Hematologic DiseasesNCT04167683
Muscle Dysfunction in Patients With Hematological Diseases Referred to Stem Cell Transplant
An observational study of Hematologic Diseases and Chronic Myeloid Leukemia, run by Copenhagen University Hospital, Rigshospitalet at 1 site in Denmark.
Trial summary
PURPOSE: To investigate the effect of the disease and HSCT on muscle dysfunction and to investigate the prognostic role of muscle dysfunction at critical decision points in patients with hematological diseases referred to hematopoietic stem cell transplant (HSCT). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hematologic Diseases, Chronic Myeloid Leukemia, Acute Myeloid Leukemia
- Type of treatment
- Injection or infusion
- How many people
- 144 participants
- Where
- 1 siteDenmark
- Study dates
- 1 Oct 2023to 1 Nov 2026
- Phase
- Observational
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Patients with malignant hematological diseases referred to myeloablative HSCT, myeloablative RTC-HSCT or non-myeloablative HSCT.
Who may be able to join
- Patients diagnosed with acute myelogenous leukaemia (AML), acute lymphatic leukaemia (ALL), chronic myelomonocytic leukaemia (CMML), myelodysplastic syndrome (MDS), chronic lymphatic leukaemia (CLL), malignant lymphomas or multiple myeloma (MM) referred to myeloablative HSCT, myeloablative RTC-HSCT or non-myeloablative HSCT at the Department of Haematology, Rigshospitalet, Blegdamsvej.
Who cannot join
- age <18; pregnancy; physical or mental disabilities precluding test of muscle function; inability to read and understand Danish
Trial locations and contacts
1 site in 1 country.
About this study
Official title: The Effect of Medical Treatment on Muscle Dysfunction and the Prognostic Role of Muscle Dysfunction at Critical Decision Points in Patients With Hematological Diseases Referred to Myeloablative Hematopoietic Stem Cell Transplant (HSCT), to Myeloablative HSCT With a "Reduced Toxicity Conditioning" Regime With Fludarabine and Treosulfane (FluTreo), or to Non-myeloablative HSCT. - A Prospective Observational Study.
PURPOSE: To investigate the effect of the disease and HSCT on muscle dysfunction and to investigate the prognostic role of muscle dysfunction at critical decision points in patients with hematological diseases referred to hematopoietic stem cell transplant (HSCT).
HSCT: Patients diagnosed with malignant hematological diseases who are referred to myeloablative HSCT, to a myeloablative "reduced toxicity conditioning" regime with Fludarabine and Treosulfane (FluTreo) or to non-myeloablative HSCT.
Read the detailed description
RATIONAL: Patients diagnosed with malignant hematological diseases undergoing HSCT are faced with poor prognosis. The treatment is demanding and associated with severe deconditioning potentially leading to worse prognostic outcomes. To what extend patients body composition at the point of referral to HSCT, as well as changes in body composition throughout the cancer continuum is associated with cancer outcomes is currently not well described, specifically if this should be part of standard clinical evaluation in order to optimize therapy-efficacy. Recent findings suggest that pathophysiological alterations in skeletal muscle mass and function can have significant implications for the risk of disease progression and long term prognosis.
- Conditions
- Hematologic Diseases, Chronic Myeloid Leukemia, Acute Myeloid Leukemia, Acute Lymphoid Leukemia, Myelodysplastic Syndromes, Non Hodgkin Lymphoma
- Being tested
- No intervention
- Keywords
- Muscle mass, Muscle strength, Complications, Patient reported outcomes, PRO, Physical function, hematopoietic stem cell transplant
- Medical subject headings
- Hematologic Diseases, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Leukemia, Myeloid, Acute, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Myelodysplastic Syndromes, Lymphoma, Non-Hodgkin, Leukemia, Lymphocytic, Chronic, B-Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
No interventionOther
No intervention
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 - Patients referred to myeloablative HSCT | These patients will undergo 5 assessments: a baseline-assessment 3-4 week prior to conditioning treatment, at discharge (in-patients) or at day +28 after stem cell infusion (out-patients) and follow-up assessments at 3 months, 6 months and 12 months. Receives: Other: No intervention | |
| Cohort 2 - Patients referred to non myeloablative HSCT | These patients will undergo 5 assessments: a baseline-assessment 3-4 week prior to conditioning treatment, at discharge (in-patients) or at day +28 after stem cell infusion (out-patients) and follow-up assessments at 3 months, 6 months and 12 months. Receives: Other: No intervention |
What is being measured
The primary outcome is the main question the study is designed to answer.
