's Hospital Medical Center, Cincinnati at 3 sites in 2 countries.
Trial summary
OBJECTIVES: I. Determine the efficacy of alendronate sodium in treating osteopenia (generalized bone density and focal bone lesions) in patients with Gaucher's disease. Read more about this study
- Who is it for?
- Ages 18 to 50 years
- Conditions the study is for
- Gaucher's Disease, Osteopenia
- What is being tested?
- alendronate sodium, calcium carbonate and cholecalciferol
- How many people
- 82 participants
- Where
- 3 sitesin 2 countries
- Study dates
- Oct 1998to Sep 2002
- Phase
- Phase 2
- 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 to 50 years
- All sexes
- No healthy volunteers
- Adult
PROTOCOL ENTRY CRITERIA:
--Disease Characteristics--
Diagnosis of type 1 Gaucher's disease enzymatically proven to have acid beta glucosidase deficiency
Must have been on stable dose (8-60 U/kg/every 2 weeks) of alglucerase enzyme therapy (Cerezyme or Ceredase) for at least 24 months
Lumbar spine bone density below the mean for age, sex, and race
--Prior/Concurrent Therapy--
At least 6 months since prior medications that directly affect skeletal metabolism including, but not limited to, bisphosphonates, calcitonin, parathyroid hormone, or estrogen
--Patient Characteristics--
Renal: No chronic renal failure; No recurrent renal stones
Esophageal: No history of dysphagia; No frequent heartburn; No esophagitis requiring treatment
Other: No untreated hyperthyroidism or hypothyroidism; No concurrent hyperparathyroidism; No concurrent malignancy; No history of alcohol or drug abuse; Not pregnant; Negative pregnancy test; Fertile patients must use effective contraception
Trial locations and contacts
3 sites in 2 countries.
3 sites in 2 countries
United States2 sites
Children's Hospital Medical Center - Cincinnati
Cincinnati, Ohio, 45229-3039
Wright State University School of Medicine
Dayton, Ohio, 45401
About this study
OBJECTIVES:
- Determine the efficacy of alendronate sodium in treating osteopenia (generalized bone density and focal bone lesions) in patients with Gaucher's disease.
Read the detailed description
PROTOCOL OUTLINE:
This is a randomized, double blind, placebo controlled study.
All patients receive oral calcium carbonate and cholecalciferol daily. Patients are randomized to receive oral alendronate sodium or placebo daily for 24 months.
Patients are followed every 6 months for 2 years.
Completion date provided represents the completion date of the grant per OOPD records
- Conditions
- Gaucher's Disease, Osteopenia
- Being tested
- alendronate sodium, calcium carbonate, cholecalciferol
- Keywords
- Gaucher's disease, inborn errors of metabolism, rare disease, sphingolipidoses
- Medical subject headings
- Gaucher Disease, Bone Diseases, Metabolic, Metabolism, Inborn Errors, Rare Diseases, Sphingolipidoses
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
alendronate sodiumDrug
calcium carbonateDrug
cholecalciferolDrug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Oct 1999 (estimated)
- Study startOct 1998
- Study completionSep 2002
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Gaucher's DiseaseStudies of the same condition, open and under way.
- More trials run by Children's Hospital Medical Center, CincinnatiThe sponsor's other registered studies.
- 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
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- Double blind
- Enrolment
- 82 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Sponsor type
- University, hospital or other
- Investigators
- Richard J. Wenstrup, Study chair, Children's Hospital Medical Center, Cincinnati
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00004488
- Sponsor's protocol ID
- 199/14269
- Other IDs
- CHMC-C-FDR001537, CHMC-C-498
- Status last verified
- Mar 2000
- First submitted
- 18 Oct 1999
- Last updated
- 25 Mar 2015
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Mar 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Randomized Study of Alendronate Sodium for Osteopenia in Patients With Gaucher's Disease
- ClinicalTrials.gov ID
- NCT00004488
- Acronym / sponsor ID
- 199/14269
- Conditions
- Gaucher's Disease, Osteopenia
- Keywords
- Gaucher's disease, inborn errors of metabolism, rare disease, sphingolipidoses
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 82 participants (estimated)
- Lead sponsor
- Children's Hospital Medical Center, CincinnatiUniversity, hospital or other
- Study start
- Oct 1998
- Study completion
- Sep 2002
- First posted
- 19 Oct 1999 (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.
OBJECTIVES:
- Determine the efficacy of alendronate sodium in treating osteopenia (generalized bone density and focal bone lesions) in patients with Gaucher's disease.
Detailed description
PROTOCOL OUTLINE:
This is a randomized, double blind, placebo controlled study.
All patients receive oral calcium carbonate and cholecalciferol daily. Patients are randomized to receive oral alendronate sodium or placebo daily for 24 months.
Patients are followed every 6 months for 2 years.
Completion date provided represents the completion date of the grant per OOPD records
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- Double blind
Who may qualify
- Ages
- 18 to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
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 |
|---|---|---|
| alendronate sodium | Drug | – |
| calcium carbonate | Drug | – |
| cholecalciferol | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Mar 2015.
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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.
Richard J. Wenstrup study chair
Children's Hospital Medical Center, Cincinnati
3 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing alendronate sodium, calcium carbonate and cholecalciferol in people with Gaucher's Disease and Osteopenia, run by Children's Hospital Medical Center, Cincinnati at 3 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
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: finished, as of the sponsor's last update on 25 Mar 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States, Israel. 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this 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.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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.
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 25 Mar 2015.