OsteopetrosisPhase 2/3NCT01087398
Hematopoietic Stem Cell Transplantation for Malignant Infantile Osteopetrosis
A Phase 2/3 study testing Busulfan, Cyclophosphamide, Thymoglobulin, Fludarabine (Conditioning regimen), Stem Cell Transplantation and Cyclosporin, Methotrexate (GVHD prophylaxis) in people with Osteopetrosis, run by Tehran University of Medical Sciences at 1 site in Iran.
Trial summary
The purpose of this study is to evaluate the efficacy and side effects of donor hematopoietic cells using chemotherapy regimen without total-body irradiation in children undergoing a hematopoietic stem cell transplant for Malignant infantile osteopetrosis. Read more about this study
- Who is it for?
- Ages Up to 5 years
- Conditions the study is for
- Osteopetrosis
- What is being tested?
- Busulfan, Cyclophosphamide, Thymoglobulin, Fludarabine (Conditioning regimen), Stem Cell Transplantation and Cyclosporin, Methotrexate (GVHD prophylaxis)
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteIran
- Study dates
- Sep 2009to Dec 2012
- 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 Up to 5 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Diagnosis of Osteopetrosis confirm by bone biopsy and radiographic imaging
- Age up to 5 year old
Who cannot join
- Carbonic Anhydrase II (CAII) deficiency osteopetrosis variant
- Creatinine clearance ≤ 40ml/min/1.73m^2 or RTA
- Bilirubin ≥ 3mg/dL
- SGPT ≥ 500 U/L
- Current severe infection
- Evidence of CNS involvement
- Morbidity such as blindness or deafness
Trial locations and contacts
1 site in 1 country.
Iran1 site, 1 recruiting
- Recruiting
Hematology-Oncology & SCT Research Center
Tehran, Tehran Province, 14114
Amir Ali Hamidieh, MD, 84902645 ext. +98-21, aahamidieh@sina.tums.ac.ir
Ardeshir Ghavamzadeh, MD
About this study
The purpose of this study is to evaluate the efficacy and side effects of donor hematopoietic cells using chemotherapy regimen without total-body irradiation in children undergoing a hematopoietic stem cell transplant for Malignant infantile osteopetrosis. The blood stem cells will be derived from either related donor or unrelated umbilical cord blood or haploidentical donor.
- Conditions
- Osteopetrosis
- Being tested
- Busulfan, Cyclophosphamide, Thymoglobulin, Fludarabine (Conditioning regimen), Stem Cell Transplantation, Cyclosporin, Methotrexate (GVHD prophylaxis)
- Keywords
- Osteopetrosis, HSCT
- Medical subject headings
- Osteopetrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Busulfan, Cyclophosphamide, Thymoglobulin, Fludarabine (Conditioning regimen)Drug
Also called Systemic chemotherapy
For sibling full match:
- Busulfan 16 mg/kg >5year - 20 mg/kg <5year po
- Cyclophosphamide 200 mg/kg iv
For other related full match, sibling or other related with one antigen mismatch and umbilical cord blood:
- Busulfan 16 mg/kg >5year - 20 mg/kg <5year po
- Cyclophosphamide 200 mg/kg iv
- ATG rabbit (Thymoglobulin) 10 mg/kg or ATG horse (Atgam) 40 mg/kg
For haploidentical:
- Busulfan 16 mg/kg >5year - 20 mg/kg <5year po
- Cyclophosphamide 200 mg/kg iv
- Fludarabine 160 mg/m^2
Stem Cell TransplantationProcedure or surgery
Also called HSCT
Patients undergoing Hematopoietic Stem Cell Transplantation from one of below source:
- Sibling full match
- Other related full match
- Sibling or other related with 1 mismatch antigen
- Cord Blood
- Haploidentical
Cyclosporin, Methotrexate (GVHD prophylaxis)Drug
Also called Graft-versus-host disease (GVHD) prophylaxis
- Cyclosporin A 1.5 mg/kg/day iv from -2, then 3 mg/kg/day iv (from +7 in PBSCT and +11 in BMT or UCBT) then 9 mg/kg/day po
- 10 mg/m^2 iv day +1 then 6 mg/m^ iv day +3 and +6 (Not for UCBT)
| Study group | Role | What they receive |
|---|---|---|
| Intervention | Experimental | Not described Receives: Drug: Busulfan, Cyclophosphamide, Thymoglobulin, Fludarabine (Conditioning regimen); Procedure: Stem Cell Transplantation; Drug: Cyclosporin, Methotrexate (GVHD prophylaxis) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Survival and Progressive Free Survival in patient with infantile Osteopetrosis who receive allogeneic HSCT
Measured: 1 year
Secondary outcomes (4)
One year overall survival after allogeneic HSCT
Measured: 1 year
One year Progressive Free Survival after allogeneic HSCT
Measured: 1 year
Transplantation Related Mortality (TRM) after allogeneic HSCT
Measured: 1 year
Acute and chronic GVHD rate after allogeneic HSCT
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 posted16 Mar 2010 (estimated)
- Study startSep 2009
- Primary completionNov 2012 (estimated)
- Study completionDec 2012 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for OsteopetrosisStudies 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
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Tehran University of Medical Sciences
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Amir Ali Hamidieh, MD, Principal investigator, Hematology-Oncology and SCT Research Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01087398
- Sponsor's protocol ID
- HORCSCT-0905
- Status last verified
- May 2012
- First submitted
- 13 Mar 2010
- Last updated
- 4 Jun 2012
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 4 Jun 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Hematopoietic Stem Cell Transplantation for Malignant Infantile Osteopetrosis
- ClinicalTrials.gov ID
- NCT01087398
- Acronym / sponsor ID
- HORCSCT-0905
- Conditions
- Osteopetrosis
- Keywords
- Osteopetrosis, HSCT
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Tehran University of Medical SciencesUniversity, hospital or other
- Study start
- Sep 2009
- Primary completion
- Nov 2012 (estimated)
- Study completion
- Dec 2012 (estimated)
- First posted
- 16 Mar 2010 (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.
