A Phase 2 interventional study of Nilotinib and Placebo to nilotinib in Pulmonary Arterial Hypertension, sponsored by Novartis Pharmaceuticals. Terminated at 13 sites in 6 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2014-05-01.
Sponsored by Novartis Pharmaceuticals · Phase 2, Interventional, and Treatment
The purpose of this trial was to establish the safety, tolerability and PK of nilotinib in this population and to test the hypothesis that 6 months treatment with nilotinib will significantly reduce pulmonary artery resistance.
The purpose of this trial was to establish the safety, tolerability and PK of nilotinib in this population and to test the hypothesis that 6 months
761 studies on the registry are indexed under Pulmonary Arterial Hypertension; 142 are open to participants now.
This study's enrollment of 23 is below the median of 38 across 509 interventional studies indexed under Pulmonary Arterial Hypertension.
Browse Pulmonary Arterial Hypertension studies →Novartis Pharmaceuticals is the lead sponsor of 2,673 studies on the registry; 228 are open to participants now.
Of its 576 completed or terminated interventional studies of FDA-regulated products, 431 (75%) have results posted.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
Other protocol-defined inclusion/exclusion criteria may apply
Participants in cohort 1 were assigned to receive nilotinib 50 mg during 14 days, followed by 150 mg during 14 days, followed by 300 mg during 140 days. Participants in cohort 2 were assigned to receive nilotinib 300 mg during 168 days
Drug: Nilotinib
Participants were assigned to receive placebo to nilotinib to match 50 mg and 150 mg capsules during 168 days.
Drug: Placebo to nilotinib
Nilotinib capsules for oral administration at 50 mg, 150 mg twice a day and 300 mg (2 capsules of 150 mg) twice a day.
Placebo to nilotinib capsules for oral administration to match 50 mg, 150 mg and 300 mg capsules twice a day
Change in Pulmonary Vascular Resistance (PVR)
Change in pulmonary vascular resistance is measured via right heart catheter assessment according to local hospital procedures. It assesses several prognostic hemodynamic variables in pulmonary hypertension, including Pulmonary Vascular Resistance (PVR). Study was prematurely terminated and not powered for efficacy.
Time frame: 168 days
Change in Six-Minute Walk Distance (6MWD) From Baseline
During standardized walk course participants are connected to a portable pulse oximeter via a finger probe and instructed to walk at a comfortable speed for as far as they could manage in 6 minutes. Study was prematurely terminated and efficacy data were not analyzed or summarized
Time frame: Baseline, 168 days
Total Number of Adverse Events and Serious Adverse Events
Adverse events were summarized by the number of patients having any adverse event overall and presented in the safety section. Study was prematurely terminated.
Time frame: 168 days
23 participants were enrolled into the study (15 in cohort 1; 8 in cohort 2) 8 participants completed cohort 1 and 6 of these participants moved into cohort 1expansion. Of the 5 participants that completed Cohort 1 expansion; 3 participants went into an Extension. None of the participants completed treatment as trial was terminated
| Milestone | Cohort 1: Nilotinib | Cohort 1: Placebo | Cohort 2: Nilotinib | Cohort 2: Placebo |
|---|---|---|---|---|
| Started | 12 | 3 | 4 | 4 |
| Completed | 7 | 1 | 0 | 0 |
| Not completed | 5 | 2 | 4 | 4 |
| Withdrew: Adverse event | 3 | 1 | 1 | 0 |
| Withdrew: Withdrew consent | 2 | 0 | 0 | 0 |
| Withdrew: Administrative problems | 0 | 0 | 2 | 4 |
| Withdrew: Death | 0 | 0 | 1 | 0 |
| Withdrew: Withdrew consent without eos 1 visit | 0 | 1 | 0 | 0 |
| Milestone | Cohort 1: Nilotinib | Cohort 1: Placebo | Cohort 2: Nilotinib | Cohort 2: Placebo |
|---|---|---|---|---|
| Started | 5 | 1 | 0 | 0 |
| Completed | 4 | 1 | 0 | 0 |
| Not completed | 1 | 0 | 0 | 0 |
| Withdrew: Death | 1 | 0 | 0 | 0 |
| Milestone | Cohort 1: Nilotinib | Cohort 1: Placebo | Cohort 2: Nilotinib | Cohort 2: Placebo |
|---|---|---|---|---|
| Started | 3 | 0 | 0 | 0 |
| Completed | 0 | 0 | 0 | 0 |
| Not completed | 3 | 0 | 0 | 0 |
| Withdrew: The study was terminated | 3 | 0 | 0 | 0 |
During standardized walk course participants are connected to a portable pulse oximeter via a finger probe and instructed to walk at a comfortable speed for as far as they could manage in 6 minutes. Study was prematurely terminated and efficacy data were not analyzed or summarized
No measurements were reported for this outcome.
