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Systemic Lupus ErythematosusPhase 2ASSISTNCT00750971

Autologous Stem Cell Transplantation for Refractory Systemic Lupus Erythematosus (ASSIST)

A Phase 2 study testing Immunoablation and Autologous Hematopoietic Stem Cell Transplantation in people with Systemic Lupus Erythematosus, run by Charite University, Berlin, Germany at 8 sites in Germany.

Unknown status

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Official record
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Trial summary

While glucocorticoids and immunosuppressants ameliorate manifestations of SLE in many patients, current therapies are insufficient to control the disease in a subset of patients, and their clinical prognosis remains poor due to the development of vital organ failure, cumulative drug toxicity and to the increased risk… Read more about this study

Who is it for?
Ages 18 to 60 years
Conditions the study is for
Systemic Lupus Erythematosus
What is being tested?
Immunoablation and Autologous Hematopoietic Stem Cell Transplantation
Type of treatment
Surgery or procedure
Could I receive a placebo?
No, everyone receives an active treatment
How many people
30 participants
Where
8 sitesGermany
Study dates
Aug 2008to Aug 2020
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 60 years
  • All sexes
  • No healthy volunteers
  • Adult

Who may be able to join

  • Diagnosis of SLE according to American College of Rheumatology (ACR) classification criteria
  • Age between 18 and 60 years, inclusive
  • Provision of informed consent
  • Active disease, refractory to standard immunosuppressive therapy defined as:
  • BILAG level A and a SLEDAI-score of at least 10, despite treatment with high-dose corticosteroids and pulse intravenous CYC at doses of 500-1000mg/m2 for at least 6 months or mycophenolate mofetil (MMF) at doses of at least 2g -
  • Lupus nephritis with renal biopsy performed within one year prior to screening showing glomerulonephritis WHO class III or IV
Show all 10Show fewer
  • Parenchymal disease of heart or lung
  • Neuropsychiatric lupus
  • Autoimmune cytopenia OR
  • recurrence of disease activity (defined as BILAG level A and a SLEDAI of at least 10) within one year after successful induction therapy with cyclophosphamide or MMF in the presence of an adequate maintenance therapy with either cyclophosphamide (at least 500mg/m2 monthly), mycophenolate mofetil (at least 2g daily), azathioprine (at least 1.5mg/kg/d), methotrexate (at least 15mg weekly), cyclosporine (at least 3mg/kg/d) in patients with persistent anti-dsDNA antibodies

Who cannot join

  • Severe concomitant disease or organ damage
  • renal: renal insufficiency with glomerular filtration rate below 40ml/min
  • cardiac: congestive heart failure, LVEF < 40% determined by echocardiogram, uncontrolled arrhythmia
  • pulmonary: mean pulmonary arterial pressure >50mmHg, DLCO < 40 % predicted
  • gastrointestinal: liver cirrhosis; SGOT, SGPT greater than 2 x the upper limit of normal, unless due to active lupus
  • Ongoing cancer or history of malignancy within 5 years of screening
Show all 12Show fewer
  • Women who are pregnant or breastfeeding or use non-reliable methods of contraception
  • Subjects with active systemic infection
  • Subjects with history of active viral infection within 6 months prior to screening, known HIV-infection or chronic Hepatitis B or Hepatitis C
  • History of allergic reaction to cyclophosphamide, G-CSF or ATG
  • Use of immunosuppressive agents for indications other than SLE
  • Any comorbidity that in the opinion of the investigator would jeopardize the ability of the subject to tolerate therapy

Trial locations and contacts

8 sites in 1 country.

Falk Hiepe, Prof. contact

+49 30 450 513026

falk.hiepe@charite.de

Renate Arnold, Prof. contact

+49 30 450-553-302

renate.arnold@charite.de

8 sites in 1 country

Germany8 sites, 8 recruiting

  • Universitätsmedizin Charité

    Berlin, 10117

    Falk Hiepe, Prof, +49 30 450 513026

    Falk Hiepe, Prof.

    Recruiting

  • Universitätsklinik Köln

    Cologne, 50937

    Andrea Rubbert-Roth, PD, +49 (0) 221-4783993

    Andrea Rubbert-Roth, PD

    Recruiting

  • Universitätsklinik Düsseldorf

    Düsseldorf, 40225

    Mathias Schneider, Prof., +49 (0) 211-8117817

    Mathias Schneider, Prof.

    Recruiting

  • Universitätsklinikum Essen

    Essen, 45239 Essen

    Christoph Specker, Prof., +49 (0)201-84081214

    Christoph Specker, Prof.

    Recruiting

  • Universitätsklinik Heidelberg

    Heidelberg, 69120

    Hanns-Marting Lorenz, Prof., +49 (0) 6221-568044

    Hanns-Martin Lorenz, Prof.

    Recruiting

  • Universitäsklinik Mainz

    Mainz, 55101

    Karin Kolbe, MD

    Karin Kolbe, MD

    Recruiting

  • Universitätsklinik Tübingen

    Tübingen, 72026

    Ina Kötter, PD, +49 (0) 7071-2984095

    Ina Kötter, PD

    Recruiting

  • Universitätsklinik Würzburg

    Würzburg, 97070

    Hans-Peter Tony, Prof., +49 (0) 931-20170420

    Hans-Peter Tony, Prof.