Cohort 1 and Cohort 2 Medical treatment complications
Incidens rate of medical complications (mortality, re-hospitalization, infections, all cause disease relapse, chronic GVHD, return to work)
Measured: From baseline to 1 year follow-up
Secondary outcomes (19)
Hospitalization duration
Total number days in hospital
Measured: From baseline to 1 year follow-up
Disease free survival
Risk of disease progression
Measured: From baseline to 1 year follow-up
Overall survival
Risk of mortality from any-cause
Measured: From baseline to 1 year follow-up
Change in whole body lean mass
Dual-energy X-ray Absorptiometry (DXA) scan
Measured: From baseline to 1 year follow-up
Change in appendicular lean mass
Dual-energy X-ray Absorptiometry (DXA) scan
Measured: From baseline to 1 year follow-up
Change in whole body fat percentage
Dual-energy X-ray Absorptiometry (DXA) scan
Measured: From baseline to 1 year follow-up
Change in visceral fat mass
Dual-energy X-ray Absorptiometry (DXA) scan
Measured: From baseline to 1 year follow-up
Change in bone mineral density
Dual-energy X-ray Absorptiometry (DXA) scan
Measured: From baseline to 1 year follow-up
Change in bone mineral content
Dual-energy X-ray Absorptiometry (DXA) scan
Measured: From baseline to 1 year follow-up
Change in walking capacity
Maximum 10 meter walking speed
Measured: From baseline to 1 year follow-up
Change in lower body physical function
30 seconds Sit-To- Stand test
Measured: From baseline to 1 year follow-up
Change in maximum leg power
Leg extensor power test
Measured: From baseline to 1 year follow-up
Change in inflammation markers
Blood values are registered from the patients hospital record in relation to assessments. C-reactive protein (CRP) and leucocytes are registered as they are inflammation markers
Measured: From baseline to 1 year follow-up
Change in creatinine and hemoglobin
Blood values are registered from the patients hospital record in relation to assessments. C-reactive protein (CRP) and leucocytes are registered as they have an influence on muscle strength
Measured: From baseline to 1 year follow-up
Change in body fat percentage, fat mass, fat-free mass, muscle mass and bone mass and total body water
Bioelectrical Impedance Analyzer
Measured: From baseline to 1 year follow-up
Change in health related quality of life
European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ-C30 Version 3.0)
Measured: From baseline to 1 year follow-up
Change in psychological distress
Hospital Anxiety and Depression Scale (HADS) questionnaire
Measured: From baseline to 1 year follow-up
Change in sleep quality
Pittsburgh Sleep Quality Index (PSQI) questionnaire
Measured: From baseline to 1 year follow-up
Change in physical activity level
International Physical Activity Questionnaire (IPAQ) short form
Measured: From baseline to 1 year follow-up
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Nov 2019
- Study start1 Oct 2023
- Primary completion1 Nov 2026 (estimated)
- Study completion1 Nov 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hematologic DiseasesStudies of the same condition, open and under way.
- More trials run by Copenhagen University Hospital, RigshospitaletThe 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 144 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Copenhagen University Hospital, Rigshospitalet
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Jan Christensen, Copenhagen University Hospital, Rigshospitalet
- Investigators
- Jan Christensen, post doc, Principal investigator, Department of Occupational- and Physiotherapy, Rigshospitalet
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04167683
- Sponsor's protocol ID
- P-2019-223
- Status last verified
- Dec 2025
- First submitted
- 15 Nov 2019
- Last updated
- 8 Dec 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: Not provided
Study at a glance
From the registry record, last updated by the sponsor on 8 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Effect of Medical Treatment on Muscle Dysfunction and the Prognostic Role of Muscle Dysfunction at Critical Decision Points in Patients With Hematological Diseases Referred to Myeloablative Hematopoietic Stem Cell Transplant (HSCT), to Myeloablative HSCT With a "Reduced Toxicity Conditioning" Regime With Fludarabine and Treosulfane (FluTreo), or to Non-myeloablative HSCT. - A Prospective Observational Study.
- ClinicalTrials.gov ID
- NCT04167683
- Acronym / sponsor ID
- P-2019-223
- Conditions
- Hematologic Diseases, Chronic Myeloid Leukemia, Acute Myeloid Leukemia, Acute Lymphoid Leukemia, Myelodysplastic Syndromes, Non Hodgkin Lymphoma
- Keywords
- Muscle mass, Muscle strength, Complications, Patient reported outcomes, PRO, Physical function, hematopoietic stem cell transplant
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 144 participants (estimated)
- Lead sponsor
- Copenhagen University Hospital, RigshospitaletUniversity, hospital or other
- Study start
- 1 Oct 2023 (actual)
- Primary completion
- 1 Nov 2026 (estimated)
- Study completion
- 1 Nov 2026 (estimated)
- First posted
- 19 Nov 2019 (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.
PURPOSE: To investigate the effect of the disease and HSCT on muscle dysfunction and to investigate the prognostic role of muscle dysfunction at critical decision points in patients with hematological diseases referred to hematopoietic stem cell transplant (HSCT).