Change in pulmonary vascular resistance is measured via right heart catheter assessment according to local hospital procedures. It assesses several prognostic hemodynamic variables in pulmonary hypertension, including Pulmonary Vascular Resistance (PVR). Study was prematurely terminated and not powered for efficacy.
No measurements were reported for this outcome.
Adverse events were summarized by the number of patients having any adverse event overall and presented in the safety section. Study was prematurely terminated.
No measurements were reported for this outcome.
Collected over Adverse events were collected over the duration of treatment 140 days for cohort 1, 168 days for cohort 2 and up to 1092 days in the extension study. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Cohort 1: Nilotinib | — | 7/12 (58.3%) | 12/12 (100%) |
| Cohort 1: Placebo | — | 1/3 (33.3%) | 1/3 (33.3%) |
| Cohort 2: Nilotinib | — | 2/4 (50%) | 3/4 (75%) |
| Cohort 2: Placebo | — | 1/4 (25%) | 4/4 (100%) |
| Event | Cohort 1: Nilotinib | Cohort 1: Placebo | Cohort 2: Nilotinib | Cohort 2: Placebo |
|---|---|---|---|---|
| Clostridial infectionInfections and infestations | 0/12 | 1/3 | 0/4 | 0/4 |
| Infusion site infectionInfections and infestations | 0/12 | 1/3 | 0/4 | 0/4 |
| SepsisInfections and infestations | 0/12 | 1/3 | 0/4 | 0/4 |
| Disseminated intravascular coagulationBlood and lymphatic system disorders | 0/12 | 0/3 | 1/4 | 0/4 |
| Cardiogenic shockCardiac disorders | 0/12 | 0/3 | 1/4 | 0/4 |
| Torsade de pointesCardiac disorders | 0/12 | 0/3 | 1/4 | 0/4 |
| Gastric ulcer haemorrhageGastrointestinal disorders | 0/12 | 0/3 | 1/4 | 0/4 |
| Cholecystitis chronicHepatobiliary disorders | 3/12 | 0/3 | 0/4 | 0/4 |
| CellulitisInfections and infestations | 0/12 | 0/3 | 1/4 | 0/4 |
| Systemic lupus erythematosusMusculoskeletal and connective tissue disorders | 0/12 | 0/3 | 0/4 | 1/4 |
| Event | Cohort 1: Nilotinib | Cohort 1: Placebo | Cohort 2: Nilotinib | Cohort 2: Placebo |
|---|---|---|---|---|
| NauseaGastrointestinal disorders | 3/12 | 1/3 | 3/4 | 1/4 |
| VomitingGastrointestinal disorders | 3/12 | 0/3 | 3/4 | 0/4 |
| HeadacheNervous system disorders | 6/12 | 1/3 | 3/4 | 1/4 |
| FatigueGeneral disorders | 6/12 | 0/3 | 0/4 | 0/4 |
| Blood bilirubin increasedInvestigations | 0/12 | 0/3 | 2/4 | 0/4 |
| SyncopeNervous system disorders | 0/12 | 0/3 | 0/4 | 2/4 |
| RashSkin and subcutaneous tissue disorders | 3/12 | 0/3 | 2/4 | 0/4 |
| PancytopeniaBlood and lymphatic system disorders | 1/12 | 1/3 | 0/4 | 0/4 |
| DiarrhoeaGastrointestinal disorders | 4/12 | 1/3 | 1/4 | 0/4 |
| Upper respiratory tract infectionInfections and infestations | 1/12 | 1/3 | 0/4 | 1/4 |
an end of study evaluation was only available for 14 of these patients
| Age, Continuous(Years) | Cohort 1: Nilotinib | Cohort 1: Placebo | Cohort 2: Nilotinib | Cohort 2: Placebo | Total |
|---|---|---|---|---|---|
| Mean | 52 ± 13.1 | 60 ± 6.1 | 31 ± 14.6 | 33 ± 10.2 | 32 ± 11.8 |
| Sex: Female, Male(Participants) | Cohort 1: Nilotinib | Cohort 1: Placebo | Cohort 2: Nilotinib | Cohort 2: Placebo | Total |
|---|---|---|---|---|---|
| Female | 10 | 3 | 4 | 3 | 20 |
| Male | 2 | 0 | 0 | 1 | 3 |
This study is terminated, as verified in Apr 2014. You cannot join it, but the record below documents what was studied.
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Pulmonary Arterial Hypertension→
Novartis Pharmaceuticals