    Recruiting

Other trials in Germany

About this study

Official title: An Open-Label, Phase II Multicenter Cohort Study of Immunoablation With Cyclophosphamide and Antithymocyte-Globulin and Transplantation of Autologous Cd34-Enriched Hemapoietic Stem Cells Versus Currently Available Immunosuppressive/Immunomodulatory Therapy for Treatment of Refractory Systemic Lupus Erythematosus

While glucocorticoids and immunosuppressants ameliorate manifestations of SLE in many patients, current therapies are insufficient to control the disease in a subset of patients, and their clinical prognosis remains poor due to the development of vital organ failure, cumulative drug toxicity and to the increased risk of cardiovascular disease and malignancy. Immunoablative chemotherapy followed by autologous hematopoietic stem cell transplantation (ASCT) has recently emerged as a promising experimental therapy for severely affected patients, providing them the potential to achieve treatment-free, long-term remission. The investigators postulate that immunoablative therapy eliminates or effectively reduces the level of autoreactive T and B lymphocytes and then regeneration of de novo immunity resets the autoreactive immune system into a self-tolerant, protective immune system resulting in prolonged and treatment-free remission.

Being tested
Immunoablation and Autologous Hematopoietic Stem Cell Transplantation
Keywords
ASSIST, SLE, Stem Cell Transplantation, Tolerance
Medical subject headings
Lupus Erythematosus, Systemic, Helping Behavior

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Immunoablation and Autologous Hematopoietic Stem Cell TransplantationProcedure or surgery

    Transplantation of purified CD34+ autologous hematopoietic stem cells mobilized with cyclophosphamide (200mg/m2)and G-CSF (10µg/kg/d) after immunoablation with cyclophosphamide (200mg/kg)and rabbit-antithymocyteglobulin (90mg/kg)

Study groups in NCT00750971
Study groupRoleWhat they receive
1ExperimentalImmunoablation and Autologous Hematopoietic Stem Cell Transplantation
Receives: Procedure: Immunoablation and Autologous Hematopoietic Stem Cell Transplantation
2Active comparatorBest currently available immunosuppressive/immunomodulatory therapy
Receives: Procedure: Immunoablation and Autologous Hematopoietic Stem Cell Transplantation

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. SLEDAI

    Measured: 48 months

Secondary outcomes (3)

  1. Serologic response (autoantibodies)

    Measured: 48 months

  2. Immune Reconstitution

    Measured: 48 months

  3. Organ-specific response parameters

    Measured: 48 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted11 Sep 2008 (estimated)
  2. Study startAug 2008
  3. Primary completionJul 2020 (estimated)
  4. Study completionAug 2020 (estimated)

Resources

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
Non-randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
30 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00750971
Sponsor's protocol ID
CT-1306
Acronym
ASSIST
Status last verified
Mar 2017
First submitted
10 Sep 2008
Last updated
20 Mar 2017
Results posted
No
Data monitoring committee
Yes

Publications

  • Thiel A, Alexander T, Schmidt CA, Przybylski GK, Kimmig S, Kohler S, Radtke H, Gromnica-Ihle E, Massenkeil G, Radbruch A, Arnold R, Hiepe F. Direct assessment of thymic reactivation after autologous stem cell transplantation. Acta Haematol. 2008;119(1):22-7. doi: 10.1159/000117824. Epub 2008 Feb 22.

    PubMed 18292651

  • Jayne D, Passweg J, Marmont A, Farge D, Zhao X, Arnold R, Hiepe F, Lisukov I, Musso M, Ou-Yang J, Marsh J, Wulffraat N, Besalduch J, Bingham SJ, Emery P, Brune M, Fassas A, Faulkner L, Ferster A, Fiehn C, Fouillard L, Geromin A, Greinix H, Rabusin M, Saccardi R, Schneider P, Zintl F, Gratwohl A, Tyndall A; European Group for Blood and Marrow Transplantation; European League Against Rheumatism Registry. Autologous stem cell transplantation for systemic lupus erythematosus. Lupus. 2004;13(3):168-76. doi: 10.1191/0961203304lu525oa.

    PubMed 15119545

  • Rosen O, Thiel A, Massenkeil G, Hiepe F, Haupl T, Radtke H, Burmester GR, Gromnica-Ihle E, Radbruch A, Arnold R. Autologous stem-cell transplantation in refractory autoimmune diseases after in vivo immunoablation and ex vivo depletion of mononuclear cells. Arthritis Res. 2000;2(4):327-36. doi: 10.1186/ar107. Epub 2000 Jun 8.

    PubMed 11056673

  • Alexander T, Thiel A, Rosen O, Massenkeil G, Sattler A, Kohler S, Mei H, Radtke H, Gromnica-Ihle E, Burmester GR, Arnold R, Radbruch A, Hiepe F. Depletion of autoreactive immunologic memory followed by autologous hematopoietic stem cell transplantation in patients with refractory SLE induces long-term remission through de novo generation of a juvenile and tolerant immune system. Blood. 2009 Jan 1;113(1):214-23. doi: 10.1182/blood-2008-07-168286. Epub 2008 Sep 29.

    PubMed 18824594

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Immunoablation and Autologous Hematopoietic Stem Cell Transplantation in people with Systemic Lupus Erythematosus, run by Charite University, Berlin, Germany at 8 sites in Germany.

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 10 inclusion and 12 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 20 Mar 2017. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 8 sites in Germany. 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?

Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

Contact Falk Hiepe, Prof. (+49 30 450 513026, falk.hiepe@charite.de), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

SLEDAI (time frame: 48 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00750971, which also lists 4 linked publications.

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 20 Mar 2017.

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