Sickle Cell DiseaseTRANSPLANTORN2NCT06351462
Comparative Health Status and Quality of Life of Patients With Sickle Cell Disease (SCD) Who Underwent Matched-sibling Hematopoietic Stem Cell Transplantation Versus Non Transplanted SCD Case-control Patients
A study testing Spermogram, Hospital Anxiety and Depression Scale (HADS) and SF36 Quality of life questionnaire in people with Sickle Cell Disease, run by Assistance Publique - Hôpitaux de Paris.
Trial summary
The long term burden of morbidity and mortality in the natural history of sickle cell disease has not been compared up to date to the risks and mortality of a curative option like bone marrow transplantation in severe sickle-cell disease patients. Read more about this study
- Who is it for?
- Ages 15 to 80 years
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- Spermogram, Hospital Anxiety and Depression Scale (HADS) and SF36 Quality of life questionnaire
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 220 participants
- Where
- No sites listed yet
- Study dates
- 1 May 2024to 1 May 2027
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
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 15 to 80 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Study population (exposed-patients), all criteria should be fulfilled:
- Patients alive with Sickle Cell Anemia (SCA, meaning SS and Sbeta0 sickle cell anemia genotype)
- Patients transplanted from allogeneic HLA-compatible sibling donor or from sibling cord blood unit from the 1st of January 2000 and the 31st of December 2012, whatever the age at transplant
- Patients having received conditioning regimen containing busulfan 1mg/kg/dose (or equivalent adjusted body-weight dosage according to recommendation) x 16 doses + cyclophosphamide 200mg/kg total dose + anti-thymoglobuline
- For patient under 18 years at time of enrolment, signed informed consent from both parental representatives
- For patient aged 18 years old : signed informed consent
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- Having an affiliation to a social security regime
- Control-population (Non-exposed patients) :
- For each allografted patient, one non-exposed patient will be matched, based on the following criteria:
- Gender
- Age at the date of transplantation of the exposed patient (+/- 1 year)
- Foetal hemoglobin (HbF) level (+/- 3%) before treatment intensification (defined as the initiation of either hydroxyurea or a transfusion program)
- Hb level (+/- 0,9 g/dl) before treatment intensification
- For patient under 18 years at time of enrolment, signed informed consent from both parental representatives
- For patient above 18 years of age: signed informed consent
- Having an affiliation of to a social security regime
Who cannot join
- Study population:
- Transplantation from donor other than sibling or related cord-blood
- Conditioning regimen other than busulfan 16mg/kg total dose + cyclophosphamide 200mg/kg total dose + anti-thymoglobuline
- For both population:
- Absence of signed informed consent
- Having any debilitating medical or psychiatric illness, which preclude understanding of the inform consent as well as optimal treatment and follow-up
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
The long term burden of morbidity and mortality in the natural history of sickle cell disease has not been compared up to date to the risks and mortality of a curative option like bone marrow transplantation in severe sickle-cell disease patients. Given this lack of data, primary-care Sickle Cell Disease (SCD) physicians and transplant physicians are prevented from a factual debate over the benefit/risk ratio for each patient and refining indications of transplant in patients. Therefore, the present study seeks to describe and compare the very long-term outcomes after either Human Leukocyte Antigen (HLA) -matched sibling transplantation (study arm) and "non-transplant care" for severe sickle cell disease SCA patients in order to yield robust comparative data regarding both arms.
The main objective is to assess the benefit of Hematopoietic stem cell transplantation (HSCT) regarding quality of life compared to standard care after 10 years, in patients with severe Sickle Cell Disease (SCD).
- Conditions
- Sickle Cell Disease
- Being tested
- Spermogram, Hospital Anxiety and Depression Scale (HADS), SF36 Quality of life questionnaire, Psychologist interview, Optional sera banking
- Keywords
- Hematopoietic stem cell transplantation
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SpermogramOther
Spermogram will be proposed to men
Hospital Anxiety and Depression Scale (HADS)Other
Anxiety and depression will be evaluated 10 years after HSCT
SF36 Quality of life questionnaireOther
Quality of life will be evaluated 10 years after HSCT
Psychologist interviewOther
During a follow-up visit
Optional sera bankingOther
One in the study
Optional DNA bankingOther
Once in the study
| Study group | Role | What they receive |
|---|---|---|
| Patients transplanted from allogeneic HLA-compatible sibling donor or from sibling cord blood unit | Other | Not described Receives: Other: Spermogram; Other: Hospital Anxiety and Depression Scale (HADS); Other: SF36 Quality of life questionnaire; Other: Psychologist interview; Other: Optional sera banking; Other: Optional DNA banking |
| Controlled patients - not transplanted | Other | Not described Receives: Other: Spermogram; Other: Hospital Anxiety and Depression Scale (HADS); Other: SF36 Quality of life questionnaire; Other: Psychologist interview; Other: Optional sera banking; Other: Optional DNA banking |
What is being measured
The primary outcome is the main question the study is designed to answer.
