Evaluate Safety and Efficacy of Intravenous Autologous ADMSc for Treatment of Idiopathic Pulmonary Fibrosis

NCT02135380 | Unknown Phase 1 DMC

• 1 other identifier: KRPL/IPF/11-12/002
• Study Type: interventional
• Target: 60
• Locations: 1 country
• Sites: 1

Brief Summary

Despite intense research efforts and clinical trials, there is still no effective treatment that can prolong the survival of patients with IPF. Conventional therapeutic approach includes combination of corticosteroids, anti-oxidants, immunodepressants and immune modulatory anti-fibrotic agents to be discontinued 20 days before screening. The only, so far, therapeutic approach that has been proven effective in terms of prolonging patient's survival is lung transplantation. Nonetheless, not all the patients with IPF are eligible for lung transplantation; there is a significant proportion of these patients that finally succumb while waiting in a lung transplantation list. Therefore, there is critical need for more effective and reliable therapeutic modalities5. Adult Stem Cells (ASCs) seem to represent one of these. Therefore, it is conceivable to assume that adult-stem cells can be easily and safely be applied as a novel therapeutic agent in chronic and fatal lung diseases, including chronic obstructive pulmonary disease (COPD) and IPF. Therefore, there is an urgent need to provide a safe, effective and affordable treatment option for IPF patients. New diagnostic, prognostic and therapeutic strategies need to be developed to reduce the burden of IPF. Given the present lack of appropriate treatment adjunctive in the therapy of IPF, adipose derived stromal vascular fraction provides new opportunities for development of the same. MSCs are having anti-fibrotic activity and hence may be excellent source to tackle pulmonary fibrosis and hence could be explored for their therapeutic potential for treating Idiopathic pulmonary fibrosis. MSC's also display membrane-bound and insoluble secreted molecules involved with cell attachment to neighbouring cells and to the extra cellular matrix.18 This cell surface configuration may enable mesenchymal stem cells to home from bloodstream to mesenchymal tissue.14 As limited clinical information is available about use of SVF and MSC in the IPF patients hence this Open Label, Prospective, Randomized multi center comparative study has been undertaken to explore the tolerability \& effectiveness of SVF in one treatment arm and MSC in second treatment arm in IPF patients. Adipose derived stromal vascular fraction and Mesenchymal Stem Cells has been found in preclinical studies to be safe and effective

Conditions

Idiopathic Pulmonary Fibrosis

Outcome Measures

Primary Outcomes (1)

• Safety

  The Incidence of treatment emergent Adverse Event (AE) in the study.

  9 Month

Secondary Outcomes (1)

• Efficacy

  9 Month

Study Arms (3)

Autologous Stromal Vascular Fraction

OTHER

Single dose of autologous adipose derived Stromal Vascular Fraction (SVF) intravenously.

Biological: Autologous Stromal Vascular Fraction (SVF)

Autologous Adipose Derived MSCs

OTHER

3 doses of 2 million per kg body weight adipose tissue derived Ex-vivo expanded Mesenchymal stem cells (MSC) intravenously each. All the three doses will be given at weekly intervals.

Biological: Autologous Adipose Derived MSCs (ADMSCs)

Control

ACTIVE COMPARATOR

* corticosteroids i.e. Prednisolone ≤10mg/day or ≤20 mg every alternate day * Immunosuppressants like Cyclophosphamide or Azathioprine at a dose of 2mg/kg/day not exceeding 150 mg/day * Antioxidants like N-acetylcysteine (NAC) at a dose upto 1800 mg/day. * Pirfenidone at dose upto 1200 to 1800 mg/day

Other: Control

Interventions

Autologous Stromal Vascular Fraction (SVF) BIOLOGICAL

Study arm A subjects will receive single dose of autologous adipose derived Stromal Vascular Fraction (SVF) intravenously.

Also known as: Autologous Stromal Vascular Fraction

Autologous Stromal Vascular Fraction

Autologous Adipose Derived MSCs (ADMSCs) BIOLOGICAL

Study arm B subjects will receive total 3 doses of 2 million per kg body weight adipose tissue derived Ex-vivo expanded Mesenchymal stem cells (MSC) intravenously each. All the three doses will be given at weekly intervals.