The purpose of this study is to evaluate the efficacy and side effects of donor hematopoietic cells using chemotherapy regimen without total-body irradiation in children undergoing a hematopoietic stem cell transplant for Malignant infantile osteopetrosis. The blood stem cells will be derived from either related donor or unrelated umbilical cord blood or haploidentical donor.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Intervention | Experimental | Drug: Busulfan, Cyclophosphamide, Thymoglobulin, Fludarabine (Conditioning regimen), Procedure: Stem Cell Transplantation, Drug: Cyclosporin, Methotrexate (GVHD prophylaxis) |
Who may qualify
- Ages
- Up to 5 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (2 listed)
- Diagnosis of Osteopetrosis confirm by bone biopsy and radiographic imaging
- Age up to 5 year old
Key exclusion criteria (7 listed)
- Carbonic Anhydrase II (CAII) deficiency osteopetrosis variant
- Creatinine clearance ≤ 40ml/min/1.73m^2 or RTA
- Bilirubin ≥ 3mg/dL
- SGPT ≥ 500 U/L
- Current severe infection
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 |
|---|---|---|
| Busulfan, Cyclophosphamide, Thymoglobulin, Fludarabine (Conditioning regimen)Also: Systemic chemotherapyFor sibling full match: * Busulfan 16 mg/kg >5year - 20 mg/kg <5year po * Cyclophosphamide 200 mg/kg iv For other related full match, sibling or other related with one antigen mismatch and umbilical cord blood: * Busulfan 16 mg/kg >5year - 20 mg/kg <5year po * Cyclophosphamide 200 mg/kg iv * ATG rabbit (Thymoglobulin) 10 mg/kg or ATG horse (Atgam) 40 mg/kg For haploidentical: * Busulfan 16 mg/kg >5year - 20 mg/kg <5year po * Cyclophosphamide 200 mg/kg iv * Fludarabine 160 mg/m^2 | Drug | Intervention |
| Stem Cell TransplantationAlso: HSCTPatients undergoing Hematopoietic Stem Cell Transplantation from one of below source: 1. Sibling full match 2. Other related full match 3. Sibling or other related with 1 mismatch antigen 4. Cord Blood 5. Haploidentical | Procedure or surgery | Intervention |
| Cyclosporin, Methotrexate (GVHD prophylaxis)Also: Graft-versus-host disease (GVHD) prophylaxis* Cyclosporin A 1.5 mg/kg/day iv from -2, then 3 mg/kg/day iv (from +7 in PBSCT and +11 in BMT or UCBT) then 9 mg/kg/day po * 10 mg/m^2 iv day +1 then 6 mg/m^ iv day +3 and +6 (Not for UCBT) | Drug | Intervention |
Primary outcome
Overall Survival and Progressive Free Survival in patient with infantile Osteopetrosis who receive allogeneic HSCT
Time frame: 1 year
4 secondary outcomes
One year overall survival after allogeneic HSCT
Time frame: 1 year
One year Progressive Free Survival after allogeneic HSCT
Time frame: 1 year
Transplantation Related Mortality (TRM) after allogeneic HSCT
Time frame: 1 year
Acute and chronic GVHD rate after allogeneic HSCT
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 4 Jun 2012.
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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.
Amir Ali Hamidieh, MD principal investigator
Hematology-Oncology and SCT Research Center
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 Busulfan, Cyclophosphamide, Thymoglobulin, Fludarabine (Conditioning regimen), Stem Cell Transplantation and Cyclosporin, Methotrexate (GVHD prophylaxis) in people with Osteopetrosis, 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 2 inclusion and 7 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 4 Jun 2012. 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. 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?
Contact Amir Ali Hamidieh, MD (84902645 ext. +98-21, aahamidieh@sina.tums.ac.ir), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall Survival and Progressive Free Survival in patient with infantile Osteopetrosis who receive allogeneic HSCT (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 4 Jun 2012.