Evaluation of quality of life assessed by SF36
It will be assessed by the Short-form 36 (SF36) scale. The Short Form (36) Health Survey is a 36-item measure if health status. The score obtained varies between 0 and 100. The higher the score the less disability.
Ware JE, Sherbourne CD. The Medical Outcomes Study 36-item short-form health survey (SF-36): I. Conceptual framework and item selection. Med Care 1992;30:473-83.
Measured: 10 years after HSCT
Secondary outcomes (3)
Evaluation of gonadal function
Spermogram in men (proposed not requested), Luteinising Hormone (LH), Follicle-Stimulating Hormone (FSH), estrogen, Anti-Müllerian Hormone (AMH) in women, testosterone in men, Amenorrhea in women.
Measured: 10 years after HSCT
Evaluation of quality of life
Questionnaire including data about employment, education level, social financial support.
Measured: 10 years after HSCT
Proportion of patients with anxiety and depression
Anxiety and depression levels will be assessed using the Hamilton Anxiety Depression scale : The HAD scale is a self-assessment scale for detecting states of depression and anxiety in the setting of an hospital medical outpatient clinic.
HADS is a self-administered scale of 14 items which assessed levels of depression and anxiety, divided into 2 subscales of 7 items (Anxiety or HADS-A, Depression or HADS-D). Each item is scored on a scale of 0 to 3. A score is generated for each of the two sub-scales (sum of the 7 items, ranging from 0 to 21). Limit scores, for each of the scores, distinguish: non-cases or asymptomatic ones (score ≤ 7); probable or borderline cases (score 8-10); clearly or clinically symptomatic cases (score ≥ 11)
Measured: 10 years after HSCT
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Apr 2024
- Study start1 May 2024 (estimated)
- Primary completion1 May 2027 (estimated)
- Study completion1 May 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- More trials run by Assistance Publique - Hôpitaux de ParisThe 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 N/A
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 220 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06351462
- Sponsor's protocol ID
- APHP200008
- Acronym
- TRANSPLANTORN2
- Status last verified
- Dec 2023
- First submitted
- 2 Apr 2024
- Last updated
- 19 Apr 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 19 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Comparative Health Status and Quality of Life of Patients With Sickle Cell Disease (SCD) Who Underwent Matched-sibling Hematopoietic Stem Cell Transplantation Versus Non Transplanted SCD Case-control Patients
- ClinicalTrials.gov ID
- NCT06351462
- Acronym / sponsor ID
- TRANSPLANTORN2 / APHP200008
- Conditions
- Sickle Cell Disease
- Keywords
- Hematopoietic stem cell transplantation
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 220 participants (estimated)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Study start
- 1 May 2024 (estimated)
- Primary completion
- 1 May 2027 (estimated)
- Study completion
- 1 May 2027 (estimated)
- First posted
- 8 Apr 2024 (actual)
- 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 long term burden of morbidity and mortality in the natural history of sickle cell disease has not been compared up to date to the risks and mortality of a curative option like bone marrow transplantation in severe sickle-cell disease patients. Given this lack of data, primary-care Sickle Cell Disease (SCD) physicians and transplant physicians are prevented from a factual debate over the benefit/risk ratio for each patient and refining indications of transplant in patients. Therefore, the present study seeks to describe and compare the very long-term outcomes after either Human Leukocyte Antigen (HLA) -matched sibling transplantation (study arm) and "non-transplant care" for severe sickle cell disease SCA patients in order to yield robust comparative data regarding both arms.
The main objective is to assess the benefit of Hematopoietic stem cell transplantation (HSCT) regarding quality of life compared to standard care after 10 years, in patients with severe Sickle Cell Disease (SCD).