Also known as: Autologous Adipose Derived MSCs

Autologous Adipose Derived MSCs

Control OTHER

Standard Therapy

Control

Eligibility Criteria

• Age: 30 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Subjects aged 30 to 70 years.
• Diagnosed subjects of IPF (HRCT scan suggestive or consistent with a probable diagnosis of usual interstitial pneumonia)
• Diagnosis of IPF ≥ three months before enrolment in the study. In addition, the following functional abnormalities must be present:
• Dyspnoea score of at least 2, on a scale of 0 (minimum) to 4 (maximum) on Modified Medical Research Council (MRC) scale
• Forced Vital Capacity (FVC) of no more than 50 to 80 percent of the predicted value
• Diffusing Lung Capacity for Carbon Monoxide (DLCO) 30 to 80 percent of the predicted value
• The subject should be stable and able to walk ≥ 50 meters in the 6MWT. If supplemental oxygen is needed, this should not exceed 4 litres per min at rest.
• Subjects with adequate subcutaneous fat available for liposuction as assessed by the Plastic Surgeon before liposuction procedure.
• Subjects who have been found medically fit by the chest physician for the use sedation and/ local anesthetic before the Liposuction procedure, INR value of below 2 before liposuction procedure
• Subject who are not currently on or have discontinued treatment with immune-suppressants and/or corticosteroids within at least 20 days prior to screening.
• Non-pregnant, non-lactating females of age ≥18 years, and woman of childbearing potential
• Men who are sexually active and agree to routinely use barrier method from screening and throughout the course of the study or who have undergone sterilization.

You may not qualify if:

• Newly diagnosed subjects of IPF who have not received any treatment for the disease or are drug naïve subjects of IPF.
• Subjects with a diagnosis of severe Pulmonary hypertension and a Mean Pulmonary Arterial Pressure (mPAP) of \>50 mm of Hg by 2D-Echo.
• Forced Vital Capacity (FVC) less than 50 percent of the predicted value
• Diffusing Lung Capacity for Carbon Monoxide (DLCO) less than 30 percent of the predicted value
• History of interstitial pulmonary fibrosis due to collagen vascular disease, connective tissue disorders and autoimmune disease
• Subjects with any type of cancer or other serious concomitant diseases including tuberculosis, granulomatous lung disease (e.g. Sarcoidosis) or any condition in the investigator's opinion that will make the ineligible for the study
• History of clinically significant environmental exposure, ingestion of a drug or cases of pulmonary fibrosis due to hypersensitivity pneumonitis
• History of unstable or deteriorating cardiac or pulmonary disease other than IPF within the 6 months prior to enrolment.
• Subjects who are pregnant, breast-feeding or have childbearing potential and have had a positive pregnancy test prior to receiving the therapy.
• Subject who has received treatment with an investigational drug within prior 3 months or is otherwise participating in another clinical study
• Subject who has undergone surgery within 30 days prior to screening or has planned major surgery.
• Subject/Subject's LAR/impartial witness not willing or able to give written informed consent to participate in the study

Sponsors & Collaborators

• Kasiak Research Pvt. Ltd.: lead

Study Sites (1)

Kasiak Research Pvt Ltd

Thane, Maharashtra, 400 610, India

RECRUITING

MeSH Terms

Conditions

Idiopathic Pulmonary Fibrosis

Condition Hierarchy (Ancestors)

Pulmonary Fibrosis; Lung Diseases, Interstitial; Lung Diseases; Respiratory Tract Diseases

Study Officials

• Dr. Ashok A Mahashur, M.B.B.S.M.D.

  P.D. Hinduja National Hospital and Medical Research Centre

  PRINCIPAL INVESTIGATOR

• Dr. Pratibha S Singhal, M.B.B.S.M.D.

  Bombay Hospital and Medical Research Council

  PRINCIPAL INVESTIGATOR

• Dr.Sujeet K Rajan, M.B.B.S.M.D.

  Bhatia General Hospital

  PRINCIPAL INVESTIGATOR

• Dr. Kartik B Shah, M.B.B.S.M.D.

  Cumballa Hill Hospital And Heart Institute

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Dr.Pankaj A Thakur, PhD

CONTACT

+91-(022)-411 73463; pankaj.thakur@kasiakresearch.com

Sainyukta S Medhekar, MSc

CONTACT

91-(022)-411 73463; sainyukta.medhekar@kasiakresearch.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 8, 2014
• First Posted: May 9, 2014
• Study Start: August 1, 2014
• Primary Completion: August 1, 2015
• Last Updated: May 13, 2014

Record last verified: 2014-05

Locations

IN (1)