HSCT: Patients diagnosed with malignant hematological diseases who are referred to myeloablative HSCT, to a myeloablative "reduced toxicity conditioning" regime with Fludarabine and Treosulfane (FluTreo) or to non-myeloablative HSCT.
Detailed description
RATIONAL: Patients diagnosed with malignant hematological diseases undergoing HSCT are faced with poor prognosis. The treatment is demanding and associated with severe deconditioning potentially leading to worse prognostic outcomes. To what extend patients body composition at the point of referral to HSCT, as well as changes in body composition throughout the cancer continuum is associated with cancer outcomes is currently not well described, specifically if this should be part of standard clinical evaluation in order to optimize therapy-efficacy. Recent findings suggest that pathophysiological alterations in skeletal muscle mass and function can have significant implications for the risk of disease progression and long term prognosis.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 - Patients referred to myeloablative HSCT | – | Other: No interventionThese patients will undergo 5 assessments: a baseline-assessment 3-4 week prior to conditioning treatment, at discharge (in-patients) or at day +28 after stem cell infusion (out-patients) and follow-up assessments at 3 months, 6 months and 12 months. |
| Cohort 2 - Patients referred to non myeloablative HSCT | – | Other: No interventionThese patients will undergo 5 assessments: a baseline-assessment 3-4 week prior to conditioning treatment, at discharge (in-patients) or at day +28 after stem cell infusion (out-patients) and follow-up assessments at 3 months, 6 months and 12 months. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Patients with malignant hematological diseases referred to myeloablative HSCT, myeloablative RTC-HSCT or non-myeloablative HSCT.
Key inclusion criteria (1 listed)
- Patients diagnosed with acute myelogenous leukaemia (AML), acute lymphatic leukaemia (ALL), chronic myelomonocytic leukaemia (CMML), myelodysplastic syndrome (MDS), chronic lymphatic leukaemia (CLL), malignant lymphomas or multiple myeloma (MM) referred to myeloablative HSCT, myeloablative RTC-HSCT or non-myeloablative HSCT at the Department of Haematology, Rigshospitalet, Blegdamsvej.
Key exclusion criteria (1 listed)
- age <18; pregnancy; physical or mental disabilities precluding test of muscle function; inability to read and understand Danish
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 |
|---|---|---|
| No interventionNo intervention | Other | Cohort 1 - Patients referred to myeloablative HSCT, Cohort 2 - Patients referred to non myeloablative HSCT |
Primary outcome
Cohort 1 and Cohort 2 Medical treatment complications
Time frame: From baseline to 1 year follow-up
Incidens rate of medical complications (mortality, re-hospitalization, infections, all cause disease relapse, chronic GVHD, return to work)
19 secondary outcomes
Hospitalization duration
Time frame: From baseline to 1 year follow-up
Disease free survival
Time frame: From baseline to 1 year follow-up
Overall survival
Time frame: From baseline to 1 year follow-up
Change in whole body lean mass
Time frame: From baseline to 1 year follow-up
Change in appendicular lean mass
Time frame: From baseline to 1 year follow-up
Change in whole body fat percentage
Time frame: From baseline to 1 year follow-up
Change in visceral fat mass
Time frame: From baseline to 1 year follow-up
Change in bone mineral density
Time frame: From baseline to 1 year follow-up
Change in bone mineral content
Time frame: From baseline to 1 year follow-up
Change in walking capacity
Time frame: From baseline to 1 year follow-up
Change in lower body physical function
Time frame: From baseline to 1 year follow-up
Change in maximum leg power
Time frame: From baseline to 1 year follow-up
Change in inflammation markers
Time frame: From baseline to 1 year follow-up
Change in creatinine and hemoglobin
Time frame: From baseline to 1 year follow-up
Change in body fat percentage, fat mass, fat-free mass, muscle mass and bone mass and total body water
Time frame: From baseline to 1 year follow-up
Change in health related quality of life
Time frame: From baseline to 1 year follow-up
Change in psychological distress
Time frame: From baseline to 1 year follow-up
Change in sleep quality
Time frame: From baseline to 1 year follow-up
Change in physical activity level
Time frame: From baseline to 1 year follow-up
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 8 Dec 2025.
- Individual participant dataSharing planned: No. Not provided
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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.
Jan Christensen, post doc principal investigator
Department of Occupational- and Physiotherapy, Rigshospitalet
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hematologic Diseases and Chronic Myeloid Leukemia, run by Copenhagen University Hospital, Rigshospitalet at 1 site in Denmark.
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 1 inclusion and 1 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: under way, not looking for participants, as of the sponsor's last update on 8 Dec 2025. 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 Denmark. 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.
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?
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?
Cohort 1 and Cohort 2 Medical treatment complications (time frame: From baseline to 1 year follow-up). 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 8 Dec 2025.