Transplantation of stem cells for the treatment of svere systemic sclerosis with a protocol which is adapted to individual manifestatons of the disease

• Register: EUCTR
• Last refreshed on: 27 November 2023
• Main ID: EUCTR2011-002434-40-DE
• Date of registration: 18/08/2011
• Prospective Registration: Yes
• Primary sponsor: University Hospital Tuebingen
• Public title: Transplantation of stem cells for the treatment of svere systemic sclerosis with a protocol which is adapted to individual manifestatons of the disease
• Scientific title: Highdose Chemotherapy and transplantation of 34+ selected stem cell for progressive systemic sclerosis Modification according to manifestation - AST-MOMA
• Date of first enrolment: 23/03/2012
• Target sample size: 44
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2011-002434-40
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: no Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

Germany

Contacts

• Name: Department Internal Medicine II
• Affiliation: University Hospital Tuebinegn

• Name: Department Internal Medicine II
• Affiliation: University Hospital Tuebinegn

Key inclusion & exclusion criteria

Inclusion criteria:
? Limited or diffuse cutaneous systemic sclerosis with organ manifestation (lung/heart or kidney)
? Progressive disease despite cyclophosphamide treatment
? Cyclophosphamide i.v.: at least 3 x 500-1000 mg/m² every 3-4 weeks or
? Oral Cyclophosphamide with at least 100mg/day for at least 2 months or
? or contraindications for a cyclophosphamide treatment
? Written informed consent

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 44
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
? Age < 18 years
? Pregnancy or unwillingsness to use contraception
? Concomittend severe disease
? Heart insuffiency with EF < 30% (echo)
? Pulmonary arterial hypertension with papSys > 50mmHg
? Kidney insiffiency with Clearance for creatinine < 30ml/min
? Severe limitation in lung function test
? IVC < 50%
? DLCO = 30%
? Bone marrow defect
? Leukopenia < 2.000/ul
? Thrombopenia < 100.000/ul
? Cyclophosphamide sum dose > 50g (relative)
? Chronic infection (Hepatitis B/C, HIV, salmonella, lues, tuberculosis (relative)
? Severe psychiatric disease
? Abuse of nicotine, alcohol or any illegal drugs
? Poor compliance



Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

Systemic sclerosis
MedDRA version: 14.1 Level: LLT Classification code 10059040 Term: Autologous peripheral haematopoietic stem cell transplant System Organ Class: 10042613 - Surgical and medical procedures

Intervention(s)

Product Name: CD34 selected autologous hematopoietic cells
Product Code: CD34+SC
Pharmaceutical Form: Suspension for infusion

Primary Outcome(s)

Timepoint(s) of evaluation of this end point: Month 6, 12, 24, 30, 36

Secondary Objective: Reduction of treatment related mortality

Main Objective: To optimise the treatment for severe systemic sclerosis with high dose chemotherapy and autologous stem cell transplantation by adapting the procedure to the individual organ manifestation.

Primary end point(s): Overall survival

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: Month 6, 12, 24, 30, 36

Secondary end point(s): Safety (treatment related mortality/toxicity)
Progression free survival
Frequency of cardiac manifestations
Efficacy (mRSS; sHAQ, DLCO/IVC)

Secondary ID(s)

AST-MOMA

Source(s) of Monetary Support

Tuebingen University Hospital

Ethics review

Status: Approved
Approval date: 15/03/2012
Contact: