The efficiency of Cell therapy for Werdnig Hoffman patients

The effectivness of allogeneic Adipose derived mesenchymal stem cells on the phenotypic changes of Werdnig Hoffman patients

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

IRCT

Registry ID

IRCT2015073023417N1

Enrollment

Registered

2015-09-12

Start date

2015-09-23

Completion date

Unknown

Last updated

2018-02-22

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Werdnig Hoffman. Infantile spinal muscular atrophy, type I [Werdnig-Hoffman]

Interventions

Treatment - Other

Intervention 1: In the intervention group that comprises of 5 patients: After genetical confirmation of disease and the electromyogram test, there will be intrathecal injections of mesenchymal stem ce

In the intervention group that comprises of 5 patients: After genetical confirmation of disease and the electromyogram test, there will be intrathecal injections of mesenchymal stem cells at 3 doses

In the control group that comprises of 5 patients: There will be no injection. The only intervention will be the electromyogram tests at the same time with intervention group.

Eligibility

Sex/Gender

All

Age

6 Months to 1 Years

Inclusion criteria

Inclusion criteria: Inclusion criteria: Age under 12 month; Weak muscle tone; Weakness in mobility; Patients sitting without full conduction of nerve; Existence of home senses; Normal Brain function; Exclusion criteria: Age beyound 12 month; Brain abnormality; Loss of sensory functions; Malignancies.

Exclusion criteria

Exclusion criteria:

Design outcomes

Primary

Measure	Time frame
Muscle Tone. Timepoint: Before intervention, 3 month after final intervention. Method of measurement: Electromyogram (EMG).	—

Secondary

Measure	Time frame
Change in patients muscle tone. Timepoint: Immediately after intervention. Method of measurement: Electromyogram.;Existence of injected cells. Timepoint: After final intervention. Method of measurement: Spinal cord fluid sampling.;Sensitivity or infection. Timepoint: After intervention. Method of measurement: Direct observation.;Patients Longevity. Timepoint: After the final intervention. Method of measurement: Direct observation.	—

Measure

Time frame

Change in patients muscle tone. Timepoint: Immediately after intervention. Method of measurement: Electromyogram.;Existence of injected cells. Timepoint: After final intervention. Method of measurement: Spinal cord fluid sampling.;Sensitivity or infection. Timepoint: After intervention. Method of measurement: Direct observation.;Patients Longevity. Timepoint: After the final intervention. Method of measurement: Direct observation.

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Countries

Iran (Islamic Republic of)

Contacts

Public ContactRashin Mohseni

School of Advanced Technologies in Medicine, Tehran University of Medical Sciences

rashin_mohseni@yahoo.com; aahamidieh@tums.ac.ir+98 21 8899 1121

Outcome results

None listed

Source: IRCT (via WHO ICTRP) · Data processed: Feb 4, 2026