Study design
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Patients transplanted from allogeneic HLA-compatible sibling donor or from sibling cord blood unit | Other | Other: Spermogram, Other: Hospital Anxiety and Depression Scale (HADS), Other: SF36 Quality of life questionnaire, Other: Psychologist interview, Other: Optional sera banking, Other: Optional DNA banking |
| Controlled patients - not transplanted | Other | Other: Spermogram, Other: Hospital Anxiety and Depression Scale (HADS), Other: SF36 Quality of life questionnaire, Other: Psychologist interview, Other: Optional sera banking, Other: Optional DNA banking |
Who may qualify
- Ages
- 15 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (16 listed)
- Patients alive with Sickle Cell Anemia (SCA, meaning SS and Sbeta0 sickle cell anemia genotype)
- Patients transplanted from allogeneic HLA-compatible sibling donor or from sibling cord blood unit from the 1st of January 2000 and the 31st of December 2012, whatever the age at transplant
- Patients having received conditioning regimen containing busulfan 1mg/kg/dose (or equivalent adjusted body-weight dosage according to recommendation) x 16 doses + cyclophosphamide 200mg/kg total dose + anti-thymoglobuline
- For patient under 18 years at time of enrolment, signed informed consent from both parental representatives
- For patient aged 18 years old : signed informed consent
Key exclusion criteria (6 listed)
- Transplantation from donor other than sibling or related cord-blood
- Conditioning regimen other than busulfan 16mg/kg total dose + cyclophosphamide 200mg/kg total dose + anti-thymoglobuline
- Absence of signed informed consent
- Having any debilitating medical or psychiatric illness, which preclude understanding of the inform consent as well as optimal treatment and follow-up
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 |
|---|---|---|
| SpermogramSpermogram will be proposed to men | Other | Controlled patients - not transplanted, Patients transplanted from allogeneic HLA-compatible sibling donor or from sibling cord blood unit |
| Hospital Anxiety and Depression Scale (HADS)Anxiety and depression will be evaluated 10 years after HSCT | Other | Controlled patients - not transplanted, Patients transplanted from allogeneic HLA-compatible sibling donor or from sibling cord blood unit |
| SF36 Quality of life questionnaireQuality of life will be evaluated 10 years after HSCT | Other | Controlled patients - not transplanted, Patients transplanted from allogeneic HLA-compatible sibling donor or from sibling cord blood unit |
| Psychologist interviewDuring a follow-up visit | Other | Controlled patients - not transplanted, Patients transplanted from allogeneic HLA-compatible sibling donor or from sibling cord blood unit |
| Optional sera bankingOne in the study | Other | Controlled patients - not transplanted, Patients transplanted from allogeneic HLA-compatible sibling donor or from sibling cord blood unit |
| Optional DNA bankingOnce in the study | Other | Controlled patients - not transplanted, Patients transplanted from allogeneic HLA-compatible sibling donor or from sibling cord blood unit |
Primary outcome
Evaluation of quality of life assessed by SF36
Time frame: 10 years after HSCT
It will be assessed by the Short-form 36 (SF36) scale. The Short Form (36) Health Survey is a 36-item measure if health status. The score obtained varies between 0 and 100. The higher the score the less disability. Ware JE, Sherbourne CD. The Medical Outcomes Study 36-item short-form health survey (SF-36): I. Conceptual framework and item selection. Med Care 1992;30:473-83.
3 secondary outcomes
Evaluation of gonadal function
Time frame: 10 years after HSCT
Evaluation of quality of life
Time frame: 10 years after HSCT
Proportion of patients with anxiety and depression
Time frame: 10 years after HSCT
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Apr 2024.
- Individual participant dataSharing planned: Undecided
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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.
Questions about this trial
What is this clinical trial studying?
A study testing Spermogram, Hospital Anxiety and Depression Scale (HADS) and SF36 Quality of life questionnaire in people with Sickle Cell Disease, run by Assistance Publique - Hôpitaux de Paris.
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 16 inclusion and 6 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: opening soon, as of the sponsor's last update on 19 Apr 2024. Status can change between updates, so confirm with the study contact before making plans.
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.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Jean-Hugues Dalle, Pr (+33140033692, jean-hugues.dalle@aphp.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Evaluation of quality of life assessed by SF36 (time frame: 10 years after HSCT). 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 19 Apr 2024.