Clinical trial
First in Human Study to Evaluate Safety of Cellavita HD Investigational Product After Intravenous Application in Participants With Huntington's Disease
Name
SAVE-DH
Description
Cellavita HD is a stem-cell therapy for Huntington's Disease. This is a first-in-human, non-randomized, phase I study in which participants with Huntington's Disease will receive three intravenous injections and will be followed for 5 years to evaluate safety and tolearability of product and preliminary evidence of effectiveness.
Trial arms
Trial start
2017-10-16
Estimated PCD
2022-12-31
Trial end
2023-12-31
Status
Active (not recruiting)
Phase
Early phase I
Treatment
Cellavita HD Lower Dose
The first three participants enrolled in the study will be assigned to the lower dose arm with staggered treatment, with an interval of 30 days between the first administration of the first participant and the first administration of the second participant assigned to this arm. All participants will receive a total of 3 intravenous administration, one every 30 days.
Arms:
Cellavita HD Lower Dose
Other names:
cellular therapy, mesenchymal stem cells
Cellavita HD Higher dose
The last three participants enrolled in the study will be assigned to the higher dose arm with staggered treatment, with an interval of 30 days between the first administration of the first participant and the first administration of the second participant assigned to this arm. All participants will receive a total of 3 intravenous administration, one every 30 days.
Arms:
Cellavita HD Higher dose
Other names:
cellular therapy, mesenchymal stem cells
Size
6
Primary endpoint
Safety of Cellavita HD by periodic monitoring changes at adverse events, vital signs, laboratory tests, ECG and incidence of benign and malignant neoplasms
first year and in the following 4 years
Eligibility criteria
Inclusion Criteria:
* Sign and date ICF;
* Ability to follow instructions as well as ability to understand and fulfill the study requirements correctly;
* Male participant aged ≥ 21 and ≤ 65;
* Participants who submit medical report (PCR) attesting Huntington's disease with a number of CAG repeats on chromosome 4, greater than or equal to 40 and less than or equal to 50 (if the participant has not performed the examination and/or if he does not have the report available, a new exam should be done);
* Score 5 points or more in motor assessment UHDRS scale (Unified Huntington's Disease Rating Scale) at the time of enrollment;
* Score between 8 and 11 points in the functional capacity of the UHDRS scale at the time of enrollment.
Exclusion Criteria:
* Participation within 12 months in any clinical trial;
* Any medical observation data (clinical and physical) that medical research judge as a risk for subject if enrollment at the study;
* Any laboratory exam data that medical research judge as a risk for subject if enrollment at the study;
* Juvenile Huntington disease diagnosis;
* History of epilepsy;
* Diagnostic of major cognitive impairment;
* Active decompensated psychiatric disease;
* Current or prior history of neoplasia;
* Current history of gastrointestinal, hepatic, renal, endocrine, pulmonary, hematologic, immune, metabolic pathology or severe and uncontrolled cardiovascular disease;
* Diagnostic of any active infection, be it viral, bacterial, fungal, or caused by another pathogen;
* Participants who have contraindication to undergo any of the tests performed in this study, for example, have pacemakers or surgical clip;
* History of alcohol or illegal drugs abusers;
* History of 1 or more episodes of suicide in the two years before Visit V-4;
* Active smoker or have stopped smoking less than six months prior to enrollment;
* Test positive in at least one of the serological tests: HIV 1 and 2 (Anti-HIV-1,2), HTLV I and II, HBV (HBsAg, anti-HBc), HCV (anti-HCV-Ab) and VDRL (Treponema pallidum);
* History of drug allergy, including contrasts for imaging, or bovine products;
* In use or expected use of immunosuppressive drugs or prohibited medicines for the first three months after the first administration of the investigational product;
* Any clinical changes that is interpreted by the medical researcher as a risk to participant's enrollment.
Protocol
{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE1'], 'designInfo': {'allocation': 'NON_RANDOMIZED', 'interventionModel': 'PARALLEL', 'primaryPurpose': 'OTHER', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 6, 'type': 'ACTUAL'}}
Updated at
2022-11-02
1 organization
1 product
1 indication
Organization
Azidus BrasilProduct
Cellavita HDIndication
Huntington's disease